Abstract Number: PB1061
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: rFVIIIFc has shown efficacy in prophylaxis of PwHA. However, in developing countries lack of access to clotting factor concentrate (CFC) lead to patients not receiving prophylaxis. We present our experience in providing prophylaxis to children with haemophilia A using rFVIIIFc provided by the World federation of Hemophilia as part of the humanitarian aid program.
Aims: To assess the annualized bleed rate in children with severe haemophilia A who are on a low dose rFVIIIFc protocol.
Methods: Children with severe haemophilia A who have been either on episodic therapy or prophylaxis with conventional half -life FVIII who were given prophylaxis with rFVIIIFc were included in this analysis. The dose was 10-15 IU/kg once a week. Annualized bleed rate (AdBR) immediately prior to inclusion in this prophylaxis program was documented. All bleeds were then prospectively documented while on prophylaxis with special emphasis on joint or muscle bleeds. Inhibitors were monitored every 3-6 months depending on number of prior exposures.
Results: A total of 25 children received prophylaxis. Their median age was 8 (range: 1-14) years. The median exposures prior to starting rFVIIIFc was 18.5 (range: 2-225). AdBR prior to prophylaxis was 8 (range: 0-15). At a median dose of 15 IU/kg/week (range: 6-31), the median AdBR was 1 (range: 0-7) at a median follow-up of 100 (range: 26-123) weeks. Thirteen patients had 0-1 AdBR.Twelve patients had low titre inhibitor (<5 BU) at enrolment. At a median exposure of 96 (range: 26-129), none developed high titre inhibitor during the study. Compliance was good with a median of 0 (range: 0-8) missed doses.
Conclusions: rFVIIIFc at lower doses of ~15 IU/kg/ week is effective in reduction of the median AdBR to ≤1/year with normal activities of daily living with the convenience of once a week dosing and lower costs.
[Median AdBR before and after starting prophylaxis]
To cite this abstract in AMA style:
Abraham A, A FN, Geevar T, Nair SC, Singh AS, Kulkarni UP, Devasia AJ, Korula A, M L K, George B, Mathews V, Srivastava A. rFVIIIFc at Lower Doses Can Achieve Clinically Meaningful Reduction of Annualized Bleed Rates in Children with Severe Haemophilia A [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/rfviiifc-at-lower-doses-can-achieve-clinically-meaningful-reduction-of-annualized-bleed-rates-in-children-with-severe-haemophilia-a/. Accessed March 21, 2024.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/rfviiifc-at-lower-doses-can-achieve-clinically-meaningful-reduction-of-annualized-bleed-rates-in-children-with-severe-haemophilia-a/