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

Research output: Contribution to journalArticle

59 Citations (Scopus)

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 cyclophosphamideprednisone 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.

Original languageEnglish
Pages (from-to)2799-2806
Number of pages8
JournalBlood
Volume122
Issue number16
DOIs
Publication statusPublished - Oct 17 2013

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Prednisone
Multiple Myeloma
Refractory materials
Cyclophosphamide
Labels
Maximum Tolerated Dose
Toxicity
Thromboembolism
Neutropenia
Thrombocytopenia
Nervous System
Disease-Free Survival
pomalidomide
Anemia
Maintenance
Survival
Therapeutics
Infection

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Pomalidomide, cyclophosphamide, and prednisone for relapsed/refractory multiple myeloma : A multicenter phase 1/2 open-label study. / Larocca, Alessandra; Montefusco, Vittorio; Bringhen, Sara; Rossi, Davide; Crippa, Claudia; Mina, Roberto; Galli, Monica; Marcatti, Magda; La Verde, Giacinto; Giuliani, Nicola; Magarotto, Valeria; Guglielmelli, Tommasina; Rota-Scalabrini, Delia; Omedé, Paola; Santagostino, Alberto; Baldi, Ileana; Carella, Angelo Michele; Boccadoro, Mario; Corradini, Paolo; Palumbo, Antonio.

In: Blood, Vol. 122, No. 16, 17.10.2013, p. 2799-2806.

Research output: Contribution to journalArticle

Larocca, A, Montefusco, V, Bringhen, S, Rossi, D, Crippa, C, Mina, R, Galli, M, Marcatti, M, La Verde, G, Giuliani, N, Magarotto, V, Guglielmelli, T, Rota-Scalabrini, D, Omedé, P, Santagostino, A, Baldi, I, Carella, AM, Boccadoro, M, Corradini, P & Palumbo, A 2013, 'Pomalidomide, cyclophosphamide, and prednisone for relapsed/refractory multiple myeloma: A multicenter phase 1/2 open-label study', Blood, vol. 122, no. 16, pp. 2799-2806. https://doi.org/10.1182/blood-2013-03-488676
Larocca, Alessandra ; Montefusco, Vittorio ; Bringhen, Sara ; Rossi, Davide ; Crippa, Claudia ; Mina, Roberto ; Galli, Monica ; Marcatti, Magda ; La Verde, Giacinto ; Giuliani, Nicola ; Magarotto, Valeria ; Guglielmelli, Tommasina ; Rota-Scalabrini, Delia ; Omedé, Paola ; Santagostino, Alberto ; Baldi, Ileana ; Carella, Angelo Michele ; Boccadoro, Mario ; Corradini, Paolo ; Palumbo, Antonio. / Pomalidomide, cyclophosphamide, and prednisone for relapsed/refractory multiple myeloma : A multicenter phase 1/2 open-label study. In: Blood. 2013 ; Vol. 122, No. 16. pp. 2799-2806.
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AU - Corradini, Paolo

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