Pomalidomide, cyclophosphamide, and prednisone for relapsed/refractory multiple myeloma: a multicenter phase 1/2 open-label study
Alessandra Larocca, Vittorio Montefusco, Sara Bringhen, Davide Rossi, Claudia Crippa, Roberto Mina, Monica Galli, Magda Marcatti, Giacinto La Verde, Nicola Giuliani, Valeria Magarotto, Tommasina Guglielmelli, Delia Rota-Scalabrini, Paola Omedé, Alberto Santagostino, Ileana Baldi, Angelo Michele Carella, Mario Boccadoro, Paolo Corradini, Antonio Palumbo, Alessandra Larocca, Vittorio Montefusco, Sara Bringhen, Davide Rossi, Claudia Crippa, Roberto Mina, Monica Galli, Magda Marcatti, Giacinto La Verde, Nicola Giuliani, Valeria Magarotto, Tommasina Guglielmelli, Delia Rota-Scalabrini, Paola Omedé, Alberto Santagostino, Ileana Baldi, Angelo Michele Carella, Mario Boccadoro, Paolo Corradini, Antonio Palumbo
Abstract
We performed a phase 1/2 trial to determine the maximum tolerated dose (MTD) of pomalidomide and to explore its efficacy when combined with cyclophosphamide-prednisone in relapsed/refractory myeloma patients. Pomalidomide was given at 1 to 2.5 mg/d, cyclophosphamide at 50 mg every other day, prednisone at 50 mg every other day, for 6 28-day cycles, followed by pomalidomide-prednisone maintenance therapy. Thromboprophylaxis was recommended. Sixty-nine patients were enrolled, 55 received the MTD (2.5 mg/d) and were evaluated. Best responses included complete response in 3 patients (5%), very good partial response in 10 (18%), partial response in 15 (27%), minimal response in 11 (20%), stable disease in 15 (27%), and progressive disease in 1 (3%), for an overall response rate of 51%. The median time-to-response was 1.83 months. After a median follow-up of 14.8 months, median progression-free survival was 10.4 months and 1-year overall survival was 69%. At the MTD, grade 3 to 4 toxicities included anemia (9%), thrombocytopenia (11%), neutropenia (42%), neurologic events (7%), dermatologic events (7%), and thromboembolism (2%). Grade 3 to 5 infections occurred in 5 patients (9%). Five patients (9%) discontinued treatment for toxicity. New grade 3 to 4 adverse events were low during maintenance. Pomalidomide-cyclophosphamide-prednisone is safe and effective in relapsed/refractory myeloma patients. This trial was registered at www.clinicaltrials.gov as #NCT01166113.
Source: PubMed