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Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Allogeneic Stem Cell Transplantation Combined With Transfusion of Mesenchymal Stem Cells in Primary Myelofibrosis: A
Qingyuan Wang1,2,3, Na Xu4, Yu Wang5,6,7
1National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China.
Background:
Allogeneic stem cell transplantation (allo-SCT) remains the only effective curative therapy for primary myelofibrosis. Utilization and efficacy of allo-SCT are limited by lethal complications, including engraftment failure, and acute (aGVHD) and chronic graft-versus-host disease (cGVHD). Several clinical trials have explored the use of mesenchymal stem cells (MSCs) in allo-SCT to prevent hematopoietic stem cell (HSC) engraftment failure and control GVHD.
Methods:
Clinical data of 17 patients with primary myelofibrosis who underwent allo-SCT combined with ex vivo expanded MSC transfusion in four centers from February 2011 to December 2018 were retrospectively analyzed.
Results:
All patients received myeloablative conditioning regimen. The median number of transplanted nucleated cells (NCs) per kilogram body weight was 11.18 × 108 (range: 2.63-16.75 × 108), and the median number of CD34+ cells was 4.72 × 106 (range: 1.32-8.4 × 106). MSCs were transfused on the day of transplant or on day 7 after transplant. The median MSC infusion number was 6.5 × 106 (range: 0.011-65 × 106). None of the patients experienced primary or secondary graft failure in the study. The median time to neutrophil engraftment was 13 days (range: 11-22 days), and the median time to platelet engraftment was 21 days (range: 12-184 days). The median follow-up time was 40.3 months (range: 1.8-127.8 months). The estimated relapse-free survival (RFS) at 5 years was 79.1%, and overall survival (OS) at 5 years was 64.7%. Analysis showed that the cumulative incidence of aGVHD grade II to IV was 36% (95% CI: 8%-55%) and that of grade III to IV was 26% (95% CI: 0%-45%) at day 100. The cumulative incidence of overall cGVHD at 2 years for the entire study population was 63% (95% CI: 26%-81%). The cumulative incidence of moderate to severe cGVHD at 2 years was 17% (95% CI: 0%-42%). Seven patients died during the study, with 5 patients succumbing from non-relapse causes and 2 from disease relapse.
Conclusion:
The findings of the study indicate that allo-SCT combined with MSC transfusion may represent an effective treatment option for primary myelofibrosis.
