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The clinical trial improves the treatment of genetic rickets



Clinical testing at the IU School of Medicine improves the treatment of genetic rickets

Erik Imel, MD. Credit: Medical Faculty of the University of Indiana

A new study shows that a drug developed jointly with researchers at the Indiana Medical University to alleviate the symptoms of a rare musculoskeletal condition is significantly more effective than conventional therapies. The findings are published in lancet,

X-linked hypophosphatemia, or XLH, is a disease causing phosphate loss that causes rickets and osteomalacia or softening of the bones and can cause short stature, bent legs, dental abscesses and bone pain. This rare genetic disease affects about 1 in every 20,000 people.

Researchers recruited 61 children aged between 1 and 12 in 16 centers worldwide, including the United States, Canada, the United Kingdom, Sweden, Australia, Japan and Korea. Children were randomized to receive either Burosumab, a two-week injection approved by the Food and Drug Administration in April 2018, or conventional therapies for taking oral phosphate and active vitamin D several times a day. The main result is an improvement in X-ray rhythm, as noted by radiologists who did not know which treatment group the participant was involved in.

Children were observed for 64 weeks, and after 40 weeks of treatment, researchers found that 72% of children receiving Burosumab achieved significant rheumatic healing, whereas only 6% of those in the conventional treatment group noticed significant treatment. Burosumab also results in greater improvements in leg deformities, growth, distance is performed in a 6-minute test and levels of serum phosphorus and active vitamin D.

"This is the first study to compare Burosumab with conventional therapy," said lead researcher Eric Mimel, MD, associate professor of medicine at the IU School of Medicine. "Now we know the scale of the benefits of Burosumab compared to the previous approach to conventional therapy." This information is crucial for physicians to make solutions for treating XLH patients.

Researchers plan to continue studying the long-term effects of Burosumab, including the effect of adult healing on children, and whether this treatment will reduce the need for leg cramps.

Burosumab blocks a protein called fibroblast growth factor 23, which was originally discovered by researchers at Indiana Medical College. Burosumab is marketed by Ultragenyx Pharmaceutical, Inc. in cooperation with Kyowa Hakko Kirin Co., Ltd. and its European subsidiary, Kyowa Kirin International PLC, under the trade name Crysvita.


Genetic rhinitis improves more with burosumab than with standard care, according to the study


More information:
Erik A Imel et al, Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomized, active-controlled, open-label, Phase 3 study, The Lancet (2019). DOI: 10.1016 / S0140-6736 (19) 30654-3

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Indiana University

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Clinical trial improves the treatment of genetic rickets (2019, 17 May)
drawn up on 17 May 2019
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