This approach stemmed from research that patients with mild-to-moderate hemophilia (or those who have clotting factor levels of 1 percent or more) rarely experience spontaneous bleeds and have less joint damage than those with severe hemophilia.
Clinicians hypothesized that if they could keep blood clotting factor levels around 1 percent with regular infusions of clotting factor concentrates, they could reduce the risk of bleeding and prevent joint damage.
Since then, several studies have shown that children who receive prophylactic treatment experience fewer bleeding episodes and have healthier joints.
The World Health Organization (WHO) and the World Federation of Hemophilia advise parents of children with hemophilia to initiate prophylactic treatment at an early age to achieve optimal results for a child with a severe form of the bleeding disorder.
Based on these positive results, prophylaxis is now the gold-standard therapy for people with severe hemophilia. Regular infusions have been shown to allow patients to remain active and participate more fully in daily life. Patients are currently advised to receive prophylaxis three times a week on average to minimize bleeds and long-term joint damage.
But because each patient has different clotting factor levels, prophylactic dosage intervals should be tailored individually, depending on the aim of the treatment, bleeding phenotype, the patient’s daily activities, and cost efficacy. The time a hemophilia patient spends with a low factor level in the blood is linked to both the number of bleeds and the number of joint bleeds.
Pharmacokinetic measurement is one tool that can help monitor and guide treatment, and to educate patients on dose escalation. There are some web-based user-friendly instruments that facilitate pharmacokinetic evaluation of changes in dose and dose interval that healthcare providers can use.
It is important to note that prophylaxis is a preventive measure and should be administered before bleeding episodes. It will not repair joints that are already damaged.
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