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CP-187 Efficacy, safety and cost of pomalidomide in relapsed and refractory multiple myeloma
  1. P Gueneau1,
  2. D Caillot2,
  3. A Cransac1,
  4. C Pernot1,
  5. M Boulin1
  1. 1University Hospital, Pharmacy Department, Dijon, France
  2. 2University Hospital, Haematology Department, Dijon, France


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

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