Abstract | OBJECTIVE: To study the efficacy of combined treatment with oral and subcutaneous iron chelators. MATERIAL AND METHODS: 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.
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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)
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Copyright | © 2014 S. Karger AG, Basel. |
Chemical References |
- Pyridones
- Siderophores
- Deferiprone
- Ferritins
- Hemoglobin E
- Creatinine
- Alanine Transaminase
- Deferoxamine
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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)
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