Burosumab, a new injection drug for the treatment of X-linked hypophosphataemia (XLH), inherited rachitis, shows a better improvement in rahitis and other outcomes compared to conventional treatment in the international phase III clinical trial in children. The results that researchers named the first study to compare a new drug with conventional treatment for this rare disease will be presented on Sunday at the ENDO 2019, the annual meeting of Endocrine companies in New Orleans, La.
"These improvements with burosumab can alter the life of children with XLH when they grow," said chief researcher Erik Imel, MD, PhD. Med., Associate Professor of Medicine and Pediatrics, Indiana University Medical School, Indianapolis, Ind.
XLH affects approximately 3,000 children and 12,000 adults in the United States, according to the Food and Drug Administration (FDA). Typically, XLH is caused by rachitis, bent legs, bone pain, and low growth.
People with XLH have a high level of hormone growth factor of fibroblasts 23 or FGF23, which causes low concentrations of phosphorus in the blood (hypophosphataemia). The usual treatment has long been the multiple daily doses of oral phosphate and active vitamin D (e.g., Calcietriol), according to Imel.
The study included 61 children infected with XLH aged 1 to 12 years who had previously received conventional therapy with oral phosphate and active vitamin D, but still had evidence of X-ray rahitis. Patients were randomized to continue this conventional treatment or switch to receive injections of burosumab for two weeks. Radiologists who did not know that the participants got drugs, examined their X-rays and gave an assessment of the improvement of rickets.
Up to 40 weeks of treatment, this improvement was more than twice as high for the burosumab group as the conventional therapeutic group. Significant healing of rickets occurred in 72% of subjects receiving burosumab (21 out of 29), compared with only 6% in the conventional treatment group (two out of 32). After a 6-minute test, Burosumab resulted in a major improvement in deformation, height and distance of the legs, and a greater increase in serum phosphorus and active vitamin D levels.
"Now we know how much the benefit of a new drug, burosumab, compared to the previous approach with conventional therapy," said Imel. "This information is essential for doctors who make treatment decisions for their patients with XLH."
Burosumab can be used by children with X-linked hypophosphataemia
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Genetic rickets is better improved with burosumab than standard care, a study (2019, March 24) states.
imposed on 24 March 2019
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