title: Pomalidomide, cyclophosphamide, and prednisone for relapsed/refractory multiple myeloma: a multicenter phase 1/2 open-label study.
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.
authors: Myeloma Unit, Division of Hematology, University of Torino, Azienda Ospedaliera Città della Salute e della Scienza di Torino, Torino, Italy;
authors: Alessandra
authors: A
authors: Larocca
authors: Vittorio
authors: V
authors: Montefusco
authors: Sara
authors: S
authors: Bringhen
authors: Davide
authors: D
authors: Rossi
authors: Claudia
authors: C
authors: Crippa
authors: Roberto
authors: R
authors: Mina
authors: Monica
authors: M
authors: Galli
authors: Magda
authors: M
authors: Marcatti
authors: Giacinto
authors: G
authors: La Verde
authors: Nicola
authors: N
authors: Giuliani
authors: Valeria
authors: V
authors: Magarotto
authors: Tommasina
authors: T
authors: Guglielmelli
authors: Delia
authors: D
authors: Rota-Scalabrini
authors: Paola
authors: P
authors: Omedé
authors: Alberto
authors: A
authors: Santagostino
authors: Ileana
authors: I
authors: Baldi
authors: Angelo Michele
authors: AM
authors: Carella
authors: Mario
authors: M
authors: Boccadoro
authors: Paolo
authors: P
authors: Corradini
authors: Antonio
authors: A
authors: Palumbo
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D003520:Cyclophosphamide / Q000008:administration & dosage*; D018572:Disease-Free Survival; D019008:Drug Resistance, Neoplasm; D005260:Female; D006801:Humans; D008297:Male; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy*; D011241:Prednisone / Q000008:administration & dosage*; D012008:Recurrence; D013792:Thalidomide / Q000008:administration & dosage / Q000031:analogs & derivatives*; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D013792:Thalidomide; D003520:Cyclophosphamide; C467566:pomalidomide; D011241:Prednisone