FROM Research Foundation, Papa Giovanni XXIII Hospital, Bergamo, Italy.
Institute of Pathology, University of Cologne, Cologne, Germany.
Blood Cancer J. 2020 Feb 25;10(2):22. doi: 10.1038/s41408-020-0290-9.
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.
在 2016 年修订的骨髓增殖性肿瘤分类中，纤维化前期原发性骨髓纤维化（pre-PMF）被认为是一种独立的实体，与原发性血小板增多症（ET）不同。由于大多数属于 pre-PMF 类别的病例以前被诊断为 ET，人们可能会质疑是否需要重新评估这两种实体的流行病学、临床和分子研究结果以及临床试验结果。基于对最近发表的研究的批判性回顾，pre-PMF 通常表现出独特的临床和血液学表现以及更高频率的全身症状。pre-PMF 患者的 JAK2V617F 和 CALR 突变与 ET 重叠，而非驱动性高危突变在 pre-PMF 中比 ET 更为丰富。血栓形成没有显著差异，而 pre-PMF 出血更为频繁。中位生存期在 pre-PMF 中显著缩短，10 年累积进展为明显骨髓纤维化的发生率为 0-1%对 10-12%，白血病转化的发生率为 1-2%对 2-6%，分别在 ET 和纤维化前期-PMF 中。大多数患者属于预后较差的 IPSS 组，仅观察即可推荐。对于有中间风险的患者，可能需要针对贫血、脾肿大或全身症状进行症状驱动的治疗，而细胞减少药物则适用于高危类别。
