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Minimal important difference in childhood interstitial lung diseases

Matthias Griese1, Nicolaus Schwerk2, Julia Carlens2

  • 1Munich University Hospital, Dr von Hauner Children's Hospital, German Center for Lung Research (DZL), Munchen, Germany matthias.griese@med.uni-muenchen.de.

Thorax
|December 26, 2022
PubMed

Insights

This study establishes minimal important differences (MIDs) for vital signs and health-related quality of life (HrQoL) in childhood interstitial lung diseases (chILD). These findings aid clinicians in monitoring chILD progression and treatment effectiveness.

Area of Science:

  • Pediatric Pulmonology
  • Clinical Research
  • Rare Diseases

Background:

  • Monitoring disease progression in childhood interstitial lung diseases (chILD) is crucial for effective patient management.
  • Minimal important difference (MID) data, representing the smallest clinically significant change, were previously unavailable for chILD vital signs and health-related quality of life (HrQoL).

Purpose of the Study:

  • To calculate and report MIDs for vital signs and HrQoL scores in a large cohort of children with chILD.
  • To provide clinicians with essential benchmarks for assessing treatment efficacy and disease progression in chILD patients.

Main Methods:

  • Utilized data from the Kids Lung Register, a web-based platform for rare pediatric lung disorders.
  • Calculated MIDs using both distribution-based (one-third SD) and anchor-based methods, with lung function parameters serving as anchors.
  • Collected data on vital signs (respiratory rate, oxygen saturation, Fan severity score) and various HrQoL questionnaires.

Main Results:

  • Established MIDs for respiratory rate (1.3 z-score), oxygen saturation (3.0%), and Fan severity score (0.2-0.4).
  • Determined MIDs for HrQoL scores, including the Health Status Questionnaire (0.4-0.8), chILD-specific questionnaire (4.4%-8.2%), and PedsQL V.4.0 subscores and total score.
  • Responsiveness analysis results generally aligned with the calculated MIDs.

Conclusions:

  • This study provides the first estimates of MIDs for vital signs and HrQoL in a substantial chILD cohort.
  • These MIDs serve as valuable tools for clinicians to interpret changes in patient status and make informed treatment decisions in chILD.
Abstract

Keywords:
paediatric interstitial lung diseaserare lung diseases

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