Loading...
Differences in initiation of enzyme replacement therapy across the United Kingdom
Ochoa-Ferraro, Antonio F. Taylor, Zoe G. Adrees, Faiza Bell, Daniel Jenkinson, Simon Akram, Siema Fondo, Ana Amado Whiting, Sam Wyllie, Thomas
Ochoa-Ferraro, Antonio F.
Taylor, Zoe G.
Adrees, Faiza
Bell, Daniel
Jenkinson, Simon
Akram, Siema
Fondo, Ana Amado
Whiting, Sam
Wyllie, Thomas
Abstract
Introduction: Lysosomal Disorders (LSDs) are a group of complex and multi-systemic inherited metabolic conditions. LSDs are characterized by an abnormal build-up of toxic materials in the bodies' cells due to a single enzyme deficiency. Some LSDs have disease specific therapies, including: enzyme replacement therapies (ERT), oral chaperone therapy and substrate reduction therapy available to patients on the National Health Service (NHS) through twelve specialized LD adult and pediatric centres that ensure patients management by an expert multi-disciplinary team. In NHS England, currently twelve ERT are funded and available to patients, access to these therapies may vary across other parts of the UK. Method This survey done with MS Forms among specialist pharmacists explores ERT initiation at LSD UK centres before transferring to homecare using the LD Homecare Framework. Results: The number of infusions received at the LD centres varies from one or 2 to more than 8 across the centres. Five of the 12 centres routinely use pre-medication when starting ERT on a treatment-naive patient, all of which are pediatric. ERT infusions are prepared exclusively at the ward/infusion suite in 9/12 centres, 1 centre prepares all its ERT infusions in the pharmacy aseptic unit and 2 centres use a combination of ward and pharmacy. Six centres are currently providing ERT under compassionate access schemes to patients, including olipudase alfa and intrathecal idursulfase. Conclusions: Initiation of ERT for patients in the UK varies across the LD centres in details such as the number of hospital-supervised infusions prior to homecare transfer, the use of pre-medication in treatment naïve patients, and where infusions are prepared for administration in hospital. These local differences in the treatments offered to patients across the UK. Pediatric LD centres routinely offer premedication and a higher number of hospital infusions than adult LD centres.
MIDER Authors
Affiliations
University Hospitals Birmingham NHS Foundation Trust; All Wales Inherited Metabolic service; Salford Royal NCA; Cambridge University Hospitals NHS Foundation Trust; Royal Free London NHS Foundation Trust; Birmingham Women's and Children's NHS Foundation Trust; University College London Hospitals NHS Foundation Trust; University Hospitals Bristol & Weston NHS Foundation Trust; University Hospital of Wales
Date
2025-02-07
Type
Conference Output
Collections
Citation
Ochoa-Ferraro, A. F., Taylor, Z. G., Adrees, F., Bell, D., Jenkinson, S., Akram, S., Fondo, A. A., Whiting, S. and Wyllie, T. (2025) 'Differences in initiation of enzyme replacement therapy across the United Kingdom.' Molecular Genetics and Metabolism, 144(2), pp. 108877. Available at: https://doi.org/10.1016/j.ymgme.2024.108877