PB1297 - A UK Single Centre Experience of Switching Adolescents with Severe Haemophilia A to Turoctocog Alfa Pegol

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Co-Author(s)

Sarah Pool, PG Cert

Specialist Nurse Practitioner
Oxford University Hospitals NHS Foundation Trust
Oxfordshire, England, United Kingdom

Co-Author(s)

AW

Alice Wilkinson

Advanced Nurse Practitioner
Oxford University Hospitals NHS Foundation Trust
Oxfordshire, England, United Kingdom
ME

Marie Eales

Advanced Nurse Practitioner
Oxford University Hospitals NHS Foundation Trust
Oxford, England, United Kingdom
AY

Ashley Yburan

Research Nurse
Oxford University Hospitals NHS Foundation Trust
Oxford, England, United Kingdom
NB

Neha Bhatnagar

Consultant paediatric haematologist
Oxford University Hospitals NHS Foundation Trust
Oxford, England, United Kingdom
GH

Georgina Hall

Consultant paediatric haematologist
Oxford University Hospitals NHS Foundation Trust
Oxford, England, United Kingdom

Presenting Author(s)

Saket Badle, MBBS MRCPCH FRCPath MD (Res)

Consultant Paediatric Haematologist
Oxford University Hospitals NHS Foundation Trust
Oxford, England, United Kingdom

Background: Turoctocog alfa pegol (TAP) is an extended half-life (EHL) factor VIII (FVIII) product. We describe loss of efficacy with TAP during the switch from standard half-life (SHL) factor.

Aims: We describe our experience of switching 4 adolescents to this product.

Methods: Case study report

Results: Four patients switched to TAP and details are summarised in Table 1.

Patient 1 has no spontaneous bleeds and continues with their new prophylactic regimen.

Patient 2 had a traumatic left elbow soft tissue bleed after ED 3 (of TAP). A 40-hour FVIII level was < 0.01IU/mL and negative FVIII inhibitor. He treated with daily TAP before returning to EOD TAP prophylaxis which continues.

Patient 3 had a spontaneous left thigh bleed after ED 6 (of TAP). A 4.5-hour FVIII level was < 0.01IU/mL and negative FVIII inhibitor. He switched to daily treatment with OA for bleed treatment then re-started TAP prophylaxis with satisfactory FVIII levels. Subsequently, he had repeat spontaneous bleeds and was switched back to OA prophylaxis.

Patient 4 presented with a traumatic right finger bleed after ED 3 (of TAP). A 36-hour FVIII level was < 0.01IU/mL and negative FVIII inhibitor. He treated with daily TAP before returning to TW TAP prophylaxis which continues.

Conclusion(s): We find that TAP is generally well tolerated. Three patients had unexpected loss of efficacy. This was investigated with FVIII measurements, troughs, peaks, inhibitors and manufacturer anti-peg antibody. Loss of efficacy in the presence of the latter has not been reported, which was also the case in our patients.

EHLs are an attractive option but need pharmacokinetic measurements to demonstrate efficacy for prophylaxis as well as around bleed episodes, some patients may need to revert to SHL-FVIII if there is no demonstrable benefit. This should be highlighted and discussed when switching patients to EHLs to aid informed treatment decisions.