In a nutshell
The authors looked at the use of busulfan (Myleran), melphalan (Alkeran), and bortezomib (Velcade) compared with melphalan only for autologous stem cell transplantation (autoSCT) in the treatment of multiple myeloma (MM). The authors concluded that using this drug combination leads to an improved survival without cancer progression in these patients.
Some background
MM is a cancer of blood cells called plasma cells. MM is incurable. SCT is an important part of the treatment for patients with MM. SCT involves getting high-dose chemotherapy and/or radiotherapy to kill the cancer cells in the bone marrow. Then, the patient receives new, healthy blood-forming stem cells. For an autoSCT, the patient’s own healthy stem cells are removed from his or her bone marrow or peripheral blood before the transplant and given back after chemotherapy.
Melphalan (Mel) in high dose (200 mg/m2) is a standard treatment before autoSCT. Recently, other medications for MM have emerged. Bortezomib (Vel) is an anti-cancer medication used in the treatment of MM. It blocks proteins found in MM cells called proteasomes. This leads to cancer cell death. Busulfan (Bu) is similar to melphalan and can also be used before autoSCT. Previously, the combination of Bu, Mel, and Vel (BuMelVel) has shown improved short-term survival without cancer worsening compared to standard high-dose Mel before autoSCT. However, the long-term outcomes for this regimen are still unknown.
Methods & findings
This study had 43 patients with MM. All patients were treated with BuMelVel before receiving autoSCT. They were followed-up for an average of 7 years. Patients were compared with 162 patients in a different trial who were treated with high-dose Mel before autoSCT.
The overall response rate for BuMelVel was 98%. After 7 years, 32% of the patients in the BuMelVel group were still alive without cancer worsening compared to 23% in the Mel group. The average length of survival without cancer worsening in the BuMelVel group was 4.58 years compared to 2.75 years in the Mel group. The 5-year survival without cancer worsening rate was 47% in the BuMelVel group compared to 30% in the Mel group.
The overall survival rate at 7 years was slightly higher in the BuMelVel group (64% vs 55%). Treatment-related side effects were similar in both groups.
The bottom line
The authors concluded that using the BuMelVel regimen before autoSCT led to an improved survival without cancer worsening in patients with MM.
The fine print
This study had a small number of participants. Also, the comparison with Mel was to a different trial, which might have influenced the results. More controlled trials are needed. This trial was funded by Otsuka Pharmaceutical, the manufacturer of busulfan.
Published By :
Leukemia & lymphoma
Date :
Aug 31, 2020
