Long-term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A

Authors: Miesbach, W., Kittler, S., Bauhofer, A., Konigs, C., Becker, T., Nemes, L., Staus, A., and Schuttrumpf, J.

Publication: Haemophilia; April 2020

Affiliations: Haemophilia Centre, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt/Main, Germany ; Biotest AG, Dreieich, Germany.

Abstract: INTRODUCTION: Prophylaxis with factor VIII (FVIII) concentrates in children with haemophilia A (HA) is current standard of care. The benefit of prophylactic treatment for adult HA patients is not commonly accepted. AIM: To investigate the benefit of prophylaxis over on-demand treatment in adult and elderly patients with severe or non-severe HA in a real-life setting. METHODS: Data from 163 patients comprising 1202 patient-years were evaluated for 7.5 (+/-5.3) years. The effects on the annual bleeding rate (ABR, including spontaneous and traumatic bleeds) of treatment with a plasma-derived FVIII concentrate, the patient’s age and disease severity were investigated. The effect of changing the treatment from on demand to continuous prophylaxis on the patients’ ABRs was further analysed. RESULTS: Prophylaxis had the greatest effect on the ABRs of patients of any age with severe or non-severe HA. The difference in ABR of all patients treated on demand (median 31.4; interquartile range (IQR) 27.6; N = 83) compared with those treated prophylactically (median 1.3; IQR 3.6; N = 122) was statistically significant (P < .05), even for patients with non-severe HA (median 8.4; IQR 15.5; N = 11) vs median 1.5; IQR 4.2 (N = 17), P < .05). Patients, aged up to 88 years, switching from on demand to continuous prophylaxis showed the lowest median ABR (1.1; N = 51) after their regimen change. CONCLUSION: Any (even low-frequency) prophylaxis results in lower ABR than on-demand treatment. Patients switching to prophylaxis benefitted the most, irrespective of age or HA severity. Prophylactic treatment-even tertiary-is the regimen of choice for patients of any age, including elderly patients, with severe or non-severe HA.