Improving outcomes and quality of life for patients with transfusion-dependent β-thalassemia: recommendations for best clinical practice and the use of novel treatment strategies.
Ali T TaherRayan Bou-FakhredinAntonios KattamisVip ViprakasitMaria Domenica CappelliniPublished in: Expert review of hematology (2021)
Approved therapies, including curative gene therapies and supportive treatments such as luspatercept, have the potential to reduce RBC transfusion burden, and improve clinical outcomes and QoL in patients with TD β-thalassemia. Cost of treatment is, however, likely to be a significant barrier for payors and patients.
Keyphrases
- clinical practice
- sickle cell disease
- end stage renal disease
- cardiac surgery
- prognostic factors
- ejection fraction
- newly diagnosed
- chronic kidney disease
- peritoneal dialysis
- type diabetes
- genome wide
- risk factors
- metabolic syndrome
- gene expression
- patient reported
- copy number
- dna methylation
- red blood cell
- climate change
- genome wide identification