An overview of the efficacy and safety of deferiprone in paediatric patients with congenital haemoglobinopathies and chronic iron overload

ABSTRACT

Introduction: Congenital hemoglobinopathies represent the most severe forms of anemia in Europe requiring treatment that include regular blood transfusions and iron chelators administered alone or in combination. However, none of the existing chelators completely covers the pediatric needs.

Areas covered: The introduction of deferiprone on the market has represented an important advancement in congenital hemoglobinopathies. Moreover, several studies have shown that deferiprone combined therapy with desferrioxamine is particularly effective in cardiac iron overload. Despite a wide experience on deferiprone use, limited data were available in the pediatric population (<10 years of age) at the time of the marketing authorization. This review investigates and discusses the evidence more recently provided by deferiprone pediatric studies.

Expert opinion: Available deferiprone efficacy and safety data in pediatrics are increasing and demonstrate that DFP is adequate and efficacious at the same dosage used in adults. No significantly increased risk for agranulocytosis and other relevant adverse events/adverse drug reactions is reported, but caution is recommended in very young children and in combined therapy if two chelators (deferiprone + desferrioxamine or deferiprone + deferasirox) are administered simultaneously. The results of larger and statistically powered trials are expected to solve the still existing controversies in pediatric uses of deferiprone.

KEYWORDS:

• Deferiprone
• pediatric population
• chronic iron overload
• congenital hemoglobinopathies
• efficacy
• safety

Article highlights

• Congenital Hemoglobinopathies (CHPs) represent the most severe forms of anemia in the European Union (EU) whose treatment include regular blood transfusions and chelation therapy.

• Currently, CHP patients have different options of chelation treatment. However, none of the existing chelators completely cover the therapeutic needs of the pediatric population.

• Deferiprone is the most widely studied chelator in the pediatric population with thousands of patients enrolled in PK, efficacy and safety trials or included in observational long-term studies.

• Based on these studies and trials, deferiprone demonstrates a favorable risk-benefit ratio in terms of ferritin level decrease and incidence of Adverse Events/Adverse Drug Reactions. However, larger and statistically more powered information is expected based on pediatric ongoing randomized clinical trials.

• Further studies are also necessary to investigate the appropriate dosage in the pediatric population in the combined therapy.

• A regular monitoring and adequate treatment schedule are still needed to avoid severe adverse events and to provide a more personalized therapy.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Supplementary material

Supplemental data for this article can be accessed here.

Additional information

Funding

This paper was not funded.