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Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
The effect of deferasirox on endocrine complications in children with thalassemia
Burçak Kurucu Bilgin1, Ayça Koca Yozgat1, Pamir Isik1
1Department of Pediatric Hematology, Ankara Dışkapı Child Health and Diseases Hematology Oncology Training and Research Hospital, Ankara, Turkey.
Insights
Long-term deferasirox treatment significantly reduced endocrine complications in beta-thalassemia patients. Existing issues improved, and new problems were less likely with this iron chelation therapy.
Area of Science:
- Pediatric Hematology
- Endocrinology
- Pharmacology
Background:
- Beta-thalassemia patients experience significant endocrine complications due to iron overload.
- Effective iron chelation is crucial for managing these complications.
Purpose of the Study:
- To evaluate the long-term impact of deferasirox on endocrine complications in children with beta-thalassemia.
- To assess changes in growth, pubertal development, bone metabolism, and hormonal functions.
Main Methods:
- Retrospective analysis of 31 transfusion-dependent beta-thalassemia patients.
- Initial evaluation (2009-2011) and reevaluation (2018) after long-term deferasirox treatment.
- Assessment of endocrine functions including growth, puberty, bone, thyroid, parathyroid, and glucose metabolism.
Main Results:
- Endocrine complications decreased from 83% to 25.8% after long-term deferasirox treatment (p < 0.05).
- 18 out of 26 patients with pre-existing endocrine issues showed recovery.
- Eight patients (25%) developed new endocrinopathies during the study period.
Conclusions:
- Long-term deferasirox therapy is effective in reducing and managing endocrine complications in beta-thalassemia.
- Deferasirox improves existing endocrine abnormalities and lowers the incidence of new-onset problems.
- This highlights the importance of continuous iron chelation for improving patient outcomes.
Abstract:
Endocrine system dysfunctions are the significant complications of excessive iron overload in beta thalassemia patients. The aim of this study was to evaluate the long-term effect of chelation with deferasirox on endocrine complications. The study group consisted of children with beta thalassemia who had been evaluated for the growth and pubertal development, bone metabolism, thyroid/parathyroid functions, glucose metabolism dysfunctions in the department of pediatric hematology of Ankara Dışkapı Child Health and Diseases Hematology Oncology Training And Research Hospital between 2009-2011 and reevaluated after deferasirox chelation therapy in 2018. Thirty-one transfusion dependent beta-thalassemia patients were enrolled for the study. Seventeen (54.8%) patients were male and the mean age was 16.9 ± 3.8 (9-23) years. Splenectomy was performed in 11 patients (35.5%). In the initial evaluation, 26 patients (84%) received deferoxamine and/or deferiprone and five (17%) patients received deferasirox as a chelator; in the final evaluation all patients were receiving deferasirox. The mean duration of deferasirox treatment was 5.9 ± 2.02 years (1-10 years). Of the 26 patients who had endocrine complications between 2009-2011, 18 were recovered. In the final evaluation, eight patients (25%) developed new endocrinopathies. The frequency of endocrine complications seen before the deferasirox treatment (83%) was higher than the frequency of complications while receiving deferasirox treatment (25.8%) (p < 0,05). In this study, it was determined that both existing endocrine abnormalities were reduced and recent developed problems were less likely with long-term deferasirox treatment in thalassemia patients.
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