Recently the results of the post-marketing safety surveillance study, which was performed by Biovitrum at almost all Nordic hemophilia clinics, were presented in an issue of Haemophilia(1).
 

All patients with severe or moderate hemophilia type A and who were treated with ReFacto at home by self-medication were asked whether they wanted to participate in this observational study, where the safety and efficacy of ReFacto were followed over 24 months. (An observational study means that the study protocol does not change anything regarding dosage, number of visits at the clinic or what is checked at the controls, in this case at the hemophilia treatment centers). ReFacto is administered via an intravenous bolus injection by the patient himself, or in the case of a young child, by a parent. After introduction of a new drug it is important to follow how the drug is used in real life and how effective and safe it is. At the present time there is a demand for so called post-marketing/authorization studies.

Children and young patients with severe hemophilia are normally treated with prophylactic injections (replacement therapy) 2-3 times weekly at home. Some adult patients continue with prophylactic injections, sometimes with less injections per week, while others as well as patients with moderate disease inject themselves in connection with bleedings (on-demand therapy).
 
Hemophilia patients on home-treatment are requested to keep a diary and record all injections taken as prophylaxis or as treatment of bleedings (spontaneous or after trauma), bleeding site as well as amount of factor concentrate given at each occasion and possible side effects. Paper diaries are normally used however; compliance is subject to huge variations and therefore far from perfect. To improve the quality as well as increase the interest for reporting, a handheld computer (Palm) was introduced in our study.
 
Results: Fifty-seven (57) patients were enrolled and were followed over 24 months.
Only 39 patients did record their injections in the diary on a regular basis. Parents to younger children were normally eager to register all injections and bleedings as well as reasons for bleedings and the overall status. In adults the compliance varied while most adolescents did not register their injections more than sporadically. This limited the number of patients with complete and reliable diary data.
 
The overall safety of treatment with ReFacto was rated as excellent or good by the clinicians for all patients at all controls.
The overall efficacy after 24 months was also assessed as excellent (74%) or good (26%). More than 50% of the patients/parents reported no absence from school or work due to bleeding episodes during the study period.
Among patients on regular prophylaxis 20% did not report any bleedings. The others on prophylaxis registered in median 4 bleedings during the 24 months and the majority of these bleedings (93%) were resolved with <2 injections of ReFacto.
 
The results of this study show that long-term treatment with ReFacto is safe and efficacious when used as prophylaxis as well as treatment of bleeding episodes.
 
Christina Rylander
 
1. Petrini P and Rylander C. Clinical safety surveillance study of the safety and efficacy of long-term home treatment with ReFacto® utilizing a computer-aided diary: a Nordic multicentre study. Haemophilia (2009), 15, 175-183.