International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia, for the IWG for Myelofibrosis Research and Treatment (IWG-MRT)
International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia, for the IWG for Myelofibrosis Research and Treatment (IWG-MRT)
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DOI：
10.1182/blood-2006-03-009746
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发表时间：
2006-09-01
期刊：
影响因子：
20.3
通讯作者：
Kantarjian, Hagop
中科院分区：
文献类型：
--
作者：
Tefferi, Ayalew;Barosi, Giovanni;Kantarjian, Hagop
与髓样变质（MMM）的骨髓纤维化是一种临床病理学实体，其特征是干细胞衍生的克隆骨髓增生，无效的erythropoiesis，dumagimandullapialdullapoiesis，外尿外造血，骨髓纤维纤维纤维纤维纤维化和骨化骨膜造成。 MMM患者的生存率缩短，其生活质量受到进行性贫血，明显的肝肺炎体系的损害，以及包括卡氏症在内的严重宪法症状。经过数十年对无效治疗的挫败感，现在通过包括同种异体造血干细胞移植和免疫调节药物在内的有希望的治疗方法为患者提供了服务。有关疾病发病机理的最新信息，包括通过功能获得的JAK2突变（JAK2（V617F））对骨髓增生性疾病表型的贡献，已恢复了靶向治疗剂以及分子监测治疗反应的前景。这种进度要求对响应标准进行标准化，以准确评估新治疗方式的价值，以便在研究之间进行准确的比较，并确保响应的定义反映了有意义的健康结果。因此，国际专家小组最近召集并描述了3个响应类别：完全缓解（CR），部分缓解（PR）和临床改进（CI）。骨髓组织学和血液学恢复分别表征了CR和CR/PR。小组同意，CI反应类别仅适用于中度至重度细胞肿瘤或脾肿大的患者。
Myelofibrosis with myeloid metaplasia (MMM) is a clinicopathologic entity characterized by stem cell-derived clonal myeloproliferation, ineffective erythropoiesis, extramedullary hematopoiesis, and bone marrow fibrosis and osteosclerosis. Patients with MMM have shortened survival and their quality of life is compromised by progressive anemia, marked hepatosplenomegaly, and severe constitutional symptoms including cachexia. After decades of frustration with ineffective therapy, patients are now being served by promising treatment approaches that include allogeneic hematopoietic stem cell transplantation and immunomodulatory drugs. Recent information regarding disease pathogenesis, including a contribution to the myeloproliferative disorder phenotype by a gain-of-function JAK2 mutation (JAK2(V617F)), has revived the prospect of targeted therapeutics as well as molecular monitoring of treatment response. Such progress calls for standardization of response criteria to accurately assess the value of new treatment modalities, to allow accurate comparison between studies, and to ensure that the definition of response reflects meaningful health outcome. Accordingly, an international panel of experts recently convened and delineated 3 response categories: complete remission (CR), partial remission (PR), and clinical improvement (CI). Bone marrow histologic and hematologic remissions characterize CR and CR/PR, respectively. The panel agreed that the CI response category is applicable only to patients with moderate to severe cytopenia or splenomegaly.