[show abstract][hide abstract] ABSTRACT: Promising new drugs are being evaluated for treatment of multiple myeloma (MM), but their impact should be measured against the expected outcome in patients failing current therapies. However, the natural history of relapsed disease in the current era remains unclear. We studied 286 patients with relapsed MM, who were refractory to bortezomib and were relapsed following, refractory to or ineligible to receive, an IMiD (immunomodulatory drug), had measurable disease, and ECOG PS of 0, 1 or 2. The date patients satisfied the entry criteria was defined as time zero (T0). The median age at diagnosis was 58 years, and time from diagnosis to T0 was 3.3 years. Following T0, 213 (74%) patients had a treatment recorded with one or more regimens (median=1; range 0–8). The first regimen contained bortezomib in 55 (26%) patients and an IMiD in 70 (33%). A minor response or better was seen to at least one therapy after T0 in 94 patients (44%) including partial response in 69 (32%). The median overall survival and event-free survival from T0 were 9 and 5 months, respectively. This study confirms the poor outcome, once patients become refractory to current treatments. The results provide context for interpreting ongoing trials of new drugs.Keywords: multiple myeloma; relapse; natural history; survival
[show abstract][hide abstract] ABSTRACT: Multiple myeloma is a rare, incurable hematologic malignancy. It has a tendency to develop subsequent relapses, so affected patients are likely to undergo more than one line of treatment. Rather than evaluating the cost-effectiveness of individual therapeutic agents, it becomes therefore recommendable for decision-makers—from both a clinical and an economic perspective—identifying an optimal sequencing of such agents.
Lenalidomide and bortezomib are the most used drugs in Italy for the treatment of relapsed/refractory multiple myeloma (rrMM). In the present work a cost-analysis was conducted, comparing two alternative strategies: bortezomib as a second-line therapy followed by lenalidomide+dexamethasone (len/dex) after progression (sequence A) vs len/dex followed by bortezomib (sequence B). Only drug costs were considered in the analysis.
Based on outcomes from phase III trials, total time to progression (TTP) was longer in sequence B (17.1+4.9=22 months) as compared to sequence A (7+10.6=17.6 months). Average costs per progression-free month in the respective sequences were quite comparable: €4,109 vs €4,061.
Sensitivity analysis on a couple of scenarios (assuming patient pooling when using bortezomib and assuming the association of bortezomib with pegylated liposomal doxorubicin) provided similar conclusions as regards costs.
Though sequences A and B look equivalent in terms of cost per progression-free month, it should be considered that with a longer total TTP (as in sequence B) costs for a fourth-line treatment, together with other medical costs (diagnostics, hospitalisations) following progression, are postponed; moreover, a longer TTP means a better quality of life for the concerned patient.
Despite the simplifications adopted in this study, len/dex as second-line treatment appears a recommendable strategy in the treatment of rrMM.
PharmacoEconomics - Italian Research Articles 15(1).