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An Organotypic High Throughput System for Characterization of Drug Sensitivity of Primary Multiple Myeloma Cells
Published on: July 15, 2015
High-dose therapy and autologous stem cell transplantation for multiple myeloma poorly responsive to initial therapy
S Kumar1, M Q Lacy, A Dispenzieri
1Division of Hematology, Department of Medicine, Mayo Clinic and Foundation, Rochester, MN 55905, USA.
Abstract:
Autologous stem cell transplant (SCT) improves survival in multiple myeloma (MM) and remains the standard of care for eligible patients. Nearly a third of patients with newly diagnosed MM fail initial therapy aimed at reducing tumor burden preceding SCT (primary refractory). It is unclear if an initial response is important for successful SCT. We evaluated our experience with SCT in 50 patients with primary refractory MM and compared it to 101 patients with chemosensitive disease receiving SCT. The study cohort had a median age of 56 years (range 29-72) consisting of 87 males (58%). A total of 46 patients (92%) in the refractory group and 100 (99%) in the chemosensitive group had a response to transplant (50% or greater reduction in the M-protein). In all, 10 refractory patients (20%) and 35 (35%) in the chemosensitive group achieved a CR (P=0.06). The 1-year estimated progression-free survival from the time of transplant for the refractory group was 70% compared to 83% for the chemosensitive group (P=0.65). The lack of response to initial induction therapy does not appear to preclude a good response to SCT. We recommend that patients with primary refractory MM be offered early SCT.
Insights
Autologous stem cell transplant (SCT) offers survival benefits for multiple myeloma (MM). Even patients with primary refractory MM, who do not respond to initial therapy, can achieve good outcomes with SCT.
Area of Science:
- Hematology
- Oncology
- Stem Cell Transplantation
Background:
- Autologous stem cell transplant (SCT) is a standard treatment for multiple myeloma (MM), improving patient survival.
- A significant proportion of newly diagnosed MM patients are primary refractory, failing to respond to initial therapies.
- The impact of initial treatment response on SCT success in primary refractory MM is not well understood.
Purpose of the Study:
- To evaluate the efficacy of SCT in patients with primary refractory multiple myeloma.
- To compare SCT outcomes in primary refractory MM versus chemosensitive MM.
- To determine if an initial response to therapy is essential for successful SCT.
Main Methods:
- Retrospective analysis of 50 patients with primary refractory MM undergoing SCT.
- Comparison with 101 patients with chemosensitive MM who received SCT.
- Assessment of response rates (≥50% M-protein reduction) and complete remission (CR) post-SCT.
- Evaluation of 1-year progression-free survival (PFS) from transplant.
Main Results:
- High response rates to SCT were observed in both groups: 92% in refractory and 99% in chemosensitive MM.
- Complete remission rates were 20% for refractory and 35% for chemosensitive MM (P=0.06).
- One-year PFS was 70% for refractory MM and 83% for chemosensitive MM (P=0.65).
Conclusions:
- Initial lack of response to induction therapy does not prevent a favorable response to autologous SCT in MM.
- Patients with primary refractory MM should be considered for early SCT.
- SCT remains a viable and recommended treatment option for eligible patients with primary refractory MM.
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