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Epidemiological data and treatment strategies in children with severe haemophilia in Italy
Francesca Abbonizio1, Hamisa J Hassan1, Elena Santagostino2
1Dipartimento di Oncologia e Medicina Molecolare, Istituto Superiore di Sanità, Rome, Italy.
Insights
Italian children with severe haemophilia show high adherence to prophylaxis, with significant use of extended half-life (EHL) clotting factor concentrates. This data provides a benchmark for future haemophilia care advancements.
Area of Science:
- Paediatric Haematology
- Haemophilia Care
- Congenital Coagulopathies
Background:
- The field of haemophilia care is undergoing significant therapeutic evolution.
- Accurate statistics on paediatric severe haemophilia populations are crucial for guiding treatment strategies.
Purpose of the Study:
- To present updated statistics on children (0-12 years) with severe haemophilia in Italy.
- To analyze current treatment adherence and factor concentrate utilization in this demographic.
Main Methods:
- Utilized data from the Italian National Registry of Congenital Coagulopathies (NRCC) survey conducted in 2017.
- Focused on children aged 0-12 years diagnosed with severe Haemophilia A (HA) or Haemophilia B (HB).
Main Results:
- The study identified 242 children with severe HA and 48 with severe HB.
- Prophylaxis was administered to 92.1% of severe HA patients and 88.6% of severe HB patients.
- Extended half-life (EHL) factor concentrates accounted for 4.6% of FVIII and 19.1% of FIX usage.
Conclusions:
- The paediatric haemophilia population in Italy demonstrates excellent adherence to prescribed therapies.
- These findings establish a valuable benchmark for evaluating the adoption of novel and alternative haemophilia treatments.
Objectives:
In a period of important therapeutic changes in the field of haemophilia care, we provide updated statistics on children with severe haemophilia (0-12 years of age) in Italy.
Methods:
Data presented are from the Italian National Registry of Congenital Coagulopathies (NRCC) - survey 2017.
Results:
Children with severe Haemophilia A (HA) were 242, those with severe haemophilia B (HB) 48. Prophylaxis was adopted in 92.1% of individuals with severe HA and 88.6% with severe HB. Thirty-nine children (14.8%) were on treatment for inhibitors. FVIII prescribed to children with severe HA represented 11.1% of the total consumption, of which 4.6% was extended half-life (EHL). FIX given to children with HB accounted for 7.2% of the total FIX, of which 19.1% was EHL-FIX.
Conclusion:
The paediatric population analysed is characterized by a great adherence to therapy, so this data may constitute a benchmark for use of new, alternative therapies in the coming years.
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