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South African Journal of Child Health

versão On-line ISSN 1999-7671
versão impressa ISSN 1994-3032


MAILULA, M T; GOGA, Y  e  JEENA, P M. The clinical profile and outcome of children with transfusion-dependent thalassaemia in KwaZulu-Natal Province, South Africa: A 16-year retrospective review. S. Afr. j. child health [online]. 2021, vol.15, n.3, pp.165-169. ISSN 1999-7671.

BACKGROUND. The clinical presentation and the outcome of patients with transfusion-dependent thalassaemia (TDT) in the province of KwaZulu-Natal, South Africa, have not been previously reported. OBJECTIVES. To document the clinical characteristics, treatment strategies, outcomes and complications from a case series of children with TDT. METHOD. A retrospective chart review of the clinical hospital records, laboratory, and blood transfusion data of patients with beta thalassaemia was performed. Data on serum ferritin (SF) levels, magnetic resonance imaging (MRI) R2 and/or T2* radiological tests to assess the presence of iron overload were collated and analysed. Iron chelator medications and haematopoietic stem cell transplantation (HSCT) outcomes were reported. RESULTS. All the 12 children enrolled with TDT were of Indian descent. All patients had regular blood transfusion and iron chelation therapy (ICT). Four patients had iron overload diagnosed on SF, liver and cardiac MRI scanning. Most of the children in this study were either initiated or changed to oral deferasirox (DFX) during treatment follow-up. One child on parenteral iron chelation deferoxamine, with high SF levels, died. Four patients successfully underwent HSCT without complications and are in remission. CONCLUSION. The study confirms that haemoglobin-level-driven blood transfusions with adequate chelation are possible in the public health service in South Africa. Close monitoring for iron overload is required, using a combination of SF and MRI scanning. Oral ICTs are the preferred agents for reducing iron overload. HSCT remains the treatment of choice for TDT when feasible.

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