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Single versus tandem high‐dose melphalan followed by autologous blood stem cell transplantation in multiple myeloma: long‐term results from the phase III GMMG ‐ HD 2 trial
Author(s) -
Mai Elias K.,
Benner Axel,
Bertsch Uta,
Brossart Peter,
Hänel Annette,
Kunzmann Volker,
Naumann Ralph,
Neben Kai,
Egerer Gerlinde,
Ho Anthony D.,
Hillengass Jens,
Raab Marc S.,
Neubauer Andreas,
Peyn Astrid,
Ko YonDschun,
Peter Norma,
Scheid Christof,
Goldschmidt Hartmut
Publication year - 2016
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.13994
Subject(s) - melphalan , medicine , multiple myeloma , autologous stem cell transplantation , population , transplantation , clinical endpoint , surgery , oncology , clinical trial , environmental health
Summary The prospective, randomized phase III trial GMMG ‐ HD 2 aimed at demonstrating non‐inferiority of single (Arm A) versus tandem (Arm B) high‐dose melphalan followed by autologous transplantation ( HDM / ASCT ) with regard to 2‐year event‐free survival ( EFS ) in newly‐diagnosed multiple myeloma ( MM ) and included 358 evaluable patients [Intention‐to‐treat population, ( ITT ), single/tandem HDM / ASCT : n = 177/181]. After a median follow‐up of more than 11 years, non‐inferiority of single versus tandem HDM / ASCT was demonstrated using the planned non‐inferiority threshold of 15% of the 2‐year EFS rate. Neither EFS ( P = 0·53) nor overall survival ( OS ) ( P = 0·33) differences were observed in the ITT population. In the tandem arm, 26% ( n = 47/181) of patients refused a second HDM / ASCT due to non‐medical reasons. A per‐protocol ( PP ) analysis, including patients who received the intervention (single/tandem HDM / ASCT : n = 156/93) and patients who did not receive a second HDM / ASCT due to medical reasons (12%, n = 22/181), did not yield differences in EFS ( P = 0·61) or OS ( P = 0·16). In the ITT and PP set of the tandem arm, the rates of complete responses increased from first to second HDM / ASCT (both P = 0·04). Ten‐year OS for the entire ITT was 34% (95% confidence interval: 29–40%). OS after first relapse was significantly shortened in the tandem arm ( P = 0·04). In this study single HDM / ASCT was non‐inferior to tandem HDM / ASCT in MM .