Department of Hematology, Amsterdam UMC, location VUmc, Vrije Universiteit, Cancer Center Amsterdam, Amsterdam, The Netherlands.
Department of Biostatistics, Erasmus MC, Rotterdam, The Netherlands.
Br J Haematol. 2022 Jan;196(2):329-335. doi: 10.1111/bjh.17892. Epub 2021 Oct 10.
Most patients with myelodysplastic syndromes (MDS) require therapeutic intervention. However, there are few approved treatments for MDS. To explore reasons, we searched clinicaltrials.gov and clinicaltrialsregister.eu for MDS trials from 2000 to 2020. We assessed which agents were under investigation and analysed clinical trial characteristics and continuation rates from phase I to II to III to approval. As such, we identified 384 unique agents in 426 phase I, 430 phase II and 48 phase III trials. Success rates for phase III trials and agents were low, and MDS trials took markedly longer to complete than the average clinical trial. Although success rates were higher when MDS-specific phase I trials were conducted, 52% of the agents had not been evaluated in a phase I trial for MDS. MDS trials often failed to include quality of life, an especially important outcome for older MDS patients. Our work identifies factors potentially contributing to the paucity of available agents for MDS. We suggest a framework to improve clinical research in MDS that might ultimately augment the number of available agents.
大多数骨髓增生异常综合征 (MDS) 患者需要治疗干预。然而，MDS 的治疗方法却寥寥无几。为了探究原因，我们在 clinicaltrials.gov 和 clinicaltrialsregister.eu 上检索了 2000 年至 2020 年的 MDS 临床试验。我们评估了正在研究的哪些药物，并分析了从 I 期到 II 期到 III 期再到批准的临床试验特征和持续率。因此，我们在 426 项 I 期、430 项 II 期和 48 项 III 期试验中确定了 384 种独特的药物。III 期试验和药物的成功率较低，而且 MDS 试验完成时间明显长于平均临床试验。尽管当进行专门针对 MDS 的 I 期试验时成功率更高，但 52%的药物尚未在 MDS 的 I 期试验中进行评估。MDS 试验往往未能纳入生活质量，这是老年 MDS 患者的一个特别重要的结果。我们的工作确定了可能导致 MDS 可用药物稀缺的因素。我们建议建立一个框架来改进 MDS 的临床研究，这可能最终会增加可用药物的数量。
