Hepatic and Cardiac Iron-load in Children on Long-term Chelation with Deferiprone for Thalassemia Major.

Abstract

OBJECTIVE:

To evaluate the efficacy of prolonged deferiprone monotherapy in patients with b-thalassemia major.

METHODS:

This cross-sectional study included 40 patients (age range 9 to 38 years) with thalassemia major receiving deferiprone for ≥5 years. Serum ferritin, and myocardial iron concentration (MIC) and liver iron concentration (LIC) assessed by T2*MRI were recorded.

RESULTS:

The patients were receiving deferiprone for a mean (SD) duration of 12.1 (4.7) years. The median (IQR) dose of deferiprone was 85 (74.3, 95) mg/kg/day. The MIC was normal or had a mild, moderate or severe elevation in 29 (72.5%), 3 (7.5%), 3 (7.5%), and 5 (12.5%) patients. The LIC was normal or had a mild, moderate or severe elevation in 2 (5%), 4 (10%), 11 (27.5%) and 23 (57.5%) patients.

CONCLUSIONS:

The majority of patients receiving deferiprone had a moderate/severe hepatic but normal cardiac iron load. Prolonged deferiprone monotherapy was suboptimal for hepatic iron load in the majority.

• Authors: Sidharth Totadri, Deepak Bansal, Amita Trehan, Alka Khadwal, Anmol Bhatia, Kushaljit Singh Sodhi, Prateek Bhatia, Richa Jain, Reena Das, Niranjan Khandelwal
• Journal: Indian pediatrics (Indian Pediatr) Vol. 55 Issue 7 Pg. 573-575 (07 15 2018) ISSN: 0974-7559 [Electronic] India
• PMID: 30129538 (Publication Type: Journal Article)

Chemical References

• Biomarkers
• Iron Chelating Agents
• Pyridones
• Deferiprone
• Iron

Topics

• Adolescent
• Adult
• Biomarkers (metabolism)
• Child
• Cross-Sectional Studies
• Deferiprone
• Drug Administration Schedule
• Female
• Humans
• Iron (metabolism)
• Iron Chelating Agents (adverse effects, therapeutic use)
• Iron Overload (chemically induced, diagnosis)
• Liver (metabolism)
• Male
• Myocardium (metabolism)
• Pyridones (adverse effects, therapeutic use)
• Treatment Outcome
• Young Adult
• beta-Thalassemia (drug therapy)