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2
Mappings
0
Definitions
0
Inheritance
6
Pathophysiology
1
Histopathology
5
Phenotypes
7
Pathograph
2
Genes
4
Treatments
3
Subtypes
0
Differentials
0
Datasets
0
Trials
0
Models
🏷

Classifications

Harrison's Chapter
cancer solid tumor
ICD-O Morphology
Embryonal Neoplasm
🔗

Mappings

MONDO
MONDO:0002728 rhabdoid tumor
skos:exactMatch MONDO
MONDO provides an exact disease term for rhabdoid tumor.
NCIT
NCIT:C3808 Rhabdoid Tumor
skos:exactMatch NCIT
NCIT provides an exact neoplasm term for rhabdoid tumor; the same identifier is used in this entry's histopathology block.
NCIT
NCIT:C3808 Rhabdoid Tumor
skos:exactMatch NCIT
NCIT provides an exact neoplasm term for rhabdoid tumor; the same identifier is used in this entry's histopathology block.

Subtypes

3
Rhabdoid Tumor of the Kidney
Renal rhabdoid tumor is the kidney-predominant form of the rhabdoid tumor spectrum, typically presenting in infancy as a rapidly progressive renal mass with a high risk of metastatic dissemination.
Show evidence (1 reference)
PMID:33249395 SUPPORT
"A total of 70 patients presented with extracranial, extrarenal tumours (eMRT) and 30 with renal rhabdoid tumours (RTK)."
EU-RHAB registry data directly identifies renal rhabdoid tumor as a major extracranial subtype within the rhabdoid tumor spectrum.
Extrarenal Malignant Rhabdoid Tumor
Extrarenal malignant rhabdoid tumor arises outside the kidney and CNS, usually in deep axial soft tissues such as the neck or paraspinal region.
Show evidence (1 reference)
PMID:16704491 SUPPORT
"Malignant rhabdoid tumor (MRT) of the soft tissue is a rare and highly aggressive tumor that occurs in infancy or childhood. It predominantly involves a deep axial location such as the neck or paraspinal region."
This review defines the extracranial soft-tissue subtype and its typical deep axial distribution.
Atypical Teratoid/Rhabdoid Tumor
Atypical teratoid/rhabdoid tumor is the CNS presentation of rhabdoid tumor, usually arising in infancy or early childhood and sharing the same SMARCB1 or SMARCA4-deficient biology as extracranial disease.
Show evidence (1 reference)
PMID:39088359 SUPPORT
"While the central nervous system is the most common site of occurrence, tumors can develop at other sites, including the kidneys and soft tissues throughout the body."
This review supports AT/RT as the CNS arm of the shared rhabdoid tumor spectrum.

Pathophysiology

6
SMARCB1 or SMARCA4 Loss
Biallelic loss of SMARCB1, and rarely SMARCA4 loss, disables the core BAF/SWI-SNF chromatin-remodeling machinery and unifies renal, soft-tissue, and CNS rhabdoid tumors as one molecular disease spectrum.
BAF complex link
chromatin remodeling link ⚠ ABNORMAL
Show evidence (2 references)
PMID:39088359 SUPPORT
"Irrespective of the site of occurrence, somatic and germline mutations in SMARCB1, and rarely in SMARCA4, underlie the entire spectrum of rhabdoid tumors."
This review directly supports SMARCB1 loss, with rare SMARCA4 loss, as the shared defining lesion across rhabdoid tumor sites.
PMID:23432645 SUPPORT
"SMARCB1 is a core subunit of the SWI/SNF chromatin remodeling complex"
This mechanistic review links the defining driver gene to the BAF/SWI-SNF complex disrupted in rhabdoid tumor.
EZH2-Driven H3K27 Methylation
SMARCB1 loss creates functional dependence on PRC2/EZH2, increasing H3K27 methylation and epigenetic gene repression that helps sustain the malignant state.
negative regulation of gene expression, epigenetic link ↑ INCREASED
histone H3K27 methyltransferase activity link ↑ INCREASED
Show evidence (1 reference)
PMID:36212776 SUPPORT In Vitro
"MRTs are characterized by loss of SMARCB1, which results in upregulated expression of enhancer of zeste homolog 2 (EZH2), which is responsible for the methylation of lysine 27 of histone H3 (H3K27me3), leading to the repression of gene expression."
This study directly supports the mechanistic link between SMARCB1 loss, EZH2 upregulation, H3K27 methylation, and epigenetic repression.
CDKN2A/p16 Epigenetic Silencing
SMARCB1 loss removes its direct transcriptional support for CDKN2A/p16(INK4a) expression, while unopposed PRC2/EZH2 activity further enforces epigenetic silencing of the locus. The net effect is loss of p16(INK4a) protein, which normally restrains CDK4-Cyclin D1 kinase activity.
CDKN2A/p16(INK4a) epigenetic silencing link ↑ INCREASED
Show evidence (1 reference)
PMID:14604992 SUPPORT In Vitro
"Following hSNF5 expression, we observed transcriptional activation of the tumor suppressor p16(INK4a) but not of p14(ARF), repression of several cyclins and CD44, a cell surface glycoprotein implicated in metastasis."
Restoring SMARCB1/hSNF5 in MRT cells transcriptionally activates p16(INK4a); SMARCB1 loss therefore silences p16(INK4a) — the single event this node captures.
CDK4-Cyclin D1 Hyperactivation and G1-S Bypass
Without p16(INK4a) restraint, CDK4-Cyclin D1 kinase activity is left unopposed, driving RB1 phosphorylation and inactivation, and bypassing the G1-S restriction point. Cyclin D1 is the critical downstream effector: its genetic ablation fully abrogates rhabdoid tumorigenesis caused by SMARCB1/Ini1 loss in vivo.
G1-S restriction-point bypass link ↑ INCREASED
CDK4-Cyclin D1 kinase activity link ↑ INCREASED
Show evidence (3 references)
PMID:14604992 SUPPORT In Vitro
"Expression of a p16(INK4a)-insensitive form of CDK4 obstructs hSNF5-induced cell cycle arrest."
A p16(INK4a)-insensitive CDK4 bypasses SMARCB1-induced arrest, establishing CDK4 as the effector kinase that drives proliferation when p16(INK4a) is lost.
PMID:12226744 SUPPORT In Vitro
"This arrest can be reverted by coexpression of cyclin D1, cyclin E or viral E1A, whereas it cannot be counteracted by pRB-binding deficient E1A mutants."
SMARCB1/INI1-induced G1 arrest is reversed by Cyclin D1 and requires functional pRB, placing the Cyclin D1-CDK4-RB1 axis directly downstream of SMARCB1 loss.
PMID:16099835 SUPPORT Model Organism
"We crossed Ini1+/- mice to Cyclin D1-/- mice and found that Ini1+/- mice with Cyclin D1 deficiency did not develop any spontaneous tumors, in contrast to the parental Ini1+/- mice."
In vivo genetic proof that Cyclin D1 is the critical effector downstream of SMARCB1/Ini1 loss — Cyclin D1 deficiency fully abrogates rhabdoid tumorigenesis.
Aberrant MRT Cell Growth
EZH1 and EZH2 activity sustains malignant rhabdoid tumor cell growth, helping maintain the aggressive proliferative phenotype downstream of SMARCB1 loss.
cell population proliferation link ↑ INCREASED
Show evidence (1 reference)
PMID:36212776 SUPPORT In Vitro
"Here, we show that EZH1, as well as EZH2, contributes to MRT cell growth and H3K27 methylation."
This study directly supports EZH-dependent malignant rhabdoid tumor cell growth downstream of the repressive chromatin state.
Blocked Cellular Differentiation
SMARCB1-deficient rhabdoid tumor cells are maintained in an undifferentiated state, and EZH2 inhibition can trigger differentiation.
cell differentiation link ↓ DECREASED
Show evidence (1 reference)
PMID:23620515 SUPPORT In Vitro
"The compound induces apoptosis and differentiation specifically in SMARCB1-deleted MRT cells."
Induction of differentiation after EZH2 inhibition supports a baseline differentiation block in SMARCB1-deleted rhabdoid tumor cells.

Histopathology

1
Rhabdoid Cell Morphology
Classic rhabdoid tumors are composed of poorly differentiated cells with eccentric nuclei, prominent nucleoli, and glassy eosinophilic cytoplasm with hyaline inclusions.
Show evidence (1 reference)
PMID:16704491 SUPPORT
"Microscopically, the tumor is composed of a diffuse proliferation of rounded or polygonal cells with eccentric nuclei, prominent nucleoli and glassy eosinophilic cytoplasm containing hyaline-like inclusion bodies, arranged in sheets and nests."
This pathology review describes the defining rhabdoid cell morphology.

Pathograph

Use the checkboxes to hide or show graph categories. Hover nodes for evidence and cross-linked metadata.
Pathograph: causal mechanism network for Rhabdoid Tumor 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

5
Digestive 2
Abdominal Mass Abdominal mass (HP:0031500)
Show evidence (1 reference)
PMID:8732342 SUPPORT Human Clinical
"Clinically, they presented with an abdominal mass but four (18%) children also had hypercalcaemia"
SIOP clinicopathological series of 22 children with rhabdoid tumour of the kidney documents abdominal mass as the dominant presenting finding.
Vomiting Vomiting (HP:0002013)
Show evidence (1 reference)
PMID:27307151 SUPPORT Human Clinical
"Patient 2, a 26-month-old girl, presented with headache and vomiting"
Same AT/RT case series documents vomiting accompanying headache, consistent with raised intracranial pressure from posterior third ventricular AT/RT.
Genitourinary 1
Hematuria Hematuria (HP:0000790)
Show evidence (1 reference)
PMID:11216700 SUPPORT Human Clinical
"Either gross or microscopic hematuria was present in 84.4% (27/32) of the patients with RTK."
National Wilms Tumor Study Group review of 50 RTK patients shows hematuria is a near-universal presenting feature, supporting its inclusion as a characteristic phenotype of renal rhabdoid tumor.
Nervous System 1
Headache Headache (HP:0002315)
Show evidence (1 reference)
PMID:27307151 SUPPORT Human Clinical
"Patient 1, a 23-month-old girl, presented with a history of gait unsteadiness and headache"
Case series of two children with intracranial AT/RT in the pineal/third ventricular region documents headache as a presenting symptom consistent with intracranial mass effect.
Neoplasm 1
Soft Tissue Mass Soft tissue neoplasm (HP:0031459)
Show evidence (1 reference)
PMID:36720509 SUPPORT Human Clinical
"We report one such rare case in a female infant presenting as left axillary mass."
Case report of extrarenal rhabdoid tumour presenting as an axillary soft-tissue mass in an infant illustrates the typical eMRT presentation as a deep soft-tissue mass.
🧬

Genetic Associations

2
SMARCB1 (Somatic/Germline Loss-of-Function)
Show evidence (2 references)
PMID:39088359 SUPPORT
"Irrespective of the site of occurrence, somatic and germline mutations in SMARCB1, and rarely in SMARCA4, underlie the entire spectrum of rhabdoid tumors."
This review supports SMARCB1 as the principal shared somatic and germline driver across renal, extrarenal, and CNS rhabdoid tumors.
PMID:23432645 SUPPORT
"Indeed, the mutation rate in RTs is among the lowest of all cancers sequenced, with loss of SMARCB1 as essentially the sole recurrent event."
This review emphasizes how dominant SMARCB1 loss is in the otherwise genetically simple rhabdoid tumor genome.
SMARCA4 (Rare Somatic/Germline Loss-of-Function)
Show evidence (1 reference)
PMID:39088359 SUPPORT
"Irrespective of the site of occurrence, somatic and germline mutations in SMARCB1, and rarely in SMARCA4, underlie the entire spectrum of rhabdoid tumors."
This review supports rare but disease-defining SMARCA4-driven rhabdoid tumors alongside the more common SMARCB1-deficient cases.
💊

Treatments

4
Multi-Agent Chemotherapy
Action: chemotherapy MAXO:0000647
Intensive multi-agent chemotherapy remains part of front-line treatment for most newly diagnosed patients, usually combined with surgery and local radiotherapy according to age, site, and resectability.
Gross Total Resection
Action: surgical procedure MAXO:0000004
Maximal safe resection is pursued whenever anatomically feasible because gross total resection is one of the strongest favorable prognostic factors in extracranial disease.
Show evidence (1 reference)
PMID:33249395 SUPPORT
"On univariate analyses, age at diagnosis (≥12 months), M0-stage, absence of synchronous tumours, absence of a GLM, gross total resection (GTR), radiotherapy and achieving a CR were significantly associated with favourable outcomes."
EU-RHAB registry data identifies gross total resection as a favorable prognostic factor in extracranial rhabdoid tumor.
Radiation Therapy
Action: radiation therapy MAXO:0000014
Radiotherapy is used for local control, especially after incomplete resection or in high-risk disease, and contributes to outcome in multimodality therapy.
Show evidence (1 reference)
PMID:33249395 SUPPORT
"On univariate analyses, age at diagnosis (≥12 months), M0-stage, absence of synchronous tumours, absence of a GLM, gross total resection (GTR), radiotherapy and achieving a CR were significantly associated with favourable outcomes."
The same EU-RHAB analysis supports radiotherapy as part of the treatment factors associated with improved extracranial outcomes.
EZH2 Inhibitor Therapy
Action: pharmacotherapy MAXO:0000058
Agent: tazemetostat
EZH2-targeted therapy is an investigational mechanism-based strategy for relapsed or refractory SMARCB1/SMARCA4-deficient disease. Preclinical data support strong EZH2 dependence, and early pediatric trials have tested tazemetostat in molecularly selected tumors.
Mechanism Target:
INHIBITS EZH2-Driven H3K27 Methylation — Tazemetostat selectively inhibits EZH2 methyltransferase activity, blocking the H3K27 methylation dependency that SMARCB1-deficient rhabdoid tumors acquire when BAF-mediated antagonism of PRC2 is lost.
Show evidence (2 references)
PMID:23620515 SUPPORT Model Organism
"These data demonstrate the dependency of SMARCB1 mutant MRTs on EZH2 enzymatic activity and portend the utility of EZH2-targeted drugs for the treatment of these genetically defined cancers."
Preclinical MRT models provide direct proof that EZH2 is a targetable dependency downstream of SMARCB1 loss.
PMID:37228094 PARTIAL
"Patients whose tumors harbored EZH2 mutations or loss of SMARCB1 or SMARCA4 by immunohistochemistry were treated with EZH2 inhibitor tazemetostat."
This pediatric MATCH trial shows that EZH2 inhibition has been clinically tested in SMARCB1/SMARCA4-deficient pediatric tumors, including rhabdoid tumors, although efficacy was limited.
🔬

Biochemical Markers

2
SMARCB1 (INI1/BAF47) Immunohistochemistry
SMARCA4 (BRG1) Immunohistochemistry
{ }

Source YAML

click to show
name: Rhabdoid Tumor
creation_date: '2026-04-12T05:17:45Z'
updated_date: '2026-05-17T14:00:00Z'
description: >-
  Rhabdoid tumor is an aggressive pediatric embryonal neoplasm spanning renal,
  extrarenal soft-tissue, and central nervous system sites. Across these anatomic
  presentations, the defining molecular lesion is loss of the BAF/SWI-SNF core
  subunits SMARCB1 and, rarely, SMARCA4. This shared chromatin-remodeling defect
  gives rise to a remarkably simple but highly lethal cancer spectrum, with
  especially poor outcomes in infants, patients with metastatic disease, or those
  with germline rhabdoid tumor predisposition syndromes.
categories:
- Pediatric Cancer
- Embryonal Neoplasm
- Molecularly Defined Tumor
- SWI/SNF-Deficient Tumor
has_subtypes:
- name: RTK
  display_name: Rhabdoid Tumor of the Kidney
  description: >-
    Renal rhabdoid tumor is the kidney-predominant form of the rhabdoid tumor
    spectrum, typically presenting in infancy as a rapidly progressive renal mass
    with a high risk of metastatic dissemination.
  evidence:
  - reference: PMID:33249395
    reference_title: Clinical and genetic risk factors define two risk groups of extracranial malignant rhabdoid tumours (eMRT/RTK).
    supports: SUPPORT
    snippet: "A total of 70 patients presented with extracranial, extrarenal tumours (eMRT) and 30 with renal rhabdoid tumours (RTK)."
    explanation: EU-RHAB registry data directly identifies renal rhabdoid tumor as a major extracranial subtype within the rhabdoid tumor spectrum.
- name: eMRT
  display_name: Extrarenal Malignant Rhabdoid Tumor
  description: >-
    Extrarenal malignant rhabdoid tumor arises outside the kidney and CNS,
    usually in deep axial soft tissues such as the neck or paraspinal region.
  evidence:
  - reference: PMID:16704491
    reference_title: "Extrarenal rhabdoid tumors of soft tissue: clinicopathological and molecular genetic review and distinction from other soft-tissue sarcomas with rhabdoid features."
    supports: SUPPORT
    snippet: "Malignant rhabdoid tumor (MRT) of the soft tissue is a rare and highly aggressive tumor that occurs in infancy or childhood. It predominantly involves a deep axial location such as the neck or paraspinal region."
    explanation: This review defines the extracranial soft-tissue subtype and its typical deep axial distribution.
- name: AT/RT
  display_name: Atypical Teratoid/Rhabdoid Tumor
  description: >-
    Atypical teratoid/rhabdoid tumor is the CNS presentation of rhabdoid tumor,
    usually arising in infancy or early childhood and sharing the same SMARCB1 or
    SMARCA4-deficient biology as extracranial disease.
  evidence:
  - reference: PMID:39088359
    reference_title: "Malignant Rhabdoid Tumor and Related Pediatric Tumors: Multimodality Imaging Review with Pathologic Correlation."
    supports: SUPPORT
    snippet: "While the central nervous system is the most common site of occurrence, tumors can develop at other sites, including the kidneys and soft tissues throughout the body."
    explanation: This review supports AT/RT as the CNS arm of the shared rhabdoid tumor spectrum.
pathophysiology:
- name: SMARCB1 or SMARCA4 Loss
  description: >-
    Biallelic loss of SMARCB1, and rarely SMARCA4 loss, disables the core
    BAF/SWI-SNF chromatin-remodeling machinery and unifies renal, soft-tissue,
    and CNS rhabdoid tumors as one molecular disease spectrum.
  protein_complexes:
  - preferred_term: BAF complex
    term:
      id: GO:0016514
      label: SWI/SNF complex
  biological_processes:
  - preferred_term: chromatin remodeling
    modifier: ABNORMAL
    term:
      id: GO:0006338
      label: chromatin remodeling
  downstream:
  - target: EZH2-Driven H3K27 Methylation
    description: Loss of BAF antagonism creates dependency on PRC2/EZH2 activity
  evidence:
  - reference: PMID:39088359
    reference_title: "Malignant Rhabdoid Tumor and Related Pediatric Tumors: Multimodality Imaging Review with Pathologic Correlation."
    supports: SUPPORT
    snippet: "Irrespective of the site of occurrence, somatic and germline mutations in SMARCB1, and rarely in SMARCA4, underlie the entire spectrum of rhabdoid tumors."
    explanation: This review directly supports SMARCB1 loss, with rare SMARCA4 loss, as the shared defining lesion across rhabdoid tumor sites.
  - reference: PMID:23432645
    reference_title: "Rhabdoid tumors: an initial clue to the role of chromatin remodeling in cancer."
    supports: SUPPORT
    snippet: "SMARCB1 is a core subunit of the SWI/SNF chromatin remodeling complex"
    explanation: This mechanistic review links the defining driver gene to the BAF/SWI-SNF complex disrupted in rhabdoid tumor.
- name: EZH2-Driven H3K27 Methylation
  description: >-
    SMARCB1 loss creates functional dependence on PRC2/EZH2, increasing H3K27
    methylation and epigenetic gene repression that helps sustain the malignant
    state.
  molecular_functions:
  - preferred_term: histone H3K27 methyltransferase activity
    modifier: INCREASED
    term:
      id: GO:0046976
      label: histone H3K27 methyltransferase activity
  biological_processes:
  - preferred_term: negative regulation of gene expression, epigenetic
    modifier: INCREASED
    term:
      id: GO:0045814
      label: negative regulation of gene expression, epigenetic
  downstream:
  - target: Aberrant MRT Cell Growth
    description: EZH1/2 activity supports continued MRT cell growth
  - target: Blocked Cellular Differentiation
    description: EZH2 dependence helps maintain an undifferentiated tumor state
  - target: CDKN2A/p16 Epigenetic Silencing
    description: Epigenetic repression by PRC2/EZH2 contributes to CDKN2A/p16(INK4a) silencing following SMARCB1 loss
  evidence:
  - reference: PMID:36212776
    reference_title: Dual targeting of EZH1 and EZH2 for the treatment of malignant rhabdoid tumors.
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: "MRTs are characterized by loss of SMARCB1, which results in upregulated expression of enhancer of zeste homolog 2 (EZH2), which is responsible for the methylation of lysine 27 of histone H3 (H3K27me3), leading to the repression of gene expression."
    explanation: This study directly supports the mechanistic link between SMARCB1 loss, EZH2 upregulation, H3K27 methylation, and epigenetic repression.
- name: CDKN2A/p16 Epigenetic Silencing
  description: >-
    SMARCB1 loss removes its direct transcriptional support for CDKN2A/p16(INK4a)
    expression, while unopposed PRC2/EZH2 activity further enforces epigenetic
    silencing of the locus. The net effect is loss of p16(INK4a) protein, which
    normally restrains CDK4-Cyclin D1 kinase activity.
  biological_processes:
  - preferred_term: CDKN2A/p16(INK4a) epigenetic silencing
    modifier: INCREASED
    term:
      id: GO:0045814
      label: negative regulation of gene expression, epigenetic
  downstream:
  - target: CDK4-Cyclin D1 Hyperactivation and G1-S Bypass
    description: Loss of p16(INK4a) restraint unleashes CDK4-Cyclin D1 kinase activity
  evidence:
  - reference: PMID:14604992
    reference_title: P16INK4a is required for hSNF5 chromatin remodeler-induced cellular senescence in malignant rhabdoid tumor cells.
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: "Following hSNF5 expression, we observed transcriptional activation of the tumor suppressor p16(INK4a) but not of p14(ARF), repression of several cyclins and CD44, a cell surface glycoprotein implicated in metastasis."
    explanation: Restoring SMARCB1/hSNF5 in MRT cells transcriptionally activates p16(INK4a); SMARCB1 loss therefore silences p16(INK4a) — the single event this node captures.
- name: CDK4-Cyclin D1 Hyperactivation and G1-S Bypass
  description: >-
    Without p16(INK4a) restraint, CDK4-Cyclin D1 kinase activity is left
    unopposed, driving RB1 phosphorylation and inactivation, and bypassing the
    G1-S restriction point. Cyclin D1 is the critical downstream effector: its
    genetic ablation fully abrogates rhabdoid tumorigenesis caused by
    SMARCB1/Ini1 loss in vivo.
  biological_processes:
  - preferred_term: G1-S restriction-point bypass
    modifier: INCREASED
    term:
      id: GO:0000082
      label: G1/S transition of mitotic cell cycle
  molecular_functions:
  - preferred_term: CDK4-Cyclin D1 kinase activity
    modifier: INCREASED
    term:
      id: GO:0004693
      label: cyclin-dependent protein serine/threonine kinase activity
  downstream:
  - target: Aberrant MRT Cell Growth
    description: Loss of the G1-S checkpoint drives unchecked rhabdoid tumor proliferation
  evidence:
  - reference: PMID:14604992
    reference_title: P16INK4a is required for hSNF5 chromatin remodeler-induced cellular senescence in malignant rhabdoid tumor cells.
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: "Expression of a p16(INK4a)-insensitive form of CDK4 obstructs hSNF5-induced cell cycle arrest."
    explanation: A p16(INK4a)-insensitive CDK4 bypasses SMARCB1-induced arrest, establishing CDK4 as the effector kinase that drives proliferation when p16(INK4a) is lost.
  - reference: PMID:12226744
    reference_title: A key role of the hSNF5/INI1 tumour suppressor in the control of the G1-S transition of the cell cycle.
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: "This arrest can be reverted by coexpression of cyclin D1, cyclin E or viral E1A, whereas it cannot be counteracted by pRB-binding deficient E1A mutants."
    explanation: SMARCB1/INI1-induced G1 arrest is reversed by Cyclin D1 and requires functional pRB, placing the Cyclin D1-CDK4-RB1 axis directly downstream of SMARCB1 loss.
  - reference: PMID:16099835
    reference_title: Genetic ablation of Cyclin D1 abrogates genesis of rhabdoid tumors resulting from Ini1 loss.
    supports: SUPPORT
    evidence_source: MODEL_ORGANISM
    snippet: "We crossed Ini1+/- mice to Cyclin D1-/- mice and found that Ini1+/- mice with Cyclin D1 deficiency did not develop any spontaneous tumors, in contrast to the parental Ini1+/- mice."
    explanation: In vivo genetic proof that Cyclin D1 is the critical effector downstream of SMARCB1/Ini1 loss — Cyclin D1 deficiency fully abrogates rhabdoid tumorigenesis.
- name: Aberrant MRT Cell Growth
  description: >-
    EZH1 and EZH2 activity sustains malignant rhabdoid tumor cell growth,
    helping maintain the aggressive proliferative phenotype downstream of
    SMARCB1 loss.
  biological_processes:
  - preferred_term: cell population proliferation
    modifier: INCREASED
    term:
      id: GO:0008283
      label: cell population proliferation
  evidence:
  - reference: PMID:36212776
    reference_title: Dual targeting of EZH1 and EZH2 for the treatment of malignant rhabdoid tumors.
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: "Here, we show that EZH1, as well as EZH2, contributes to MRT cell growth and H3K27 methylation."
    explanation: This study directly supports EZH-dependent malignant rhabdoid tumor cell growth downstream of the repressive chromatin state.
- name: Blocked Cellular Differentiation
  description: >-
    SMARCB1-deficient rhabdoid tumor cells are maintained in an undifferentiated
    state, and EZH2 inhibition can trigger differentiation.
  biological_processes:
  - preferred_term: cell differentiation
    modifier: DECREASED
    term:
      id: GO:0030154
      label: cell differentiation
  evidence:
  - reference: PMID:23620515
    reference_title: Durable tumor regression in genetically altered malignant rhabdoid tumors by inhibition of methyltransferase EZH2.
    supports: SUPPORT
    evidence_source: IN_VITRO
    snippet: "The compound induces apoptosis and differentiation specifically in SMARCB1-deleted MRT cells."
    explanation: Induction of differentiation after EZH2 inhibition supports a baseline differentiation block in SMARCB1-deleted rhabdoid tumor cells.
histopathology:
- name: Rhabdoid Cell Morphology
  finding_term:
    preferred_term: Rhabdoid Tumor
    term:
      id: NCIT:C3808
      label: Rhabdoid Tumor
  description: >-
    Classic rhabdoid tumors are composed of poorly differentiated cells with
    eccentric nuclei, prominent nucleoli, and glassy eosinophilic cytoplasm with
    hyaline inclusions.
  evidence:
  - reference: PMID:16704491
    reference_title: "Extrarenal rhabdoid tumors of soft tissue: clinicopathological and molecular genetic review and distinction from other soft-tissue sarcomas with rhabdoid features."
    supports: SUPPORT
    snippet: "Microscopically, the tumor is composed of a diffuse proliferation of rounded or polygonal cells with eccentric nuclei, prominent nucleoli and glassy eosinophilic cytoplasm containing hyaline-like inclusion bodies, arranged in sheets and nests."
    explanation: This pathology review describes the defining rhabdoid cell morphology.
phenotypes:
- category: Genitourinary
  name: Abdominal Mass
  subtype: RTK
  diagnostic: true
  description: >-
    Renal rhabdoid tumors commonly present as a large abdominal or flank mass in
    infancy.
  phenotype_term:
    preferred_term: Abdominal mass
    term:
      id: HP:0031500
      label: Abdominal mass
  evidence:
  - reference: PMID:8732342
    reference_title: "Rhabdoid tumour of the kidney: a clinicopathological study of 22 patients from the International Society of Paediatric Oncology (SIOP) nephroblastoma file."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: "Clinically, they presented with an abdominal mass but four (18%) children also had hypercalcaemia"
    explanation: SIOP clinicopathological series of 22 children with rhabdoid tumour of the kidney documents abdominal mass as the dominant presenting finding.
- category: Genitourinary
  name: Hematuria
  subtype: RTK
  description: >-
    Gross or microscopic hematuria can accompany kidney involvement and collecting
    system invasion.
  phenotype_term:
    preferred_term: Hematuria
    term:
      id: HP:0000790
      label: Hematuria
  evidence:
  - reference: PMID:11216700
    reference_title: "Clinical presentation of rhabdoid tumors of the kidney."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: "Either gross or microscopic hematuria was present in 84.4% (27/32) of the patients with RTK."
    explanation: National Wilms Tumor Study Group review of 50 RTK patients shows hematuria is a near-universal presenting feature, supporting its inclusion as a characteristic phenotype of renal rhabdoid tumor.
- category: Musculoskeletal
  name: Soft Tissue Mass
  subtype: eMRT
  diagnostic: true
  description: >-
    Extrarenal disease often presents as a rapidly enlarging deep axial soft-tissue
    mass.
  phenotype_term:
    preferred_term: Soft tissue neoplasm
    term:
      id: HP:0031459
      label: Soft tissue neoplasm
  evidence:
  - reference: PMID:36720509
    reference_title: "Extrarenal rhabdoid tumour of axillary soft tissue: a diagnostic challenge resolved by immunohistochemistry."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: "We report one such rare case in a female infant presenting as left axillary mass."
    explanation: Case report of extrarenal rhabdoid tumour presenting as an axillary soft-tissue mass in an infant illustrates the typical eMRT presentation as a deep soft-tissue mass.
- category: Neurological
  name: Headache
  subtype: AT/RT
  description: >-
    AT/RT commonly causes headache from intracranial mass effect or obstructive
    hydrocephalus.
  phenotype_term:
    preferred_term: Headache
    term:
      id: HP:0002315
      label: Headache
  evidence:
  - reference: PMID:27307151
    reference_title: "Atypical teratoid/rhabdoid tumors with multilayered rosettes in the pineal region."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: "Patient 1, a 23-month-old girl, presented with a history of gait unsteadiness and headache"
    explanation: Case series of two children with intracranial AT/RT in the pineal/third ventricular region documents headache as a presenting symptom consistent with intracranial mass effect.
- category: Neurological
  name: Vomiting
  subtype: AT/RT
  description: >-
    Vomiting, often worse in the morning, reflects increased intracranial pressure
    in CNS rhabdoid tumor.
  phenotype_term:
    preferred_term: Vomiting
    term:
      id: HP:0002013
      label: Vomiting
  evidence:
  - reference: PMID:27307151
    reference_title: "Atypical teratoid/rhabdoid tumors with multilayered rosettes in the pineal region."
    supports: SUPPORT
    evidence_source: HUMAN_CLINICAL
    snippet: "Patient 2, a 26-month-old girl, presented with headache and vomiting"
    explanation: Same AT/RT case series documents vomiting accompanying headache, consistent with raised intracranial pressure from posterior third ventricular AT/RT.
biochemical:
- name: SMARCB1 (INI1/BAF47) Immunohistochemistry
  notes: >-
    Loss of nuclear INI1/BAF47 staining is the canonical diagnostic biomarker
    for most rhabdoid tumors and mirrors biallelic SMARCB1 inactivation.
- name: SMARCA4 (BRG1) Immunohistochemistry
  notes: >-
    Rare SMARCA4-deficient rhabdoid tumors show loss of BRG1 staining and should
    prompt consideration of rhabdoid tumor predisposition syndrome type 2.
genetic:
- name: SMARCB1
  gene_term:
    preferred_term: SMARCB1
    term:
      id: hgnc:11103
      label: SMARCB1
  association: Somatic/Germline Loss-of-Function
  notes: >-
    SMARCB1 is the dominant driver gene across the rhabdoid tumor spectrum. Both
    somatic and germline loss can occur, and germline variants define rhabdoid
    tumor predisposition syndrome type 1.
  evidence:
  - reference: PMID:39088359
    reference_title: "Malignant Rhabdoid Tumor and Related Pediatric Tumors: Multimodality Imaging Review with Pathologic Correlation."
    supports: SUPPORT
    snippet: "Irrespective of the site of occurrence, somatic and germline mutations in SMARCB1, and rarely in SMARCA4, underlie the entire spectrum of rhabdoid tumors."
    explanation: This review supports SMARCB1 as the principal shared somatic and germline driver across renal, extrarenal, and CNS rhabdoid tumors.
  - reference: PMID:23432645
    reference_title: "Rhabdoid tumors: an initial clue to the role of chromatin remodeling in cancer."
    supports: SUPPORT
    snippet: "Indeed, the mutation rate in RTs is among the lowest of all cancers sequenced, with loss of SMARCB1 as essentially the sole recurrent event."
    explanation: This review emphasizes how dominant SMARCB1 loss is in the otherwise genetically simple rhabdoid tumor genome.
- name: SMARCA4
  gene_term:
    preferred_term: SMARCA4
    term:
      id: hgnc:11100
      label: SMARCA4
  association: Rare Somatic/Germline Loss-of-Function
  notes: >-
    SMARCA4 loss is an uncommon alternative driver in rhabdoid tumor and is the
    basis of rhabdoid tumor predisposition syndrome type 2.
  evidence:
  - reference: PMID:39088359
    reference_title: "Malignant Rhabdoid Tumor and Related Pediatric Tumors: Multimodality Imaging Review with Pathologic Correlation."
    supports: SUPPORT
    snippet: "Irrespective of the site of occurrence, somatic and germline mutations in SMARCB1, and rarely in SMARCA4, underlie the entire spectrum of rhabdoid tumors."
    explanation: This review supports rare but disease-defining SMARCA4-driven rhabdoid tumors alongside the more common SMARCB1-deficient cases.
treatments:
- name: Multi-Agent Chemotherapy
  description: >-
    Intensive multi-agent chemotherapy remains part of front-line treatment for
    most newly diagnosed patients, usually combined with surgery and local
    radiotherapy according to age, site, and resectability.
  treatment_term:
    preferred_term: chemotherapy
    term:
      id: MAXO:0000647
      label: chemotherapy
- name: Gross Total Resection
  description: >-
    Maximal safe resection is pursued whenever anatomically feasible because gross
    total resection is one of the strongest favorable prognostic factors in
    extracranial disease.
  treatment_term:
    preferred_term: surgical procedure
    term:
      id: MAXO:0000004
      label: surgical procedure
  evidence:
  - reference: PMID:33249395
    reference_title: Clinical and genetic risk factors define two risk groups of extracranial malignant rhabdoid tumours (eMRT/RTK).
    supports: SUPPORT
    snippet: "On univariate analyses, age at diagnosis (≥12 months), M0-stage, absence of synchronous tumours, absence of a GLM, gross total resection (GTR), radiotherapy and achieving a CR were significantly associated with favourable outcomes."
    explanation: EU-RHAB registry data identifies gross total resection as a favorable prognostic factor in extracranial rhabdoid tumor.
- name: Radiation Therapy
  description: >-
    Radiotherapy is used for local control, especially after incomplete resection
    or in high-risk disease, and contributes to outcome in multimodality therapy.
  treatment_term:
    preferred_term: radiation therapy
    term:
      id: MAXO:0000014
      label: radiation therapy
  evidence:
  - reference: PMID:33249395
    reference_title: Clinical and genetic risk factors define two risk groups of extracranial malignant rhabdoid tumours (eMRT/RTK).
    supports: SUPPORT
    snippet: "On univariate analyses, age at diagnosis (≥12 months), M0-stage, absence of synchronous tumours, absence of a GLM, gross total resection (GTR), radiotherapy and achieving a CR were significantly associated with favourable outcomes."
    explanation: The same EU-RHAB analysis supports radiotherapy as part of the treatment factors associated with improved extracranial outcomes.
- name: EZH2 Inhibitor Therapy
  description: >-
    EZH2-targeted therapy is an investigational mechanism-based strategy for
    relapsed or refractory SMARCB1/SMARCA4-deficient disease. Preclinical data
    support strong EZH2 dependence, and early pediatric trials have tested
    tazemetostat in molecularly selected tumors.
  treatment_term:
    preferred_term: pharmacotherapy
    term:
      id: MAXO:0000058
      label: pharmacotherapy
    therapeutic_agent:
    - preferred_term: tazemetostat
      term:
        id: NCIT:C107506
        label: Tazemetostat
  target_mechanisms:
  - target: EZH2-Driven H3K27 Methylation
    treatment_effect: INHIBITS
    description: >-
      Tazemetostat selectively inhibits EZH2 methyltransferase activity, blocking
      the H3K27 methylation dependency that SMARCB1-deficient rhabdoid tumors
      acquire when BAF-mediated antagonism of PRC2 is lost.
  evidence:
  - reference: PMID:23620515
    reference_title: Durable tumor regression in genetically altered malignant rhabdoid tumors by inhibition of methyltransferase EZH2.
    supports: SUPPORT
    evidence_source: MODEL_ORGANISM
    snippet: "These data demonstrate the dependency of SMARCB1 mutant MRTs on EZH2 enzymatic activity and portend the utility of EZH2-targeted drugs for the treatment of these genetically defined cancers."
    explanation: Preclinical MRT models provide direct proof that EZH2 is a targetable dependency downstream of SMARCB1 loss.
  - reference: PMID:37228094
    reference_title: "Tazemetostat for tumors harboring SMARCB1/SMARCA4 or EZH2 alterations: results from NCI-COG pediatric MATCH APEC1621C."
    supports: PARTIAL
    snippet: "Patients whose tumors harbored EZH2 mutations or loss of SMARCB1 or SMARCA4 by immunohistochemistry were treated with EZH2 inhibitor tazemetostat."
    explanation: This pediatric MATCH trial shows that EZH2 inhibition has been clinically tested in SMARCB1/SMARCA4-deficient pediatric tumors, including rhabdoid tumors, although efficacy was limited.
disease_term:
  preferred_term: rhabdoid tumor
  term:
    id: MONDO:0002728
    label: rhabdoid tumor
mappings:
  mondo_mappings:
  - term:
      id: MONDO:0002728
      label: rhabdoid tumor
    mapping_predicate: skos:exactMatch
    mapping_source: MONDO
    mapping_justification: MONDO provides an exact disease term for rhabdoid tumor.
  ncit_mappings:
  - term:
      id: NCIT:C3808
      label: Rhabdoid Tumor
    mapping_predicate: skos:exactMatch
    mapping_source: NCIT
    mapping_justification: NCIT provides an exact neoplasm term for rhabdoid tumor; the same identifier is used in this entry's histopathology block.
classifications:
  icdo_morphology:
    classification_value: Embryonal Neoplasm
  harrisons_chapter:
  - classification_value: cancer
  - classification_value: solid tumor
review_notes: >-
  This entry deliberately models the multisite rhabdoid tumor spectrum rather than
  a single organ-specific disease. Shared chromatin-remodeling mechanisms are
  represented at the umbrella level, while clinical features are tagged to RTK,
  eMRT, and AT/RT subtypes because presentation and local therapy are strongly
  site dependent.