Related and unrelated donor transplantation for β-thalassemia major: results of an international survey

Li, Chunfu; Mathews, Vikram; Kim, Soyoung; George, Biju; Hebert, Kyle; Jiang, Hua; Li, Changgang; Zhu, Yiping; Keesler, Daniel A.; Boelens, Jaap Jan; Dvorak, Christopher C.; Agarwal, Rajni; Auletta, Jeffery J.; Goyal, Rakesh; Hanna, Rabi; Kasow, Kimberly A.; Shenoy, Shalini; Smith, Angela R.; Walters, Mark C.; Eapen, Mary

doi:10.1182/bloodadvances.2019000291

Cited by 63 publications

(39 citation statements)

References 28 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The 5-year probabilities of overall survival (OS) after HLA-matched relative, HLA-mismatched relative, HLA-matched unrelated, and HLA-mismatched unrelated donor transplants were reported to be 89%,73%, 87%, and 83%, respectively. The 5-year probabilities of EFS after HLA-matched relative, HLA-mismatched relative, HLAmatched unrelated, and HLA-mismatched unrelated donor transplants were 86%, 70%, 82%, and 78%, respectively [23]. This report shows comparable event-free and OS after HLA-matched related and HLA-matched unrelated donor transplantation.…”

Section: Bone Marrow Transplantationmentioning

confidence: 51%

Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation

Motta

Bou‐Fakhredin

Taher

et al. 2020

Drugs

View full text Add to dashboard Cite

Hemoglobinopathies are among the most common monogenic diseases worldwide. Approximately 1-5% of the global population are carriers for a genetic thalassemia mutation. The thalassemias are characterized by autosomal recessive inherited defects in the production of hemoglobin. They are highly prevalent in the Mediterranean, Middle East, Indian subcontinent, and East and Southeast Asia. Due to recent migrations, however, the thalassemias are now becoming more common in Europe and North America, making this disease a global health concern. Currently available conventional therapies in thalassemia have many challenges and limitations. A better understanding of the pathophysiology of β-thalassemia in addition to key developments in optimizing transfusion programs and iron-chelation therapy has led to an increase in the life span of thalassemia patients and paved the way for new therapeutic strategies. These can be classified into three categories based on their efforts to address different features of the underlying pathophysiology of β-thalassemia: correction of the globin chain imbalance, addressing ineffective erythropoiesis, and improving iron overload. In this review, we provide an overview of the novel therapeutic approaches that are currently in development for β-thalassemia.

show abstract

Section: Bone Marrow Transplantationmentioning

confidence: 51%

Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation

Motta

Bou‐Fakhredin

Taher

et al. 2020

Drugs

View full text Add to dashboard Cite

show abstract

“…They reported that the 5-year probabilities of OS after HLA-matched relative, HLA-mismatched relative, HLA-matched unrelated, and HLA-mismatched unrelated donor transplants were 89%, 73%,87% and 83%, respectively. The 5-year probabilities of EFS after HLA-matched relative, HLA-mismatched relative, HLA-matched unrelated, and HLA-mismatched unrelated donor transplants were 86%, 70% and 82%, 78%, respectively 10. This report is the first demonstrating comparable event-free and overall survival after HLA-matched related and HLA-matched unrelated donor transplantation.…”

Section: Bone Marrow Transplantation (Bmt)mentioning

confidence: 61%

Curing Hemoglobinopathies: Challenges and Advances of Conventional and New Gene Therapy Approaches.

Motta

Ghiaccio

Cosentino

et al. 2019

Mediterr J Hematol Infect Dis

View full text Add to dashboard Cite

Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD), are the most common monogenic diseases worldwide, with a global carrier frequency of over 5%.1 With migration, they are becoming more common worldwide, making their management and care an increasing concern for health care systems.BT is characterized by an imbalance in the α/β-globin chain ratio, ineffective erythropoiesis, chronic hemolytic anemia, and compensatory hemopoietic expansion.1 Globally, there are over 25,000 births each year with transfusion-dependent thalassemia (TDT). The currently available treatment for TDT is lifelong transfusions and iron chelation therapy or allogenic bone marrow transplantation as a curative option. SCD affects 300 million people worldwide2 and severely impacts the quality of life of patients who experience unpredictable, recurrent acute and chronic severe pain, stroke, infections, pulmonary disease, kidney disease, retinopathy, and other complications. While survival has been dramatically extended, quality of life is markedly reduced by disease- and treatment-associated morbidity.The development of safe, tissue-specific and efficient vectors, and efficient gene-editing technologies have led to the development of several gene therapy trials for BT and SCD. However, the complexity of the approach presents its hurdles. Fundamental factors at play include the requirement for myeloablation on a patient with benign disease, the age of the patient, and the consequent bone marrow microenvironment. A successful path from proof-ofconcept studies to commercialization must render gene therapy a sustainable and accessible approach for a large number of patients. Furthermore, the cost of these therapies is a considerable challenge for the health care system. While new promising therapeutic options are emerging,3,4 and many others are on the pipeline,5 gene therapy can potentially cure patients. We herein provide an overview of the most recent, likely potentially curative therapies for hemoglobinopathies and a summary of the challenges that these approaches entail.

show abstract

“…Overall survival (OS) and disease‐free survival (DFS) were highest in children aged ≤ 6 years who received grafts from HLA‐matched siblings or HLA‐matched unrelated donors. Five‐year OS rates in patients aged ≤ 6 years, 7–15 years, and 16–25 years were 91%, 83%, and 55%, respectively, and corresponding DFS rates were 87%, 79%, and 55%, respectively 13 …”

Section: Treatmentmentioning

confidence: 99%

Epidemiology and treatment of beta thalassemia major in China

Wang

2019

Pediatric Investigation

View full text Add to dashboard Cite

Thalassemia, classified as the main types α‐ and β‐thalassemia, is a single gene disorder resulting from globin chain synthesis impairment through the mutation or deletion of globin genes. The incidence of thalassemia is high worldwide, with high associated mortality. Therefore, treatment is important to improve patient outcomes. This paper reviews the current status of β‐thalassemia major in China, including its epidemiology and treatment.

show abstract

Related and unrelated donor transplantation for β-thalassemia major: results of an international survey

Cited by 63 publications

References 28 publications

Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation

Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation

Curing Hemoglobinopathies: Challenges and Advances of Conventional and New Gene Therapy Approaches.

Epidemiology and treatment of beta thalassemia major in China

Contact Info

Product

Resources

About