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Burosumab, a new injectable drug to treat X-linked hypophosphatemia (XLH), a hereditary form of rickets, presents a superior improvement in rickets and other outcomes compared to conventional treatment in the part of an international phase 3 clinical trial conducted in the child. The findings of what investigators have called the first comparative study comparing the new drug and conventional treatment for this rare disease will be presented Sunday at the annual ENDO 2019 conference, hosted by the Society of Endocrinology in New Orleans.
"These improvements with burosumab could potentially change the lives of children with XLH as they grow up," said lead researcher Erik Imel, MD, badociate professor of medicine and pediatrics at Indiana University School. of Medicine in Indianapolis, Ind.
XLH affects approximately 3,000 children and 12,000 adults in the United States, according to estimates by the Food and Drug Administration (FDA). As a rule, XLH causes rickets, arched legs, bone pain and a short stature.
People with XLH have high levels of the hormone, fibroblast growth factor 23, or FGF23, which causes low blood levels of phosphorus (hypophosphatemia). Conventional therapy has long been several daily doses of oral phosphate and active vitamin D (eg, calcitriol), according to Imel.
The study involved 61 children aged 1 to 12 years with XLH who had previously received standard oral phosphate and active vitamin D, but who still showed signs of X-rickets. were randomly badigned to continue this conventional treatment or to receive injections of burosumab every two weeks. Radiologists who were unaware of the drug badignments of the participants examined their X-rays and badigned a rickets improvement score.
After 40 weeks of treatment, this improvement was more than twice as high for the burosumab group compared to the conventional treatment group, said Imel. The researchers reported that 72% of participants receiving burosumab (21 out of 29) healed substantially, compared to only 6% in the conventional treatment group (two out of 32). Burosumab would also have resulted in greater improvement in leg deformities, height and distance traveled during a 6-minute test, as well as a greater increase in serum phosphorus and vitamin levels. D active.
"We now know the magnitude of the benefits of the new drug, burosumab, compared to the previous conventional treatment approach," Imel said. "This information is essential for physicians to make therapeutic decisions about their XLH patients."
Burosumab may be beneficial for children with X-linked hypophosphatemia
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Genetic rickets improve with burosumab more than conventional care, study reveals (March 24, 2019)
recovered on March 24, 2019
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