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Efficacy and Safety of Combined Oral Chelation with Deferiprone and Deferasirox on Iron Overload in Transfusion Dependent Children with Thalassemia – A Prospective Observational Study

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Indian Journal of Pediatrics Aims and scope Submit manuscript

Abstract

Objectives

To assess the efficacy and safety of dual oral iron chelation therapy (deferiprone and deferasirox) in decreasing iron overload status, using serum ferritin and liver and cardiac MRI as indicators, in transfusion dependent thalassemic children.

Methods

This was a prospective observational study conducted in a tertiary care hospital for a period of one year. Children with thalassemia between 2 and 18 y of age with serum ferritin above 1500 ng/ml were started on oral deferiprone and deferasirox. They were followed up for one year. Serum ferritin and MRI quantification of liver and cardiac iron concentration was done at enrolment and end of 12 mo. They were also monitored monthly for any adverse effects.

Results

Twenty one thalassemic children with mean age of 7.8 y (range 4–12 y) and a mean ferritin value of 3129 + 1231.5 ng/ml were enrolled. Mean serum ferritin decreased by 1226.3 ng/ml (p = 0.047, 95% CI =10.2, 1504.3) with 16.8% fall from baseline. The reduction in ferritin correlated significantly with the initial ferritin level (spearman’s rho = 0.742, p = 0.001). Mean liver iron concentration and myocardial iron concentration did not change significantly. Red color urine, transient rise in creatinine and liver enzymes were noted during the study period.

Conclusions

Combined oral chelation with deferiprone and deferasirox significantly decreases the serum ferritin level in children with severe iron overload. The drugs were tolerated well without any serious adverse effects.

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Authors and Affiliations

  1. Department of Pediatrics, Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER), Puducherry, 605006, India

    Rajeswari Rethinaswamy DivakarJose & C. G. Delhikumar

  2. Department of Radiodiagnosis, Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER), Puducherry, India

    G. Ram Kumar

Authors
  1. Rajeswari Rethinaswamy DivakarJose
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  2. C. G. Delhikumar
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  3. G. Ram Kumar
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Contributions

CGDK and RRDJ: Conceptualized the study. RRDJ: Collected the data and drafted the initial manuscript. CGDK and GRK: Analysis of data and literature search; GRK: Intellectual input in final drafting and overall supervision. All authors contributed to the critical revision of the article. Dr. Niranjan Biswal is the Guarantor for this paper.

Corresponding author

Correspondence to C. G. Delhikumar.

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Ethics Committee Approval

Ethics Committee approval was obtained for the study from JIPMER, Institute Ethics committee (Human studies) (JIP/IEC/2017/0292).

Conflict of Interest

None.

Informed Consent

Detailed information about the study was given to the subjects who accepted to participate in the study as well as to their parents and they were asked to sign an informed consent form.

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DivakarJose, R.R., Delhikumar, C.G. & Ram Kumar, G. Efficacy and Safety of Combined Oral Chelation with Deferiprone and Deferasirox on Iron Overload in Transfusion Dependent Children with Thalassemia – A Prospective Observational Study. Indian J Pediatr 88, 330–335 (2021). https://doi.org/10.1007/s12098-020-03442-5

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  • DOI: https://doi.org/10.1007/s12098-020-03442-5

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