Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia: the applicability of the pediatric item banks

Lorynn Teela; Michiel A J Luijten; Isolde A R Kuijlaars; Tessa C M van Gastel; Evelien S van Hoorn; Samantha C Gouw; Karin C J Fijnvandraat; Kathelijn Fischer; Marjon H Cnossen; Sasja Andeweg; Carolien van der Velden-van 't Hoff; Corinne Liem; Margreet E Jansen-Zijlstra; Marjolein Peters; Lotte Haverman

doi:10.1016/j.rpth.2023.102159

Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia: the applicability of the pediatric item banks

Res Pract Thromb Haemost. 2023 Aug 2;7(6):102159. doi: 10.1016/j.rpth.2023.102159. eCollection 2023 Aug.

Authors

Lorynn Teela^{1

2

3}, Michiel A J Luijten^{1

3

4

5}, Isolde A R Kuijlaars⁶, Tessa C M van Gastel^{1

3

7}, Evelien S van Hoorn⁸, Samantha C Gouw⁹, Karin C J Fijnvandraat⁹, Kathelijn Fischer⁶, Marjon H Cnossen¹⁰, Sasja Andeweg¹⁰, Carolien van der Velden-van 't Hoff¹⁰, Corinne Liem¹¹, Margreet E Jansen-Zijlstra¹², Marjolein Peters⁹, Lotte Haverman^{1

2

3}

Affiliations

¹ Amsterdam UMC location University of Amsterdam, Emma Children's Hospital, Child and Adolescent Psychiatry & Psychosocial Care, Amsterdam, The Netherlands.
² Amsterdam Public Health, Mental health and Digital health, Amsterdam, The Netherlands.
³ Amsterdam Reproduction and Development, Child development, Amsterdam, The Netherlands.
⁴ Amsterdam Public Health, Mental health and Methodology, Amsterdam, The Netherlands.
⁵ Epidemiology and Data Science, Amsterdam UMC location Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.
⁶ Centre for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands.
⁷ Amsterdam Public Health, Mental health and Health Behaviours & Chronic Diseases, Amsterdam, The Netherlands.
⁸ Department of Public Health, Erasmus MC, Erasmus University Medical Centre Rotterdam, The Netherlands.
⁹ Amsterdam UMC location University of Amsterdam, Emma Children's Hospital, Paediatric Haematology, Amsterdam, The Netherlands.
¹⁰ Department of Paediatric Haematology, Erasmus MC Sophia Children's Hospital, Erasmus University Medical Centre Rotterdam, The Netherlands.
¹¹ Department of Pediatric Haemato-Oncology, Radboud University Medical Centre, Nijmegen, The Netherlands.
¹² Department of Pediatric Haematology, University Medical Centre Groningen, Groningen, The Netherlands.

Abstract

Background: The use of patient-reported outcomes measures (PROMs) is important in hemophilia care, as it facilitates communication between patients and clinicians and promotes patient-centered care. Currently, a variety of PROMs with insufficient psychometric properties are used. Patient-reported outcomes measurement information system (PROMIS) measures, including Computer Adaptive Tests, were designed to measure generically and more efficiently and, therefore, are an alternative for the existing PROMs.

Objectives: To assess the feasibility, measurement properties, and outcomes of 8 PROMIS pediatric measures for boys with hemophilia.

Methods: In this multicenter study, boys with hemophilia completed 8 PROMIS measures and 2 legacy instruments. Feasibility was determined by the number of completed items and floor or ceiling effects (percentage of participants that achieved the lowest or highest possible score). Reliability was assessed as the percentage of scores with a SE ≤ 4.5. Construct validity was evaluated by comparing the PROMIS measures with the legacy instruments. Mean PROMIS T-scores were calculated and compared with the Dutch general population.

Results: In total, 77 boys with hemophilia participated. Reliability was good for almost all PROMIS measures and legacy instruments. The total number of completed items varied from 49 to 90 for the PROMIS pediatric measures, while the legacy instruments contained 117 to 130 items. Floor and ceiling effects were observed in both the PROMIS measures (0-39.5%) and legacy instruments (0-66.7%), but were higher for the legacy instruments.

Conclusions: The PROMIS pediatric measures are feasible to use for boys with hemophilia. With the use of the PROMIS measures in clinical care and research, a step toward worldwide standardization of PROM administration can be taken.

Keywords: feasibility studies; hemophilia; patient-reported outcome measures; pediatrics; psychometrics.