Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
Nanfang Hospital, Southern Medical University, Guangzhou, China.
Acta Haematol. 2023;146(4):293-306. doi: 10.1159/000529750. Epub 2023 Feb 22.
Genetic landscape, disease characteristics, and clinical outcomes of young adults with myeloproliferative neoplasms (MPNs) were reported. However, data on patient-reported outcomes (PROs) in young adults with MPNs were rare.
We conducted a multicenter, cross-sectional study to compare the PROs in respondents with thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF) by age at survey, including the young group (18-40 years), middle-aged group (41-60 years), and elderly group (>60 years).
Of the 1,664 respondents with MPNs, 349 (21.0%) were young including 244 (69.9%) with ET, 34 (9.7%) with PV, and 71 (20.3%) with MF. In multivariate analyses, the young groups with ET and MF were associated with the lowest MPN-10 scores among the 3 age groups; those with MF, highest proportion of reporting negative impact of disease and therapy on their daily life and work. The young groups with MPNs had the highest physical component summary scores but the lowest mental component summary scores in those with ET. The young groups with MPNs were most concerned about fertility; those with ET, treatment-related adverse events and long-term efficacy of treatment.
We concluded that young adults with MPNs have different PROs compared with middle-aged and elderly patients.
报道了年轻成年人骨髓增殖性肿瘤（MPN）的遗传图谱、疾病特征和临床结局。然而，关于年轻成年人 MPN 患者报告的结果（PROs）的数据很少。
我们进行了一项多中心、横断面研究，按调查时的年龄比较了有血小板增多症（ET）、真性红细胞增多症（PV）和骨髓纤维化（MF）的应答者的 PROs，包括年轻组（18-40 岁）、中年组（41-60 岁）和老年组（>60 岁）。
在 1664 名 MPN 应答者中，有 349 名（21.0%）为年轻人，包括 244 名（69.9%）ET、34 名（9.7%）PV 和 71 名（20.3%）MF。在多变量分析中，年轻 ET 和 MF 组在 3 个年龄组中与 MPN-10 评分最低相关；MF 组报告疾病和治疗对日常生活和工作影响负面的比例最高。在 ET 中，年轻 MPN 组的生理成分综合评分最高，但心理成分综合评分最低。年轻 MPN 组最关心生育问题；ET 组最关注治疗相关不良反应和长期治疗效果。
我们的结论是，与中年和老年患者相比，年轻成年人 MPN 的 PROs 不同。
