Pomalidomide Plus Low-Dose Dexamethasone in Relapsed/Refractory Multiple Myeloma Patients: Results of the Real-World "POWERFUL" Study

Evangelos Terpos, Panagiotis Repousis, Chrysavgi Lalayanni, Evdoxia Hatjiharissi, Theodora Assimakopoulou, Georgios Vassilopoulos, Anastasia Pouli, Emmanouil Spanoudakis, Eurydiki Michalis, Gerassimos Pangalis, Ioannis Ntanasis-Stathopoulos, Christos Poziopoulos, Marie-Christine Kyrtsonis, Vasiliki Pappa, Argiris Symeonidis, Christos Georgopoulos, Panagiotis M Zikos, Maria Gavriatopoulou, Helen A Papadaki, Magdalini Dadakaridou, Kiki Karvounis-Marolachakis, Eirini Katodritou, Evangelos Terpos, Panagiotis Repousis, Chrysavgi Lalayanni, Evdoxia Hatjiharissi, Theodora Assimakopoulou, Georgios Vassilopoulos, Anastasia Pouli, Emmanouil Spanoudakis, Eurydiki Michalis, Gerassimos Pangalis, Ioannis Ntanasis-Stathopoulos, Christos Poziopoulos, Marie-Christine Kyrtsonis, Vasiliki Pappa, Argiris Symeonidis, Christos Georgopoulos, Panagiotis M Zikos, Maria Gavriatopoulou, Helen A Papadaki, Magdalini Dadakaridou, Kiki Karvounis-Marolachakis, Eirini Katodritou

Abstract

The "POWERFUL" multicenter, retrospective, and prospective study investigated the effectiveness of pomalidomide plus low-dose dexamethasone (POM/LoDex) therapy in relapsed/refractory multiple myeloma in routine care in Greece. Ninety-nine eligible adult patients treated with POM/LoDex according to the approved label after having received ≥2 prior therapies, including lenalidomide and bortezomib, were consecutively enrolled between 16 November 2017 and 21 February 2019 in 18 hematology departments. Fifty patients (50.5%) started POM/LoDex as third-line treatment. During the treatment period (median: 8.3 months; range: 0.3-47.6 months), the median POM dose was 4 mg/day, and 31.3% of the patients received additional antimyeloma agents. The overall response rate was 32.3%. During a median follow-up period of 13.8 months (Kaplan-Meier estimate), the median progression-free survival (PFS) was 10.5 months (95% CI: 7.4-14.4). The PFS was not significantly different between patients receiving POM/LoDex in the third versus later line of therapy, nor between patients receiving concomitant antimyeloma therapy versus POM/LoDEx doublet. During the prospective safety data collection period (median: 7.6 months) among patients with prospective follow-up (N = 75), POM-related adverse event incidence rate was 42.7% (serious: 18.7%; grade ≥ 3 hematological POM-related adverse events: 8.0%). Only neutropenia (13.3%) was reported at a frequency ≥10%. In conclusion, in this real-world study, POM/LoDex displayed a long PFS with no new safety signals emerging.

Keywords: ORR; PFS; duration of response; lenalidomide; multiple myeloma; pomalidomide; refractory.

Conflict of interest statement

E.T.: Amgen: research funding, honoraria, Janssen: research funding, honoraria, Takeda: research funding, honoraria, Genesis Pharma: research funding, honoraria, Celgene: honoraria, Sanofi: honoraria, BMS: honoraria; P.R.: Genesis Pharma: research funding, advisory board; CL: Genesis Pharma SA: honoraria, research funding, Janssen: advisory board, Amgen: honoraria, Astellas: honoraria, Abbvie: advisory board; E.H.: Genesis Pharma SA: honoraria, research funding, advisory board, Janssen: honoraria, advisory board, Abbvie: honoraria, Gilead: advisory board, Roche: honoraria; T.A.: Genesis Pharma SA: research funding; G.V.: Genesis Pharma SA: advisory board, research funding, honoraria, Janssen: advisory board, Novartis: advisory board, Abbvie: advisory board, Gilead: research funding; A.P.: Genesis Pharma SA: research funding; E.S.: Genesis Pharma SA: research funding; E.M.: Genesis Pharma SA: research funding, Takeda: research funding; G.P.: Genesis Pharma SA: research funding; C.P.: Genesis Pharma SA: research funding; M.-C.K.: Genesis Pharma SA: research funding, advisory board, honoraria; V.P.: Genesis Pharma SA: research funding; A.S.: Genesis Pharma SA: research funding, honoraria, advisory board; C.G.: Genesis Pharma SA: research funding; P.M.Z.: Genesis Pharma SA: research funding, advisory board; M.G.: Amgen, Karyopharm, Genesis Pharma, Janssen, and Takeda; HAP: Genesis Pharma SA: research funding, Gilead: advisory board, Abbvie: advisory board, Janssen research and development, LLC: research grant; M.D.: Genesis Pharma SA: research funding; K.K.-M.: Genesis Pharma SA: employment; E.K.: Genesis Pharma: honoraria, research funding, Amgen: honoraria, advisory boards, research funding, Janssen-Cilag: honoraria, advisory boards, research funding, Takeda: honoraria, research funding. The other authors report no conflict of interest.

Figures

Figure 1
Figure 1
Patient disposition in the study.
Figure 2
Figure 2
Reasons for pomalidomide (POM) treatment modifications and permanent discontinuation. (a) Reasons for POM dose reductions; (b) reasons for POM temporary interruptions; (c) distribution of patients who permanently discontinued POM/low-dose dexamethasone (LoDex) by reason for discontinuation.
Figure 3
Figure 3
Progression-free survival (PFS) estimation by Kaplan–Meier analysis: (a) PFS analysis in the overall eligible population; (b) PFS analysis in subpopulations of patients initiated on POM/LoDex in the third versus a later line setting; (c) PFS analysis in subpopulations of patients receiving POM/LoDex only versus those receiving additional antimyeloma agents.

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