Multiple Myeloma Population in Clinical Trials Does Not Reflect Real World Patients

Previous research has indicated that many patient groups are underrepresented in cancer clinical trials. Researchers analyzed patient eligibility in the INSIGHT MM study, the largest prospective, observational multiple myeloma (MM) trial to date and found that nearly 40% of INSIGHT MM patients would be ineligible for randomized, controlled trials (RCTs), which is a higher rate than has been previously reported. The results of the study were presented by Dawn Marie Stull, PharmD, BCOP, scientific director within global medical affairs oncology at Takeda Oncology, Millennium Pharmaceuticals, Inc., at HOPA’s 16th Annual Conference.

The INSIGHT MM trial included more than 4,300 patients with MM from 15 countries; at data cutoff in the current study, 3,201 patients were eligible for analysis (1,761 with newly diagnosed MM and 1,440 with relapsed/refractory MM). The researchers reviewed patient data and compared it with 20 standard RCT laboratory and performance status and medical history exclusion criteria.

Among the INSIGHT MM cohort, 39.2% of patients would have been ineligible for RCTs for not meeting one or more eligibility criteria, including 38.8% of newly diagnosed patients and 39.7% of relapsed/refractory patients.

The most common exclusionary criteria were another prior malignancy (7.5%), creatine clearance ≤30 mL/min (6.4%), cardiac arrhythmias (5.4%), platelets ≤75,000/mm3 (5.1%), hemoglobin <8.0 g/dL (4.8%), and chronic pulmonary disease (4.7%).

The researchers noted that when applying International Myeloma Working Group frailty index parameters, 320 patients (10%) were deemed frail in the INSIGHT MM trial, and 74.7% would have been ineligible for RCTs according to the 20 exclusionary criteria studied.

Among the 126 evaluable frail patients receiving first-line therapy, 30 (24%) received doublet therapy and 83 (66%) received triplet therapy; median durations of treatment (DOT) were 5.4 months and 6.2 months, respectively. Among the 48 frail patients receiving second- through fourth-line treatment, 26 (54%) and 21 (44%) received doublet and triplet therapy, and median DOT were 4.4 months and 5.3 months, respectively.

“These findings emphasize the importance of real-world data for evaluating effectiveness of treatment options,” the researchers concluded. “Continued initiatives to broaden oncology clinical trial eligibility are important.”

Hungria VTM, Lee HC, Abonour R, et al. Analysis of over 3,000 patients from the INSIGHT MM global, prospective, observational study shows real-world multiple myeloma patients are under-represented in clinical trials based on standard laboratory parameters and baseline characteristics. Presented at HOPA 16th Annual Conference. March 2020, Tampa, Florida.