Clinician Investigator Hospital for Sick Children Toronto, Canada
The management of hemophilia has always involved regular visits by the patient and his family to comprehensive care hemophilia treatment centers (HTCs). The establishment of comprehensive care HTCs was critical to the evolution of hemophilia care. HTCs comprise a multidisciplinary group of professionals (physicians, nurses, physiotherapists, social workers) dedicated to the evaluation and care of patients with hemophilia and other bleeding disorders. Regular visits to HTC's have allowed patients to be informed of different therapies, allowed patients to be evaluated on a regular basis and to have their treatments adjusted based on the status of the patient. This has been particularly important in managing pediatric patients with hemophilia. Regular visits to HTCs have been crucial for teaching families all about the condition of hemophilia and what to expect as the child ages. It has allowed for appropriate teaching of bleed recognition and how to respond to bleeds if, and when, they occur. Critically regular visits to HTCs have been crucial to teaching patients/families how to infuse factor, both to treat bleeds and for purposes of prophylaxis. This has allowed families to become more independent and to be able to respond to bleeds quicker thus resulting in better long-term outcomes. Regular visits to HTCs have been important to undertake regular inhibitor screening particularly during a child’s first few years of life when inhibitors are much more likely to occur.
Nowadays with the movement away from prophylaxis with factor concentrates to prophylaxis with non-factor therapies there is a concern that this will result in the perception that regular visits to HTCs are less important. Prophylaxis with emicizumab (the first of likely many non-factor therapies) has been shown to be highly effective with a low rate of bleeding. With non-factor therapies patients/families are for the most part not learning self-(IV) infusion skills and thus are not having to come to HTCs to the same degree as in the past. Additionally in the future if patients undergo gene therapy there may also be a tendency for them to be seen less frequently in HTCs.
Yet given that non-factor therapies do not, as yet completely abolish the risk of bleeding and that their mechanism of action is much more complex it is even more imperative that at least for the near future patients continue with regular visits to the HTCs.
Learning Objectives:
The value of regular patient/family visits to HTC will be discussed particularly in the context of pediatric hemophilia care. The concern is that with lower rates of bleeding and the use of non-factor therapies that patients may not value regular visits to HTCs in the same way as they did in the past. All of this will be extensively discussed.
