The proportion of non-transplant-eligible (NTE) newly diagnosed multiple myeloma (NDMM) patients excluded from clinical trials (CTs) and their prognosis is unknown. CT results may not be generalizable to real-world practice due to strict recruitment criteria. We analyzed causes of NTE-NDMM patient exclusion form CTs and their outcomes. A total of 211 NTE-NDMM patients were included. They were divided into three periods: 2003–2007, 2008–2012, and 2013–2017. Overall, 50% received non-trial treatment (NCT), while 50% participated in a CT (20% control group (CG) and 30% experimental group (EG)). Main causes for exclusion from CTs were comorbidities, ECOG > 2, and renal insufficiency. In the first two periods, the CR rate was similar regardless of treatment type, but in the last period, the EG group showed improved CR. Median PFS was similar in the first two periods, with a benefit seen only in the EG in the last period. The median OS was significantly longer in CT-included patients compared to NCT group in the last two periods. Conclusions: The presence of comorbidities and worsened ECOG were the main reasons for CT exclusion. Patients included in CTs had a longer OS than NCT. This OS benefit may be influenced by a selection bias, making it challenging to generalize CT results to real clinical practice.
不适合移植的新诊断多发性骨髓瘤患者被临床试验排除的比例及其预后尚不明确。由于严格的入组标准，临床试验结果可能无法推广至真实世界临床实践。本研究分析了不适合移植新诊断多发性骨髓瘤患者被临床试验排除的原因及其临床结局。共纳入211例不适合移植新诊断多发性骨髓瘤患者，按时间分为三个时期：2003-2007年、2008-2012年及2013-2017年。总体而言，50%患者接受非临床试验治疗，50%参与临床试验（其中20%进入对照组，30%进入实验组）。排除临床试验的主要原因为合并症、ECOG评分＞2及肾功能不全。前两个时期中，无论治疗类型如何，完全缓解率相似；但在最后一个时期，实验组显示出更高的完全缓解率。前两个时期的中位无进展生存期相似，仅在最后一个时期的实验组观察到生存获益。在后两个时期中，纳入临床试验的患者中位总生存期显著长于非临床试验组。结论：合并症的存在及ECOG评分恶化是排除临床试验的主要原因。纳入临床试验的患者总生存期优于非临床试验组。这种总生存获益可能受选择偏倚影响，使得临床试验结果难以推广至真实临床实践。
肿瘤(癌症)患者之家