Efficace Fabio, Santini Valeria, La Nasa Giorgio, Cottone Francesco, Finelli Carlo, Borin Lorenza, Quaresmini Giulia, Di Tucci Anna Angela, Volpe Antonio, Cilloni Daniela, Quarta Giovanni, Sanpaolo Grazia, Rivellini Flavia, Salvi Flavia, Molteni Alfredo, Voso Maria Teresa, Alimena Giuliana, Fenu Susanna, Mandelli Franco, Angelucci Emanuele
Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy.
Functional Unit of Haematology, Azienda Ospedaliero Universitaria Careggi, University of Florence, Florence, Italy.
BMJ Support Palliat Care. 2016 Mar;6(1):80-8. doi: 10.1136/bmjspcare-2014-000726. Epub 2014 Sep 9.
The primary objective of this study was to evaluate the health-related quality of life (HRQOL) in lower-risk, transfusion-dependent patients with myelodysplastic syndromes (MDS) treated with deferasirox. A secondary objective was to investigate the relationship between HRQOL, serum ferritin levels and transfusion dependency.
This was a prospective multicentre study enrolling 159 patients, of whom 152 received at least one dose of deferasirox. HRQOL was assessed with the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) at baseline and then at 3, 6, 9 and 12 months. Primary analysis was performed estimating mean HRQOL scores over time by a linear mixed model on selected scales.
The median age of treated patients was 72 years (range 24-87 years). No statistically significant changes over time were found in mean scores for global health status/quality of life (p=0.564), physical functioning (p=0.409) and fatigue (p=0.471) scales. Also, no significant changes were found for constipation (p=0.292), diarrhoea (p=0.815) and nausea and vomiting (p=0.643). Serum ferritin levels were not associated with HRQOL outcomes. A higher patient-reported baseline pain severity was an independent predictive factor of an earlier achievement of transfusion independence with a HR of 1.032 (99% CI 1.004 to 1.060; p=0.003).
HRQOL of transfusion-dependent patients with MDS receiving deferasirox therapy remains stable over time. HRQOL assessment might also provide important predictive information on treatment outcomes.
NCT00469560.
本研究的主要目的是评估接受地拉罗司治疗的低风险、依赖输血的骨髓增生异常综合征（MDS）患者的健康相关生活质量（HRQOL）。次要目的是研究HRQOL、血清铁蛋白水平与输血依赖性之间的关系。
这是一项前瞻性多中心研究，纳入159例患者，其中152例接受了至少一剂地拉罗司。在基线时以及随后的3、6、9和12个月，使用欧洲癌症研究与治疗组织生活质量核心问卷30（EORTC QLQ-C30）评估HRQOL。通过线性混合模型对选定量表上的平均HRQOL得分随时间进行估计，进行初步分析。
接受治疗患者的中位年龄为72岁（范围24 - 87岁）。在全球健康状况/生活质量（p = 0.564）、身体功能（p = 0.409）和疲劳（p = 0.471）量表的平均得分上，未发现随时间有统计学显著变化。此外，在便秘（p = 0.292）、腹泻（p = 0.815）以及恶心和呕吐（p = 0.643）方面也未发现显著变化。血清铁蛋白水平与HRQOL结果无关。患者报告的基线疼痛严重程度较高是较早实现输血独立的独立预测因素，风险比为1.032（99%置信区间1.00
