Analysis of Common Eligibility Criteria of Randomized Controlled Trials in Newly Diagnosed Multiple Myeloma Patients and Extrapolating Outcomes

作者：Shah Jatin J; Abonour Rafat; Gasparetto Christina; Hardin James W; Toomey Kathleen; Narang Mohit; Srinivasan Shankar; Kitali Amani; Zafar Faiza; Flick E Dawn; Rifkin Robert M^*

来源：Clinical Lymphoma Myeloma & Leukemia, 2017, 17(9): 575-+.

DOI：10.1016/j.clml.2017.06.013

• Baseline disease characteristics
• Clinical trial design
• Community practice
• Connect MM registry
• NDMM

The strict exclusion criteria of clinical trials in multiple myeloma (MM) limit the enrollment of patients reflective of a general patient population. Using the Connect MM Registry data representative of an unselected newly diagnosed MM population, 563 of 1406 patients (40.0%) were identified as ineligible for clinical trials. This study provides insight into potential modifications of standard eligibility criteria that can lead to improved trial design.
Background: The performance of multiple myeloma (MM) therapies in a general patient population and specific eligibility criteria that might limit enrollment into randomized controlled trials (RCTs) have not been evaluated in depth. This study aimed to determine if improvements seen with MM therapies in RCTs are reflected in the general patient population and to identify eligibility criteria that can be modified to increase enrollment. Patients and Methods: The Connect MM Registry is a prospective observational cohort study of patients with newly diagnosed MM (NDMM) in the United States. Using common RCT exclusion criteria collected from 16 published studies, patients in the registry were categorized according to their eligibility for inclusion in RCTs. Results: On the basis of common criteria, 563 of 1406 of registry patients (40.0%) are ineligible for RCTs. Criteria leading to exclusion included M-protein <= 1.0 g/dL (25.2%), creatinine > 2.5 mg/dL (13.9%), low absolute neutrophil count (10.0%), and low hemoglobin (9.6%). Significantly more RCT-ineligible versus RCT-eligible patients had hypercalcemia (11.0% vs. 5.5%), elevated creatinine levels (38.9% vs. 6.2%), low hemoglobin levels (59.5% vs. 39.5%), or International Staging System stage III disease (40.1% vs. 22.1%; P <.001 for all comparisons). RCT-ineligible patients had a lower 3-year survival rate than RCT-eligible patients (63% vs. 70%). The incidence of serious adverse events was similar between groups. Conclusion: Of patients with NDMM enrolled in the Connect MM Registry, 40% are ineligible for RCTs. This study provides insight into potential modifications of standard eligibility criteria that can lead to improved RCT design and accelerated enrollment.

• 出版日期2017-9