Publications by authors named "A A Portale"
Article Synopsis
- In patients with X-linked hypophosphatemia (XLH), traditional treatments can lead to nephrocalcinosis, but the connections between XLH, its treatment, and kidney function are still unclear.
- A study analyzed kidney health in 196 children and 318 adults with XLH, with findings showing a significant prevalence of nephrocalcinosis, especially in children, and its association with reduced kidney function.
- Results indicated that nephrocalcinosis was common in both age groups, with children showing more symptoms related to kidney function compared to adults, highlighting the need for deeper understanding and monitoring of kidney health in XLH patients.*
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.
Context: Burosumab was developed as a treatment option for patients with the rare, lifelong, chronically debilitating, genetic bone disease X-linked hypophosphatemia (XLH).
Objective: Collect additional information on the safety, immunogenicity, and clinical response to long-term administration of burosumab.
Methods: UX023-CL203 (NCT02312687) was a Phase 2b, open-label, single-arm, long-term extension study of adult subjects with XLH who participated in KRN23-INT-001 or KRN23-INT-002 studies.
Article Synopsis
- * A subgroup analysis of data from a phase 3 study involving 134 adults revealed no significant differences in treatment benefits among 14 clinically relevant subgroups, including variations in sex, pain levels, and physical function.
- * Overall, burosumab consistently showed better outcomes compared to placebo, suggesting it is beneficial for all symptomatic adults with active XLH, although some treatment effects were small and not statistically significant across subgroups. *