文章：

新的世界卫生组织原发性血小板增多症分类要求对现有证据进行修订

The new WHO classification for essential thrombocythemia calls for revision of available evidences

原文发布日期：2020-02-25

DOI: 10.1038/s41408-020-0290-9

类型: Review Article

开放获取: 是

英文摘要：

In the 2016 revised classification of myeloproliferative neoplasms pre-fibrotic primary myelofibrosis (pre-PMF) was recognized as a separate entity, distinct from essential thrombocythemia (ET). Owing that the majority of cases falling in the pre-PMF category were previously diagnosed as ET, one may question about the need to re-evaluate the results of epidemiologic, clinical, and molecular studies, and the results of clinical trials in the two entities. Based on a critical review of recently published studies, pre-PMF usually presents with a distinct clinical and hematological presentation and higher frequency of constitutional symptoms. JAK2V617F and CALR mutations in pre-PMF patients are superimposable to ET, whereas non-driver high-risk mutations are enriched in pre-PMF compared with ET. Thrombosis is not significantly different, whereas bleeding is more frequent in pre-PMF. Median survival is significantly shorter in pre-PMF and 10-year cumulative rates progression to overt myelofibrosis is 0–1% vs. 10–12%, and leukemic transformation is 1–2% vs. 2–6%, in ET and pre-fibrotic-PMF, respectively. Most patients fall in the lower prognostic IPSS group in which observation alone can be recommended. Patients at intermediate risk may require a symptom-driven treatment for anemia, splenomegaly or constitutional symptoms while cytoreductive drugs are indicated in the high-risk category.
 

摘要翻译： 

在2016年修订的骨髓增殖性肿瘤分类中，前纤维化原发性骨髓纤维化被确认为一个独立病种，与原发性血小板增多症截然不同。鉴于大多数归类为前纤维化原发性骨髓纤维化的病例先前被诊断为原发性血小板增多症，人们可能会质疑是否需要重新评估这两种实体在流行病学、临床和分子学研究以及临床试验的结果。基于对近期发表研究的重要综述，前纤维化原发性骨髓纤维化通常呈现独特的临床血液学表现和较高频率的全身性症状。该病患者中的JAK2V617F和CALR突变与原发性血小板增多症患者相似，而非驱动性高危突变在前纤维化原发性骨髓纤维化中较原发性血小板增多症更为富集。两者在血栓形成方面无显著差异，而前纤维化原发性骨髓纤维化的出血事件更为频繁。前纤维化原发性骨髓纤维化的中位生存期显著较短，其10年进展为显著骨髓纤维化的累积率分别为0-1%对10-12%，白血病转化率为1-2%对2-6%（对应原发性血小板增多症和前纤维化原发性骨髓纤维化）。大多数患者属于较低风险的IPSS预后分组，对此仅建议观察随访。中危患者可能需要针对贫血、脾肿大或全身性症状进行对症治疗，而高危患者则需使用细胞减灭药物。

原文链接：

The new WHO classification for essential thrombocythemia calls for revision of available evidences