Oral melphalan, prednisone, and thalidomide in elderly patients with multiple myeloma: updated results of a randomized, controlled trial.

Author(s): Palumbo A, Bringhen S, Liberati AM, Caravita T, Falcone A, Callea V, Montanaro M, Ria R, Capaldi A, Zambello R, Benevolo G, Derudas D, Dore F, Cavallo F, Gay F, Falco P, Ciccone G, Musto P, Cavo M, Boccadoro M

Affiliation(s): Divisione di Ematologia dellUniversita di Torino, Azienda Ospedaliera S Giovanni Battista, Torino, Italy.

Publication date & source: 2008-05-27, Blood., [Epub ahead of print]

Publication type:

Rationale: The initial analysis of the oral combination melphalan, prednisone, and thalidomide (MPT) in newly diagnosed patients with myeloma showed significantly higher response rate and longer progression-free survival (PFS) than did the standard melphalan and prednisone (MP) combination and suggested a survival advantage. In this updated analysis, efficacy and safety endpoints were updated. Objectives: Patients were randomly assigned to receive oral MPT or MP alone. Updated analysis was by intention-to-treat and included PFS, overall survival (OS), and survival after progression. Findings: After a median follow-up of 38.1 months, the median PFS was 21.8 months for MPT and 14.5 months for MP (P = .0004). The median OS was 45.0 months for MPT and 47.6 months for MP (P = .79). In different patient subgroups, MPT improved PFS irrespective of age, serum concentrations of b2-microglobulin, or high International Staging System. Thalidomide or bortezomib administration as salvage regimens significantly improved survival after progression in the MP group (P = .0002) but not in the MPT group (P = .34). Conclusion: These data confirm activity of MPT for PFS but failed to show any survival advantage. New agents in the management of relapsed disease could explain this finding. The study is registered at ClinicalTrial.gov, number NCT00232934.