BACKGROUND: High-dose therapy with autologous stem cell transplantation (HDT-ASCT) and maintenance treatment with novel agents are the best options for patients with newly diagnosed multiple myeloma, increasing the rate of complete response (CR) and prolonging progression-free survival (PFS) and overall survival (OS). Indeed, the achievement of a CR is a predictor of long-term survival among transplant-eligible patients. However, it is unclear whether patients reaching a CR after induction treatment could receive less intense consolidation or avoid maintenance therapy. PATIENTS AND METHODS: We analyzed CR patients treated in 2 phase III trials, GIMEMA-RV-MM-PI-209 and RV-MM-EMN-441, to compare HDT-ASCT with an R-Alk (lenalidomide, alkylator) regimen as consolidation, and lenalidomide (R) maintenance with no maintenance. The primary endpoints were PFS, second PFS (PFS2), and OS from consolidation and maintenance (_m). RESULTS: Overall, the data from 166 patients in CR were analyzed, 95 in the HDT-ASCT group and 71 in the R-Alk group. The CR patients who received HDT-ASCT had a better PFS (hazard ratio [HR], 0.55; P = .01), PFS2 (HR, 0.46; P = .02), and OS (HR, 0.42; P = .03) compared with patients randomized to R-Alk. The survival benefit with HDT-ASCT was confirmed among all the subgroups, according to age, International Staging System (ISS stage, cytogenetic profile, and receipt of maintenance therapy. CR patients who received lenalidomide maintenance had a better PFS_m (4 years: 54% vs. 19%; HR, 0.43; P = .02) compared with those who received no maintenance. However, no difference was observed in terms of PFS2_m (4 years: 72% vs. 58%; HR, 0.83; P = .67) and OS_m (4 years: 79% vs. 72%; HR, 0.82; P = .73) with maintenance therapy. CONCLUSION: Even in CR patients, outcomes were improved by an intensified approach with HDT-ASCT consolidation and lenalidomide-based maintenance therapy.

Clinica Ematologica Azienda Sanitaria Universitaria Integrata DAME Università di Udine Udine Italy

Department of Biomedical Science University of Bari Aldo Moro Medical School Internal Medicine G Baccelli Policlinico Bari Italy

Department of Haematology Alfred Health Monash University Melbourne Victoria Australia

Department of Haematooncology University Hospital Ostrava and Faculty of Medicine University of Ostrava Ostrava Czech Republic

Department of Oncology and Hematology University of Milano Milano Italy

Dipartimento di Medicina Ematologia e Immunologia Clinica Padua Italy

Dipartimento di Oncologia e Emato oncologia Università degli Studi Fondazione IRCCS Cà Granda OM Policlinico Milano Italy

Division of Hematology AOU Policlinico OVE University of Catania Catania Italy

Division of Hematology Chaim Sheba Medical Center Tel Hashomer Israel

Division of Hematology Department of Translational Medicine University of Eastern Piedmont Novara Italy

Ematologia AUSL IRCCS Reggio Emilia Italy

Ematologia Casa Sollievo Della Sofferenza IRCCS Hospital San Giovanni Rotondo Italy

Ematologia Ospedale San Luigi Gonzaga Orbassano Italy

Hematology Department of Cellular Biotechnologies and Hematology Sapienza University of Rome Rome Italy

Myeloma Unit Division of Hematology University of Torino Azienda Ospedaliero Universitaria Città della Salute e della Scienza di Torino Torino Italy

Reparto Ematologia e Centro TMO Ospedale Centrale Bolzano Bolzano Italy

SC Hematology AO Città della Salute e della Scienza Turin Italy

Scientific Direction IRCCS CROB Referral Cancer Center of Basilicata Rionero in Vulture Italy

UOC di Ematologia Azienda Ospedaliera Papardo Messina Italy

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