Real World Results May Not Mirror Clinical Study Outcomes in Multiple Myeloma
New MM treatment modalities have largely been evaluated in prospective clinical studies with strict inclusion criteria for trial-eligible patients.
Survival outcomes in “real world” practice may differ among nontransplant patients with multiple myeloma who receive routine care compared with clinical trials, according to a study published in the Annals of Hematology.
Although the treatment of multiple myeloma has advanced in recent years, new treatment modalities have largely been evaluated in prospective clinical studies with strict inclusion criteria for trial-eligible patients. How much these results translate to real world practice requires further investigation.
For the prospective TLN cohort study, researchers assessed the outcomes of 3795 patients with hematological malignancies after first- or second-line therapy; 285 non-transplant patients received systemic treatment for multiple myeloma. Trial ineligibility criteria included heart/renal failure, liver/renal diseases, polyneuropathy, and HIV-positive status; the remaining study patients (30%) were considered trial-eligible.
Regardless of the intervention, the median progression-free survival (PFS) and overall survival (OS) were significantly prolonged among patients who were trial-eligible. The PFS of trial ineligible patients was 16.2 months compared with 27.3 months for those who were trial eligible.
Median OS was 34.2 months vs 58.6 months among trial-ineligible and eligible patients, respectively, and the 3-year OS rate was 44.4% and 69.4%, respectively.
Although treatment algorithms were similar, survival outcomes varied significantly between trial-eligible and trial-ineligible patients. Results may not always mirror clinical studies, especially when viewed through the scope of real world application and a more diverse study population. The authors concluded that “clinical trials specifically addressing this commonly excluded comorbid group of patients are needed to develop optimized treatment strategies.”
Knauf W, Aldaoud A, Hutzschenreuter U, et al. Survival of non-transplant patients with multiple myeloma in routine care differs from that in clinical trials—data from the prospective German Tumour Registry Lymphatic Neoplasms[published online August 1, 2018]. Ann Hematol. doi: 10.1007/s00277-018-3449-8