Prospective Study of Allogeneic Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide and Antithymocyte Globulin from HLA-Mismatched Related Donors for Nonmalignant Diseases |
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| Affiliation: | 1. Children''s Cancer Center, National Center for Child Health and Development, Tokyo, Japan;2. Department of Pediatric Hematology and Oncology Research, National Research Institute for Child Health and Development, Tokyo, Japan;3. Department of Clinical Research Promotion, Clinical Research Center, National Center for Child Health and Development, Tokyo, Japan;4. Department of Human Genetics, National Research Institute for Child Health and Development, Tokyo, Japan;5. Division of Immunology, National Center for Child Health and Development, Tokyo, Japan;6. Showa University Research Administration Center, Showa University, Tokyo, Japan;7. Department of Childhood Cancer Data Management, National Center for Child Health and Development, Tokyo, Japan;8. Center for Pediatric Inflammatory Bowel Disease, Division of Gastroenterology, National Center for Child Health and Development, Tokyo, Japan;9. Depertment of Advanced Medicine for Virus Infections, National Center for Child Health and Development, Tokyo, Japan;10. Department of Clinical Pharmaceutics, Faculty of Pharmaceutical Sciences, Doshisha Women''s College of Liberal Arts, Kyoto, Japan;11. Department of Community Pediatrics, Perinatal and Maternal Medicine, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University, Tokyo, Japan;12. Department of Hematology and Oncology, Tokyo Metropolitan Children''s Medical Center, Tokyo, Japan;13. Department of Child Health and Development, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University, Tokyo, Japan |
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| Abstract: | Allogeneic hematopoietic stem cell transplantation (HSCT) is performed as a curative treatment for children with nonmalignant diseases, such as bone marrow failure syndromes and primary immunodeficiencies. Because graft-versus-host-disease (GVHD) is a major factor affecting survival probability and quality of life after HSCT, the availability of HLA-matched donors restricts the application of HSCT. Recently, HSCT with post-transplantation cyclophosphamide (PTCy) has emerged as a potent method to prevent GVHD after HSCT from HLA-haploidentical donors, and some studies have suggested the safety of PTCy-HSCT for nonmalignant diseases. We conducted a prospective clinical trial aiming to help confirm the safety of HSCT and further reduction of GVHD using a combination of PTCy and low-dose antithymocyte globulin (ATG) from HLA-mismatched related donors for children with nonmalignant diseases. Six patients underwent HSCT and achieved engraftment at a median of 14.5 days, and no patient developed severe acute GVHD. All patients had sustained donor chimerism without developing chronic GVHD at the last follow-up. In conclusion, HSCT with PTCy and low-dose ATG from an HLA-mismatched related donor were feasible to control GVHD for nonmalignant diseases in the children involved in our study.© 2020 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. |
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