Palumbo Antonio, Bringhen Sara, Liberati Anna M, Caravita Tommaso, Falcone Antonietta, Callea Vincenzo, Montanaro Marco, Ria Roberto, Capaldi Antonio, Zambello Renato, Benevolo Giulia, Derudas Daniele, Dore Fausto, Cavallo Federica, Gay Francesca, Falco Patrizia, Ciccone Giovannino, Musto Pellegrino, Cavo Michele, Boccadoro Mario
Divisione di Ematologia dell'Università di Torino, Azienda Ospedaliera S. Giovanni Battista, Torino, Italy.
Blood. 2008 Oct 15;112(8):3107-14. doi: 10.1182/blood-2008-04-149427. Epub 2008 May 27.
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 end points were updated. 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. 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 = .004). 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 beta(2)-microglobulin, or high International Staging System. Thalidomide or bortezomib administration as salvage regimens significantly improved survival after progression in the MP group (P = .002) but not in the MPT group (P = .34). 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 www.clinicaltrials.gov as #NCT00232934.
对新诊断的骨髓瘤患者口服美法仑、泼尼松和沙利度胺（MPT）联合用药的初步分析显示，与标准的美法仑和泼尼松（MP）联合用药相比，其缓解率显著更高，无进展生存期（PFS）更长，提示有生存优势。在本次更新分析中，对疗效和安全性终点进行了更新。患者被随机分配接受口服MPT或单独的MP。更新分析采用意向性治疗，包括PFS、总生存期（OS）和疾病进展后的生存期。中位随访38.1个月后，MPT组的中位PFS为21.8个月，MP组为14.5个月（P = .004）。MPT组的中位OS为45.0个月，MP组为47.6个月（P = .79）。在不同的患者亚组中，无论年龄、β2微球蛋白血清浓度或国际分期系统如何，MPT均可改善PFS。作为挽救方案使用沙利度胺或硼替佐米显著改善了MP组疾病进展后的生存期（P = .002），但MPT组未改善（P = .34）。这些数据证实了MPT对PFS的有效性，但未显示出任何生存优势。复发疾病管理中的新药物可能可以解释这一发现。该研究已在www.clinicaltrials.gov注册，编号为#NCT00232934。
