Modified conditioning regimen improves outcomes of unrelated donor peripheral blood stem cell transplantation for β‐thalassaemia major patients

Huang, Ke; Zhou, Dun-Hua; Li, Yang; Xu, Huilin; Que, Li-Ping; Chen, Chun; Hou, Xue; Guo, Haixia; Weng, Wen-Jun; Huang, Shao-Liang; Fang, Jian-Pei

doi:10.1002/pbc.27026

Cited by 7 publications

(7 citation statements)

References 22 publications

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“…The majority of patients treated with allo-HSCT show a good response to treatment, and recent advancements in transplantation technology have improved the OS and Original Article Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts TFS rates to around 90% and 80%, respectively (4,5). Most transplants use HLA fully-matched sibling donors, but limitations in the number of matched related donors has led to the use of mismatched related donors, matched unrelated donors and mismatched unrelated donors (6).…”

Section: Introductionmentioning

confidence: 99%

Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

Huang¹,

Qu²,

Li³

et al. 2021

Transl Pediatr

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Background: Until now, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only effective method to cure Thalassemia major. However, it has not been determined whether similar results can be obtained with the same conditioning regimen for both fully-matched and mismatched donors grafts.We hypothesized that using modified NF-08-TM conditioning regimen could achieve similar results for both fully and mismatched donors grafts.Methods: This retrospective cohort study included patients with β-thalassemia major who underwent HSCT with modified NF-08-TM conditioning regimen at Guangzhou Women and Children's Medical

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Section: Introductionmentioning

confidence: 99%

Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

Huang¹,

Qu²,

Li³

et al. 2021

Transl Pediatr

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“…Recent reports of alternative HCT donors are summarised in Table 1. [32][33][34][35][36][37][38] aGVHD: Acute graft-versus-host disease; ATG: anti-thymocyte globulin; Az: azathioprine; Bu: busulfan; cGVHD: chronic graft-versus-host disease; CsA: cyclosporin A; Cy: cyclophosphamide; Dxm: dexamethasone; Flu: fludarabine; GF: graft failure; GVHD: graft-versus-host disease; HLA: human leukocyte antigen; Hu: hydroxyurea; MAC: myeloablative conditioning; MMF: mycophenolate mofetil; MMUD: mismatched unrelated donor; MP: methylprednisolone; MTX : methotrexate; MUD: matched unrelated donor; N/A: not applicable; NR: not reported; OS: overall survival; PT-Cy: post-transplant cyclophosphamide; Rit: rituximab; TFS: thalassaemia-free survival; TT: thiotepa; UCB: umbilical cord blood; Vel: velcade.…”

Section: Allogeneic Haemopoietic Cell Transplantationmentioning

confidence: 99%

Current and Future Therapies for β-Thalassaemia: A Review Article

Giannoni

Angelucci

2021

EMJ Hematol

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This article will review recent and forthcoming advances in the treatment of thalassaemia. Prognosis of thalassaemia has dramatically improved in the last 50 years with the development of regular and safe blood transfusions and iron chelation. Almost 20 years ago, development of oral chelators, and more recently the improvement in the knowledge and understanding of iron pathophysiology, have led to optimal iron toxicity prevention and treatment. These considerable advancements in medical therapy have transformed transfusion-dependent thalassaemia from a lethal childhood disease to a chronic disease with an open prognosis, even in those individuals over 50 years of age, and with the disease being, in some instances, curable. In the 1980s, the introduction of allogeneic haematopoietic cell transplantation provided the possibility of curing the congenital disease for the first time. More recent developments include an improved understanding of erythropoiesis, which led to the development of new erythroid-stimulating factors effective in thalassaemia, an expansion of donor pull for transplantation, and the approach of the long-term promised gene therapy in clinical practice. Moreover, ongoing trials of gene editing and agents modulating iron metabolism promise new improvements. Today, patients with thalassaemia have several weapons in their therapeutic arsenal and, hopefully, will have much more to come. As usual in medical practice, new advancements provide new challenges for the medical community, and it is the duty of this community to clearly understand the benefits and challenges of any new approach in order to provide the highest clinical benefit to patients.

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“…Risk for marrow transplanta;on in thalassemia pa;ents can be categorized into three classes; class I, II and III (Lucarelli et al, 1993& Lucarelli et al, 2002 with class III has the greatest risk (Lucarelli et al, 1996). The survival outcome of treated BTM pa;ents with allo-HSCT has been improved especially aaer recent progress of transplanta;on technologies as the overall survival (OS) and thalassemia free survival (TFS) rates reach around 90% and 80%, respec;vely (Choudhary et al, 2019 andHuang et al, 2018). The transplant outcomes are affected by several factors; include age at transplanta;on, donor type, degree of HLA matching, pre-transplant risk class, source of stem cell, and type of condi;oning regimen (Issaragrisil and Kunacheewa, 2016).…”

Section: Introductionmentioning

confidence: 99%

Outcome of Beta Thalassemia Major in Paediatric Patients Allografted from Fully Matched Related Donors; in Egypt

Shibl,

Sayed,

Zakaria

et al. 2024

Egyptian Journal of Cancer and Biomedical Research

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Background: Thalassemia is the most common genetic disease worldwide. Allogeneic hemopoietic stem cell transplantation (HSCT) is a method available to treat transfusion-dependent thalassemia major patients. Aim: To report incidence of post-transplant complications and survival outcome with factors affecting it in beta thalassemia major patients. Patients and Methods: We conducted this retrospective study on pediatric patients (≤18 years old) who underwent their first allo-HSCT for 5 years (2015)(2016)(2017)(2018)(2019). All patients received allografts from fully matched related donors. A total of 115 BTM patients' data were collected and classified into 3 classes according to Pesaro classification. Results: Post-transplant complications were reported in 80% of all patients where the most common one was infections (63.5%) which seen more in class III (64.7%). Also, acute graft versus host disease (AGVHD), chronic GVHD, cardiovascular toxicity, endocrinopathies and graft failure were more common in class III patients (35.3%, 11.8%, 11.8%, 17.6% and 11.8%, respectively). While pulmonary complications, neurotoxicity, and hemorrhagic cystitis were reported more in class II patients (40%, 21.8% and 21.8% respectively). Three-year overall survival (OS) and thalassemia free survival (TFS) of all patients were 80% and 77.4%, with the worst outcome reported in class III patients (58.8% for both OS and TFS). Patients' age group 5-10 years and received MTX included GVHD prophylaxis were associated with better survival outcomes than others. Conclusion: Addition of fludarabine to the conditioning regimen in class III BTM patients didn't improve the occurrence of GVHD, graft failure or survival outcomes in comparison to class I and II patients.

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Modified conditioning regimen improves outcomes of unrelated donor peripheral blood stem cell transplantation for β‐thalassaemia major patients

Abstract: This modified conditioning protocol effectively improved outcomes of UD-PBSCT for patients with β-TM.

Cited by 7 publications

References 22 publications

Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

Current and Future Therapies for β-Thalassaemia: A Review Article

Outcome of Beta Thalassemia Major in Paediatric Patients Allografted from Fully Matched Related Donors; in Egypt

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