A study comparing the efficacy of burosumab with conventional treatment—oral phosphate and active vitamin D—found that burosumab, which blocks FGF23, was significantly more effective in alleviating symptoms.
A study comparing burosumab (Crysvita) with conventional treatment for a rare musculoskeletal disease found that burosumab was significantly more effective in alleviating symptoms of the disease.
X-linked hypophosphatemia (XLH) is a rare phosphate-wasting genetic disease that affects 1 in every 20,000 people and causes rickets and osteomalacia, or softening of the bone. The disease is characterized by elevated serum concentrations of fibroblast growth factor 23, rickets, bowed legs, and growth impairment. In April 2018, the FDA approved burosumab for the treatment XLH in adults and children aged 1 year or older.
“This is the first study comparing burosumab head-to-head with conventional therapy,” Erik Imel, MD, associate professor of medicine at Indiana University School of Medicine, said in a statement. “We now know the magnitude of benefit from burosumab over the prior approach with conventional therapy. This information is critical for doctors to make treatment decisions for patients with XLH.”
Between August 3, 2016, and May 8, 2017, the researchers recruited 61 children between the ages of 1 and 12 years at 16 centers around the globe, including in the United States, Canada, the United Kingdom, Sweden, Australia, Japan, and Korea. A total of 32 patients received conventional therapy—oral phosphate and active vitamin D—while 29 received burosumab 0.8 mg/kg every 2 weeks.
Observed for a total of 64 weeks, 72% of children who received burosumab achieved significant relief of rickets by 40 weeks of treatment, compared with just 6% of children receiving conventional therapy. The treatment also led to greater improvements in leg deformities, growth, distance walked in a 6-minute test, and serum phosphorus and active vitamin D levels.
Treatment-emergent adverse events considered possibly, probably, or definitely related to treatment were more frequently observed among patients receiving burosumab compared with conventional treatment (59% vs 22%). There were 3 serious adverse events that occurred in each group, all of which were unrelated to treatment.
The researchers noted that they plan on continuing to study the long-term effects of burosumab, including the effect on height outcomes as an adult and whether the treatment will decrease the need for surgeries to correct bowed legs.
Reference:
Imel E, Glorieux F, Whyte M, et al. Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial [published online May 16, 2019]. Lancet. doi: 10.1016/S0140-6736(19)30654-3.
NCCN Guidelines Update Adds Momelotinib Below Ruxolitinib for High-, Low-Risk Myelofibrosis
January 23rd 2024Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
Read More
Oncology Onward: A Conversation With Dr Shereef Elnahal, Under Secretary for Health
April 20th 2023Shereef Elnahal, MD, MBA, under secretary for health at the Veterans Health Administration (VHA), sat for a conversation with our hosts Emeline Aviki, MD, MBA, Memorial Sloan Kettering Cancer Center, and Stephen Schleicher, MD, MBA, Tennessee Oncology, that covered the cancer footprint of the VHA.
Listen
Interventions Needed to Increase DMT Uptake in Sickle Cell Disease
December 26th 2023A recent study found that uptake of disease-modifying therapies (DMTs) has been low among patients with sickle cell disease, suggesting that more interventions that consider individual patient characteristics are needed to improve adoption.
Read More
Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals
May 3rd 2022On this episode of Managed Care Cast, Ari D. Panzer, BS, lead author and researcher, then at Tufts Medical Center—now at Duke University—discusses the findings from his team’s investigation into coverage decisions by health plan insurers of the 66 drugs approved by the FDA in 2018.
Listen
Exagamglogene Autotemcel Meets End Points in Severe Sickle Cell Disease, β-Thalassemia
December 7th 2023Two posters set to be presented at the 65th American Society of Hematology Annual Meeting & Exposition met their primary and secondary end points regarding exagamglogene autotemcel therapy for sickle cell disease and β-thalassemia.
Read More