Study Shows Positive Outcomes for Early Prophylaxis in Children

Horizons in Hemophilia, March 2011

Contributed by the National Hemophilia Foundation

A group of European researchers published a study in January 2011 affirming that prophylactic, or preventive, treatment of children with hemophilia A, particularly when initiated at an early age (at or before 36 months), can prevent bleeds and joint disease. The lead investigator of the study was Alessandro Gringeri, MD, MSc, Department of Medicine and Medical Specialities, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico and University of Milan, Italy.

Gringeri and colleagues from institutions in Italy, Sweden and Germany, conducted a 10-year study of 45 children with severe hemophilia A, ranging in age from 1 to 7 years old. The median age was 4 years old. When evaluated through radiological imaging and clinical exams prior to the study, the children exhibited no signs of joint damage. Each child has experienced at least one bleed in the six months prior to the study. Of the children, 21 were put on a prophylactic therapy regimen of three factor infusions per week; 19 were treated episodically--after a bleed had begun.

The purpose of the study was to determine if quality of life improved for children on prophylaxis. The results showed that the prophylactic group experienced significantly fewer joint bleeds than the children receiving episodic treatment. Radiology images showed signs of arthropathy, or joint disease, in 29% of the children on prophylaxis vs. 74% of those receiving on-demand treatment. Investigators also reported that prophylaxis was even more effective when started at or before the age of 36 months, with fewer joint bleeds and no radiologic signs of arthropathy.

“Early prophylaxis is more efficacious than delayed prophylaxis, even though the latter still generates substantial benefits to these patients. The higher costs of prophylaxis compared to episodic therapy are balanced by a better orthopedic outcome and hence a better quality of life," wrote the authors.

The study, “A Randomized Clinical Trial of Prophylaxis in Children with Hemophilia A,” was published online January 21, 2011, in the Journal of Thrombosis and Haemostasis.

Source: Modern Medicine (online), February 3, 2011