Gaucher Unit.
School of Medicine, Hebrew University, Jerusalem, Israel.
Hematology Am Soc Hematol Educ Program. 2020 Dec 4;2020(1):389-394. doi: 10.1182/hematology.2020000123.
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.
戈谢病(GD)患者是一种罕见的常染色体隐性糖脂贮积病,常因不明原因的脾肿大、血小板减少、贫血和骨骼症状而就诊于血液科医生。GD 患者可能会出现其他表现,如自身免疫性血小板减少症、单克隆丙种球蛋白病、多发性骨髓瘤,甚至更罕见的其他血液系统恶性肿瘤;有时,他们在评估这些疾病时被首次诊断。尽管过去 30 年来 GD 的诊断和治疗已有显著进展,但仍有一些患者对 GD 特异性治疗反应不佳,需要新型和研究性治疗。理想情况下,像其他罕见病患者一样,GD 患者应由具有不同临床表现和潜在 GD 相关或非相关合并症的多学科团队专家进行管理。血液学领域的专业人员应了解 GD 相关的血液学并发症和合并症的表现和多样性。