Optimizing bisphosphonate therapy for young people with brittle bones
Research type
Research Study
Full title
Improving treatment pathways for children and young people with osteogenesis imperfecta: Moving towards individualized bisphosphonate therapy
IRAS ID
292768
Contact name
Emma Webb webb
Contact email
Sponsor organisation
Norfolk and Norwich University Hospitals NHS Foundation Trust
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
Research Summary
Some children have medical conditions, including brittle bone disease, which leads to their bones breaking more easily. To help strengthen their bones they receive regular bisphosphonate infusions. We measure their bone strength using a DEXA scan to decide how often they need their infusions. In future we would also like to be able to measure how much bisphosphonate medicine is still present in their body before their next infusion to help us decide on the timing of the infusion.
The purpose of this study is to collect a blood sample from children aged 0-18 years at the same time as their planned bisphosphonate infusion. This will help us to assess whether we can measure the amount of bisphosphonate still present in children before their next infusion. In the future we hope that this information can be used to help us to improve our care for children with brittle bones.
Summary of Results
Children and young people with low bone density are given treatment with a medicine called a bisphosphonate to help improve their bone strength. To help decide how often we should give this medicine and what dose we should use we measure children's bone strength using a DEXA scan . In future we would also like to be able to measure how much bisphosphonate medicine is still present in their body before their next infusion to help us decide on the timing of the infusion. The purpose of this study was to collect a blood sample from children and young people aged 0-18 years at the same time as their planned bisphosphonate infusion to assess whether we could identify and measure the amount of bisphosphonate still present in young people before their next infusion. The results of this study showed that we could still measure bisphosphonates a long time after the medicine had been given and that in some patients the level of bisphosphonates present was still very high. The next steps following this study are to look in more detail at how individuals process this medicine so that we can individualise treatment plans avoiding over/under treatment.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
21/YH/0076
Date of REC Opinion
16 Apr 2021
REC opinion
Further Information Favourable Opinion