Overall and event-free survival are not improved by the use of myeloablative therapy following intensified chemotherapy in previously untreated patients with multiple myeloma: a prospective randomized phase 3 study

We compared the efficacy of intensified chemotherapy followed by myeloablative therapy and autologous stem cell rescue with intensified chemotherapy alone in patients newly diagnosed with multiple myeloma. There were 261 eligible pa- tients younger than 66 years with stage II/III multiple myeloma who were random- ized after remission induction therapy with vincristine, adriamycin, dexametha- sone (VAD) to receive intensified chemo- therapy, that is, melphalan 140 mg/m2 administered intravenously in 2 doses of 70 mg/m2 (intermediate-dose melphalan phamide, total body irradiation, and au- tologous stem cell reinfusion (n 132). Interferon--2a was given as mainte- nance. Of the eligible patients, 79% re- ceived both cycles of IDM and 79% of allocated patients actually received my- eloablative treatment. The response rate (complete remission (CR) plus partial re- mission (PR)) was 88% in the intensified chemotherapy group versus 95% in the myeloablative treatment group. CR was significantly higher after myeloablative therapy (13% versus 29%; P.002). With a median follow-up of 33 months (range, 8-65 months), the event-free survival (EFS) was not different between the treatments (median 21 months versus 22 months; P.28). Time to progression (TTP) was significantly longer after myeloablative treatment (25 months versus 31 months; P.04). The overall survival (OS) was not different (50 months versus 47 months; P.41). Intensified chemo- therapy followed by myeloablative therapy as first-line treatment for multiple my- eloma resulted in a higher CR and a longer TTP when compared with intensi- fied chemotherapy alone. However, it did not result in a better EFS and OS. (Blood. 2003;101:2144-2151)