Combined chelation therapy with daily oral deferiprone and twice-weekly subcutaneous infusion of desferrioxamine in children with β-thalassemia: 3-year experience.
OBJECTIVE
To study the efficacy of combined treatment with oral and subcutaneous iron chelators.


METHODS
50-100 mg/kg/day of oral deferiprone (DFP) combined with 40 mg/kg/dose s.c. desferrioxamine (DFO) twice weekly were given to transfusion-dependent β-thalassemia children.


RESULTS
Enrolled patients (9 with β-thalassemia major and 33 with β-thalassemia hemoglobin E), ranging from 3 to 18 years in age, were divided into 3 groups; group 1 ferritin ≥1,000-2,500 ng/ml (n = 10), group 2 ferritin >2,500-4,000 ng/ml (n = 23) and group 3 ferritin >4,000 ng/ml (n = 9). Of the 42 patients, 28 reached the 36-month follow-up. Ten patients whose ferritin declined <15% while receiving 100 mg/kg/day of DFP were considered nonresponders. The median age and previous transfusion duration before enrollment were significantly higher in nonresponders than responders (p = 0.04 and 0.003, respectively). The responders exhibited a significant fall in median ferritin levels from 2,954.6 to 936.6 ng/ml (p < 0.001). Time to a significant decrease in serum ferritin among responders was 6 months. In 13 patients, 16 episodes of adverse events occurred: hemophagocytosis with cytopenia (n = 1), neutropenia (n = 2), thrombocytopenia (n = 2), elevated alanine aminotransferase (n = 5), elevated serum creatinine (n = 1), proteinuria (n = 1) and gastrointestinal discomfort (n = 4).


CONCLUSIONS
Combination therapy with daily oral DFP and subcutaneous DFO twice weekly is a safe and effective alternative to chelation monotherapy in β-thalassemia children.
Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
Duantida
D
Songdej
Nongnuch
N
Sirachainan
Pakawan
P
Wongwerawattanakoon
Werasak
W
Sasanakul
Praguywan
P
Kadegasem
Witaya
W
Sungkarat
Ampaiwan
A
Chuansumrit
D000284:Administration, Oral; D000293:Adolescent; D000410:Alanine Transaminase / Q000097:blood; D002648:Child; D002675:Child, Preschool; D003404:Creatinine / Q000097:blood; D000077543:Deferiprone; D003676:Deferoxamine / Q000008:administration & dosage* / Q000009:adverse effects; D004359:Drug Therapy, Combination / Q000379:methods; D005260:Female; D005293:Ferritins / Q000097:blood; D006446:Hemoglobin E / Q000378:metabolism; D006801:Humans; D055104:Infusions, Subcutaneous; D008297:Male; D009503:Neutropenia / Q000097:blood / Q000139:chemically induced; D011728:Pyridones / Q000008:administration & dosage* / Q000009:adverse effects; D017262:Siderophores / Q000008:administration & dosage* / Q000009:adverse effects; D013921:Thrombocytopenia / Q000097:blood / Q000139:chemically induced; D017086:beta-Thalassemia / Q000097:blood / Q000188:drug therapy*
D011728:Pyridones; D017262:Siderophores; D000077543:Deferiprone; D005293:Ferritins; D006446:Hemoglobin E; D003404:Creatinine; D000410:Alanine Transaminase; D003676:Deferoxamine