Safety profile of a liquid formulation of deferiprone in young children with transfusion-induced iron overload: a 1-year experience.

Abstract

BACKGROUND:

Data on the use of deferiprone in young children with iron overload are limited.

OBJECTIVE:

To study the safety profile of a liquid formulation of deferiprone in chelating young children with transfusion-induced iron overload.

PATIENTS AND METHODS:

A daily dose of 50-100 mg/kg BW in three divided doses of oral deferiprone was given to young patients who had received at least ten packed red cell transfusions and achieved a serum ferritin level >1000 μg/L during a 12-month period from 2011 to 2012.

RESULTS:

Nine children (four males) diagnosed with various types of thalassaemia (n = 8) and hereditary spherocytosis (n = 1) were enrolled. Their mean (SD) age was 4.5 (1.9) years. The patients received 15-20 ml/kg BW of packed red cell transfusions every 4-8 weeks from a mean (SD) age of 2.1 (1.7) years to maintain a pre-transfusion haematocrit at 27%. A mean (SD) total of packed red cells of 5132 (2725) ml were given within a mean (SD) duration of 2.4 (1.1) years before the study. During the 1-year study period, they received a mean (SD) total of packed red cells of 2194 (680) ml or 138 (50) ml/kg BW with a mean (SD) daily iron load of 0.29 (0.12) mg/kg BW. The pre-treatment geometric mean of serum ferritin of 1863.8 μg/L decreased to 1279.7 μg/L after 1 year of treatment (P = 0.05). All patients tolerated the liquid formulation well and did not experience any gastro-intestinal discomfort, nausea or vomiting.

CONCLUSION:

The liquid formulation of deferiprone is safe in young children with transfusion-induced iron overload.

• Authors: Ampaiwan Chuansumrit, Duantida Songdej, Nongnuch Sirachainan, Pakawan Wongwerawattanakoon, Praguywan Kadegasem, Werasak Sasanakul
• Journal: Paediatrics and international child health (Paediatr Int Child Health) Vol. 36 Issue 3 Pg. 209-13 (Aug 2016) ISSN: 2046-9055 [Electronic] England
• PMID: 26052612 (Publication Type: Journal Article)

Chemical References

• Iron Chelating Agents
• Pyridones
• Deferiprone
• Ferritins

Topics

• Anemia, Hemolytic (therapy)
• Child
• Child, Preschool
• Deferiprone
• Female
• Ferritins (blood)
• Humans
• Iron Chelating Agents (administration & dosage, adverse effects)
• Iron Overload (drug therapy, etiology)
• Male
• Pyridones (administration & dosage, adverse effects)
• Transfusion Reaction