HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe

C Oikonomopoulou, E Goussetis - Bone Marrow Transplantation, 2021 - nature.com
Patients with β-thalassemia suffer from severe anemia, iron overload and multiple
complications, that affect their quality of life and well-being. Allogeneic hematopoietic stem …

[HTML][HTML] Advancing the care of β-thalassaemia patients with novel therapies

R Bou-Fakhredin, I Motta, MD Cappellini - Blood Transfusion, 2022 - ncbi.nlm.nih.gov
The β-thalassaemias are a group of inherited disorders of haemoglobin synthesis
characterised by chronic anaemia of varying severity. Currently available conventional …

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

C Huang, Y Qu, S Liu, S Nie, H Jiang - Translational Pediatrics, 2021 - ncbi.nlm.nih.gov
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 …

[HTML][HTML] Matched family versus alternative donor hematopoietic stem cell transplantation for patients with thalassemia major: experience from a tertiary referral center …

VV Swaminathan, R Uppuluri, S Patel… - Biology of Blood and …, 2020 - Elsevier
Hematopoietic stem cell transplantation (HSCT) is the only curative option available for
patients with thalassemia major in India with increasing access to alternate donor …

Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression

B Meissner, P Lang, P Bader, M Hoenig… - Bone Marrow …, 2024 - nature.com
We performed a retrospective analysis on 124 patients with transfusion-dependent
thalassemia who were registered in the German pediatric registry for stem cell …

Chronic graft-versus-host disease in children and adolescents with thalassemia after hematopoietic stem cell transplantation

O Lertkovit, U Anurathapan, S Hongeng… - International Journal of …, 2021 - Springer
Data on chronic graft-versus-host disease (cGVHD) in patients with thalassemia after
hematopoietic stem cell transplantation (HSCT) have not been specifically explored. The …

[HTML][HTML] Hematopoietic stem cell transplantation and results in pediatric patients with thalassemia major: single-center study

A Ayçiçek, Ş Kalkan, EP Uysalol… - Turkish Archives of …, 2022 - ncbi.nlm.nih.gov
Objective: This study aimed to reveal whether patients with thalassemia major, who were
followed up in our clinic, were given information about hematopoietic stem cell …

Research advances in transplantation for thalassemia major

CW Huang, H Jiang - Zhongguo Dang dai er ke za zhi= Chinese …, 2020 - europepmc.org
地中海贫血是因调节血红蛋白的基因突变导致珠蛋白链形成障碍的遗传性血液疾病.
目前异基因造血干细胞移植是公认的唯一治愈手段, 其经历了预处理方案的演进 …

[HTML][HTML] The Thalassemia Syndromes: New Insights

S Singh, CK Sri, A Bakane - 2023 - intechopen.com
Thalassemia is characterized by impaired synthesis of globin chains in hemoglobin.
Supportive care for this condition includes regular transfusions and adequate iron chelation …

[HTML][HTML] 重型地中海贫血的移植进展

黄楚雯, 江华 - 中国当代儿科杂志, 2020 - zgddek.com
地中海贫血是因调节血红蛋白的基因突变导致珠蛋白链形成障碍的遗传性血液疾病.
目前异基因造血干细胞移植是公认的唯一治愈手段, 其经历了预处理方案的演进 …