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Journal Article
Observational Study
Outcomes of Haematopoietic Stem Cell Transplantation in Beta Thalassemia Major with Fully Matched Parents as Donor.
OBJECTIVE: To determine the outcome of beta thalassemia major (BTM) patients undergoing haematopoietic stem cells (HSCT), with fully matched parents as donors vs. matched sibling donors (MSD).
STUDY DESIGN: Observational Study. Place and Duration of the Study: Department of Clinical Haematology and Bone Marrow Transplantation Centre, Rawalpindi, Pakistan, from January 2013 to July 2023.
METHODOLOGY: Group A consisted of BTM patients who underwent HSCT with fully matched siblings as donors, and Group B consisted of BTM patients who underwent HSCT with fully matched parents as donors. Study data included the age and gender of both recipients and donors, source and dose of stem cells infused, and stage and grades of acute and chronic graft versus host disease (GvHD). All patients received Myeloablative conditioning regimen (MAC). Data were collected to assess patients' demographics, response to HSCT, remission rate, disease free survival (DFS), relapse, and GvHD free survival (GRFS), and overall survival (OS).
RESULTS: The mean age of the 54 patients was 5.90 ± 3.29 years. The mean TNC and CD34 doses were 4.99 + 1.13 and 5.42 + 3.70, respectively. Mean time for neutrophil engraftment in both groups was 14.88 + 4.51 days and platelets engraftment was 23.0 + 5.35 days. Most common cause of death was neutropenic sepsis followed by aGVHD. Seven patients had graft rejection. There was no significant association found between graft rejection with donor relation though graft rejection was higher in OS in this study was 70.4%. OS was equal in both groups. Disease free survival was superior in MSD (63%) than parent group (57.7%).
CONCLUSION: Allogenic bone marrow transplantation with parents as donors in BTM patients yields outcomes comparable to those with matched sibling donors. This finding is especially relevant in regions like Pakistan, where donor registries and high-resolution HLA typing may be limited.
KEY WORDS: Beta thalassemia major, Haematopoietic stem cell transplant, Post-transplant outcome.
STUDY DESIGN: Observational Study. Place and Duration of the Study: Department of Clinical Haematology and Bone Marrow Transplantation Centre, Rawalpindi, Pakistan, from January 2013 to July 2023.
METHODOLOGY: Group A consisted of BTM patients who underwent HSCT with fully matched siblings as donors, and Group B consisted of BTM patients who underwent HSCT with fully matched parents as donors. Study data included the age and gender of both recipients and donors, source and dose of stem cells infused, and stage and grades of acute and chronic graft versus host disease (GvHD). All patients received Myeloablative conditioning regimen (MAC). Data were collected to assess patients' demographics, response to HSCT, remission rate, disease free survival (DFS), relapse, and GvHD free survival (GRFS), and overall survival (OS).
RESULTS: The mean age of the 54 patients was 5.90 ± 3.29 years. The mean TNC and CD34 doses were 4.99 + 1.13 and 5.42 + 3.70, respectively. Mean time for neutrophil engraftment in both groups was 14.88 + 4.51 days and platelets engraftment was 23.0 + 5.35 days. Most common cause of death was neutropenic sepsis followed by aGVHD. Seven patients had graft rejection. There was no significant association found between graft rejection with donor relation though graft rejection was higher in OS in this study was 70.4%. OS was equal in both groups. Disease free survival was superior in MSD (63%) than parent group (57.7%).
CONCLUSION: Allogenic bone marrow transplantation with parents as donors in BTM patients yields outcomes comparable to those with matched sibling donors. This finding is especially relevant in regions like Pakistan, where donor registries and high-resolution HLA typing may be limited.
KEY WORDS: Beta thalassemia major, Haematopoietic stem cell transplant, Post-transplant outcome.
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