Incidence and Outcome of Discontinuation of Prophylactic Treatment among Young Adults with Severe Hemophilia.

Introduction

Most of the current discussion about prophylaxis for severe hemophilia patients is on the dose and when to start. However, as hemophilia is a chronic disease, it is important to evaluate the duration of prophylaxis. The aim of this study was to study and compare adherence to prophylaxis and outcome of severe hemophilia patients.

Methods

All patients with severe hemophilia A and B (factor VIII/IX<0.01 IU/ml), born between 1970 and 1980 and treated in Copenhagen, Århus (Denmark) or the Van Creveldkliniek, Utrecht (The Netherlands) were studied. Data on treatment were collected from the patients’ files from 1972 until 2003. In addition, a questionnaire on adherence to prophylaxis was used. For assessment of outcome the clinical score according to Gilbert and the radiological Pettersson score were used.

Patients were categorized according to adherence to prophylactic treatment: patients who never discontinued prophylaxis (never), patients who temporarily discontinued prophylaxis (temporarily) and patients had switched to on demand regimen (permanently).

Results

83 patients were studied. Median follow up was 19 years (range 6–29). Median age at start of prophylaxis was 5.9 years (interquartile range (IQR) 4.0–8.7). 34% of patients stopped taking prophylaxis temporarily and 35% stopped taking prophylaxis permanently at a median age of 21.5 years (IQR 18.4–24.4). Follow up since the last stop was 3.6 years (IQR 1.4–7.9), the annual number of joint bleeds on on demand treatment was 3.0 (IQR 1.4–8.7).

The median clinical score was 3.0 points (IQR 1.0–6.0) in patients who never or temporarily stopped and 4.0 (IQR 0.0–6.3) in patients who permanently stopped taking prophylaxis. Pettersson scores were available for the Dutch patients and the median Pettersson score was 13 points (IQR 5–23) for patients who never or temporarily stopped and 13 (IQR 1–24) for patients who stopped permanently. The proportion of patients who discontinued prophylaxis and outcome parameters were similar for the Dutch and Danish patients.

Conclusion

Two thirds of young adults with severe hemophilia on prophylaxis discontinue prophylaxis at least once. One third permanently stop taking prophylaxis, while maintaining a low joint bleed frequency. Four years after switching to on demand treatment, outcome in these patients is similar to outcome in patients who continue taking prophylaxis.