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A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?
- Source :
- Blood Reviews. 32:300-311
- Publication Year :
- 2018
- Publisher :
- Elsevier BV, 2018.
-
Abstract
- Beta-thalassaemia causes defective haemoglobin synthesis leading to ineffective erythropoiesis, chronic haemolytic anaemia, and subsequent clinical complications. Blood transfusion and iron chelation allow long-term disease control, and haematopoietic stem cell transplantation offers a potential cure for some patients. Nonetheless, there are still many challenges in the management of beta-thalassaemia. The main treatment option for most patients is supportive care; furthermore, the long-term efficacy and safety of current therapeutic strategies are limited and adherence is suboptimal. An increasing understanding of the underlying molecular and cellular disease mechanisms plus an awareness of limitations of current management strategies are driving research into novel therapeutic options. Here we provide an overview of the current pathophysiology, clinical manifestations, and global burden of beta-thalassaemia. We reflect on what has been achieved to date, describe the challenges associated with currently available therapy, and discuss how these issues might be addressed by novel therapeutic approaches in development.
- Subjects :
- Ineffective erythropoiesis
medicine.medical_specialty
Chronic haemolytic anaemia
Blood transfusion
medicine.medical_treatment
Disease
medicine.disease_cause
03 medical and health sciences
0302 clinical medicine
medicine
Animals
Humans
Erythropoiesis
Intensive care medicine
business.industry
beta-Thalassemia
Disease mechanisms
Disease Management
Hematology
Combined Modality Therapy
Transplantation
Beta-thalassaemia
Phenotype
Oncology
030220 oncology & carcinogenesis
Paradigm shift
Quality of Life
Symptom Assessment
business
030215 immunology
Subjects
Details
- ISSN :
- 0268960X
- Volume :
- 32
- Database :
- OpenAIRE
- Journal :
- Blood Reviews
- Accession number :
- edsair.doi.dedup.....1a414cad6d1e184f0a251801ae90a88a