Elbahy, S., ElBakry, M., Abdulsamea, S., Wynn, R. (2021). Investigation of the Influences on Decision-Making Algorithms in Allogeneic Stem Cell Transplantation in Different Groups of Pediatric Nonmalignant Diseases. Benha Medical Journal, 38(1), 196-209. doi: 10.21608/bmfj.2020.132363
Samar Elbahy; Mohamed ElBakry; Sameh Abdulsamea; Robert Wynn. "Investigation of the Influences on Decision-Making Algorithms in Allogeneic Stem Cell Transplantation in Different Groups of Pediatric Nonmalignant Diseases". Benha Medical Journal, 38, 1, 2021, 196-209. doi: 10.21608/bmfj.2020.132363
Elbahy, S., ElBakry, M., Abdulsamea, S., Wynn, R. (2021). 'Investigation of the Influences on Decision-Making Algorithms in Allogeneic Stem Cell Transplantation in Different Groups of Pediatric Nonmalignant Diseases', Benha Medical Journal, 38(1), pp. 196-209. doi: 10.21608/bmfj.2020.132363
Elbahy, S., ElBakry, M., Abdulsamea, S., Wynn, R. Investigation of the Influences on Decision-Making Algorithms in Allogeneic Stem Cell Transplantation in Different Groups of Pediatric Nonmalignant Diseases. Benha Medical Journal, 2021; 38(1): 196-209. doi: 10.21608/bmfj.2020.132363
Investigation of the Influences on Decision-Making Algorithms in Allogeneic Stem Cell Transplantation in Different Groups of Pediatric Nonmalignant Diseases
1Department of pediatrics, Benha faculty of medicine, Banha University, Egypt.
2Bone Marrow transplant unit Royal Manchester Children's Hospital, Manchester, United Kingdom
Abstract
Background: Haematopoietic stem cell transplantation (HSCT) represents the definitive treatment for many non-malignant diseases. Treosulfan is considered a safe and effective conditioning drug compared to other conventional myeloablative conditioning (MAC) regimens, especially in patients with comorbidities. Aim of the work: To evaluate the safety and efficacy of Fludarabine-Treosulfan-Thiotepa reduced toxicity conditioning in different paediatric nonmalignant disease. Patients and methods: 54 patients (55 transplants) were reviewed retrospectively, they had metabolic, immunodeficiency, BM failure, hemoglobinopathy and other nonmalignant diseases. All had the same conditioning. 96% received serotherapy (Alemtuzumab/ATG). post-transplant Graft-versus-Host disease (GVHD) prophylaxis was given in all patients, based mostly on ciclosporin. 90% of the patients were fully HLA-matched. Results: Median age at transplant was six years. No primary graft failure, 4%(n=2) had secondary graft failure. Overall survival at a median follow-up of 15months was 90.9%. Neutrophil engraftment occurred at a median of 12 days, Platelet engraftment occurred at a median of 19 days. immune reconstitution was achieved at a median time of nine Chimerism was full donor in 64%(n=35), high donor in 18%(n=10), and mixed donor in 6%(n=3). 60%(n=33) developed GVHD but only 4%(n=2) had acute severe GVHD, another 4%(n=2) had severe chronic GVHD. One patient had severe VOD. Conclusion: This study demonstrates that Fludarabine-Treosulfan-Thiotepa regimen is a safe and effective conditioning that can achieve engraftment, with very low rates of graft failure, transplant-related mortality and morbidity, even if it is used twice in the same patient.
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