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Thalassemia: Common Clinical Queries in Management

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Abstract

Beta thalassemia major (TM) is the most frequent form of transfusion-dependent inherited anemia in India. The thalassemia syndromes exhibit enormous variability in their genetic basis and phenotypic expression. The authors recommend that the diagnosis of TM or non-transfusion-dependent thalassemia (NTDT) should not be based on a one-time assessment. Many patients have a chronic anemia that is not severe enough to justify regular transfusions, but the clinical course can evolve with age. Continued observation may reveal that some patients who are considered NTDT will benefit from transfusions later in life. Clinical decision making can be influenced by the perceived difficulty in access to a safe blood supply and the cost of therapy. Here, authors present selected case scenarios that address common issues in the management of TM or NTDT. The recommendations are based on published evidence where available or on the authors’ shared experience. Among the topics under discussion are deciding when to start regular transfusions, the role of hydroxyurea in TM, the procedure for blood administration, the use of deferasirox for chelation and monitoring of side effects, the role of splenectomy, and the prospects for gene therapy. In order to achieve an optimal outcome with blood transfusions and chelation therapy over the lifetime, it is essential to adhere to the current guidelines for the management of thalassemia.

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Authors and Affiliations

  1. Hematology/Oncology, Department of Pediatrics, University of California, San Francisco, California, USA

    Ashutosh Lal

  2. Pediatric Hematology/Oncology Unit, Department of Pediatrics, Postgraduate Institute of Medical Education and Research, Chandigarh, India

    Deepak Bansal

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  1. Ashutosh Lal
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The authors contributed to formulation of questions and discussion of recommended management. DB is the guarantor of this paper.

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Correspondence to Ashutosh Lal.

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Lal, A., Bansal, D. Thalassemia: Common Clinical Queries in Management. Indian J Pediatr 87, 75–81 (2020). https://doi.org/10.1007/s12098-019-03065-5

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