Table 1 Rates of clinical trial eligibility reported from real-world and registry studies of patients with MM.
Reference | Study/Registry | N | Region | Criteria used | Ineligible patients, % | Outcomes: ineligible vs eligible patients |
|---|---|---|---|---|---|---|
Shah et al.6 | Connect MM® | 1406 | United States | Common exclusion criteria from 24 RCTs, including absence of measurable disease, inadequate renal/hepatic/hematologic function, ECOG performance status 3/4, and history of other malignancy | 40.0–56.8a | 3-Year OS: 63% vs 70% (HR 0.81, 95% CI 0.67–0.99, p = 0.0392) |
Fiala et al.8 | CoMMpass | 848 | 76 Sites worldwide, primarily in the United States, Canada, Italy, and Spain† | ECOG performance status 3/4, inadequate renal/hematologic function | 22 | 91% Increase in risk of progression or death in ineligible patients (adjusted HR 1.91, 95% CI 1.45–2.52, p < 0.0001) |
Chari et al.7 | EHR database analysis | 1265 | United States | Eligibility criteria for ASPIRE, TOURMALINE-MM1, POLLUX, ELOQUENT-2, CASTOR, ENDEAVOR | 47.9–72.3 | With Rd treatment: 3-year OS: 63.5% vs 74.7% (mortality HR 1.46, 95% CI 1.03–2.07, p = 0.0329) With Vd treatment: 3-year OS: 46.2% vs 61.0% (mortality HR 1.51, 95% CI 1.14–2.01, p = 0.0045) |
Knauf et al.9 | German Tumour Registry Lymphatic Neoplasms | 285 | Germany | Heart failure, renal failure, other renal diseases, liver diseases, polyneuropathy, HIV positive | 32 | 3-Year PFS: 18.8% vs 39.9% 3-year OS: 44.4% vs 69.4% 2-year DSS: 69.3% vs 88.4% |
Hungria et al.10 | INSIGHT MM | 3201 | EMEA 50.4%, North America 31.3%, Latin America 11.5%, Asia-Pacific 6.8% | 20 Standard RCT ineligibility criteria, including inadequate renal/hepatic/hematologic function, ECOG performance status >2, hypercalcemia, cardiac medical history, chronic pulmonary disease, cerebrovascular disease, and history of other malignancy | 39.2 | Median TTNT (patients with CRAB symptoms): NDMM patients: 18.0 months vs NE RRMM patients: 14.9 vs 21.2 months |
Klausen et al.11 | Danish Multiple Myeloma Registry | 1425 | Denmark | Common eligibility criteria for non-transplant/elderly patients in the HOVON 87, VISTA, FIRST, and SWOG S0777 phase 3 studies | 63.4 | Median OS: 21.3 vs 44.0 months (HR 1.7, 95% CI 1.5–1.9, p < 0.0001) |
Common eligibility criteria for non-transplant/elderly patients in the ALCYONE phase 3 study | 66.2 | HR 1.7, 95% CI 1.5–1.9, p < 0.0001 | ||||
764 | Denmark | Common eligibility criteria for transplant-eligible patients in the STaMINA, IFM2013–04, IFM DFCI 2009, and EMN02 phase 3 studies | 54.8 | Median OS: 65.5 vs 78.4 months (HR 1.3, 95% CI 1.0–1.5, p = 0.021) |
