Effectiveness of Oral Deferiprone (GPO-L-ONE®) in Pediatric Thalassemia Patients with Iron Overload, in Krabi Provincial Hospital

Authors

  • Sunjutha Sudjan Krabi Hospital, Krabi Province

Keywords:

deferiprone, effectiveness, thalassemia, iron overload

Abstract

Objective: To study the effectiveness of oral deferiprone (GPO-L-ONE) in pediatric thalassemia patients with iron overload.

Method: A retrospective descriptive study was conducted in pediatric thalassemia patients who had serum ferritin levels above 1,000 µg/L treated with GPO-L-ONE® chelation since 2018 to 2021. Serum ferritin levels were measured every six months and patients were followed up for 30 months. In addition, laboratories and clinical safety monitoring were performed. Paired t–test and repeated measurement ANOVA were used to analyze data.

Result: One hundred and eighty pediatric thalassemia patients were included in this study and they had 7.53 ± 3.73 years of average age with either β-thalassemia/Hb E disease (50%) or Homozygous β-thalassemia (46.11%) or Hb H Constant Spring (3.89%). After treatment for 12, 18, 24 and 30 months, serum ferritin levels were decreased significantly (p=0.004, 0.014, 0.02 and 0.02, Mean difference = -118, -137, -170 and -207 µg/L respectively). Only 56 patients were completely followed up every 6 months for 30 months and the serum ferritin levels at before and after treatment of this group were compared and the levels were nosignificantly decreased from 1282±216 to 1075±619 µg/L. The most common adverse drug reactions observed in patients were increased transaminase followed by neutropenia.

Conclusion: GPO-L-ONE® is an effective oral iron-chelating agent for treatment in pediatric thalassemia with iron overload. However, safety monitoring especially increased transaminase and neutropenia should be closely observed.

References

Wilkins SN. Iron chelation therapy in thalassemia. J Government Pharmaceutical Organization 2017;24(2):8-10.

Viprakasit V, Tanphaichitr V, Mahasandana C, Assteerawatt A, Suwantol L, Veerakul G, et al. Linear growth in homozygous beta-thalassemia and beta-thalassemia/hemoglobin E patients under different treatment regimens. J Med Assoc Thai 2001;84(7):929-41.

Brittenham GM, Griffith PM, Nienhuis AW, McLaren CE, Young NS, Tucker EE, et al. Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. NEJM 1994;331(9):567-73.

Ceci A, Baiardi P, Catapano M, Felisi M, Cianciulli P, De Sanctis V, et al. Risk factors for death in patients with beta-thalassemia major: results of a case-control study. Haematologica 2006;91(10):1420-1.

Telfer P, Coen PG, Christou S, Hadjigavriel M, Kolnakou A, Pangalou E, et al. Survival of medically treated thalassemia patients in Cyprus. Trends and risk factors over the period 1980-2004. Haematologica 2006;91(9):1187-92.

Yamane T. Statistics–An Introductory Analysis. Tokyo: John Weather Hill. Inc; 1970.

Jamuar SS, Lai AH. Safety and efficacy of iron chelation therapy with deferiprone in patients with transfusion-dependent thalassemia. Ther Adv Hematol 2012;3(5):299-307.

Chueamuangphan N. Effectiveness of Deferiprone (GPO-L-ONE®) Chelation Therapy in adult thalassemia patients with iron overload. J Hematol Transfus Med 2012;22(3):189-94.

Pienwittayapun R. Safety and effectiveness of oral deferiprone (GPO-L-ONE®) in Iron-overload children patient with thalassemia at Queen sirikit national institute of child health. J DMS 2018;43(3):103-13.

Glickstein H, El RB, Link G, Breuer W, Konijn AM, Hershko C, et al. Action of chelators in iron-loaded cardiac cells: accessibility to intracellular labile iron and functional consequences. Blood 2006;108(9):3195-203.

Galanello R. Deferiprone in the treatment of transfusion-dependent thalassemia: a review and perspective. Ther Clin Risk Manag 2007;3(5):795-805.

Viprakasit V. Clinical practice guideline for deferiprone in patients with iron overload. Bangkok : Faculty of Medicine Siriraj Hospital; 2019.

Department of medical services. Clinical practice guidline for diagnosis and management of thalassemia syndromes Nonthaburi: Veterans Affairs Printing House; 2017.

Hoffbrand AV. Deferiprone therapy for transfusional iron overload. Best Practice Res Clin Haematol 2005;18(2):299-317.

Cohen AR, Galanello R, Piga A, Dipalma A, Vullo C, Tricta F. Safety profile of the oral iron chelator deferiprone: a multicentre study. Br J Haematol 2000;108(2):305-12.

Naithani R, Chandra J, Sharma S. Safety of oral iron chelator deferiprone in young thalassaemics. Eur J Haematol 2005;74(3):217-20.

Published

2023-02-24

How to Cite

1.
Sudjan S. Effectiveness of Oral Deferiprone (GPO-L-ONE®) in Pediatric Thalassemia Patients with Iron Overload, in Krabi Provincial Hospital. SRIMEDJ [Internet]. 2023 Feb. 24 [cited 2024 Jun. 16];38(1):29-38. Available from: https://li01.tci-thaijo.org/index.php/SRIMEDJ/article/view/256498

Issue

Section

Original Articles