Investigation of the Influences on Decision-Making Algorithms in Allogeneic Stem Cell Transplantation in Different Groups of Pediatric Nonmalignant Diseases
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. EKB Journal Management System, 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". EKB Journal Management System, 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', EKB Journal Management System, 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. EKB Journal Management System, 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

Benha Medical Journal
Article 17, Volume 38, Issue 1, April 2021, Page 196-209  XML PDF (588.57 K)
Document Type: Original Article
DOI: 10.21608/bmfj.2020.132363
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Authors
Samar Elbahyorcid 1; Mohamed ElBakry1; Sameh Abdulsameaorcid 1; Robert Wynn2
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.
Keywords
Bone marrow transplantation; nonmalignant; conditioning
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