TY - JOUR
T1 - Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia
T2 - the applicability of the pediatric item banks
AU - Teela, Lorynn
AU - Luijten, Michiel A. J.
AU - Kuijlaars, Isolde A. R.
AU - van Gastel, Tessa C. M.
AU - van Hoorn, Evelien S.
AU - Gouw, Samantha C.
AU - Fijnvandraat, Karin C. J.
AU - Fischer, Kathelijn
AU - Cnossen, Marjon H.
AU - Andeweg, Sasja
AU - van der Velden – van ‘t Hoff, Carolien
AU - Liem, Corinne
AU - Jansen-Zijlstra, Margreet E.
AU - Peters, Marjolein
AU - Haverman, Lotte
N1 - Funding Information:
We would like to thank all participants in this study. In addition, we acknowledge professor, Dr Caroline Terwee for her help in interpreting the results. M.P. and L.H. conceived the study. L.T. I.A.R.K. E.Sv.H. S.C.G. K.C.J.F. K.F. and M.H.C contributed to study design. Data collection was led by L.T. and supported by M.H.C. S.A. Cvd.Vvt.H. C.L. M.E.J.Z. L.T. performed the statistical analyses and M.A.J.L. T.C.Mv.G. M.P. LH contributed to the interpretation of the data and modification of statistical analyses. The first draft of the paper was written by L.T. M.P.and L.H. were responsible for the supervision. All authors critically revised the manuscript for intellectual content and approved the final version of the manuscript. This study was funded with an unrestricted research grant from Pfizer. MAJL and LH are members of the Dutch-Flemish PROMIS group. SCG received an unrestricted research grant from Sobi. The other authors have no conflict of interest regarding this work/project. The study was approved by the Medical Ethic Research board from all participating centers. All procedures performed in this study were in accordance with the ethical standards of the international and/or national research committee (Medical Ethics Committee of the Amsterdam UMC – W19_349 # 21.111) and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. Informed consent was provided by all patients for the use of their data for this study.
Funding Information:
This study was funded with an unrestricted research grant from Pfizer .
Publisher Copyright:
© 2023 The Author(s)
PY - 2023/8/1
Y1 - 2023/8/1
N2 - 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.
AB - 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.
KW - feasibility studies
KW - hemophilia
KW - patient-reported outcome measures
KW - pediatrics
KW - psychometrics
UR - https://www.scopus.com/pages/publications/85171420847
U2 - 10.1016/j.rpth.2023.102159
DO - 10.1016/j.rpth.2023.102159
M3 - Article
C2 - 37753226
SN - 2475-0379
VL - 7
JO - Research and practice in thrombosis and haemostasis
JF - Research and practice in thrombosis and haemostasis
IS - 6
M1 - 102159
ER -