OBJECTIVES:

Despite new treatments, Relapsed-Refractory Multiple Myeloma (RRMM) remains an incurable disease with survival less than 12 months¹. In a recent study by Moreau, et al, a relationship between response and survival was demonstrated in RRMM patients treated with pomalidomide². Understanding the relationships between initial response and long-term prognosis can inform patient treatment or guide new compound development. Expanding on the work of Moreau, et al, we explore this relationship in a standardized clinical trial patient pool to assess applicability of findings across multiple studies.

METHODS: A retrospective pooled analysis was conducted in a Study Data Tabulation Model (SDTM) dataset from the Medidata Enterprise Data Store. Subjects were selected based on inclusion/exclusion criteria from the NIMBUS trial². Response, Progression-free survival (PFS), and Overall Survival (OS) were extracted. Patients were stratified by covariates including age, gender, and prior regimens. Log-rank tests were conducted to compare PFS and OS in patient sub-populations. Survival was assessed at 90, 180, and 240 days from most recent treatment start. Cox proportional hazard models assessed survival predictors. Rates were estimated for common adverse events, including leukopenia, neutropenia, and thrombocytopenia. Factors associated with neutropenia were assessed using logistic regression.

RESULTS: Within the pooled analysis, survival rates were consistent with published literature rates, at ~4 months and ~12 months, respectively. Pooled analysis demonstrated a significant association between response, PFS, and OS. Results were consistent with findings of Moreau, et al, showing little difference between Stable Disease and Partial Response, and lower overall survival in patients with Progressive Disease versus Stable Disease. Neutropenia was seen in one-fourth of overall patients, and was associated with male gender, age, and treatment regimen.

CONCLUSIONS: The use of SDTM for pooled clinical trial analyses can overcome individual trial limitations, expanding sample size, population ranges, relative treatment outcomes, and safety event rates that can be studied