• María-Victoria Mateos, Miguel-Teodoro Hernández, Pilar Giraldo, Javier de la Rubia, Felipe de Arriba, Lucía López Corral, Laura Rosiñol, Bruno Paiva, Luis Palomera, Joan Bargay, Albert Oriol, Felipe Prosper, Javier López, Eduardo Olavarría, Nuria Quintana, José-Luis García, Joan Bladé, Juan-José Lahuerta, and Jesús-F San Miguel.
• Hospital Universitario de Salamanca, Instituto de Biología Molecular y Celular del Cáncer, Universidad de Salamanca-Consejo Superior de Investigaciones Científicas, Salamanca, Spain.
• N. Engl. J. Med.. 2013 Aug 1;369(5):438-47.

BackgroundFor patients with smoldering multiple myeloma, the standard of care is observation until symptoms develop. However, this approach does not identify high-risk patients who may benefit from early intervention.MethodsIn this randomized, open-label, phase 3 trial, we randomly assigned 119 patients with high-risk smoldering myeloma to treatment or observation. Patients in the treatment group received an induction regimen (lenalidomide at a dose of 25 mg per day on days 1 to 21, plus dexamethasone at a dose of 20 mg per day on days 1 to 4 and days 12 to 15, at 4-week intervals for nine cycles), followed by a maintenance regimen (lenalidomide at a dose of 10 mg per day on days 1 to 21 of each 28-day cycle for 2 years). The primary end point was time to progression to symptomatic disease. Secondary end points were response rate, overall survival, and safety.ResultsAfter a median follow-up of 40 months, the median time to progression was significantly longer in the treatment group than in the observation group (median not reached vs. 21 months; hazard ratio for progression, 0.18; 95% confidence interval [CI], 0.09 to 0.32; P<0.001). The 3-year survival rate was also higher in the treatment group (94% vs. 80%; hazard ratio for death, 0.31; 95% CI, 0.10 to 0.91; P=0.03). A partial response or better was achieved in 79% of patients in the treatment group after the induction phase and in 90% during the maintenance phase. Toxic effects were mainly grade 2 or lower.ConclusionsEarly treatment for patients with high-risk smoldering myeloma delays progression to active disease and increases overall survival. (Funded by Celgene; ClinicalTrials.gov number, NCT00480363.).

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