201 The care pathway for young children with hemophilia in the Netherlands 7 INTRODUCTION Hemophilia A and B are inherited bleeding disorders caused by a deficiency in coagulation factor VIII (FVIII) or factor IX (FIX), respectively 1. Hemophilia A is more common than hemophilia B and is estimated to account for 80-85 percent of all hemophilia cases 2. Disease severity is traditionally classified according to residual factor levels into severe (FVIII or FIX < 0.01 IU/mL), moderate (FVIII or FIX 0.01-0.05 IU/mL), and mild (FVIII or FIX 0.06-0.40 IU/mL) hemophilia 2-4. Bleeding phenotype, however, does not always coincide with the disease severity classification 5. Symptoms range from easy bruising, soft tissue bleeding or mucosal bleeding to more severe symptoms such as bleeding in joints, muscles or intracranial bleeding, which may lead to disability 6. To prevent bleeding and disability due to hemophilic arthropathy, patients with severe and sometimes with moderate hemophilia A and B are treated prophylactically. Prophylactic treatment may consist of factor replacement therapy involving frequent intravenous injections of standard or extended half-life FVIII or FIX concentrates, non-factor replacement therapies that can be administrated subcutaneously or, in the near future, other novel therapies including gene therapy 7,8. The evolving hemophilia landscape requires hemophilia treatment centers (HTCs) and their multidisciplinary teams to regularly adapt their care provision. (Inter)national treatment guidelines can guide HTCs to use new treatment modalities as well as other interventions that improve hemophilia care 9,10. However, the extent to which each HTC in each country is able to follow these guidelines and how they choose to adjust their hemophilia care provision may differ due to geographical and resource constraints or sociocultural and political factors 9. Insight into the care pathway, i.e. the healthcare processes and the care trajectories that patients and their caregivers follow within a HTC is essential to organize, standardize, alter and overall improve care for patients with hemophilia 1. Moreover, insight into the current care pathway is necessary to evaluate and improve the cost-effectiveness of treatment and to facilitate the tracking of treatment utilization 11,12. Until now, insight into the care pathway for patients with hemophilia is limited. This study aims to identify and visualize the care pathway that young children aged 0-8 years with hemophilia and their caregivers follow in Dutch HTCs.
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