Page 228 - Let them speak Towards a patient reported outcome measure set for

Chapter 8
References
1. Valderas JM, Alonso J. Patient reported outcome measures: a model-based classification
system for research and clinical practice. QualLife Res. 2008;17(9):1125–35.
2. Wilson IB, Cleary PD. Linking clinical variables with health-related quality of life. A conceptual
model of patient outcomes. JAMA. 1995;273.
3. Ustun TB, Chatterji S, Bickenbach J, Kostanjsek N, Schneider M. The international classification
of functioning, disability and health: A new tool for understanding disability and health.
Disability and rehabilitation. 2003;25.
4. Cella D, Yount S, Rothrock N, Gershon R, Cook K, Reeve B, et al. The Patient-Reported Outcomes
Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group
during its first two years. Medical Care. 2007;45(5 Suppl 1):S3–s11.
5. Oude Voshaar M, Terwee CB, Haverman L, van der Kolk B, Harkes M, van Woerden CS, et al.
Development of a standard set of PROs and generic PROMs for Dutch medical specialist care:
Recommendations from the Outcome-Based Healthcare Program Working Group Generic
PROMs. Quality of Life Research. 2023:1595–605.
6. van Silfhout NY, van Muilekom MM, van Karnebeek CD, Daams JG, Haverman L, van Eeghen AM.
Patient reported outcomes used in clinical trials and core outcome sets for individuals with genetic
intellectual disability: a scoping review. Journal of Neurodevelopmental Disorders. 2025;17(1):43.
7. van Silfhout NY, van Muilekom MM, van Karnebeek CD, Haverman L, van Eeghen AM. Patient
reported outcomes that matter to individuals with (genetic) intellectual disabilities: a qualitative
study. Journal of Neurodevelopmental Disorders. 2025;17(1):57.
8. Garayzábal E, Hidalgo I, de Souza ALDM, Da Silva NC, Giacheti CM, Pinato L. Sleep disturbances
and behavior in Smith-Magenis syndrome. Research in developmental disabilities.
2022;128:104286.
9. Boot E, Bassett AS, Marras C. 22q11. 2 deletion syndrome–associated Parkinson’s disease.
Movement disorders clinical practice. 2019;6(1):11–6.
10. van Silfhout NY, van Muilekom MM, Menke LA, van Karnebeek CD, Haverman L, van Eeghen
AM. What Matters Most? Developing a Core Patient Reported Outcome Set for Individuals With
Genetic Intellectual Disabilities: An International Delphi Study. J Intellect Disabil Res. 2026.
11. Kang S, Jones A, Shaffer RC, Erickson CA, Schmitt LM. Developing improved outcome
measures in FXS: Key stakeholder feedback. Research in developmental disabilities.
2023;137:104502.
12. Administration USFaD. Clinical Outcome Assessment (COA): Frequently Asked Questions
2020 [Available from: https://www.fda.gov/about-fda/clinical-outcome-assessment-coa-
frequently-asked-questions#COADefinition.
13. Cella D, Gershon R, Lai J-S, Choi S. The future of outcomes measurement: Item banking,
tailored short-forms, and computerized adaptive assessment. Quality of Life Research.
2007;16(SUPPL. 1):133–41.
14. Fries JF, Witter J, Rose M, Cella D, Khanna D, Morgan-DeWitt E. Item response theory,
computerized adaptive testing, and PROMIS: assessment of physical function. The Journal of
Rheumatology. 2014;41(1):153–8.
15. Choi SW, Reise SP, Pilkonis PA, Hays RD, Cella D. Efficiency of static and computer adaptive
short forms compared to full-length measures of depressive symptoms. Qual Life Res.
2010;19(1):125–36.
16. Greenberg KK, Schwartz AE, Kramer JM. Adoption of patient‐reported outcome measures
with youth with intellectual/developmental disabilities: Contextual influences and practice
patterns. Child: Care, Health and Development. 2021;47(4):501–8.
17. Maguire S, Davison J, McLaughlin M, Simms V, Bunting B. Exploring the psychometric properties
of self‐report instruments used to measure health‐related quality of life and subjective
wellbeing of adolescents with intellectual disabilities: A Consensus‐based Standards for
the Selection of Health Measurement Instruments (COSMIN) systematic review. Journal of
Applied Research in Intellectual Disabilities. 2023;36(5):899–915.
18. Dhaenens BA, Rietman A, Husson O, Oostenbrink R. Health-related quality of life of children
with neurofibromatosis type 1: analysis of proxy-rated PedsQL and CHQ questionnaires.
European Journal of Paediatric Neurology. 2023;45:36–46.
226