Retrospective Evaluation of Secondary Episodic Prophylaxis with rFVIIa in Hemophilia Patients with Inhibitor

Sedano C, Altisent C, Garcia-Candel F, Gimenez F, Mingot ME, Nunez R, Paloma MJ, Soto I, Velasco F, Cid AR, Jimenez V, Fernandez-Lopez MF, Prieto M.  Retrospective Evaluation of Secondary Episodic Prophylaxis with rFVIIa in Hemophilia Patients with Inhibitor.  Journal of Coagulation Disorders, July 2010; 2(2):  1-5

REVIEW ARTICLE

C. Sedano^1, C. Altisent^2, F. García-Candel^3, F. Giménez^4, E. Mingot^5, R. Núñez^6, M. Paloma^7, I. Soto^8, F. Velasco^9, A.R. Cid^10, V. Jiménez^11, M. López-Fernández¹² and M. Prieto¹³

Affiliations: ¹Haematology, Hospital Universitario Marqués de Valdecilla, Santander, ²Haemophilia Unit, Hospital Vall d'Hebron, Barcelona, ³Haematology, Hospital Virgen de la Arrixaca, Murcia, ⁴Haematology, Hospital Torrecárdenas, Almería, ⁵Haematology, Hospital Carlos Haya, Málaga, ⁶Haemophilia Unit, Hospital Virgen del Rocío, Sevilla, ⁷Haematology, Hospital Virgen del Camino, Pamplona, ⁸Haematology, Hospital Central de Asturias, Oviedo, ⁹Haematology, Hospital Reina Sofía, Córdoba, ¹⁰Congenital Coagulophaties Unit, Hospital la Fe, Valencia, ¹¹Haemophilia Unit, Hospital La Paz, Madrid, ¹²Haematology, Complejo Hospitalario A Coruña, A Coruña, ¹³Haematology, Hospital General Yagüe, Burgos, Spain and the Working Group for the Treatment of Haemophilia Patients with Inhibitor from Sociedad Española de Trombosis y Hemostasia (SETH, Spanish Society of Thrombosis and Hemostasis)


ABSTRACT

BACKGROUND
Hemophilic arthropathy is a significant cause of morbidity in hemophilia patients. Prevention of this condition is now a major aim of hemophilia treatment. In patients with high-responding inhibitors, prophylactic administration of bypassing agents may be of benefit, as has been seen in hemophilia without inhibitors.

OBJECTIVES
This retrospective study assesses the reduction in the number of hemarthroses after secondary prophylaxis with rFVIIa is started in hemophilia patients with high-responding inhibitors and initial or advanced arthropathy.

PATIENTS AND METHODS
Nine patients were included: eight with hemophilia A and one with hemophilia B. The number of hemarthrosis episodes occurring in the 3 months following initiation of the prophylaxis program was compared with those in the previous 3 months.

RESULTS
The rFVIIa dose was ≥90 µg/kg, except in two patients who received 60 µg/kg, administered as a single daily dose in six patients, every 2 days in one patient, and three times a week in two patients. Duration of prophylaxis was 1-19 months. In four patients, no hemarthroses occurred during the prophylaxis period. One patient presented only one hemarthrosis, versus eight who had previously . Three patients did not experience any improvement, and one presented a partial response. The overall reduction in the number of joint bleeds in the series was 54% (22 episodes, versus 48 episodes during on-demand treatment).

CONCLUSIONS
These results suggest that secondary rFVIIa prophylaxis reduces the number of hemarthrosis episodes in hemophilia patients with high-responding inhibitors.

Keywords: rFVIIa, hemarthroses, hemophilia, inhibitors, prophylaxis

Correspondence: Carmen Sedano,  Hospital  Marqués de Valdecilla, Servicio de Haematologia-Coagulacion, Av. Valdecilla 25, 39008Santander(Cantabria), Tel: +34 942203816 . Fax: +34 942202559
email: hemsbc@humv.es