Efficacy and safety of deferiprone (Ferriprox), an oral iron-chelating agent, in pediatric patients

Sung Chul Won; Dong Kyun Han; Jong Jin Seo; Nak Gyun Chung; Sang Kyu Park; Kyung Bae Park; Hoon Kook; Chuhl Joo Lyu

doi:10.5045/kjh.2010.45.1.58

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Original Article

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Korean J Hematol 2010; 45(1):

Published online March 31, 2010

https://doi.org/10.5045/kjh.2010.45.1.58

Efficacy and safety of deferiprone (Ferriprox), an oral iron-chelating agent, in pediatric patients

Sung Chul Won¹, Dong Kyun Han², Jong Jin Seo³, Nak Gyun Chung⁴, Sang Kyu Park⁵, Kyung Bae Park⁶, Hoon Kook², and Chuhl Joo Lyu^1*

Author Info. +

¹Department of Pediatrics, College of Medicine, Yonsei University, Seoul, Korea.

²Department of Pediatrics, College of Medicine, Chonnam National University Medical School, Hwasun, Korea.

³Department of Pediatrics, College of Medicine, Asan Medical Center, University of Ulsan, Seoul, Korea.

⁴Department of Pediatrics, College of Medicine, The Catholic University of Korea, Seoul, Korea.

⁵Department of Pediatrics, College of Medicine, University of Ulsan, Ulsan, Korea.

⁶Department of Pediatrics, College of Medicine, Soonchunhyang University, Cheonan, Korea.

Correspondence to : Correspondence to Chuhl Joo Lyu, M.D., Ph.D. Department of Pediatrics, College of Medicine, Yonsei University, 134 Shinchon-dong, Seodaemun-gu, Seoul 120-752, Korea. Tel: +82-2-2228-2060, Fax: +82-2-393-9118, cj@yuhs.ac

Received: February 28, 2010; Revised: March 5, 2010; Accepted: March 8, 2010

This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.

Abstract

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Abstract

Background

Iron overload is a predictable and life-threatening complication in patients dependent on the regular transfusion of RBCs. The aims of this study were to investigate the efficacy and safety of deferiprone in a variety of pediatric hematologic and/or oncologic patients with a high iron overload.

Methods

Seventeen patients (age: 1.1-20.4 years; median: 10.6 years) from 7 hospitals who were treated with deferiprone from 2006 to 2009 were enrolled in this study. Medical records of enrolled patients were reviewed retrospectively.

Results

Serum ferritin levels were 4,677.8±1,130.9 µg/L at baseline compared to 3,363.9±1,149.7 µg/L at the end of deferiprone treatment (P=0.033). Only 1 patient developed neutropenia as a complication.

Conclusion

Deferiprone treatment is relatively safe for pediatric patients suffering from various hematologic and oncologic diseases that require RBC transfusions as part of treatment. However, the potential development of critical complications such as agranulocytosis and/or neutropenia remains a concern.

Keywords Deferiprone, Iron overload, Transfusion, Neutropenia

Article

Original Article

Korean J Hematol 2010; 45(1): 58-61

Published online March 31, 2010 https://doi.org/10.5045/kjh.2010.45.1.58

Efficacy and safety of deferiprone (Ferriprox), an oral iron-chelating agent, in pediatric patients

Sung Chul Won¹, Dong Kyun Han², Jong Jin Seo³, Nak Gyun Chung⁴, Sang Kyu Park⁵, Kyung Bae Park⁶, Hoon Kook², and Chuhl Joo Lyu^1*

¹Department of Pediatrics, College of Medicine, Yonsei University, Seoul, Korea.

²Department of Pediatrics, College of Medicine, Chonnam National University Medical School, Hwasun, Korea.

³Department of Pediatrics, College of Medicine, Asan Medical Center, University of Ulsan, Seoul, Korea.

⁴Department of Pediatrics, College of Medicine, The Catholic University of Korea, Seoul, Korea.

⁵Department of Pediatrics, College of Medicine, University of Ulsan, Ulsan, Korea.

⁶Department of Pediatrics, College of Medicine, Soonchunhyang University, Cheonan, Korea.

Correspondence to:Correspondence to Chuhl Joo Lyu, M.D., Ph.D. Department of Pediatrics, College of Medicine, Yonsei University, 134 Shinchon-dong, Seodaemun-gu, Seoul 120-752, Korea. Tel: +82-2-2228-2060, Fax: +82-2-393-9118, cj@yuhs.ac

Received: February 28, 2010; Revised: March 5, 2010; Accepted: March 8, 2010

Abstract

Background

Methods

Results

Serum ferritin levels were 4,677.8±1,130.9 µg/L at baseline compared to 3,363.9±1,149.7 µg/L at the end of deferiprone treatment (P=0.033). Only 1 patient developed neutropenia as a complication.

Conclusion

Keywords: Deferiprone, Iron overload, Transfusion, Neutropenia

Abstract

Fig 1.

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Figure 1.

Serum ferritin concentrations of all patients at baseline (■) versus end of study (▲). Compared to baseline, the decline in serum ferritin levels at the end of the study was statistically significant (P=0.033).

Blood Research 2010; 45: 58-61https://doi.org/10.5045/kjh.2010.45.1.58

Table 1 . Patient characteristics..

Table 2 . Changes in biochemical markers of the hematologic, hepatic, and renal systems at baseline and end of deferiprone treatment..

^a)Student's t-test for statistical analysis..