In a nutshell
This study compared autologous stem cell transplant (SCT) to treatment with the proteasome inhibitor and chemotherapy regimen VMP (bortezomib, melphalan, prednisone) for patients with multiple myeloma (MM). It found that SCT led to better outcomes for the initial treatment of MM.
Some background
MM is a cancer of the plasma cells, a type of white blood cell which creates antibodies. Autologous SCT (auto-SCT) is a cancer treatment that involves isolating stem cells from the blood or bone marrow. Stem cells have the ability to become multiple types of blood cells, including plasma cells. The patient is given high doses of chemotherapy to kill off the MM cancer cells. Then, the stem cells are reintroduced in the body to repopulate normal blood cells.
Auto-SCT has been the gold standard for the initial treatment of MM for patients who could support high doses of chemotherapy. However, targeted treatments have greatly improved outcomes for patients with MM who are not treated with SCT. Bortezomib (Velcade) is a targeted therapy that encourages cancer cells to undergo self-destruction. Bortezomib can be used in multi-drug regimens including VMP.
It is unclear whether auto-SCT is still the best initial therapy for patients with MM, or whether VMP is an equally effective treatment.
Methods & findings
This study included 1197 patients newly diagnosed with active MM. 702 patients were randomly assigned to auto-SCT, and 495 to VMP. After initial treatment, some patients also had consolidation treatment (kills any remaining cancer cells after initial therapy). Consolidation therapy involved VRD (bortezomib, lenalidomide, and dexamethasone). All patients were given maintenance therapy (prevents cancer from returning) with lenalidomide (Revlimid). The patients were followed for an average of 60.3 months.
Patients treated with auto-SCT had a significantly longer time before the cancer progressed (worsened) (56.7 vs. 41.9 months). Among patients predicted to have a poorer response to treatment and whose cancer was in stage 3, auto-SCT led to a 52% lower risk of progression. Patients whose MM cancer cells showed high-risk gene markers had a 37% lower risk of progression when treated with auto-SCT compared to VMP. The overall 5-year survival was similar in both groups (75% vs. 72%).
34% of patients treated with auto-SCT and 27% of those treated with VMP having at least one serious side effect. Patients treated with auto-SCT more often had serious low white blood cell count (79% vs. 29%) or infection (30% vs. 4%).
A subgroup of 877 patients was used to examine whether consolidation treatment was beneficial. 449 patients were randomly assigned to consolidation treatment with VRD. 428 patients did not receive consolidation treatment. Patients treated with VRD consolidation went significantly longer before the cancer progressed (58.9 months vs. 45.5 months).
The bottom line
This study found that patients newly diagnosed with MM who were treated with auto-SCT had better outcomes than those treated with VMP. It also found that consolidation treatment with VRD was beneficial.
The fine print
63% of patients in the VMP group underwent auto-SCT upon relapse. The use of multiple treatments makes interpreting overall survival more difficult.
Published By :
The Lancet. Haematology
Date :
Apr 30, 2020