PulseExploreJournal ClubDebatesTrendingResearchersJournals
Instagram
HomeExploreJournal ClubTrending
Synapse
⌘+K
Synapse
April 13, 2026American Journal of Medical Genetics Part A0 citations

CBL Syndrome With Granular Cell Tumor and café au lait macules: Expansion of the Phenotype

View Full Paper
CHCaitlin HarringtonEKEmily KohlZGZainab Gilitwala

Key Points

  • To present phenotypic variability in CBL syndrome, particularly involving granular cell tumors.
  • Case report of a patient with CBL syndrome
  • Genetic testing for pathogenic variants
  • Clinical assessment of pheno-typic manifestations
  • Patient exhibited a germline likely pathogenic variant in CBL
  • Documented presence of ten café au lait macules
  • Recurrent granular cell tumor not previously described in CBL syndrome

Abstract

CBL syndrome is a RASopathy with phenotypic variability including neurodevelopmental differences, cardiac defects, growth delay, dysmorphic and cutaneous findings, hematologic, immunologic, and vascular manifestations, and predisposition to juvenile myelomonocytic leukemia (JMML). We present a patient with a germline likely pathogenic variant in CBL with ten café au lait macules and a recurrent granular cell tumor. Granular cell tumors have not been described in this condition. This case broadens the recognized clinical spectrum of CBL syndrome and challenges the existing genetic testing approach for patients with greater than six café au lait macules.

Ask AI
Helpful
Bookmark
Share
View Full Paper

Cite This Study

Harrington et al. (2026) studied this question.

synapsesocial.com/papers/69dc89473afacbeac03eb15chttps://doi.org/10.1002/ajmg.a.70165
Ask AI
Helpful
Bookmark
Share
View Full Paper

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Immunological and hematological findings as major features in a patient with a new germline pathogenic <scp><i>CBL</i></scp> variant2024
  2. 2Chronic myeloproliferative neoplasm in adulthood in CBL syndrome harboring a splice‐site <i>CBL</i> variant alongside a novel constitutional <i>CSF3R</i> variant2024 · 1 citations
  3. 3Severe ADEM‐Like Neuroinflammatory Disease and Cerebrovascular Fragility With Recurrent Pseudoaneurysms and Moyamoya in a Familial Germline <i>CBL</i> Mutation: Expanding the Clinical Phenotype2026
  4. 4CBL mutations in chronic myelomonocytic leukemia often occur in the RING domain with multiple subclones per patient: Implications for targeting2024 · 1 citations
  5. 5Abstract A023-PR006: CBL mutations in pediatric solid and CNS tumours are a marker of receptor tyrosine kinase activation and a potential therapeutic target2025