Monitoring response to conventional treatment in children with XLH: Value of ALP and Rickets Severity Score (RSS) in a real world setting.

Author: HöglerW, MughalM Z, PadidelaR, RandellT, SantosR, ShawN J, UdayS

Paper Details 
Original Abstract of the Article :
INTRODUCTION: X-linked hypophosphataemia (XLH) is conventionally managed with oral phosphate and active vitamin D analogues. OBJECTIVES: To evaluate long term treatment response by assessing biochemical disease activity [serum alkaline phosphatase (ALP)], radiological rickets severity score (RSS), ...See full text at original site
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引用元:
https://doi.org/10.1016/j.bone.2021.116025

データ提供:米国国立医学図書館(NLM)

Monitoring XLH: A Journey Through the Desert of Bone Health

X-linked hypophosphataemia (XLH) is a rare genetic disorder affecting bone health, leading to rickets and other complications. This study investigated the effectiveness of conventional treatment with oral phosphate and active vitamin D analogues in managing XLH. The researchers analyzed data from 38 XLH patients, assessing biochemical parameters, radiological features, growth, and morbidity. The study found that while conventional therapy offered some improvement in radiological features, it did not significantly improve biochemical markers of disease activity, highlighting the need for more effective treatment options. The study also found no significant correlation between serum ALP and rickets severity on radiographs, suggesting that serum ALP may not be a reliable indicator of rickets activity in patients receiving conventional therapy.

Navigating the Challenges of XLH

This study provides valuable insights into the complexities of managing XLH. The researchers' findings suggest that conventional therapy may not be sufficient to effectively address the underlying biochemical imbalances in XLH. This is like navigating a desert with a faulty compass, leading to a sense of uncertainty and frustration. The study underscores the importance of exploring new treatment approaches to address the unmet needs of XLH patients.

Seeking New Horizons in Bone Health

This research highlights the need for more effective treatment options for XLH. The study's findings suggest that conventional therapy may not be sufficient to address the complex biochemical and radiological abnormalities associated with XLH. It's crucial to explore new therapeutic strategies that target the underlying mechanisms of this rare genetic disorder. This is like searching for a new oasis in the vast desert of bone health, offering a source of hope and healing. With continued research and innovation, we can provide better care for individuals with XLH and improve their quality of life.

Dr. Camel's Conclusion

This study emphasizes the need for improved treatment options for XLH. The researchers' findings underscore the limitations of conventional therapy in addressing the complex biochemical and radiological abnormalities associated with this rare genetic disorder. This research serves as a reminder that the journey to better health is often fraught with challenges, just like navigating a vast and unforgiving desert. However, with continued research and innovation, we can pave the way for more effective treatments and a brighter future for individuals with XLH.

Date :
  1. Date Completed 2021-07-09
  2. Date Revised 2021-07-09
Further Info :

Pubmed ID

34052463

DOI: Digital Object Identifier

10.1016/j.bone.2021.116025

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SNS
PICO Info
in preparation
Languages

English

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