Prophylaxis
Prophylaxis treatment of hemophilia is defined as a course of regular factor VIII
infusions with the goal of preventing bleeding episodes1. Studies have
linked prophylaxis to decreases in bleeding episodes and long-term morbidity,2-4
reductions in emergency room visits and hospitalizations,4 and improvements
in capacity for physical activity and academic performance.1,2 Due to
these demonstrable benefits of prophylaxis, the Medical and Scientific Advisory
Council (MASAC) has recommended that prophylaxis be considered optimal therapy for
those with severe hemophilia A.5
Initially, prophylactic regimens of care were supported by the early observation
that patients with moderate or mild hemophilia had a much decreased incidence of
musculoskeletal complications.6 Therefore, the hypothesis was developed
that if one could modulate a patient with severe hemophilia through regular prophylactic
infusion therapy, perhaps the incidence of joint damage in young boys with Hemophilia
A could be altered.7
Based on the clinical experience and excellent outcomes of prophylaxis that were
reported from Sweden, beginning in the late 1950s, the World Federation of Hemophilia
(WFH), the National Hemophilia Foundation (NHF), and the World Health Organization
(WHO) have recommended the use of prophylaxis.
Prophylaxis in the United States
The use of prophylaxis in the United States has gradually increased over the years.
Of approximately 7,000 patients with severe hemophilia A, approximately half are
on prophylaxis8. Adherence and cost are seen as being 2 significant barriers
to prophylaxis. The Centers for Disease Control and Prevention(CDC) reports that
13.4% of adults with Hemophilia are on prophylaxis vs 42.2% in the 6- to 12-year
age group8.
FAQs on Prophylaxis
What does the Helixate® FS indication for prophylaxis include?
Helixate® FS is indicated for routine prophylaxis in children with
hemophilia A with no pre-existing joint damage.
When did CSL Behring receive FDA approval for this indication?
Helixate® FS was approved for this indication on July 30, 2009. Read full press release.
Which clinical trial demonstrated adequate efficacy for Helixate®
FS in a prophylactic regimen?
This indication was granted by the FDA based on data from a multicenter, randomized,
open-label trial was conducted in 65 boys with severe hemophilia A between April
1996 and April 2005. The results of this trial have been published in The New England
Journal of Medicine under the title "Prophylaxis versus Episodic Treatment
to Prevent Joint Disease in Boys with Severe Hemophilia" Marilyn
J. Manco-Johnson, M.D., Thomas C. Abshire, M.D. et al.
What was the conclusion of this study?
Primary outcome of the study:
Prophylaxis is effective in preventing hemarthroses and structural joint damage
in young boys with hemophilia A.
Prophylaxis is superior to enhanced episodic therapy or on-demand therapy in preventing
joint disease
Are all factor concentrates indicated for prophylaxis?
No, not all factor concentrates are indicated for prophylactic use. Read the indications
section in the package insert of the product in use to find out whether the product
is indicated for prophylaxis
References:
1. Shapiro AD, Donfield SM, Lynn HS, et al. Defining the impact of hemophilia: the
Academic Achievement in Children with Hemophilia Study. Pediatrics. 2001;108:e105.
2. Thornburg CD, Pipe SW. Adherence to prophylactic infusions of factor VIII or
factor IX for haemophilia. Haemophilia. 2006;12:198-199
3. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic
treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med.
2007;357:535-544.
4. Panicker J, Warrier I, Thomas R, Lusher JM. The overall effectiveness of prophylaxis
in severe haemophilia. Haemophilia. 2003;9:272-278.
5. National Hemophilia Foundation. Medical and Scientific Advisory Committee (MASAC)
Recommendation Concerning Prophylaxis (Regular Administration of Clotting Factor
Concentrate to Prevent Bleeding), MASAC Document #179, November 4, 2007
6. Ahlberg A. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of
arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta
Orthop Scand Suppl. 1965;suppl 77:3-132.
7. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience
of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992;232:25-32.
8. Centers for Disease Control and Prevention - Universal Data Collection database