Publikationsdetails

Zitat:	S. Uday, N J Shaw, M Z Mughal, T Randell, W Högler, R Santos, R Padidela, "Monitoring response to conventional treatment in children with XLH: Value of ALP and Rickets Severity Score (RSS) in a real world setting" , in Bone, 10-2021, ISSN: 1873-2763
Original Titel:	Monitoring response to conventional treatment in children with XLH: Value of ALP and Rickets Severity Score (RSS) in a real world setting
Sprache des Titels:	Englisch
Original Kurzfassung:	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), growth and morbidity in patients with XLH on conventional therapy and assess the correlation between serum ALP and RSS. Methods: XLH patients from 3 UK tertiary centres with ?3 radiographs one year apart were included. Data was collected retrospectively. The RSS was assessed from routine hand and knee radiographs and ALP z scores were calculated using age-specific reference data. Results: Thirty-eight (male = 12) patients met the inclusion criteria. The mean ± SD knee, wrist and total RSS at baseline (median age 1.2 years) were 2.0 ± 1.2, 1.9 ± 1.2 and 3.6 ± 1.3 respectively; and at the most recent clinic visit (median age 9.0 years, range 3.3-18.9) were 1.6 ± 1.0, 1.0 ± 1.0 and 2.5 ± 1.5 respectively. The mean ± SD serum ALP z scores at baseline and the most recent visit were 4.2 ± 2.3 and 4.0 ± 3.3. Median height SDS at baseline and most recent visit were -1.2 and -2.1 (p = 0.05). Dental abscess, craniosynostosis, limb deformity requiring orthopaedic intervention and nephrocalcinosis were present in 31.5%, 7.9%, 31.6% and 42.1% of the cohort respectively. There was no statistically significant (p > 0.05) correlation between ALP z scores and knee (r = 0.07) or total (r = 0.12) RSS. Conclusions: Conventional therapy was not effective in significantly improving biochemical and radiological features of disease. The lack of association between serum ALP and rickets severity on radiographs limits the value of ALP as the sole indicator of rickets activity in patients receiving conventional therapy. Keywords: Hypomineralisation; Hypophosphataemia; Osteomalacia; Radiograph; Rickets. Copyright © 2021. Published by Elsevier Inc.
Sprache der Kurzfassung:	Englisch
Journal:	Bone
Erscheinungsmonat:	10
Erscheinungsjahr:	2021
ISSN:	1873-2763
Anzahl der Seiten:	1
DOI:	10.1016/j.bone.2021.116025
URL zu weiteren Infos:	https://pubmed.ncbi.nlm.nih.gov/34052463/
Reichweite:	international
Publikationstyp:	Aufsatz / Paper in SCI-Expanded-Zeitschrift
Autoren:	S. Uday, N J Shaw, M Z Mughal, T Randell, W Högler, R Santos, R Padidela
Forschungseinheiten:	Universitätsklinik für Kinder- und Jugendheilkunde

Wissenschaftsgebiete:	Kinder- und Jugendheilkunde (ÖSTAT:302035)

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