Yueyang Hospital of Integrated Traditional Chinese and Western Medicine, Shanghai University of Traditional Chinese Medicine, Shanghai, China.
Ann Hematol. 2022 Oct;101(10):2219-2229. doi: 10.1007/s00277-022-04950-4. Epub 2022 Aug 17.
Thrombocytopenia is a common and unsolved problem in myelodysplastic syndrome (MDS) patients; we aimed to summarize the evidence of TPO-RA treatment for heath-related quality of life (HRQoL) and platelet transfusion burden of MDS patients. We searched Pubmed, Web of Science, EMBASE, and CENTRAL for randomized clinical trials (RCTs) comparing TPO-RA to placebo in MDS published until July 31, 2021. A random-effect model was used. Eight RCTs with 908 patients were identified. Only three RCTs involving eltrombopag reported HRQoL, and all three studies treated HRQoL as a secondary outcome. In these three RCTs, the HRQoL instruments used in each study were different. However, this outcome cannot be meta-analyzed because some studies did not provide complete data. Subsequent clinical trials should pay more attention to this. Compared to placebo, TPO-RA did not affect platelet transfusion incidence 0.83 (95% CI 0.60-1.15). There was no evidence for subgroup differences in the analyses of different types of TPO-RA, different additional agent, and different types of MDS risk groups. However, platelet transfusion units (RR = 0.68, 95% CI 0.53 to 0.84) were significantly decreased. The RR of patients who did not require platelet transfusion for 56 or more consecutive days was not different between groups (RR = 0.98, 95% CI 0.41 to 2.34). TPO-RA may decrease platelet transfusion units in MDS patients with thrombocytopenia. But the significance of this finding should be interpreted with caution, because too few studies were meta-analyzed.
血小板减少症是骨髓增生异常综合征(MDS)患者中常见且未解决的问题;我们旨在总结 TPO-RA 治疗 MDS 患者健康相关生活质量(HRQoL)和血小板输注负担的证据。我们在 Pubmed、Web of Science、EMBASE 和 CENTRAL 中搜索了截至 2021 年 7 月 31 日比较 TPO-RA 与安慰剂治疗 MDS 的随机临床试验(RCT)。使用随机效应模型。共确定了 8 项涉及 908 名患者的 RCT。只有 3 项涉及艾曲泊帕的 RCT 报告了 HRQoL,并且所有 3 项研究均将 HRQoL 作为次要结局。在这 3 项 RCT 中,每项研究使用的 HRQoL 工具不同。然而,由于一些研究没有提供完整的数据,因此无法对此进行荟萃分析。后续临床试验应更加关注这一点。与安慰剂相比,TPO-RA 并未影响血小板输注发生率 0.83(95%CI 0.60-1.15)。在分析不同类型的 TPO-RA、不同的附加药物和不同类型的 MDS 风险组时,没有证据表明存在亚组差异。然而,血小板输注单位(RR=0.68,95%CI 0.53 至 0.84)显著减少。不需要连续 56 天或更长时间输注血小板的患者比例在两组之间没有差异(RR=0.98,95%CI 0.41 至 2.34)。TPO-RA 可能减少 MDS 血小板减少症患者的血小板输注单位。但应谨慎解释这一发现的意义,因为进行荟萃分析的研究太少。