Eculizumab in patients with classic PNH: A cost effectivity study.

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare acquired benign stem cell disorder characterized by hemolysis, anaemia, abdominal pain and thrombophilia. The sypmtoms interfere strongly with patient's social and working life. The newly registered orphan drug eculizumab has changed the lives of PNH patients significantly. Patients have less fatigue and a remarkably improved quality-of-life. Administration of eculizumab is per i.v. infusion once every week. In the Netherlands PNH patients travel to Nijmegen for each eculizumab infusion. In this study patients already on eculizumab treatment and new patients after at least 3 months of treatment at the out-patient clinics of the Radboud University Nijmegen Medical Center will be offered eculizumab treatment by specialized nurses at home. Cost effectiveness, expressed as cost and patient satisfaction will be compared per patient over 6 months of treatment in the expert center and 6 months at home.

Evaluation of eculizumab treatment in PNH patients at home compared to treatment at the center of excellence in the Netherlands.

1. 6 months treatment in the expert center;

2. 6 months treatment at home;

3. Patients fill in questionnaire every 1,5 months.

A questionnaire is filled in by patients. No intervention will be given because the patients are already using eculizumab.