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注射用低甲基化剂治疗骨髓增生异常综合征:患者对治疗的看法。

Injectable Hypomethylating Agents for Management of Myelodysplastic Syndromes: Patients' Perspectives on Treatment.

机构信息

Section of Hematology, Department of Medicine, Yale School of Medicine, and Yale Cancer Center, New Haven, CT.

OPEN Health, Bethesda, MD.

出版信息

Clin Lymphoma Myeloma Leuk. 2022 Mar;22(3):e185-e198. doi: 10.1016/j.clml.2021.09.009. Epub 2021 Sep 11.

DOI:10.1016/j.clml.2021.09.009
PMID:34674983
Abstract

BACKGROUND

Until recently, patients with MDSs could receive HMAs via intravenous (IV) or subcutaneous (SC) administration. An oral HMA was recently approved as an alternative to IV/SC administration. This study assessed the impact of IV/SC HMA on MDS patients, and their experience of, challenges with, and views about oral MDS treatment.

PATIENTS AND METHODS

We conducted an online cross-sectional survey among adult MDS patients (or caregivers as proxies) invited by 2 U.S. MDS patient advocacy groups. Patients were required to have received IV/SC HMA (ie, azacitidine or decitabine) within 6 months of the survey.

RESULTS

The survey was completed by 141 participants (120 patients, 21 caregiver proxies). Median patient age was 63.0 years, 53.9% were women, and 19.8%, 62.4%, and 17.7% had lower-, higher-, or unknown risk scores, respectively. HMA treatments received included SC azacitidine (37%), IV azacitidine (36%), and IV decitabine (27%). Among 89 IV HMA recipients, 74.2% and 69.7% reported treatment-related interference with their social and daily activities, respectively, and 66.3% reported pain related to treatment administration. Following an injection, SC HMA recipients reported pain (94.2%) and interference with daily (86.5%) and social (80.8%) activities. Among the 49.6% of patients who were working, 61.4% felt less productive due to treatment. Most (69.5%) MDS patients indicated they would prefer oral MDS treatment to IV/SC therapies.

CONCLUSION

Patients receiving IV/SC HMAs experienced pain/discomfort and interference with social and daily activities. The introduction of an oral HMA may alleviate some treatment challenges for MDS patients.

摘要

背景

直到最近,骨髓增生异常综合征 (MDS) 患者还可以通过静脉 (IV) 或皮下 (SC) 给药接受 HMAs。最近批准了一种口服 HMA 作为 IV/SC 给药的替代方案。本研究评估了 IV/SC HMA 对 MDS 患者的影响,以及他们对 MDS 口服治疗的体验、面临的挑战和观点。

患者和方法

我们邀请了两个美国 MDS 患者倡导组织的成年 MDS 患者(或作为代理人的护理人员)进行了一项在线横断面调查。患者需要在调查前 6 个月内接受过 IV/SC HMA(即阿扎胞苷或地西他滨)治疗。

结果

共有 141 名参与者(120 名患者,21 名护理人员代理人)完成了调查。患者的中位年龄为 63.0 岁,53.9%为女性,分别有 19.8%、62.4%和 17.7%的患者具有低、中高和未知风险评分。接受的 HMA 治疗包括 SC 阿扎胞苷(37%)、IV 阿扎胞苷(36%)和 IV 地西他滨(27%)。在 89 名接受 IV HMA 治疗的患者中,分别有 74.2%和 69.7%的患者报告治疗相关的社会和日常活动干扰,分别有 66.3%的患者报告与治疗管理相关的疼痛。在接受注射后,SC HMA 治疗的患者报告疼痛(94.2%)和日常活动(86.5%)和社会活动(80.8%)干扰。在 49.6%的工作患者中,61.4%的患者因治疗而工作效率降低。大多数(69.5%) MDS 患者表示,他们希望 MDS 口服治疗能替代 IV/SC 治疗。

结论

接受 IV/SC HMAs 治疗的患者经历了疼痛/不适和社会及日常活动干扰。口服 HMA 的引入可能会减轻 MDS 患者的一些治疗挑战。

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