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The Thalassemia Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
General Updates | A potential novel treatment option for patients with thalassemia and bone disease
In this video, Julian Waung, BM, PhD, Whittington Hospital, London, UK, introduces romosozumab, a monoclonal antibody that inhibits sclerostin and increases bone density. Romosozumab is approved for use in postmenopausal women who have experienced a recent osteoporotic fracture. Although no published data exists in patients with thalassemia, Dr Waung notes that he has observed a substantial increase in bone mineral density in some of his patients treated with this agent. This interview took place virtually.
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Transcript
So romosozumab is a monoclonal antibody to sclerostin, which is produced by the osteocytes, which are bone cells. And it’s a negative… sclerostin is a negative inhibitor of bone formation. So what this antibody does is it both increases bone formation and reduces bone resorption at the same time, and it’s given as a subcutaneous injection once a month for 12 months...
So romosozumab is a monoclonal antibody to sclerostin, which is produced by the osteocytes, which are bone cells. And it’s a negative… sclerostin is a negative inhibitor of bone formation. So what this antibody does is it both increases bone formation and reduces bone resorption at the same time, and it’s given as a subcutaneous injection once a month for 12 months. It’s currently licensed for postmenopausal women who have had a fracture, an osteoporotic fracture, within the last 24 months. And it massively increases bone mass and reduces fracture risk at all sites; so vertebral fractures, major osteoporotic fractures, hip fractures. But until now, there’s no… well there’s still no published data at all about it. And in my patient cohort, there have been mixed results, but some [patients] with very substantial increase in bone mineral density up to about 15% in the spine, which is highly significant.
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