A PROSPECTIVE, RANDOMIZED TRIAL OF AUTOLOGOUS BONE MARROW TRANSPLANTATION AND CHEMOTHERAPY IN MULTIPLE MYELOMA

TLDR

Patients with multiple myeloma have a median survival of three years or less after conventional chemotherapy, but high‑dose therapy with autologous bone marrow transplantation has shown promising results. The study aimed to compare conventional chemotherapy with high‑dose therapy plus autologous transplantation in newly diagnosed patients. Two hundred untreated patients under 65 were randomized at diagnosis to receive either conventional chemotherapy or high‑dose therapy followed by autologous bone marrow transplantation. High‑dose therapy achieved an 81 % response rate versus 57 % for conventional therapy, and produced 5‑year event‑free survival of 28 % versus 10 % and overall survival of 52 % versus 12 %, with similar treatment‑related mortality, demonstrating superior outcomes. Published in N Engl J Med 1996;335:91‑7.

Abstract

Background The median survival of patients with myeloma after conventional chemotherapy is three years or less. Promising results have been reported with high-dose therapy supported by autologous bone marrow transplantation. We conducted a randomized study comparing conventional chemotherapy and high-dose therapy. Methods Two hundred previously untreated patients under the age of 65 years who had myeloma were randomly assigned at the time of diagnosis to receive either conventional chemotherapy or highdose therapy and autologous bone marrow transplantation. Results The response rate among the patients who received high-dose therapy was 81 percent (including complete responses in 22 percent and very good partial responses in 16 percent), whereas it was 57 percent (complete responses in 5 percent and very good partial responses in 9 percent) in the group treated with conventional chemotherapy (P � 0.001). The probability of event-free survival for five years was 28 percent in the high-dose group and 10 percent in the conventional-dose group (P � 0.01); the overall estimated rate of survival for five years was 52 percent in the high-dose group and 12 percent in the conventional-dose group (P � 0.03). Treatment-related mortality was similar in the two groups. Conclusions High-dose therapy combined with transplantation improves the response rate, eventfree survival, and overall survival in patients with myeloma. (N Engl J Med 1996;335:91-7.) ©1996, Massachusetts Medical Society.