Validation of the Radiographic Global Impression of Change (RGI-C) score to assess healing of rickets in pediatric X-linked hypophosphatemia (XLH)

Ruth Lim, Randheer Shailam, Rebecca Hulett, Alison Skrinar, Annabel Nixon, Angela Williams, Mark Nixon, Tom D. Thacher

Research output: Contribution to journalArticlepeer-review

8 Scopus citations

Abstract

Background: Rickets is a primary manifestation of pediatric X-linked hypophosphatemia (XLH) – a rare progressive hereditary phosphate-wasting disease. Severity is quantified from radiographs using the Rickets Severity Scale (RSS). The Radiographic Global Impression of Change (RGI-C) is a complementary assessment in which a change score is assigned based on differences in the appearance of rickets on pairs of radiographs compared side by side. Objective: The current study evaluated the reliability, validity, and sensitivity to change of the RGI-C specifically in pediatric XLH. Methods: The reliability, validity, and sensitivity to change of the RGI-C were evaluated using data from two studies in pediatric XLH (113 children aged 1–12 years) in which burosumab treatment significantly improved rickets severity. Intra-rater and inter-rater reliability were assessed by three pediatric radiologists. Results: Intra-rater reliability for RGI-C global score was >90% for agreement within 1 point, with weighted kappa values >0.5, indicating moderate to almost perfect agreement. Inter-rater reliability was also >90% (0.47–0.52 for all reader pairs; moderate agreement). The RGI-C global score showed significant relationships with changes from baseline to week 64 in serum phosphorus (r = −0.397), alkaline phosphatase (−0.611), total RSS (−0.672), standing height (0.268), and patient-reported global functioning (0.306) and comfort/pain functioning (0.409). Based on standardized response means, RGI-C global scores were sensitive to change in RSS, differentiating between those considered improved and greatly improved. Results for validity and sensitivity to change were similar for the RGI-C wrist, knee, and standing long leg scores. Conclusion: The RGI-C is a reliable, valid, and sensitive measure in pediatric XLH, and complementary to the RSS.

Original languageEnglish
Article number115964
JournalBone
Volume148
DOIs
StatePublished - Jul 2021

Keywords

  • Clinical outcome assessment (COA)
  • Metabolic bone disease
  • Pediatric radiology
  • Reliability
  • Sensitivity to change
  • Treatment

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