| Literature DB >> 33275748 |
Ari Zimran1,2, Rosa Ruchlemer3,2, Shoshana Revel-Vilk1,2.
Abstract
Patients with Gaucher disease (GD), a rare autosomal recessive glycosphingolipid storage disease, commonly present to hematologists with unexplained splenomegaly, thrombocytopenia, anemia, and bone symptoms. Patients with GD may develop other manifestations, such as autoimmune thrombocytopenia, monoclonal gammopathy, multiple myeloma, or, even more rarely, other hematological malignancies; sometimes they are first diagnosed during an assessment of those disorders. Although the diagnosis and management of patients with GD have significantly evolved over the last 30 years, some patients remain poor responders to GD-specific therapy, needing novel and investigational therapies. Ideally, patients with GD, like patients with other rare diseases, should be managed by a multidisciplinary team expert with the diverse clinical manifestations and potential GD-related or -unrelated comorbidities. The hematology community should be knowledgeable regarding the presentation and the variety of hematologic complications and comorbidities associated with Gaucher disease.Entities:
Mesh:
Year: 2020 PMID: 33275748 PMCID: PMC7727517 DOI: 10.1182/hematology.2020000123
Source DB: PubMed Journal: Hematology Am Soc Hematol Educ Program ISSN: 1520-4383