Methodological approach for determining the minimal important difference and minimal important change scores for the European Organisation for Research and Treatment of Cancer Head and Neck Cancer Module (EORTC QLQ-HN43) exemplified by the swallowing scale

Research output: Contribution to journalArticlepeer-review

Authors

  • EORTC Quality of Life Group
  • EORTC Head and Neck Cancer Group
  • Susanne Singer
  • Eva Hammerlid
  • Iwona M. Tomaszewska
  • Cecilie Delphin Amdal
  • Kristin Bjordal
  • Bente Brokstad Herlofson
  • Marcos Santos
  • Joaquim Castro Silva
  • Hisham Mehanna
  • Amy Fullerton
  • Christine Brannan
  • Loreto Fernandez Gonzalez
  • Johanna Inhestern
  • Monica Pinto
  • Juan I. Arraras
  • Noam Yarom
  • Pierluigi Bonomo
  • Ingo Baumann
  • Razvan Galalae
  • Ourania Nicolatou-Galitis
  • Naomi Kiyota
  • Judith Raber-Durlacher
  • Dina Salem
  • Alexander Fabian
  • Andreas Boehm
  • Sanja Krejovic-Trivic
  • Wei-Chu Chie
  • Christian Simon
  • Lisa Licitra
  • Allen C. Sherman

Abstract

Purpose
The aim of this study was to explore what methods should be used to determine the minimal important difference (MID) and minimal important change (MIC) in scores for the European Organisation for Research and Treatment of Cancer Head and Neck Cancer Module, the EORTC QLQ-HN43.

Methods
In an international multi-centre study, patients with head and neck cancer completed the EORTC QLQ-HN43 before the onset of treatment (t1), three months after baseline (t2), and six months after baseline (t3). The methods explored for determining the MID were: (1) group comparisons based on performance status; (2) 0.5 and 0.3 standard deviation and standard error of the mean. The methods examined for the MIC were patients' subjective change ratings and receiver-operating characteristics (ROC) curves, predictive modelling, standard deviation, and standard error of the mean. The EORTC QLQ-HN43 Swallowing scale was used to investigate these methods.

Results
From 28 hospitals in 18 countries, 503 patients participated. Correlations with the performance status were |r|< 0.4 in 17 out of 19 scales; hence, performance status was regarded as an unsuitable anchor. The ROC approach yielded an implausible MIC and was also discarded. The remaining approaches worked well and delivered MID values ranging from 10 to 14; the MIC for deterioration ranged from 8 to 16 and the MIC for improvement from − 3 to − 14.

Conclusions
For determining MIDs of the remaining scales of the EORTC QLQ-HN43, we will omit comparisons of groups based on the Karnofsky Performance Score. Other external anchors are needed instead. Distribution-based methods worked well and will be applied as a starting strategy for analyses. For the calculation of MICs, subjective change ratings, predictive modelling, and standard-deviation based approaches are suitable methods whereas ROC analyses seem to be inappropriate.

Background
Quality of Life (QoL) domains are usually reported in terms of scores. In order to assess the effects of a new drug or intervention, researchers must determine the minimal difference in these scores deemed clinically important. Only by knowing that, can they calculate the sample size for a trial and interpret which results are clinically meaningful.

Details

Original languageEnglish
JournalQuality of Life Research
Early online date16 Jul 2021
Publication statusE-pub ahead of print - 16 Jul 2021

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