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

Abstract background The performance of multiple myeloma (MM) therapies in a general patient population and specific eligibility criteria that may limit enrollment into randomized controlled trials (RCTs) have not been evaluated in depth. This study aimed to determine if improvements seen with MM the...

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Published in:	Clinical lymphoma, myeloma and leukemia myeloma and leukemia, 2017-09, Vol.17 (9), p.575-583.e2
Main Authors:	Shah, Jatin J., MD, Abonour, Rafat, MD, Gasparetto, Christina, MD, Hardin, James W., PhD, Toomey, Kathleen, MD, Narang, Mohit, MD, Srinivasan, Shankar, PhD, Kitali, Amani, MPH, Zafar, Faiza, MPAS, Flick, E. Dawn, PhD, Rifkin, Robert M., MD
Format:	Article
Language:	eng
Subjects:	Adult Aged Aged, 80 and over Baseline disease characteristics Biomarkers Clinical trial design Community practice Connect MM registry Female Guideline Adherence Hematology, Oncology and Palliative Medicine Humans Male Middle Aged Multiple Myeloma - diagnosis Multiple Myeloma - epidemiology Multiple Myeloma - mortality Multiple Myeloma - therapy NDMM Neoplasm Staging Randomized Controlled Trials as Topic Registries Research Design Treatment Outcome Young Adult
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Summary:	Abstract background The performance of multiple myeloma (MM) therapies in a general patient population and specific eligibility criteria that may 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 US. 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, 40.0% (563/1406) of registry patients 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 vs RCT-eligible patients had hypercalcemia (11.0% vs 5.5%), elevated creatinine (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.
ISSN:	2152-2650 2152-2669