BSPED2024 Oral Communications Endocrine Oral Communications 1 (9 abstracts)
Oral risedronate therapy in duchenne muscular dystrophy: the john walton muscular dystrophy centre experience
Elisabeth Wray 1 , Ruth Owen 2 , Matthew Chakraverty 3 , Sze Choong. Wong 4 , Nicola Crabtree 5 , Michela Guglieri 6 , Kate Owen 1 , Tim Cheetham 1 & Claire Wood 1
1Royal Victoria Infirmary, Newcastle upon Tyne, Newcastle, United Kingdom; 2Hull York Medical School, York, United Kingdom; 3Newcastle University, Newcastle, United Kingdom; 4Royal Hospital for Children, University of Glasgow, Glasgow, United Kingdom; 5Birmingham Women and Children Foundation Hospitals Trust and Children’s Hospital, Birmingham, United Kingdom; 6John Walton Muscular Dystrophy Research Centre, Newcastle, United Kingdom
Introduction: International standards of care for DMD recommend yearly spinal imaging for those on GC and the use of bisphosphonates (BP) for those with symptomatic or moderate asymptomatic vertebral fractures (VF). In view of the significant morbidity associated with VF and the potential for VF cascade, however, there may be justification for prophylactic BP in DMD. Although IV BP are used for treatment, there may be practical and cost advantages of using oral BP prophylactically, but there is limited evidence for this. From January 2008, oral BPs (risedronate) were offered to all boys starting or treated with daily GC attending the John Walton Muscular Dystrophy Centre in Newcastle upon Tyne, UK. This was done routinely until around 2015 but smaller numbers have also continued to be offered it since; this audit describes that cohort.
Methods: A retrospective notes and radiology review of the 92 patients who were started on oral risedronate between 2008 and 2022 was performed. Time to VF was calculated from start of GC to censor date of either date of x-ray when VF was first reported or last x-ray date without a VF.
Results: Spinal imaging was available for 76/92 patients. 47/76 sustained at least 1 VF during follow-up, of these 39/47 had started risedronate before first VF. 19 patients started risedronate within first year of GC exposure and had longitudinal XR FU with no VF at baseline. Of these, 6/19 had a subsequent VF (mean follow-up (FU) 9.7 years, range 6.1-11.9y), while 13/19 had no VF after 9.6 years FU (range 3.7-13.1y). 53/92 stopped risedronate during FU: (17 changed to zoledronate after VF, 5 to alendronate, 14 stopped BP due to SE including swallowing difficulties, 5 stopped due to improvement in bone density or stopping GCs, unknown in 12).
Conclusions: This audit suggests that oral risedronate is generally well tolerated and may be useful prophylactically as the time to first VF in the prophylactic cohort is significantly longer than reported in other DMD datasets. A well-designed prospective study is needed to further evaluate the utility of prophylactic BP in DMD.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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