Combined chelation therapy with daily oral deferiprone and twice-weekly subcutaneous infusion of desferrioxamine in children with β-thalassemia: 3-year experience.

Abstract

OBJECTIVE:

To study the efficacy of combined treatment with oral and subcutaneous iron chelators.

MATERIAL AND 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).

CONCLUSION:

Combination therapy with daily oral DFP and subcutaneous DFO twice weekly is a safe and effective alternative to chelation monotherapy in β-thalassemia children.

• Authors: Duantida Songdej, Nongnuch Sirachainan, Pakawan Wongwerawattanakoon, Werasak Sasanakul, Praguywan Kadegasem, Witaya Sungkarat, Ampaiwan Chuansumrit
• Journal: Acta haematologica (Acta Haematol) Vol. 133 Issue 2 Pg. 226-36 ( 2015) ISSN: 1421-9662 [Electronic] Switzerland
• PMID: 25376266 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2014 S. Karger AG, Basel.

Chemical References

• Pyridones
• Siderophores
• Deferiprone
• Ferritins
• Hemoglobin E
• Creatinine
• Alanine Transaminase
• Deferoxamine

Topics

• Administration, Oral
• Adolescent
• Alanine Transaminase (blood)
• Child
• Child, Preschool
• Creatinine (blood)
• Deferiprone
• Deferoxamine (administration & dosage, adverse effects)
• Drug Therapy, Combination (methods)
• Female
• Ferritins (blood)
• Hemoglobin E (metabolism)
• Humans
• Infusions, Subcutaneous
• Male
• Neutropenia (blood, chemically induced)
• Pyridones (administration & dosage, adverse effects)
• Siderophores (administration & dosage, adverse effects)
• Thrombocytopenia (blood, chemically induced)
• beta-Thalassemia (blood, drug therapy)