(MED-TH-04.2) Real life experience of emicizumab in pediatric population

Emicizumab has transformed how very young children with hemophilia A are managed. Prior to the advent of emicizumab young children with severe hemophilia A would initially be treated on demand and would not be started on prophylaxis until at least 1-2 years of age. It was always recognized that this was not ideal, and most clinicians wished to start prophylaxis earlier, but the problem was always IV access in very young children. Recognizing the value of prophylaxis in preventing all forms of bleeds including intracranial hemorrhages central venous access devices (CVADs) would often be inserted to permit starting prophylaxis at early ages. Not only did this require hospitalizations and intense exposure to factor which sometimes resulted in inhibitor development but additionally CVADs are prone to complications (thrombosis, mechanical failure and infections). Emicizumab, being a non-factor therapy which is given infrequently and subcutaneously has revolutionized the management of all patients with hemophilia but particularly that in very young children.

Most hemophilia treatment centers (HTCs) in Europe, North America (including my own) and many other parts of the world are now primarily commencing young children with severe hemophilia A (PUPS; previously untreated patients) on emicizumab and doing so at much younger ages – sometimes in the first few weeks of life. Until recently there was no published data on the use of emicizumab in children < 1 year of age but recently this data has emerged, and emicizumab has been found to be equally efficacious in preventing bleeds in such young children as it was in older children in the initial HAVEN studies.
HTCs, such as my own are now putting most/all of their PUPs on emicizumab outside of studies; meaning in real life. The outcomes have appeared to be extremely good with most young children experiencing very few, or no bleeds. The consequences of this have also been profound with respect to inhibitor development and now very little inhibitor development is occurring in very young children. There is a fear that we are however postponing inhibitor development to later ages, but most pediatric treaters would be content avoiding inhibitor development in very young children and postponing this to later ages when children would have better IV access. This might still permit immune tolerance induction (ITI) – a process that involves frequent IV infusions of Factor VIII.
In this presentation real-life experiences from my own HTCs will be utilized to demonstrate the benefits but also some potentially troubling issues of starting PUPs with SHA on emicizumab or other non-factor therapies (patients/families not acquiring IV access skills, poorer recognition of bleeds, delays in treating bleeds, etc.).