PB0531 - Prophylaxis in boys with severe haemophilia A: Patient and parent questionnaire on treatment choices. A single centre experience.

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

JA

Jayanthi Alamelu

Consultant Paediatric Haematologist
Evelina London Haemophilia Centre
London, England, United Kingdom

Co-Author(s)

CJ

Clare James

Clinical Nurse Specialist
Evelina London Children's Hospital
London, England, United Kingdom

Background: Majority of boys with severe haemophilia A in our centre are now on prophylaxis with Emicizumab, many of whom have only had minimal exposure to FVIII concentrate.

Aims: An internal service evaluation project was carried out to evaluate the direction of treatment choices and implications of this on staffing.

Methods: A simple survey was undertaken on patients on Emicizumab; previously untreated/ minimally treated patients (PUPs/MTPs) and previously treated patients (PTPs). Patients with inhibitors were not included. Responses were also collected from boys that were able to answer the questions. Questions included satisfaction and pain during injection as well as switching to FVIII injections. Quantitative scale was graded between 0 (denoting poor response for satisfaction and extremely painful for injection associated discomfort) to 5 (denoting excellent response for satisfaction and insignificant injection associated discomfort).

Results: Questionnaires were collected from 6 PUPs/MTPs and 10 PTPs. All PUP questionnaires were filled by parents as the boys were too young. Of the 6 PUPs, satisfaction was graded as 5 in all. Pain causing a concern was rated 0-3. Parent responses on FVIII injections being introduced was agreeable in 3/6 but for consideration when the child was older. In PTPs, 8/10 were also answered by the child. 9/10 PTP parents graded overall satisfaction as 4 or 5. Pain causing a concern ranged between 0-5. 7 of the 8 boys graded satisfaction as 5/5 and pain 0-4. The option of switching to an IV FVIII injection was acceptable to just 1/10 parents of PTPs and 0/8 PTPs. There was no difference to this response with once weekly FVIII injection.

Conclusion(s): Emicizumab is the preferred option among parents and patients with none of our patients opting to switch to Factor VIII prophylaxis. Half of the parents of PUPs were open to FVIII introduction when the child was older.