Background Patients with relapsed and refractory multiple myeloma (RRMM) have a median survival of about 3–6 months.¹

Purpose The aim of the study was to analyse efficacy, safety and cost of pomalidomide in patients with RRMM.

Material and methods All patients in whom a treatment by pomalidomide was initiated between August 2013 and October 2015 for a RRMM in our hospital were included. Outcomes were predictive factors of early pomalidomide discontinuation (before the third month), overall response rate (ORR, using International Multiple Myeloma Working Group criteria²), overall survival (OS), safety and treatment cost.

Results 63 patients (mean age 61 years) were included. All patients received pomalidomide with dexamethasone. Pomalidomide was discontinued early in 17 (27%) patients. Time from diagnosis to pomalidomide initiation <3 years was independently associated with early pomalidomide discontinuation (OR=5.82; 95%CI 1.51–22.4; p=0.01). At 3 months, ORR was 51%. After a median follow-up of 15.1 months, 54 (86%) patients had discontinued pomalidomide. Median OS from pomalidomide initiation was 6.4 (95% CI 3.7–not achieved) months in patients who discontinued pomalidomide early versus 17.1 (95% CI 9.4–not achieved) months in patients with a stable disease versus not achieved (95% CI 9.4–not achieved) in responders (log rank; p=0.004). The independent risk factors of mortality from pomalidomide initiation were: early pomalidomide discontinuation (hazard ratio 6.8 vs no early discontinuation; 95% CI 2.3–19.6; p<10^-3) and a haemoglobin level below 11g/dL (hazard ratio 2.7 vs ≥11 g/dL; 95% CI 1.0–7.0; p=0.04). The most common grade ≥3 adverse events were neutropenia (14%) and infections (25%). The mean pomalidomide cost per patient was €79 717±46 296 (range €17 850–214 200).

Conclusion Compared with the MM-003 phase III trial,³ we reported similar safety data but a higher ORR (51% vs 21%). We demonstrated the long term favourable safety and efficacy profile of pomalidomide in RRMM patients, even in those with stable disease.

References and/or acknowledgements

1. Kumar SK, et al. Risk of progression and survival in multiple myeloma relapsing after therapy with ImiDs and bortezomib. Leukaemia2001;226:149–57.

2. Rajkumar SV, et al. Consensus recommendations for the uniform reporting of clinical trials. Blood2011;117:4691–5.

3. Miguel JS, et al. Pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone alone for patients with relapsed and refractory multiple myeloma (MM-003). Lancet Oncol2013;14:1055–66.

References and/or acknowledgementsNo conflict of interest