ICCBH2019 Poster Presentations (1) (226 abstracts)
Burosumab initiation in a UK X-linked hypophosphataemia cohort: real-world use resonates with research evidence
Poonam Dharmaraj 1 , Christine Burren 2 , Moira S Cheung 3 , Raja Padidela 4 , Zulf Mughal 4 , Nick Shaw 5 , Vrinda Saraff 5 , Ruchi Nadar 5 , Tabitha Randell 6 , Talat Mushtaq 7 , Renuka Ramakrishnan 1 , Senthil Sennipathan 1 , Sophia Sakka 3 , Louise Bath 8 , Daniela Elleri 8 , Justin H Davies 9 , John Barton 2 , Ian Tucker 2 , Lauren Rayner 4 , Paul Arundel 10 , Robyn Gilbey-Cross 3 , Alexander M Tothill 11 , James Philip 12 , Nadine Sawoky 12 , Paul Connor 12 & Leigh Mathieson 12
1Alder Hey Children’s Hospital, Liverpool, UK; 2Bristol Royal Hospital for Children, Bristol, UK; 3Evelina Children’s Hospital, London, UK; 4Royal Manchester Children’s Hospital, Manchester, UK; 5Birmingham Children’s Hospital, Birmingham, UK; 6Nottingham Children’s Hospital, Nottingham, UK; 7Leeds Teaching Hospital, Nottingham, UK; 8Royal Hospital for Sick Children, Edinburgh, UK; 9Southampton University Hospital, Southampton, UK; 10Sheffield Children’s Foundation Trust, Sheffield, UK; 11MAP BioPharma, Papworth Everard, Cambridge, UK; 12Kyowa Kirin, Galashiels, UK.
Objectives: X-linked hypophosphataemia (XLH) is a rare inherited form of osteomalacia characterised by low blood phosphate levels which lead to inadequate mineralisation of bone resulting in rickets, skeletal abnormalities, physical impairment, weakness, and pain. Burosumab is an anti-FGF23 fully human monoclonal-antibody, and the first treatment to target the underlying pathophysiology of XLH. Real-world evidence is important in validating the findings of clinical studies. We report relevant real-world biochemical data following the first 6 months of burosumab treatment.
Methods: An early access program (EAP) for burosumab was made available for children in the UK with XLH in 12 specialist centres. Inclusion criteria for the EAP included radiographic evidence of disease, XLH confirmed by genetic PHEX mutation or familial X-linked inheritance of mutation or family history. Patients must have also had an unsatisfactory response to conventional treatment. EAP enrolment was between January and March 2018. 135 of 142 applications were approved. 132 have commenced treatment (dose in accordance with EMA marketing authorisation), of whom 31 have completed a median of 24 weeks (22–26 weeks) of burosumab treatment.
Results: Mean age enrolled was 7.2 years (range 1.6–14.7), 68% female and 32% male. Mean dose administered by subcutaneous injection was 0.51 mg/kg (0.28–0.95 mg/kg) at week 0 and 0.89 mg/kg (0.25–2.01 mg/kg) at week 24 (22–26 weeks). Mean fasting serum phosphorus was 0.73 mmol/l (0.5–0.91 mmol/l) in week 0 rising to 1.06 mmol/l (0.77–1.48 mmol/l) at week 24 (22–26 weeks) representing a 45% increase in serum phosphate. Mean serum ALP fell from 591.5 IU/l (261–4089 IU/l) at week 0 to 353.2 IU/l (190–733 IU/l) at week 24 (22–26 weeks), representing 40% decrease in ALP. No patients discontinued treatment due to adverse events.
Conclusions: Early data from treating children and young people with XLH with burosumab in a real-world UK setting demonstrate that key biochemical responses are aligned with the clinical research program findings. Ongoing monitoring and research is required to confirm the biochemical response translates to the expected subsequent impact on skeletal and non-skeletal outcomes, including linear growth and deformities.
Disclosure: The authors declared no competing interests.
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