Division of Hematology, Department of Internal and Laboratory Medicine, Mayo Clinic, Rochester, MN.
Division of Hematopathology, Department of Internal and Laboratory Medicine, Mayo Clinic, Rochester, MN.
Mayo Clin Proc. 2019 Apr;94(4):599-610. doi: 10.1016/j.mayocp.2018.08.022. Epub 2019 Feb 26.
To document the Mayo Clinic decades-long experience with myeloproliferative neoplasms (MPNs) and provide mature risk-stratified survival data and disease complication estimates.
All Mayo Clinic patients with World Health Organization-defined MPNs constituted the core study group and included those with polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF).
A total of 3023 consecutive patients (median age, 62 years; range, 18-96 years) were considered: 665 PV, 1076 ET, and 1282 PMF. From October 27, 1967, through December 29, 2017, 1631 deaths (54%), 183 leukemic transformations (6%), 244 fibrotic progressions (14%), and 516 thrombotic events (17%) were recorded. Median overall survival (OS) was 18 years for ET, 15 years for PV, and 4.4 years for PMF (P<.05 for all intergroup comparisons). Inferior survival was documented in patients with ET diagnosed more recently (post-1990) (P<.001), whereas survival data were time independent in PV and PMF. After conventional risk stratification, OS in low-risk ET and low-risk PV were superimposed (P=.89) but each differed significantly from that of age- and sex-matched controls (P<.001). Leukemia-free survival was similar for ET and PV (P=.22) and significantly worse with PMF (P<.001). Compared with ET, PV was associated with higher risk of fibrotic progression (P<.001). Thrombosis risk after diagnosis was highest in PV and lowest in PMF (P=.002 for PV vs ET; P=.56 for ET vs PMF; and P=.001 for PV vs PMF).
This study provides the most mature survival and outcomes data in MPNs and highlights MPN subgroup risk categorization as key in appraising disease natural history. The OS was only marginally better in ET compared with PV, and PV displayed a higher risk of thrombosis and fibrotic progression.
记录梅奥诊所长达数十年的骨髓增殖性肿瘤（MPN）经验，并提供成熟的风险分层生存数据和疾病并发症估计。
所有符合世界卫生组织定义的 MPN 的梅奥诊所患者均构成核心研究组，包括真性红细胞增多症（PV）、特发性血小板增多症（ET）和原发性骨髓纤维化（PMF）患者。
共纳入 3023 例连续患者（中位年龄 62 岁；范围 18-96 岁）：665 例 PV、1076 例 ET 和 1282 例 PMF。从 1967 年 10 月 27 日至 2017 年 12 月 29 日，记录了 1631 例死亡（54%）、183 例白血病转化（6%）、244 例纤维化进展（14%）和 516 例血栓事件（17%）。ET 的中位总生存期（OS）为 18 年，PV 为 15 年，PMF 为 4.4 年（所有组间比较均<.05）。最近诊断（1990 年后）的 ET 患者生存情况较差（P<.001），而 PV 和 PMF 的生存数据与时间无关。经过传统风险分层，低危 ET 和低危 PV 的 OS 重叠（P=.89），但与年龄和性别匹配的对照组相比差异均有统计学意义（P<.001）。ET 和 PV 的白血病无进展生存率相似（P=.22），而 PMF 则显著较差（P<.001）。与 ET 相比，PV 与纤维化进展风险较高相关（P<.001）。诊断后血栓形成风险在 PV 中最高，在 PMF 中最低（PV 与 ET 相比，P=.002；ET 与 PMF 相比，P=.56；PV 与 PMF 相比，P=.001）。
本研究提供了 MPN 中最成熟的生存和结局数据，并强调 MPN 亚组风险分类是评估疾病自然史的关键。与 PV 相比，ET 的 OS 仅略有改善，而 PV 的血栓形成和纤维化进展风险更高。
Zotero 插件