Deferiprone effectiveness in thalassemia major children with or without hepatitis B or C virus infection: a non-randomized study
Abstract
Background A high incidence rate of hepatitis B or C virus
infection is found among thalassemia children in Indonesia. This
may influence deferiprone effectiveness.
Objective To determine the effectiveness of deferiprone in
thalassemia children with or without hepatitis B or C virus
infection.
Methods A non-randomized clinical study was performed at
Thalassemia Center Jakarta. Subjects were thalassemia children
with serum ferritin level > 1000 ng/mL who had hepatitis B or
C virus infection. A match control pair was recruited based on
similar duration of transfusion therapy, thalassemia type, and
initial serum ferritin level. All subjects received initial deferiprone
dose of 50 mg/kg/day for 3 months. Those whose ferritin decreased
2:: 10% continued to receive deferiprone of 50 mg/kg/day for the
following 3 months. Otherwise, deferiprone dose was adjusted
to 75 mg/kg/day.
Results Forty-eight subjects were recruited. After 3 months of
treatment, 16/24 subjects without and 6/24 subjects with hepatitis
B or C had their ferritin level decreased 2:: 10%. Mean ferritin
serum level of all subjects after 6 months was significantly reduced
from 4734 (SD 2116) to 3695 (SD 1709) ng/mL. Lower mean
deferiprone dose, lower mean post- study ferritin serum level and
higher mean percentage of ferritin serum level decrement were
found in subjects without hepatitis B or C infection than those
with infection.
Conclusions Deferiprone 50-7 5 mg/kg/day for 6 months is effective
in reducing serum ferritin level of thalassemia major children; it
is more effective in thalassemia children without hepatitis B or C
virus infection.
References
2005;353: 1135-46.
2. Data of Thalassemia Center Cipto Mangunkusumo Hospital,
Jakarta December 2008.
3. Permono B, Ugrasena IDG. Thalassemia. In: Permono B,
Sutaryo, Ugrasena IDG, Windiastuti E, Abdulsalam M,
editors. Buku ajar hematologi-onkologi anak (Textbook of
Pediatric Hematology-Oncology). Jakarta: Ikatan Dokter
Anak Indonesia, 2005; p. 64-84.
4. Cohen AR, Galanello R, Pennel DJ, Cunningham MJ,
Vichinsky E. Thalassemia. Hematology. 2004;1:14-32.
5. Thalassemia International Federation. Guidelines for the
clinical management of thalassemia. Athens: Thalassemia
International Federation; 2000.
6. Wahidiyat PA. Personal communication.Jakarta, 19 June 2008.
7. Anderson L, Holden S, Boris B, CardiovascularT2-star (T2*)
magnetic resonance for the early diagnosis of myocardial iron
overload. Eur Heart J. 2001 ;22 :2171-9.
8. Olivieri NF, Brittenham GM, Matsui D, Berkovitch M,
Blendis LM, Cameron RG, et al. Iron-chelation therapy with
oral deferiprone in patients with thalassemia major. N Engl]
Med. 1995;332:918-22.
9. Cohen AR, Galanello R, Piga A, DiPalma A, Vullo C,
Tricta F. Safety profile of the oral iron chelator deferiprone:
a multicentre study. Br] Haematol. 2000;108:305-12.
10. Olivieri NF, Brittenham GM, McLaren CE, Templeton
DM, Cameron RG, McClelland RA. Long-term safety and
effectiveness of iron-chelation therapy with deferiprone for
thalassemia major. N Engl J Med. 1998;339:417-23 .
11. Addis A, Loebstein R, Koren G, Einarson TR. Meta-analytic
review of the clinical effectiveness of oral deferiprone. Eur J
Clin Pharmacol. 1999;55:1-6.
12. Pootrakul P, Sirankapracha P, Sankote J, Kachintorn U,
Maungsub W, Sriphen K, et al. Clinical trial of deferiprone
iron chelation therapy in ()-thalassemia /hemoglobin E
patients in Thailand. Cr J Haematol. 2003;122:305-10.
13. Kowdley KV, Kaplan MM. Iron chelation therapy with
oral deferiprone: toxicity or lack of efficacy? N Engl J Med.
1998;339:468-9.
14. Mazza P, Amurri B, Lazzari G, Masi C, Palazzo G, Spartera
MA, et al. Oral iron chelating therapy. A single center interim
report on deferiprone (Ll) in thalassemia. Haemotologica.
1998;83:496-501.
15. Ced A, Baiardi P, Felisi M, Cappellini MD, Carnelli V, De
Sanctis V, et al. The safety and effectiveness of deferiprone in
a large-scale, 3-year study in Italian patients. Br J Haematol.
2002; 118:33 0-6.
16. Maggio A, D'Amico G, Morabito A, Capra M, Ciaccio C,
Cianciulli P. Deferiprone versus deferoxamine in patients
with thalassemia major: a randomized clinical trial. Blood
Cell Mol Dis. 2002;28: 196-208.
17. Chen AC, Peng CT, Wu SF, Wu KH, Chiang IP, Tsai CH.
Effect of deferiprone on liver iron overload and fibrosis
in hepatitis-C-virus-infected thalassemia. Hemoglobin.
112 • Paediatr lndones, Vol. 50, No. 2, March 2010
2006;30:209-14.
18. Olivieri NF, Brittenham GM. Iron-chelating therapy and the
treatment of thalassemia. Blood. 1997;89:739-61.
Authors who publish with this journal agree to the following terms:
Authors retain copyright and grant the journal right of first publication with the work simultaneously licensed under a Creative Commons Attribution License that allows others to share the work with an acknowledgement of the work's authorship and initial publication in this journal.
Authors are able to enter into separate, additional contractual arrangements for the non-exclusive distribution of the journal's published version of the work (e.g., post it to an institutional repository or publish it in a book), with an acknowledgement of its initial publication in this journal.
Accepted 2017-01-29
Published 2010-04-30
