Simon Laurence, Leblond Véronique
Service d'hématologie clinique, groupe hospitalier La Pitié-Salpêtrière, Paris, France.
Rev Prat. 2018 Sep;68(7):797-802.
Waldenström macroglobulinaemia. Waldenström macroglobulinaemia (WM) is a rare, indolent B-cell lymphoproliferative disorder characterized by cellular involvement in bone marrow and monoclonal IgM production. Symptoms can be related to cytopenias, tumoural involvement, or IgM-related disorders. Somatic mutations in the MYD88 gene have been described in the majority of WM cases. Treatments are started when WM is symptomatic, following systematic biological and morphological assessments. Therapeutic choice depends on age, frailty and urgent efficacy need. In first line, the majority of patients are treated with monoclonal anti- CD20 antibody-based regimens combined with cytotoxic chemotherapy. Rituximab, cyclophosphamide and dexamethasone remain the most commonly used regimen with good safety. Nevertheless, increasing numbers of new drugs are becoming available or are in development. Bruton tyrosine kinase (BTK) inhibitor demonstrated excellent results and is now available for relapse/ refractory disease or as first line for some patients.
华氏巨球蛋白血症。华氏巨球蛋白血症（WM）是一种罕见的惰性B细胞淋巴增殖性疾病，其特征是骨髓细胞受累和单克隆IgM产生。症状可能与血细胞减少、肿瘤累及或IgM相关疾病有关。大多数WM病例中已发现MYD88基因的体细胞突变。在进行系统的生物学和形态学评估后，当WM出现症状时开始治疗。治疗选择取决于年龄、身体虚弱程度和紧急疗效需求。一线治疗中，大多数患者接受基于抗CD20单克隆抗体的方案联合细胞毒性化疗。利妥昔单抗、环磷酰胺和地塞米松仍然是最常用的方案，安全性良好。然而，越来越多的新药可供使用或正在研发中。布鲁顿酪氨酸激酶（BTK）抑制剂已显示出优异的效果，现在可用于复发/难治性疾病或作为一些患者的一线治疗药物。
