Hematopoietic Stem Cell Transplantation
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Scientific Literature
Optimizing Outcomes snd Accessibility of Matched Sibling Donor Transplant for Transfusion Dependent Thalassemia in LMICs
Abstract Severe thalassemia is a life-threatening condition common in many low- and middle-income countries. While bone marrow transplantation (BMT) offers a curative option, its accessibility remains limited due to cost…
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Scientific Literature
A Multi-Center Clinical Trial of Allogeneic Hematopoietic Stem Cell Transplantation in Transfusion-Dependent Thalassemia
Allogeneic stem cell transplantation (allo-HSCT) has recently been approved as standard therapy for transfusion-dependent thalassemia (TDT) but remains limited to the use of HLA-matched sibling donors (MSDs), due to a…
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Scientific Literature
Hoping for a Normal Life: Decision‐Making on Hematopoietic Stem Cell Transplantation by Patients With a Hemoglobinopathy and Their Caregivers
Background: To provide insight into the perspectives of children and young adults with transfusion-dependent thalassemia and sickle cell disease and their caregivers regarding the decision for hematopoietic stem cell transplantation…
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Scientific Literature
Health-Related Quality-of-Life Profile of Pediatric Patients with β-Thalassemia after Hematopoietic Stem Cell Transplantation
Matched hematopoietic stem cell transplantation (HSCT) is a feasible and curative treatment in pediatric patients with beta thalassemia major (β-TM). However, little data are available regarding patients and their parents’…
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Scientific Literature
How to Facilitate Decision-Making for Hematopoietic Stem Cell Transplantation in Patients With Hemoglobinopathies. The Perspectives of Healthcare Professionals
Hematopoietic stem cell transplantation decision-making for hemoglobinopathy patients is a complex process, and it remains difficult for health care professionals to decide whether and when a hematopoietic stem cell transplantation…
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Scientific Literature
Setting up and Sustaining Blood and Marrow Transplant Services for Children in Middle-Income Economies: An Experience-Driven Position Paper on Behalf of the EBMT PDWP
Severe blood disorders and cancer are the leading cause of death and disability from noncommunicable diseases in the global pediatric population and a major financial burden. The most frequent of…
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