Although several treatment options for multiple myeloma (MM) have become available in recent years, there is limited evidence regarding real-world treatment outcomes associated with these treatments. PREAMBLE (Prospective Research Assessment in Multiple Myeloma: An Observational Evaluation), a multinational observational study, seeks to address this evidence gap by assessing the impact of prior transplantation experience on real-world clinical effectiveness of immunomodulatory drugs (IMiDs), proteasome inhibitors (PIs), and combination therapy for relapsed/refractory MM (RRMM). The results of preliminary efficacy analyses were reported at the ASH 2016 Annual Meeting.
This analysis included patients with a confirmed diagnosis of RRMM with 1 prior treatment who started treatment with an IMiD, PI, or IMiD+PI. Response rate, time in response, progression-free survival (PFS), and overall survival (OS) were evaluated from patient data collected at each healthcare provider visit for a follow-up period of 3 years. Of 924 enrolled and treated patients, 395 (43%) had 1 prior line of therapy (median age, 70 years; 56% male) with an IMiD (n = 208), a PI (n = 156), or IMiD+PI (n = 31). Median follow-up was 19 months, and 229 remained on study. Discontinuations due to death occurred for 100 patients. Most of these were due to disease progression; none were caused by drug toxicity. Approximately 30% had prior transplantation experience. In patients without transplantation experience (n = 257; 65%), the response rate (95% confidence interval) was similar between patients with (43%) and without prior transplantation experience (46%). Overall, the median time in response was longer in patients with prior transplantation (21 months) than in those with prior transplantation (16 months). Patients receiving an IMiD as index therapy had better outcomes than patients receiving a PI, regardless of prior transplantation status. Patients with prior transplantation achieved longer median PFS than patients without (14.1 vs 12.2 months). OS rate at 12 months was similar regardless of prior transplantation status.
Researchers observed loss of response over time, highlighting the continuing unmet medical need in RRMM, regardless of index therapy type or prior transplantation experience. Collectively, the data underscore the importance of novel therapies that can provide durable responses and improve treatment outcomes for patients with RRMM. The PREAMBLE study is ongoing, and additional findings will be reported in the future.
Durie B, et al. ASH 2016. Abstract 2403.