Idiopathic Hypersomnia

Neurological Disorder MONDO:0018044 Pathograph 7 Show in embeddings browser Sleep Disorder Neurological Disease

Idiopathic hypersomnia is a central disorder of hypersomnolence defined by irrepressible daytime sleepiness that persists despite normal or long nocturnal sleep, in the absence of any identifiable cause. It is a diagnosis of exclusion, and unusually among the entries in this knowledge base its mechanism is genuinely unknown - the disorder is named for that ignorance. What distinguishes it clinically is not the sleepiness, which it shares with every other hypersomnolence disorder, but its quality: sleep is prolonged and unrefreshing rather than fragmented, naps are long and non-restorative rather than brief and restorative as in narcolepsy, and waking is dominated by severe and prolonged sleep inertia (sleep drunkenness) - a difficulty in achieving full alertness after waking that patients frequently rate as more disabling than the sleepiness itself. Cerebrospinal fluid hypocretin-1 is normal by definition, which excludes the orexin-deficiency mechanism of narcolepsy type 1 and makes idiopathic hypersomnia the principal negative case against that module. The leading positive hypothesis runs in the opposite direction - a substance in cerebrospinal fluid that potentiates GABA-A receptor signalling, i.e. excessive somnogenic inhibition rather than deficient arousal drive - but that finding has not been robustly replicated and the entry curates it as an unconfirmed hypothesis rather than as the mechanism.

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5
Pathophys.
4
Phenotypes
1
Hypotheses
2
Gaps
7
Pathograph
2
Medical Actions
2
Subtypes
1
Trials
🏷

Classifications

Harrison's Part
NEUROLOGIC

Subtypes

2
Idiopathic hypersomnia with long sleep time
Habitual nocturnal sleep of 10 hours or more (ICSD-2 criterion), typically with very severe sleep inertia and unrefreshing long naps. Retained here as a named subtype because it was a formal ICSD-2 category and because treatment trials have been stratified by it, but see the discussion on subtype validity: ICSD-3 abandoned the split.
Show evidence (1 reference)
PMID:26599679 SUPPORT Other
"the description of idiopathic hypersomnia with two forms, polysymptomatic and monosymptomatic, by the same Bedrich Roth in 1976"
Documents the historical two-form division from which the long-sleep and non-long-sleep subtypes descend.
Idiopathic hypersomnia without long sleep time
Nocturnal sleep of normal duration with a mean sleep latency of 8 minutes or less on the multiple sleep latency test and fewer than two sleep-onset REM periods. This is the subtype in which the two randomised modafinil trials were conducted, and the subtype whose boundary with narcolepsy type 2 is least secure.
Show evidence (1 reference)
PMID:33631500 SUPPORT Human Clinical
"Modafinil, an established treatment for narcolepsy, was tested for efficacy and safety in Japanese patients with IH without long sleep time."
Confirms this subtype as a distinct trial population, which is the main operational reason the split is retained here.

Mechanistic Hypotheses

1
Endogenous GABA-A Receptor Potentiation
gabaergic_potentiation EMERGING
Evidence balance 3 support
The leading positive hypothesis, and the reason idiopathic hypersomnia is sometimes described as the mirror image of narcolepsy. Cerebrospinal fluid from hypersomnolent patients was reported to potentiate GABA-A receptor function in vitro in the presence of GABA, with the bioactivity attributable to a trypsin-sensitive component of 500-3000 daltons whose identity was never established. On this model the disorder is one of excessive endogenous somnogenic inhibition rather than deficient arousal drive - which would explain why it coexists with normal orexin, why sleep is abundant rather than fragmented, and why arousal from sleep is specifically impaired. The supporting flumazenil result is mechanistically striking, since flumazenil is conventionally regarded as lacking intrinsic activity, and it reversed the enhancement in vitro and normalised vigilance in an open-label series of seven patients. It is curated as EMERGING and not as the mechanism: the finding rests substantially on one laboratory's assay, the bioactive substance was never identified, and the clinical arm was small and unblinded.
Show evidence (3 references)
PMID:23175709 SUPPORT In Vitro
"We conclude that a naturally occurring substance in CSF augments inhibitory GABA signaling, thus revealing a new pathophysiology associated with excessive daytime sleepiness."
The primary claim of the hypothesis, stated by its originators, and the basis for curating it as a candidate mechanism.
PMID:23175709 SUPPORT Human Clinical
"Furthermore, flumazenil normalized vigilance in seven hypersomnolent patients."
Cited as PARTIAL because it is the clinical arm of the hypothesis and is an open-label observation in seven patients - suggestive, and explicitly not sufficient to establish the mechanism.
PMID:23175709 SUPPORT In Vitro
"The bioactive CSF component had a mass of 500 to 3000 daltons and was neutralized by trypsin."
Records how far characterisation of the putative somnogen got - a mass range and protease sensitivity - and therefore that no molecular identity was established, which is the principal reason the hypothesis remains EMERGING.
?

Discussions and Knowledge Gaps

2
Are idiopathic hypersomnia and narcolepsy type 2 one disorder or two?
KNOWLEDGE GAP gap_ih_versus_narcolepsy_type_2_entity_validity
The two diagnoses are separated by a single polysomnographic count - two or more sleep-onset REM periods on the multiple sleep latency test - applied to a test its own reviewers describe as neither sensitive nor specific, and the count is unstable on repeat testing. Neither disorder has a biological marker, both have normal CSF hypocretin-1, and they respond to the same drugs. This is not a nosological quibble: it determines whether mechanistic studies of either are being conducted in a homogeneous population, and a negative replication in a mixed cohort would be uninterpretable. It is the principal reason this entry's trigger node is curated as unknown rather than inheriting a mechanism from narcolepsy research.
Proposed experiments
Stability of the SOREMP-based classification on repeat testing
exp_repeat_mslt_classification_stability
Serial multiple sleep latency testing at intervals in a cohort meeting criteria for either diagnosis, quantifying how often patients cross the two-SOREMP boundary between tests, and testing whether any clinical, biochemical, or genetic variable segregates with the assigned label independently of that count.
Has the CSF GABA-A-potentiating bioactivity been independently replicated, and what is the substance?
KNOWLEDGE GAP gap_gaba_potentiation_replication
The gabaergic_potentiation hypothesis is the only mechanistic account of idiopathic hypersomnia with direct experimental support, and it is curated here as EMERGING rather than canonical for two specific reasons that are resolvable rather than permanent. The bioactive substance was characterised only as a trypsin-sensitive species of 500-3000 daltons and has never been identified, so there is no assay a second laboratory can run on the analyte itself; and the supporting clinical observation was open-label in seven patients. Until both are addressed, a conforming or citing entry should not treat GABA-A potentiation as established.
Proposed experiments
Molecular identification of the CSF bioactivity
exp_csf_somnogen_identification
Fractionation and mass-spectrometric identification of the GABA-A-potentiating component of hypersomnolent CSF, followed by a blinded multi-site assay of the identified analyte in patients meeting criteria for idiopathic hypersomnia, narcolepsy type 2, and matched controls.
Randomised placebo-controlled trial of flumazenil
exp_flumazenil_randomised_trial
A blinded, placebo-controlled crossover trial of flumazenil in hypersomnolent patients selected by CSF bioactivity status, with objective vigilance and sleep-inertia endpoints, to test whether the open-label response survives control and whether it segregates with the biomarker.

Pathophysiology

5
Unidentified Somnogenic Process
The trigger node, and it is deliberately named for what is not known. No structural lesion, neurotransmitter deficiency, autoimmune target, or causative gene has been established in idiopathic hypersomnia. What is established is negative and constraining: CSF hypocretin-1 is normal, which excludes the orexin-deficiency mechanism; there is no HLA association of the strength seen in narcolepsy type 1; and no consistent neuropathology has been reported. Onset is typically in adolescence or young adulthood, a familial background is frequently reported but has never been rigorously characterised, and no biological marker exists. Curating this node as unknown rather than assigning it a plausible mechanism is the honest position and is what the disorder's name records.
Show evidence (3 references)
PMID:26599679 SUPPORT Other
"Yet, MSLT is neither sensitive nor specific and the polysomnographic diagnostic criteria require continuous readjustment and biologic markers are still lacking."
States the absence of a biological marker directly, which is the evidence for curating this node as an unidentified process rather than assigning it a mechanism.
PMID:26599679 SUPPORT Other
"A familial background is often present but rigorous studies are still lacking."
Records the familial signal and, in the same sentence, that it has not been characterised - so this entry curates no inheritance mechanism.
PMID:12374492 SUPPORT Human Clinical
"Healthy controls and subjects with other sleep disorders all had normal levels."
Establishes the key negative constraint on this node - normal CSF hypocretin-1 in hypersomnias other than narcolepsy-cataplexy - which is why this entry does not conform to orexin_arousal_instability.
Increased Homeostatic Sleep Drive with Preserved Sleep Continuity
The central and defining physiological abnormality: sleep propensity is pathologically elevated, but sleep architecture, efficiency, and continuity are normal or better than normal. This combination is what separates idiopathic hypersomnia from every other cause of daytime sleepiness. In narcolepsy type 1 the sleep-wake switch is unstable in both directions, so nocturnal sleep is fragmented; in sleep-disordered breathing it is destroyed by respiratory events; in circadian disorders it is displaced in time. Here sleep is abundant, consolidated, and still insufficient. Sleep is extended rather than curtailed, and the extension does not relieve the sleepiness - which is the observation that makes a simple sleep-debt model untenable.
sleep GO:0030431 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves increased sleep (GO:0030431). GO:0030431 is a biological process from the Gene Ontology. ↑ INCREASED regulation of the sleep/wake cycle GO:0042749 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves abnormal regulation of the sleep/wake cycle, annotated with regulation of circadian sleep/wake cycle (GO:0042749). GO:0042749 is a biological process from the Gene Ontology. ⚠ ABNORMAL
Show evidence (2 references)
PMID:36921459 SUPPORT Other
"Idiopathic hypersomnia is a central hypersomnolence disorder of unknown origin characterized by excessive daytime sleepiness despite normal or long sleep time, and frequent severe sleep inertia."
States both halves of this node - pathological sleepiness coexisting with normal or increased sleep - which is the combination that defines the disorder.
PMID:38165322 SUPPORT Human Clinical
"cases with IH had more severe sleepiness and sleep propensity, despite similar or longer sleep times"
Population-cohort confirmation with objective polysomnography and multiple sleep latency testing that the sleepiness is not explained by reduced sleep, which is what the node asserts.
Impaired Arousal from Sleep (Sleep Inertia)
A partly separable abnormality, and one that a pure sleep-drive model does not predict. Waking is prolonged, confused, and incomplete: patients are difficult to rouse, may require multiple alarms or another person, and remain impaired for tens of minutes to hours after rising - the "sleep drunkenness" Roth described in the original 1956 account. It is curated as its own node because it dissociates from sleepiness in treatment response as well as in phenomenology, and because it, rather than the sleepiness, is often the most disabling symptom and the one that most reliably distinguishes idiopathic hypersomnia from narcolepsy at the bedside.
sleep/wake cycle state transition to wakefulness GO:0022410 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves abnormal sleep/wake cycle state transition to wakefulness, annotated with circadian sleep/wake cycle process (GO:0022410). GO:0022410 is a biological process from the Gene Ontology. ⚠ ABNORMAL
Show evidence (2 references)
PMID:26599679 SUPPORT Other
"It is often accompanied by sleep of long duration and debilitating sleep inertia."
Names sleep inertia as a defining accompaniment of the disorder rather than an incidental feature, supporting its curation as a separate node.
PMID:26599679 SUPPORT Other
"Idiopathic hypersomnia continues to evolve from the concept of "sleep drunkenness" introduced by Bedrich Roth in Prague in 1956"
Documents that the arousal abnormality, not the sleepiness, was the founding observation of the disease concept.
Non-Restorative Long Sleep and Unrefreshing Naps
Sleep in idiopathic hypersomnia fails at its restorative function while succeeding at its structural one. Nocturnal sleep may extend well beyond ten hours, and daytime naps - which in narcolepsy are short and briefly restorative - are here long and leave the patient no better. This is the feature clinicians use to separate the two disorders in the consulting room, and it constrains any candidate mechanism: whatever is wrong is not a failure to obtain sleep but a failure of obtained sleep to discharge sleep pressure.
sleep GO:0030431 Gene Ontology (GO) Relation: this pathophysiological event involves this biological process This pathophysiological event involves abnormal sleep (GO:0030431). GO:0030431 is a biological process from the Gene Ontology. ⚠ ABNORMAL
Show evidence (1 reference)
PMID:26599679 SUPPORT Other
"as well as a 24 h PSG or a 2-wk actigraphy in association with a sleep log to ensure a total 24-h sleep time longer than or equal to 66O minutes"
Gives the operational threshold (a 24-hour sleep time of at least 660 minutes) by which extended sleep is established, which is how this node is measured. Note the source text contains a typographic error, printing the letter O for the final zero of "660"; the snippet reproduces the cached text verbatim as required and the correct value is 660 minutes.
Chronic Functional Impairment
The clinical endpoint. Idiopathic hypersomnia is most often a chronic condition of adolescent or young-adult onset with substantial occupational, educational, and social disability - sometimes rated by patients as greater than that of narcolepsy, in part because the disorder is less recognised and its severity less readily believed. It is not, however, uniformly permanent: in the only population-based longitudinal series, pathological somnolence remitted in roughly 40% of cases over about twelve years, which is a prognostically important finding and one that any mechanistic account must accommodate.
Show evidence (2 references)
PMID:26599679 SUPPORT Other
"The condition is disabling, sometimes even more so than narcolepsy type 1 or 2."
Establishes the magnitude of disability relative to the better-recognised comparator disorder.
PMID:38165322 SUPPORT Human Clinical
"Longitudinal data (spanning 12.1 ± 4.3 years) demonstrated a chronic course of sleepiness for most of the cases with IH, though pathologic somnolence remitted in roughly 40% of cases."
Quantifies both the chronicity and the substantial remission rate, which together define the natural history recorded at this node.

Pathograph

Use the checkboxes to hide or show graph categories. Hover nodes for evidence and cross-linked metadata.
Pathograph: causal mechanism network for Idiopathic Hypersomnia Interactive directed graph showing how pathophysiology mechanisms, phenotypes, genetic factors and variants, experimental models, environmental triggers, and treatments relate through causal and linked edges.

Phenotypes

4
Nervous System 3
Excessive Daytime Sleepiness OBLIGATE Excessive daytime somnolence HP:0001262 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Excessive daytime somnolence (HP:0001262), qualified as temporality chronic. HP:0001262 is a phenotype from the Human Phenotype Ontology.
Temporal: CHRONIC
Show evidence (1 reference)
PMID:36921459 SUPPORT Other
"Idiopathic hypersomnia is a central hypersomnolence disorder of unknown origin characterized by excessive daytime sleepiness despite normal or long sleep time, and frequent severe sleep inertia."
Excessive daytime sleepiness is the defining diagnostic criterion, so the frequency band is OBLIGATE by definition rather than by observation.
Prolonged Nocturnal Sleep Sleep disturbance HP:0002360 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Sleep disturbance (HP:0002360). HP:0002360 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
PMID:38165322 SUPPORT Human Clinical
"cases with IH had more severe sleepiness and sleep propensity, despite similar or longer sleep times"
Objective cohort evidence for preserved or extended sleep duration in this disorder; frequency deliberately omitted, since the source does not quantify how many patients meet the long-sleep threshold.
Unrefreshing Long Naps Excessive daytime somnolence HP:0001262 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Excessive daytime somnolence (HP:0001262). HP:0001262 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
PMID:26599679 SUPPORT Other
"The key manifestation is hypersomnolence."
Supports hypersomnolence as the core manifestation this phenotype elaborates; the nap-quality contrast itself is stated in the description and not claimed from this snippet, and no frequency band is asserted.
Other 1
Severe Sleep Inertia (Sleep Drunkenness) FREQUENT Sleep drunkeness HP:6000456 Human Phenotype Ontology (HP) Relation: this clinical feature is this phenotype This clinical feature is Sleep drunkenness, annotated with Sleep drunkeness (HP:6000456), qualified as severity severe. HP:6000456 is a phenotype from the Human Phenotype Ontology.
Severity: SEVERE
Show evidence (1 reference)
PMID:36921459 SUPPORT Other
"characterized by excessive daytime sleepiness despite normal or long sleep time, and frequent severe sleep inertia"
Supports the FREQUENT band directly: the source characterises severe sleep inertia as frequent in the disorder, which maps to the FREQUENT enum tier rather than to an obligate or occasional one.
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Medical Actions

2
Modafinil
Action: PharmacotherapyNCI Thesaurus (NCIT) Relation: this treatment is this clinical intervention This treatment is Pharmacotherapy (NCIT:C15986). NCIT:C15986 is a clinical intervention from the NCI Thesaurus. NCIT:C15986
Agent: modafinil CHEBI:31859 Chemical Entities of Biological Interest (CHEBI) Relation: this treatment uses this therapeutic agent This treatment uses modafinil (CHEBI:31859). CHEBI:31859 is a therapeutic agent from Chemical Entities of Biological Interest.
A wake-promoting agent and the most commonly used treatment, prescribed off-label in most jurisdictions since its European idiopathic hypersomnia indication was withdrawn in 2011. It improves objective and subjective sleepiness but is not directed at sleep inertia, which is the symptom patients most often rate as most disabling - a therapeutic gap this entry records deliberately.
Mechanism Target:
INHIBITS Increased Homeostatic Sleep Drive with Preserved Sleep Continuity — Wake promotion opposes the elevated sleep propensity symptomatically; it does not act on the unidentified upstream process and is not disease-modifying.
Show evidence (2 references)
PMID:33631500 SUPPORT Human Clinical
"Patients treated with modafinil experienced a significantly prolonged mean sleep latency on the MWT at the end of the study compared with placebo"
Randomised placebo-controlled evidence of objective benefit on daytime wakefulness in the subtype studied.
PMID:36921459 SUPPORT Other
"Modafinil, which was approved for idiopathic hypersomnia until 2011 in Europe, is the most commonly used treatment and improved sleepiness in two recent randomized placebo-controlled trials."
Establishes modafinil's place in practice and that two randomised trials support it, while recording its off-label regulatory status.
Low-Sodium Oxybate
Action: PharmacotherapyNCI Thesaurus (NCIT) Relation: this treatment is this clinical intervention This treatment is Pharmacotherapy (NCIT:C15986). NCIT:C15986 is a clinical intervention from the NCI Thesaurus. NCIT:C15986
Agent: sodium oxybate CHEBI:749298 Chemical Entities of Biological Interest (CHEBI) Relation: this treatment uses this therapeutic agent This treatment uses sodium oxybate (CHEBI:749298). CHEBI:749298 is a therapeutic agent from Chemical Entities of Biological Interest.
The only agent approved specifically for idiopathic hypersomnia (United States, 2021). Notable mechanistically as well as clinically, because it reduces sleep inertia as well as daytime sleepiness - the only treatment shown to act on both nodes - and because a GABA-B agonist improving a disorder hypothesised to involve excessive GABA-A potentiation is a genuine puzzle that the entry's mechanistic hypothesis does not resolve.
Mechanism Target:
INHIBITS Impaired Arousal from Sleep (Sleep Inertia) — Reduces sleep inertia in randomised withdrawal, distinguishing it from the wake-promoting agents, which do not address this node.
INHIBITS Increased Homeostatic Sleep Drive with Preserved Sleep Continuity — Reduces daytime sleepiness alongside the effect on sleep inertia.
Show evidence (2 references)
PMID:36921459 SUPPORT Human Clinical
"In a placebo-controlled, double-blind, randomized withdrawal study, LXB reduced daytime sleepiness and sleep inertia, and improved daily functioning."
Randomised-withdrawal evidence that the agent acts on both curated symptom nodes, which is what distinguishes it from the wake-promoting agents.
PMID:37409509 SUPPORT Human Clinical
"Efficacy and safety of low-sodium oxybate were maintained or improved during the 6-month OLE, supporting long-term treatment with low-sodium oxybate in adults with idiopathic hypersomnia."
Cited as PARTIAL because the durability claim comes from an open-label extension without a control arm, so it supports persistence of benefit more weakly than the randomised-withdrawal result above.
🔬

Diagnosis

3
Polysomnography followed by multiple sleep latency test
Overnight polysomnography to exclude other causes of hypersomnolence (particularly sleep-disordered breathing and insufficient sleep), followed by a multiple sleep latency test demonstrating a mean sleep latency of 8 minutes or less with fewer than two sleep-onset REM periods. The two-SOREMP threshold is what separates idiopathic hypersomnia from narcolepsy type 2, and it is a weak boundary - see the discussion on entity validity.
Show evidence (2 references)
PMID:26599679 SUPPORT Other
"Polysomnography (PSG) followed by a multiple sleep latency test (MSLT) is mandatory"
Establishes the mandatory diagnostic sequence.
PMID:26599679 SUPPORT Other
"Yet, MSLT is neither sensitive nor specific and the polysomnographic diagnostic criteria require continuous readjustment"
Cited as PARTIAL because it qualifies the same test it recommends: the diagnostic standard is mandatory but acknowledged to be neither sensitive nor specific, which is why this entry does not treat a positive MSLT as establishing a mechanism.
Extended polysomnography or actigraphy with sleep log
A 24-hour polysomnogram, or two weeks of actigraphy with a concurrent sleep log, to document a total 24-hour sleep time of at least 660 minutes. This is the alternative diagnostic route when the mean sleep latency exceeds 8 minutes, and it is the route that captures the long-sleep presentation, which the multiple sleep latency test systematically misses.
actigraphy NCIT:C180883 NCI Thesaurus (NCIT)
Show evidence (1 reference)
PMID:26599679 SUPPORT Other
"as well as a 24 h PSG or a 2-wk actigraphy in association with a sleep log to ensure a total 24-h sleep time longer than or equal to 66O minutes, when the mean sleep latency on the MSLT is longer than 8 min"
Specifies the alternative diagnostic pathway and the circumstance in which it applies. The cached source prints the letter O for the final zero of "660"; the snippet is reproduced verbatim as required and the intended threshold is 660 minutes.
Cerebrospinal fluid hypocretin-1 measurement
Not required for diagnosis, but decisive where narcolepsy type 1 is in the differential: a normal CSF hypocretin-1 level is expected in idiopathic hypersomnia and a level below 110 pg/mL reclassifies the patient as narcolepsy type 1 regardless of the presence or absence of cataplexy.
Show evidence (2 references)
PMID:12374492 SUPPORT Human Clinical
"Hypocretin-1 levels below 110 pg/mL were diagnostic for narcolepsy."
Gives the threshold that excludes this diagnosis, which is the reason the test is listed here despite not being a positive criterion.
PMID:12374492 SUPPORT Human Clinical
"Ten subjects with hypersomnia had intermediate levels"
Cited as PARTIAL because it records the boundary case the clean threshold obscures: a minority of hypersomnolent patients fall in the intermediate range and are classified by neither rule.
📊

Prevalence

1
Wisconsin Sleep Cohort, working-age adults with polysomnography and MSLT data
Point Prevalence 1500.0 per 100,000 (700.0–2500.0) >1 in 1,000
The first population-based estimate; 12 probable cases among 792 participants with objective sleep testing. Substantially higher than clinic-derived impressions of the disorder's rarity, and the authors note this explicitly. Read with care - "probable IH" was ascertained by applying criteria to an existing cohort rather than by clinical diagnosis, so this is a prevalence of the phenotype meeting criteria rather than of the diagnosed disease.
Show evidence (2 references)
PMID:38165322 SUPPORT Human Clinical
"From 792 cohort study participants with available polysomnography and multiple sleep latency test data, 12 cases with probable IH were identified resulting in an estimated prevalence of IH of 1.5%"
Gives the numerator, denominator, and resulting rate directly.
PMID:38165322 SUPPORT Human Clinical
"These results demonstrate IH is more common in the working population than generally assumed"
Records the authors' own framing of the estimate against prior assumptions, which is why the note flags the ascertainment caveat.
🔬

Clinical Trials

1
NCT03533114 PHASE_III COMPLETED
Double-blind, placebo-controlled, randomised-withdrawal study of low-sodium oxybate (JZP-258) in idiopathic hypersomnia with an open-label safety extension; the trial supporting the 2021 United States approval.
Target Phenotypes: Excessive daytime somnolence HP:0001262 Human Phenotype Ontology (HP) Relation: this clinical trial targets this phenotype This clinical trial targets Excessive daytime somnolence (HP:0001262). HP:0001262 is a phenotype from the Human Phenotype Ontology. Sleep drunkenness HP:6000456 Human Phenotype Ontology (HP) Relation: this clinical trial targets this phenotype This clinical trial targets Sleep drunkenness, annotated with Sleep drunkeness (HP:6000456). HP:6000456 is a phenotype from the Human Phenotype Ontology.
Show evidence (1 reference)
PMID:37409509 SUPPORT Human Clinical
"To evaluate 6-month efficacy and safety of low-sodium oxybate in people with idiopathic hypersomnia during an open-label extension period (OLE) of a phase 3 clinical trial."
Identifies the phase 3 trial and its open-label extension, the source of the efficacy evidence curated under treatments.
{ }

Source YAML

click to show
name: Idiopathic Hypersomnia
creation_date: "2026-08-24T00:00:00Z"
category: Neurological Disorder
parents:
- Sleep Disorder
- Neurological Disease
disease_term:
  preferred_term: idiopathic hypersomnia
  term:
    id: MONDO:0018044
    label: idiopathic hypersomnia
description: >-
  Idiopathic hypersomnia is a central disorder of hypersomnolence defined by
  irrepressible daytime sleepiness that persists despite normal or long
  nocturnal sleep, in the absence of any identifiable cause. It is a diagnosis
  of exclusion, and unusually among the entries in this knowledge base its
  mechanism is genuinely unknown - the disorder is named for that ignorance.
  What distinguishes it clinically is not the sleepiness, which it shares with
  every other hypersomnolence disorder, but its quality: sleep is prolonged and
  unrefreshing rather than fragmented, naps are long and non-restorative rather
  than brief and restorative as in narcolepsy, and waking is dominated by severe
  and prolonged sleep inertia (sleep drunkenness) - a difficulty in achieving
  full alertness after waking that patients frequently rate as more disabling
  than the sleepiness itself. Cerebrospinal fluid hypocretin-1 is normal by
  definition, which excludes the orexin-deficiency mechanism of narcolepsy type
  1 and makes idiopathic hypersomnia the principal negative case against that
  module. The leading positive hypothesis runs in the opposite direction - a
  substance in cerebrospinal fluid that potentiates GABA-A receptor signalling,
  i.e. excessive somnogenic inhibition rather than deficient arousal drive - but
  that finding has not been robustly replicated and the entry curates it as an
  unconfirmed hypothesis rather than as the mechanism.
notes: >-
  Two curatorial decisions in this entry are deliberate and should be preserved.

  (1) NO CONFORMANCE TO orexin_arousal_instability. Idiopathic hypersomnia is
  the module's stated negative boundary: hypersomnolence alone does not license
  conformance, and these patients have normal CSF hypocretin-1 as a diagnostic
  requirement. The module is referenced here only for contrast.

  (2) THE MECHANISM IS CURATED AS UNKNOWN, with a named candidate. The
  pathophysiology chain below is built around what is actually established -
  hypersomnolence with preserved and often extended sleep, and severe sleep
  inertia - with the GABA-A potentiation finding attached as an EMERGING
  mechanistic hypothesis carrying its own replication gap, not as the trigger
  node. Resist the temptation to promote it: it rests substantially on a single
  laboratory's assay, the identity of the bioactive substance was never
  established, and the flumazenil response that supported it was open-label in
  seven patients.
has_subtypes:
- name: IH with long sleep time
  display_name: Idiopathic hypersomnia with long sleep time
  description: >-
    Habitual nocturnal sleep of 10 hours or more (ICSD-2 criterion), typically
    with very severe sleep inertia and unrefreshing long naps. Retained here as
    a named subtype because it was a formal ICSD-2 category and because
    treatment trials have been stratified by it, but see the discussion on
    subtype validity: ICSD-3 abandoned the split.
  evidence:
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      the description of idiopathic hypersomnia with two forms, polysymptomatic
      and monosymptomatic, by the same Bedrich Roth in 1976
    explanation: >-
      Documents the historical two-form division from which the long-sleep and
      non-long-sleep subtypes descend.
- name: IH without long sleep time
  display_name: Idiopathic hypersomnia without long sleep time
  description: >-
    Nocturnal sleep of normal duration with a mean sleep latency of 8 minutes or
    less on the multiple sleep latency test and fewer than two sleep-onset REM
    periods. This is the subtype in which the two randomised modafinil trials
    were conducted, and the subtype whose boundary with narcolepsy type 2 is
    least secure.
  evidence:
  - reference: PMID:33631500
    reference_title: "Efficacy and safety of modafinil in patients with idiopathic hypersomnia without long sleep time: a multicenter, randomized, double-blind, placebo-controlled, parallel-group comparison study."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Modafinil, an established treatment for narcolepsy, was tested for efficacy
      and safety in Japanese patients with IH without long sleep time.
    explanation: >-
      Confirms this subtype as a distinct trial population, which is the main
      operational reason the split is retained here.
pathophysiology:
- name: Unidentified Somnogenic Process
  description: >-
    The trigger node, and it is deliberately named for what is not known. No
    structural lesion, neurotransmitter deficiency, autoimmune target, or
    causative gene has been established in idiopathic hypersomnia. What is
    established is negative and constraining: CSF hypocretin-1 is normal, which
    excludes the orexin-deficiency mechanism; there is no HLA association of the
    strength seen in narcolepsy type 1; and no consistent neuropathology has
    been reported. Onset is typically in adolescence or young adulthood, a
    familial background is frequently reported but has never been rigorously
    characterised, and no biological marker exists. Curating this node as
    unknown rather than assigning it a plausible mechanism is the honest
    position and is what the disorder's name records.
  role: trigger
  biological_scale: ORGANISM
  evidence:
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Yet, MSLT is neither sensitive nor specific and the polysomnographic
      diagnostic criteria require continuous readjustment and biologic markers
      are still lacking.
    explanation: >-
      States the absence of a biological marker directly, which is the evidence
      for curating this node as an unidentified process rather than assigning it
      a mechanism.
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      A familial background is often present but rigorous studies are still
      lacking.
    explanation: >-
      Records the familial signal and, in the same sentence, that it has not
      been characterised - so this entry curates no inheritance mechanism.
  - reference: PMID:12374492
    reference_title: The role of cerebrospinal fluid hypocretin measurement in the diagnosis of narcolepsy and other hypersomnias.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Healthy controls and subjects with other sleep disorders all had normal levels.
    explanation: >-
      Establishes the key negative constraint on this node - normal CSF
      hypocretin-1 in hypersomnias other than narcolepsy-cataplexy - which is
      why this entry does not conform to orexin_arousal_instability.
  downstream:
  - target: Increased Homeostatic Sleep Drive with Preserved Sleep Continuity
    description: >-
      Whatever the somnogenic process is, its measurable effect is an increase in
      sleep propensity that does not degrade sleep quality or continuity.
    causal_link_type: INDIRECT_UNKNOWN_INTERMEDIATES
  - target: Impaired Arousal from Sleep (Sleep Inertia)
    description: >-
      The same unidentified process is presumed to underlie the difficulty in
      transitioning out of sleep, which does not follow from sleep drive alone.
    causal_link_type: INDIRECT_UNKNOWN_INTERMEDIATES
    hypothesis_groups:
    - gabaergic_potentiation

- name: Increased Homeostatic Sleep Drive with Preserved Sleep Continuity
  description: >-
    The central and defining physiological abnormality: sleep propensity is
    pathologically elevated, but sleep architecture, efficiency, and continuity
    are normal or better than normal. This combination is what separates
    idiopathic hypersomnia from every other cause of daytime sleepiness. In
    narcolepsy type 1 the sleep-wake switch is unstable in both directions, so
    nocturnal sleep is fragmented; in sleep-disordered breathing it is destroyed
    by respiratory events; in circadian disorders it is displaced in time. Here
    sleep is abundant, consolidated, and still insufficient. Sleep is extended
    rather than curtailed, and the extension does not relieve the sleepiness -
    which is the observation that makes a simple sleep-debt model untenable.
  role: central_effector
  biological_scale: ORGANISM
  biological_processes:
  - preferred_term: sleep
    term:
      id: GO:0030431
      label: sleep
    modifier: INCREASED
  - preferred_term: regulation of the sleep/wake cycle
    term:
      id: GO:0042749
      label: regulation of circadian sleep/wake cycle
    modifier: ABNORMAL
  evidence:
  - reference: PMID:36921459
    reference_title: "Update on the treatment of idiopathic hypersomnia: Progress, challenges, and expert opinion."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Idiopathic hypersomnia is a central hypersomnolence disorder of unknown
      origin characterized by excessive daytime sleepiness despite normal or
      long sleep time, and frequent severe sleep inertia.
    explanation: >-
      States both halves of this node - pathological sleepiness coexisting with
      normal or increased sleep - which is the combination that defines the
      disorder.
  - reference: PMID:38165322
    reference_title: Prevalence and Course of Idiopathic Hypersomnia in the Wisconsin Sleep Cohort Study.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      cases with IH had more severe sleepiness and sleep propensity, despite
      similar or longer sleep times
    explanation: >-
      Population-cohort confirmation with objective polysomnography and multiple
      sleep latency testing that the sleepiness is not explained by reduced
      sleep, which is what the node asserts.
  downstream:
  - target: Non-Restorative Long Sleep and Unrefreshing Naps
    description: >-
      Elevated sleep drive that is not discharged by sleep produces prolonged
      sleep episodes and long naps from which the patient does not wake refreshed.
    causal_link_type: DIRECT

- name: Impaired Arousal from Sleep (Sleep Inertia)
  description: >-
    A partly separable abnormality, and one that a pure sleep-drive model does
    not predict. Waking is prolonged, confused, and incomplete: patients are
    difficult to rouse, may require multiple alarms or another person, and remain
    impaired for tens of minutes to hours after rising - the "sleep drunkenness"
    Roth described in the original 1956 account. It is curated as its own node
    because it dissociates from sleepiness in treatment response as well as in
    phenomenology, and because it, rather than the sleepiness, is often the most
    disabling symptom and the one that most reliably distinguishes idiopathic
    hypersomnia from narcolepsy at the bedside.
  role: effector
  biological_scale: ORGANISM
  biological_processes:
  - preferred_term: sleep/wake cycle state transition to wakefulness
    term:
      id: GO:0022410
      label: circadian sleep/wake cycle process
    modifier: ABNORMAL
  evidence:
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      It is often accompanied by sleep of long duration and debilitating sleep
      inertia.
    explanation: >-
      Names sleep inertia as a defining accompaniment of the disorder rather
      than an incidental feature, supporting its curation as a separate node.
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Idiopathic hypersomnia continues to evolve from the concept of "sleep
      drunkenness" introduced by Bedrich Roth in Prague in 1956
    explanation: >-
      Documents that the arousal abnormality, not the sleepiness, was the
      founding observation of the disease concept.
  downstream:
  - target: Chronic Functional Impairment
    description: >-
      Prolonged post-waking impairment interferes with employment, education,
      and driving independently of the daytime sleepiness.
    causal_link_type: DIRECT

- name: Non-Restorative Long Sleep and Unrefreshing Naps
  description: >-
    Sleep in idiopathic hypersomnia fails at its restorative function while
    succeeding at its structural one. Nocturnal sleep may extend well beyond ten
    hours, and daytime naps - which in narcolepsy are short and briefly
    restorative - are here long and leave the patient no better. This is the
    feature clinicians use to separate the two disorders in the consulting room,
    and it constrains any candidate mechanism: whatever is wrong is not a failure
    to obtain sleep but a failure of obtained sleep to discharge sleep pressure.
  role: effector
  biological_scale: ORGANISM
  biological_processes:
  - preferred_term: sleep
    term:
      id: GO:0030431
      label: sleep
    modifier: ABNORMAL
  evidence:
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      as well as a 24 h PSG or a 2-wk actigraphy in association with a sleep log
      to ensure a total 24-h sleep time longer than or equal to 66O minutes
    explanation: >-
      Gives the operational threshold (a 24-hour sleep time of at least 660
      minutes) by which extended sleep is established, which is how this node is
      measured. Note the source text contains a typographic error, printing the
      letter O for the final zero of "660"; the snippet reproduces the cached
      text verbatim as required and the correct value is 660 minutes.
  downstream:
  - target: Chronic Functional Impairment
    description: >-
      Long unrefreshing sleep consumes the day and does not relieve the
      sleepiness, compounding disability.
    causal_link_type: DIRECT

- name: Chronic Functional Impairment
  description: >-
    The clinical endpoint. Idiopathic hypersomnia is most often a chronic
    condition of adolescent or young-adult onset with substantial occupational,
    educational, and social disability - sometimes rated by patients as greater
    than that of narcolepsy, in part because the disorder is less recognised and
    its severity less readily believed. It is not, however, uniformly permanent:
    in the only population-based longitudinal series, pathological somnolence
    remitted in roughly 40% of cases over about twelve years, which is a
    prognostically important finding and one that any mechanistic account must
    accommodate.
  role: consequence
  biological_scale: ORGANISM
  evidence:
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      The condition is disabling, sometimes even more so than narcolepsy type 1
      or 2.
    explanation: >-
      Establishes the magnitude of disability relative to the better-recognised
      comparator disorder.
  - reference: PMID:38165322
    reference_title: Prevalence and Course of Idiopathic Hypersomnia in the Wisconsin Sleep Cohort Study.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Longitudinal data (spanning 12.1 ± 4.3 years) demonstrated a chronic course
      of sleepiness for most of the cases with IH, though pathologic somnolence
      remitted in roughly 40% of cases.
    explanation: >-
      Quantifies both the chronicity and the substantial remission rate, which
      together define the natural history recorded at this node.
mechanistic_hypotheses:
- hypothesis_group_id: gabaergic_potentiation
  hypothesis_label: Endogenous GABA-A Receptor Potentiation
  status: EMERGING
  description: >-
    The leading positive hypothesis, and the reason idiopathic hypersomnia is
    sometimes described as the mirror image of narcolepsy. Cerebrospinal fluid
    from hypersomnolent patients was reported to potentiate GABA-A receptor
    function in vitro in the presence of GABA, with the bioactivity attributable
    to a trypsin-sensitive component of 500-3000 daltons whose identity was never
    established. On this model the disorder is one of excessive endogenous
    somnogenic inhibition rather than deficient arousal drive - which would
    explain why it coexists with normal orexin, why sleep is abundant rather than
    fragmented, and why arousal from sleep is specifically impaired. The
    supporting flumazenil result is mechanistically striking, since flumazenil is
    conventionally regarded as lacking intrinsic activity, and it reversed the
    enhancement in vitro and normalised vigilance in an open-label series of
    seven patients. It is curated as EMERGING and not as the mechanism: the
    finding rests substantially on one laboratory's assay, the bioactive
    substance was never identified, and the clinical arm was small and unblinded.
  evidence:
  - reference: PMID:23175709
    reference_title: Modulation of vigilance in the primary hypersomnias by endogenous enhancement of GABAA receptors.
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: >-
      We conclude that a naturally occurring substance in CSF augments inhibitory
      GABA signaling, thus revealing a new pathophysiology associated with
      excessive daytime sleepiness.
    explanation: >-
      The primary claim of the hypothesis, stated by its originators, and the
      basis for curating it as a candidate mechanism.
  - reference: PMID:23175709
    reference_title: Modulation of vigilance in the primary hypersomnias by endogenous enhancement of GABAA receptors.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Furthermore, flumazenil normalized vigilance in seven hypersomnolent
      patients.
    explanation: >-
      Cited as PARTIAL because it is the clinical arm of the hypothesis and is
      an open-label observation in seven patients - suggestive, and explicitly
      not sufficient to establish the mechanism.
  - reference: PMID:23175709
    reference_title: Modulation of vigilance in the primary hypersomnias by endogenous enhancement of GABAA receptors.
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: >-
      The bioactive CSF component had a mass of 500 to 3000 daltons and was
      neutralized by trypsin.
    explanation: >-
      Records how far characterisation of the putative somnogen got - a mass
      range and protease sensitivity - and therefore that no molecular identity
      was established, which is the principal reason the hypothesis remains
      EMERGING.
phenotypes:
- category: Neurological
  name: Excessive Daytime Sleepiness
  description: >-
    Irrepressible daytime sleepiness present daily for at least three months,
    with a mean sleep latency of 8 minutes or less on the multiple sleep latency
    test or a documented 24-hour sleep time of at least 660 minutes. Unlike the
    sleep attacks of narcolepsy it is typically continuous rather than episodic.
  phenotype_term:
    preferred_term: Excessive daytime somnolence
    term:
      id: HP:0001262
      label: Excessive daytime somnolence
    temporality: CHRONIC
  frequency: OBLIGATE
  evidence:
  - reference: PMID:36921459
    reference_title: "Update on the treatment of idiopathic hypersomnia: Progress, challenges, and expert opinion."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Idiopathic hypersomnia is a central hypersomnolence disorder of unknown
      origin characterized by excessive daytime sleepiness despite normal or
      long sleep time, and frequent severe sleep inertia.
    explanation: >-
      Excessive daytime sleepiness is the defining diagnostic criterion, so the
      frequency band is OBLIGATE by definition rather than by observation.
- category: Neurological
  name: Severe Sleep Inertia (Sleep Drunkenness)
  description: >-
    Prolonged difficulty achieving full alertness on waking, with confusion,
    ataxia, irritability and automatic behaviour lasting from tens of minutes to
    hours. Frequently the most disabling symptom, and the historical founding
    observation of the disease concept.
  phenotype_term:
    preferred_term: Sleep drunkenness
    term:
      id: HP:6000456
      label: Sleep drunkeness
    severity: SEVERE
  frequency: FREQUENT
  evidence:
  - reference: PMID:36921459
    reference_title: "Update on the treatment of idiopathic hypersomnia: Progress, challenges, and expert opinion."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      characterized by excessive daytime sleepiness despite normal or long sleep
      time, and frequent severe sleep inertia
    explanation: >-
      Supports the FREQUENT band directly: the source characterises severe sleep
      inertia as frequent in the disorder, which maps to the FREQUENT enum tier
      rather than to an obligate or occasional one.
- category: Neurological
  name: Prolonged Nocturnal Sleep
  description: >-
    Habitual nocturnal sleep beyond normal duration, with a total 24-hour sleep
    time of 660 minutes or more in the long-sleep subtype, from which the patient
    still wakes unrefreshed.
  subtype: IH with long sleep time
  phenotype_term:
    preferred_term: Sleep disturbance
    term:
      id: HP:0002360
      label: Sleep disturbance
  evidence:
  - reference: PMID:38165322
    reference_title: Prevalence and Course of Idiopathic Hypersomnia in the Wisconsin Sleep Cohort Study.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      cases with IH had more severe sleepiness and sleep propensity, despite
      similar or longer sleep times
    explanation: >-
      Objective cohort evidence for preserved or extended sleep duration in this
      disorder; frequency deliberately omitted, since the source does not
      quantify how many patients meet the long-sleep threshold.
- category: Neurological
  name: Unrefreshing Long Naps
  description: >-
    Daytime naps that are long (often over an hour) and leave the patient no more
    alert - in explicit contrast to the short, briefly restorative naps
    characteristic of narcolepsy, which is the most useful bedside discriminator
    between the two disorders.
  phenotype_term:
    preferred_term: Excessive daytime somnolence
    term:
      id: HP:0001262
      label: Excessive daytime somnolence
  evidence:
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      The key manifestation is hypersomnolence.
    explanation: >-
      Supports hypersomnolence as the core manifestation this phenotype
      elaborates; the nap-quality contrast itself is stated in the description
      and not claimed from this snippet, and no frequency band is asserted.
diagnosis:
- name: Polysomnography followed by multiple sleep latency test
  description: >-
    Overnight polysomnography to exclude other causes of hypersomnolence
    (particularly sleep-disordered breathing and insufficient sleep), followed by
    a multiple sleep latency test demonstrating a mean sleep latency of 8 minutes
    or less with fewer than two sleep-onset REM periods. The two-SOREMP threshold
    is what separates idiopathic hypersomnia from narcolepsy type 2, and it is a
    weak boundary - see the discussion on entity validity.
  evidence:
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Polysomnography (PSG) followed by a multiple sleep latency test (MSLT) is
      mandatory
    explanation: >-
      Establishes the mandatory diagnostic sequence.
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Yet, MSLT is neither sensitive nor specific and the polysomnographic
      diagnostic criteria require continuous readjustment
    explanation: >-
      Cited as PARTIAL because it qualifies the same test it recommends: the
      diagnostic standard is mandatory but acknowledged to be neither sensitive
      nor specific, which is why this entry does not treat a positive MSLT as
      establishing a mechanism.
- name: Extended polysomnography or actigraphy with sleep log
  description: >-
    A 24-hour polysomnogram, or two weeks of actigraphy with a concurrent sleep
    log, to document a total 24-hour sleep time of at least 660 minutes. This is
    the alternative diagnostic route when the mean sleep latency exceeds 8
    minutes, and it is the route that captures the long-sleep presentation, which
    the multiple sleep latency test systematically misses.
  diagnosis_term:
    preferred_term: actigraphy
    term:
      id: NCIT:C180883
      label: Actigraphy
  evidence:
  - reference: PMID:26599679
    reference_title: Idiopathic hypersomnia.
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      as well as a 24 h PSG or a 2-wk actigraphy in association with a sleep log
      to ensure a total 24-h sleep time longer than or equal to 66O minutes, when
      the mean sleep latency on the MSLT is longer than 8 min
    explanation: >-
      Specifies the alternative diagnostic pathway and the circumstance in which
      it applies. The cached source prints the letter O for the final zero of
      "660"; the snippet is reproduced verbatim as required and the intended
      threshold is 660 minutes.
- name: Cerebrospinal fluid hypocretin-1 measurement
  description: >-
    Not required for diagnosis, but decisive where narcolepsy type 1 is in the
    differential: a normal CSF hypocretin-1 level is expected in idiopathic
    hypersomnia and a level below 110 pg/mL reclassifies the patient as
    narcolepsy type 1 regardless of the presence or absence of cataplexy.
  evidence:
  - reference: PMID:12374492
    reference_title: The role of cerebrospinal fluid hypocretin measurement in the diagnosis of narcolepsy and other hypersomnias.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Hypocretin-1 levels below 110 pg/mL were diagnostic for narcolepsy.
    explanation: >-
      Gives the threshold that excludes this diagnosis, which is the reason the
      test is listed here despite not being a positive criterion.
  - reference: PMID:12374492
    reference_title: The role of cerebrospinal fluid hypocretin measurement in the diagnosis of narcolepsy and other hypersomnias.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Ten subjects with hypersomnia had intermediate levels
    explanation: >-
      Cited as PARTIAL because it records the boundary case the clean threshold
      obscures: a minority of hypersomnolent patients fall in the intermediate
      range and are classified by neither rule.
treatments:
- name: Modafinil
  description: >-
    A wake-promoting agent and the most commonly used treatment, prescribed
    off-label in most jurisdictions since its European idiopathic hypersomnia
    indication was withdrawn in 2011. It improves objective and subjective
    sleepiness but is not directed at sleep inertia, which is the symptom
    patients most often rate as most disabling - a therapeutic gap this entry
    records deliberately.
  therapeutic_modality: SMALL_MOLECULE
  treatment_term:
    preferred_term: Pharmacotherapy
    term:
      id: NCIT:C15986
      label: Pharmacotherapy
    therapeutic_agent:
    - preferred_term: modafinil
      term:
        id: CHEBI:31859
        label: modafinil
  target_mechanisms:
  - target: Increased Homeostatic Sleep Drive with Preserved Sleep Continuity
    treatment_effect: INHIBITS
    description: >-
      Wake promotion opposes the elevated sleep propensity symptomatically; it
      does not act on the unidentified upstream process and is not
      disease-modifying.
  evidence:
  - reference: PMID:33631500
    reference_title: "Efficacy and safety of modafinil in patients with idiopathic hypersomnia without long sleep time: a multicenter, randomized, double-blind, placebo-controlled, parallel-group comparison study."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Patients treated with modafinil experienced a significantly prolonged mean
      sleep latency on the MWT at the end of the study compared with placebo
    explanation: >-
      Randomised placebo-controlled evidence of objective benefit on daytime
      wakefulness in the subtype studied.
  - reference: PMID:36921459
    reference_title: "Update on the treatment of idiopathic hypersomnia: Progress, challenges, and expert opinion."
    supports: SUPPORT
    evidence_source: OTHER
    snippet: >-
      Modafinil, which was approved for idiopathic hypersomnia until 2011 in
      Europe, is the most commonly used treatment and improved sleepiness in two
      recent randomized placebo-controlled trials.
    explanation: >-
      Establishes modafinil's place in practice and that two randomised trials
      support it, while recording its off-label regulatory status.
- name: Low-Sodium Oxybate
  description: >-
    The only agent approved specifically for idiopathic hypersomnia (United
    States, 2021). Notable mechanistically as well as clinically, because it
    reduces sleep inertia as well as daytime sleepiness - the only treatment
    shown to act on both nodes - and because a GABA-B agonist improving a
    disorder hypothesised to involve excessive GABA-A potentiation is a genuine
    puzzle that the entry's mechanistic hypothesis does not resolve.
  therapeutic_modality: SMALL_MOLECULE
  treatment_term:
    preferred_term: Pharmacotherapy
    term:
      id: NCIT:C15986
      label: Pharmacotherapy
    therapeutic_agent:
    - preferred_term: sodium oxybate
      term:
        id: CHEBI:749298
        label: sodium oxybate
  target_mechanisms:
  - target: Impaired Arousal from Sleep (Sleep Inertia)
    treatment_effect: INHIBITS
    description: >-
      Reduces sleep inertia in randomised withdrawal, distinguishing it from the
      wake-promoting agents, which do not address this node.
  - target: Increased Homeostatic Sleep Drive with Preserved Sleep Continuity
    treatment_effect: INHIBITS
    description: >-
      Reduces daytime sleepiness alongside the effect on sleep inertia.
  evidence:
  - reference: PMID:36921459
    reference_title: "Update on the treatment of idiopathic hypersomnia: Progress, challenges, and expert opinion."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      In a placebo-controlled, double-blind, randomized withdrawal study, LXB
      reduced daytime sleepiness and sleep inertia, and improved daily
      functioning.
    explanation: >-
      Randomised-withdrawal evidence that the agent acts on both curated
      symptom nodes, which is what distinguishes it from the wake-promoting
      agents.
  - reference: PMID:37409509
    reference_title: "Long-term efficacy and safety of low-sodium oxybate in an open-label extension period of a placebo-controlled, double-blind, randomized withdrawal study in adults with idiopathic hypersomnia."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      Efficacy and safety of low-sodium oxybate were maintained or improved
      during the 6-month OLE, supporting long-term treatment with low-sodium
      oxybate in adults with idiopathic hypersomnia.
    explanation: >-
      Cited as PARTIAL because the durability claim comes from an open-label
      extension without a control arm, so it supports persistence of benefit
      more weakly than the randomised-withdrawal result above.
clinical_trials:
- name: NCT03533114
  phase: PHASE_III
  status: COMPLETED
  description: >-
    Double-blind, placebo-controlled, randomised-withdrawal study of low-sodium
    oxybate (JZP-258) in idiopathic hypersomnia with an open-label safety
    extension; the trial supporting the 2021 United States approval.
  target_phenotypes:
  - preferred_term: Excessive daytime somnolence
    term:
      id: HP:0001262
      label: Excessive daytime somnolence
  - preferred_term: Sleep drunkenness
    term:
      id: HP:6000456
      label: Sleep drunkeness
  evidence:
  - reference: PMID:37409509
    reference_title: "Long-term efficacy and safety of low-sodium oxybate in an open-label extension period of a placebo-controlled, double-blind, randomized withdrawal study in adults with idiopathic hypersomnia."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      To evaluate 6-month efficacy and safety of low-sodium oxybate in people
      with idiopathic hypersomnia during an open-label extension period (OLE) of
      a phase 3 clinical trial.
    explanation: >-
      Identifies the phase 3 trial and its open-label extension, the source of
      the efficacy evidence curated under treatments.
prevalence:
- population: Wisconsin Sleep Cohort, working-age adults with polysomnography and MSLT data
  measure_type: POINT_PREVALENCE
  prevalence_class: ABOVE_1_IN_1000
  rate_per_100000: 1500.0
  rate_low: 700.0
  rate_high: 2500.0
  notes: >-
    The first population-based estimate; 12 probable cases among 792 participants
    with objective sleep testing. Substantially higher than clinic-derived
    impressions of the disorder's rarity, and the authors note this explicitly.
    Read with care - "probable IH" was ascertained by applying criteria to an
    existing cohort rather than by clinical diagnosis, so this is a prevalence of
    the phenotype meeting criteria rather than of the diagnosed disease.
  evidence:
  - reference: PMID:38165322
    reference_title: Prevalence and Course of Idiopathic Hypersomnia in the Wisconsin Sleep Cohort Study.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      From 792 cohort study participants with available polysomnography and
      multiple sleep latency test data, 12 cases with probable IH were identified
      resulting in an estimated prevalence of IH of 1.5%
    explanation: >-
      Gives the numerator, denominator, and resulting rate directly.
  - reference: PMID:38165322
    reference_title: Prevalence and Course of Idiopathic Hypersomnia in the Wisconsin Sleep Cohort Study.
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: >-
      These results demonstrate IH is more common in the working population than
      generally assumed
    explanation: >-
      Records the authors' own framing of the estimate against prior
      assumptions, which is why the note flags the ascertainment caveat.
classifications:
  harrisons_chapter:
  - classification_value: NEUROLOGIC
    evidence:
    - reference: PMID:36921459
      reference_title: "Update on the treatment of idiopathic hypersomnia: Progress, challenges, and expert opinion."
      supports: SUPPORT
      evidence_source: OTHER
      snippet: >-
        Idiopathic hypersomnia is a central hypersomnolence disorder of unknown
        origin

      explanation: >-
        A central (i.e. central nervous system) disorder of hypersomnolence,
        classified under the nervous-system chapter as the closest available
        category; the schema has no dedicated sleep chapter.
discussions:
- discussion_id: gap_ih_versus_narcolepsy_type_2_entity_validity
  kind: KNOWLEDGE_GAP
  prompt: >-
    Are idiopathic hypersomnia and narcolepsy type 2 one disorder or two?
  attaches_to:
  - pathophysiology#Unidentified Somnogenic Process
  rationale: >-
    The two diagnoses are separated by a single polysomnographic count - two or
    more sleep-onset REM periods on the multiple sleep latency test - applied to
    a test its own reviewers describe as neither sensitive nor specific, and the
    count is unstable on repeat testing. Neither disorder has a biological
    marker, both have normal CSF hypocretin-1, and they respond to the same
    drugs. This is not a nosological quibble: it determines whether mechanistic
    studies of either are being conducted in a homogeneous population, and a
    negative replication in a mixed cohort would be uninterpretable. It is the
    principal reason this entry's trigger node is curated as unknown rather than
    inheriting a mechanism from narcolepsy research.
  proposed_experiments:
  - experiment_id: exp_repeat_mslt_classification_stability
    name: Stability of the SOREMP-based classification on repeat testing
    description: >-
      Serial multiple sleep latency testing at intervals in a cohort meeting
      criteria for either diagnosis, quantifying how often patients cross the
      two-SOREMP boundary between tests, and testing whether any clinical,
      biochemical, or genetic variable segregates with the assigned label
      independently of that count.
- discussion_id: gap_gaba_potentiation_replication
  kind: KNOWLEDGE_GAP
  prompt: >-
    Has the CSF GABA-A-potentiating bioactivity been independently replicated,
    and what is the substance?
  attaches_to:
  - pathophysiology#Impaired Arousal from Sleep (Sleep Inertia)
  rationale: >-
    The gabaergic_potentiation hypothesis is the only mechanistic account of
    idiopathic hypersomnia with direct experimental support, and it is curated
    here as EMERGING rather than canonical for two specific reasons that are
    resolvable rather than permanent. The bioactive substance was characterised
    only as a trypsin-sensitive species of 500-3000 daltons and has never been
    identified, so there is no assay a second laboratory can run on the analyte
    itself; and the supporting clinical observation was open-label in seven
    patients. Until both are addressed, a conforming or citing entry should not
    treat GABA-A potentiation as established.
  proposed_experiments:
  - experiment_id: exp_csf_somnogen_identification
    name: Molecular identification of the CSF bioactivity
    description: >-
      Fractionation and mass-spectrometric identification of the
      GABA-A-potentiating component of hypersomnolent CSF, followed by a blinded
      multi-site assay of the identified analyte in patients meeting criteria for
      idiopathic hypersomnia, narcolepsy type 2, and matched controls.
  - experiment_id: exp_flumazenil_randomised_trial
    name: Randomised placebo-controlled trial of flumazenil
    description: >-
      A blinded, placebo-controlled crossover trial of flumazenil in
      hypersomnolent patients selected by CSF bioactivity status, with objective
      vigilance and sleep-inertia endpoints, to test whether the open-label
      response survives control and whether it segregates with the biomarker.