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Öğe Outcomes of hematopoietic stem cell transplantation in patients with thalassemia major: how do anti-HLA antibodies impact?(Wiley, 2024) Ersoy, Gizem Zengin; Aksoy, Başak Adaklı; Erdem, Melek; Karataş, Lokman; Aydoğdu, Selime; Öner, Özlem Başoğlu; Dikme, Guercan; Bozkurt, Ceyhun; Fışgın, TunçAimTo investigate the effects of anti-human Leucocyte Antigen (HLA) antibody positivity on early hematopoietic stem cell transplantation (HSCT) results in patients with thalassemia major (TM).MethodsOne hundred and twenty-four HLA-matched HSCTs were performed in patients with TM between 2015 and 2022. Ninety-one patients were screened for anti-HLA antibodies by testing panel reactive antigens (PRA). Demographic and transplantation characteristics of patients were recorded. The presence of PRA was tested with the Antibody Testing Assay (Luminex LIFECODES HLA Antibody Identification System).ResultsThe number of PRA-positive patients was 54. There was no relationship between acute graft versus host disease (GVHD), chronic GVHD, grade of GVHD, and viral reactivation of the patients. However, platelet engraftment took around 3 days longer in the PRA-positive group (p = 0.05). The median number of erythrocyte transfusions was significantly higher in PRA-positive patients in the post-transplant period (p = 0.003), as was the median number of platelet transfusions (p = 0.003). Treosulfan conditioning increased the stable mixed chimerism (MC) rate by 3.8-fold (p = 0.011). In contrast, reduced rates of MC were found in patients who received matched unrelated donor cells or peripherally derived stem cells (p = 0.011 and p = 0.039, respectively) in the posttransplantation period in TM patients. PRA-positivity did not affect MC (p = 0.478). However, 80% of patients who had primary graft failure (n = 5; p = 0.59) and 75% of patients who died (n = 4) were PRA positive (p = 0.64), but these results were statistically insignificant due to the low number of patients.ConclusionAnti-HLA antibodies primarily delayed platelet engraftment in TM patients and increased the erythrocyte and thrombocyte transfusion requirements. Although PRA positivity was more common in patients with primary graft failure or who died, there was no statistically significant impact of PRA positivity on chimerism, acute or chronic GVHD, viral activation, or mortality rates.Öğe Outcomes of hematopoietic stem cell transplantation in patients with thalassemia major: how do anti-HLA antibodies impact?: the impact of anti-HLA antibodies on transplantation outcomes in thalassemia major(2024) Ersoy, Gizem Zengin; Aksoy, Başak Adaklı; Erdem, Melek; Karataş, Lokman; Aydoğdu, Selime; Başoğlu Öner, Özlem; Dikme, Gürcan; Bozkurt, Ceyhun; Fışgın, TunçAim: To investigate the effects of anti-human Leucocyte Antigen (HLA) antibody positivity on early hematopoietic stem cell transplantation (HSCT) results in patients with thalassemia major (TM). Methods: One hundred and twenty-four HLA-matched HSCTs were performed in patients with TM between 2015 and 2022. Ninety-one patients were screened for anti-HLA antibodies by testing panel reactive antigens (PRA). Demographic and transplantation characteristics of patients were recorded. The presence of PRA was tested with the Antibody Testing Assay (Luminex LIFECODES HLA Antibody Identification System). Results: The number of PRA-positive patients was 54. There was no relationship between acute graft versus host disease (GVHD), chronic GVHD, grade of GVHD, and viral reactivation of the patients. However, platelet engraftment took around 3 days longer in the PRA-positive group (p = 0.05). The median number of erythrocyte transfusions was significantly higher in PRA-positive patients in the post-transplant period (p = 0.003), as was the median number of platelet transfusions (p = 0.003). Treosulfan conditioning increased the stable mixed chimerism (MC) rate by 3.8-fold (p = 0.011). In contrast, reduced rates of MC were found in patients who received matched unrelated donor cells or peripherally derived stem cells (p = 0.011 and p = 0.039, respectively) in the posttransplantation period in TM patients. PRA-positivity did not affect MC (p = 0.478). However, 80% of patients who had primary graft failure (n = 5; p = 0.59) and 75% of patients who died (n = 4) were PRA positive (p = 0.64), but these results were statistically insignificant due to the low number of patients. Conclusion: Anti-HLA antibodies primarily delayed platelet engraftment in TM patients and increased the erythrocyte and thrombocyte transfusion requirements. Although PRA positivity was more common in patients with primary graft failure or who died, there was no statistically significant impact of PRA positivity on chimerism, acute or chronic GVHD, viral activation, or mortality rates.Öğe The impact of Treosulfan-based conditioning for inborn errors of immunity: Is dose monitoring crucial?(2023) Ersoy, Gizem Zengin; Çipe, Funda; Fışgın, Tunç; Adaklı Aksoy, Başak; Başoğlu Öner, Özlem; Hashemi, Nazlı; Aydoğdu, Selime; Erdem, Melek; Dikme, Gürcan; Murat, Koza; Bozkurt, CeyhunIntroduction: In children with inborn errors of immunity (IEI) who will receive a hematopoietic stem cell transplant (HSCT) treosulfan-based conditioning is currently preferred. The aim of this study was to investigate early and late outcomes in pediatric IEI patients receiving pre-HSCT treosulfan and to examine the effect of treosulfan dose monitoring on outcomes. Methods: Seventy-three pediatric patients receiving this management between 2015 and 2022 were included. Results: Overall survival rate was 80%, and event-free survival was 67.8%. A larger treosulfan dose AUC after first application increased the rate of early toxicity (p = .034) and slowed lymphocyte engraftment (r = .290; p = .030). Underlying disease, treosulfan AUC, donor type, stem cell type, number of immunosuppressive agents, the dose of anti-thymocyte globulin, and post-transplantation cyclophosphamide did not to increase risk of acute graft-versus-host disease. The risk of mixed chimerism (MC) in patients with autoimmune lymphoproliferative syndrome and leukocyte adhesion deficiency were higher than those with severe combined immunodeficiency (p = .021 and p = .014, respectively). The risk of MC was lower in those receiving peripheral blood stem cells (SC) compared with bone marrow derived SC (OR = .204, p = .022). Conclusion: The AUC of the treosulfan dose was not associated with poorer late outcomes. Treosulfan is an agent that can be used safely in the IEI patient group, level measurement appears essential to identify early toxicities. Prospective studies with more extended follow-up periods are needed.Öğe The impact of treosulfan-based conditioning for primary immune deficiencies : single center experience(2023) Ersoy, Gizem Zengin; Çipe, Funda; Aksoy, Başak Adaklı; Hashemi, Nazlı; Öner, Özlem Başoğlu; Aydoğdu, Selime; Dikme, Gürcan; Erdem, Melek; Bozkurt, Ceyhun; Fışgın, Tunç...Öğe Would monitorizing treosulfan levels in patients transplanted for transfusion dependent thalassemia be beneficial in terms of chimerizm? a single center experience(2023) Aksoy, Başak Adaklı; Ersoy, Gizem Zengin; Elsayed, Mariam; Öner, Özlem Başoğlu; Aydoğdu, Selime; Dikme, Gürcan; Erdem, Melek; Fışgın, Tunç; Bozkurt, Ceyhun...
