Boquoi Amelie, Rings Veronika, Mohring Annemarie, Savickaite Ingrida, Zukovs Romans, Strapatsas Judith, Nachtkamp Kathrin, Kobbe Guido, Germing Ulrich, Fenk Roland
Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Heinrich Heine University, 40225 Duesseldorf, Germany.
Cancers (Basel). 2022 Apr 26;14(9):2147. doi: 10.3390/cancers14092147.
Randomized controlled trials (RCT) are the driver of therapeutic innovations. However, it has been frequently shown that less than 5% of adult cancer patients enroll in clinical trials, although 70% of patients are considered as being willing to participate. Barriers to trial participation have been extensively studied. Although there is evidence that trial participation correlates with improved survival and reduced mortality, the rate of participation has not changed substantially. We provide retrospective data from a single-center analysis of 411 patients with multiple myeloma (MM) who were treated at the University Hospital Duesseldorf in Germany between January 2014 and December 2016. Each patient was analyzed for the real-world possibility of participating in a clinical study, based on the inclusion and exclusion (I/E) criteria and the recruiting period of open studies. The overall rate of study participation was 19%. A total of 53% of NDMM patients were eligible for first-line studies (GMMG-HD6, LenaMain). Of these, 80% consented to enrolment (42% of all). In contrast, only 38% of the RRMM population was eligible (GMMG-Relapse, Castor, Tourmaline, Admyre). Of these, only 22% (7% of all) consented. This was confirmed by virtual analysis, showing that only 29% of all RRMM patients would have been eligible for six internationally recruiting trials leading to later drug approval. The majority of cases were rendered ineligible by only one I/E criterion. The most common criteria were study-specific (prior therapies or refractory disease to a specific drug), kidney disease, and previous malignancy, followed by internal, neurologic, and infectious disease. In summary, this single-center analysis showed that I/E criteria permit study participation for most NNDM patients, with a dramatic decrease in the RRMM population. This is aggravated by the fact that the willingness for study participation also significantly declines in RRMM. Thus, addressing patient expectations and priorities seems to be the most promising approach to increasing patient enrollment in clinical trials.
随机对照试验（RCT）是治疗创新的驱动力。然而，经常有研究表明，尽管70%的成年癌症患者被认为愿意参与临床试验，但实际参与临床试验的患者不到5%。试验参与的障碍已得到广泛研究。虽然有证据表明参与试验与生存率提高和死亡率降低相关，但参与率并未有实质性变化。我们提供了对411例多发性骨髓瘤（MM）患者的单中心回顾性分析数据，这些患者于2014年1月至2016年12月在德国杜塞尔多夫大学医院接受治疗。根据纳入和排除（I/E）标准以及开放研究的招募期，对每位患者参与临床研究的现实可能性进行了分析。总体研究参与率为19%。共有53%的新诊断多发性骨髓瘤（NDMM）患者符合首次研究（GMMG-HD6，LenaMain）的条件。其中，80%同意入组（占全部患者的42%）。相比之下，复发/难治性多发性骨髓瘤（RRMM）患者中只有38%符合条件（GMMG-复发，Castor，Tourmaline，Admyre）。其中，只有22%（占全部患者的7%）同意入组。虚拟分析证实，在所有RRMM患者中，只有29%符合六项导致后续药物获批的国际招募试验的条件。大多数病例仅因一项I/E标准而不符合条件。最常见的标准是研究特定标准（先前的治疗或对特定药物的难治性疾病）、肾脏疾病和既往恶性肿瘤，其次是内科疾病、神经疾病和感染性疾病。总之，这项单中心分析表明，I/E标准允许大多数NDMM患者参与研究，但RRMM患者群体的参与率大幅下降。RRMM患者参与研究的意愿也显著下降，这加剧了这一情况。因此，满足患者的期望和优先事项似乎是增加患者参与临床试验的最有前景的方法。
