Publications by authors named "F H Glorieux"
Article Synopsis
- * The phase 2b Asteroid study tested the efficacy and safety of the drug setrusumab in adults with OI, providing different doses of the drug or a placebo over 12 months.
- * Results showed significant improvements in bone strength measures like failure load and stiffness for certain doses, and although there were some serious adverse reactions, the drug shows promise for further research in phase 3 trials.
Article Synopsis
- Osteogenesis imperfecta (OI) is a genetic bone disorder that causes low bone density and frequent fractures, primarily treated with bisphosphonates in children, while adult treatment options are limited.
- The ASTEROID trial tested a new treatment, setrusumab, which showed improvements in bone density and strength for adults with OI types I, III, and IV.
- Analysis of bone biopsies revealed that while treatment with setrusumab did not significantly change the bone matrix properties, it did increase bone mass, suggesting that the fragility associated with OI may be due to other factors beyond just mineral content and matrix structure.
In a randomized, open-label phase 3 study of 61 children aged 1-12 years old with X-linked hypophosphatemia (XLH) previously treated with conventional therapy, changing to burosumab every 2 weeks (Q2W) for 64 weeks improved the phosphate metabolism, radiographic rickets, and growth compared with conventional therapy. In this open-label extension period (weeks 64-88), 21 children continued burosumab Q2W at the previous dose or crossed over from conventional therapy to burosumab starting at 0.8 mg/kg Q2W with continued clinical radiographic assessments through week 88.
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- * The analysis compared the effects of switching to burosumab versus maintaining higher or lower doses of conventional therapy on skeletal responses, focusing on the improvement in radiographic assessments of rickets at 64 weeks.
- * Results showed that children receiving burosumab had significantly higher improvements in their rickets and lower levels of serum alkaline phosphatase than those continuing conventional therapy, regardless of their previous phosphate or vitamin D dosages.
Background: Osteogenesis imperfecta (OI) is associated with short stature, which is mild, severe and moderate in OI types I, III and IV, respectively. Standardized OI type- and sex-specific growth charts across all pediatric ages do not exist.
Methods: We assessed 573 individuals with OI (type I, III or IV), each with at least one height measurement between ages 3 months and 20 years (total 6523 observations).