Page 74 - Let them speak Towards a patient reported outcome measure set for
P. 74

Chapter 3
22. Müller AR, Luijten MA, Haverman L, de Ranitz-Greven WL, Janssens P, Rietman AB, et al.
Understanding the impact of tuberous sclerosis complex: development and validation of the
TSC-PROM. BMC medicine. 2023;21(1):1-15.
23. Beurskens AJ, de Vet HC, Kökeb AJ, Lindeman E, van der Heijden GJ, Regtope W, Knipschild
PG. A patient-specific approach for measuring functional status in low back pain. Journal of
manipulative and physiological therapeutics. 1999;22(3):144-8.
24. Churruca K, Pomare C, Ellis LA, Long JC, Henderson SB, Murphy LE, et al. Patient‐reported
outcome measures (PROMs): A review of generic and condition‐specific measures and a
discussion of trends and issues. Health Expectations. 2021.
25. 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.
26. Gibbons C, Porter I, Goncalves-Bradley DC, Stoilov S, Ricci-Cabello I, Tsangaris E, et al. Routine
provision of feedback from patient-reported outcome measurements to healthcare providers
and patients in clinical practice. Cochrane Database Syst Rev. 2021;10:CD011589.
27. Basch E, Deal AM, Kris MG, Scher HI, Hudis CA, Sabbatini P, et al. Symptom monitoring with
patient-reported outcomes during routine cancer treatment: a randomized controlled trial.
Journal of Clinical Oncology. 2016;34(6):557.
28. 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.
29. Benjamin K, Vernon MK, Patrick DL, Perfetto E, Nestler-Parr S, Burke L. Patient-reported
outcome and observer-reported outcome assessment in rare disease clinical trials: an ISPOR
COA emerging good practices task force report. Value in Health. 2017;20(7):838-55.
30. Slade A, Isa F, Kyte D, Pankhurst T, Kerecuk L, Ferguson J, et al. Patient reported outcome
measures in rare diseases: a narrative review. Orphanet Journal of Rare Diseases. 2018;13:1-
9.
31. Kölker S, Gleich F, Mütze U, Opladen T. Rare disease registries are key to evidence-based
personalized medicine: highlighting the European experience. Frontiers in Endocrinology.
2022;13:832063.
32. Basch E, Torda P, Adams K. Standards for patient-reported outcome–based performance
measures. Jama. 2013;310(2):139-40.
33. Rivera SC, Kyte DG, Aiyegbusi OL, Slade AL, McMullan C, Calvert MJ. The impact of patient-
reported outcome (PRO) data from clinical trials: a systematic review and critical analysis.
Health and quality of life outcomes. 2019;17:1-19.
34. Müller AR, van Silfhout NY, den Hollander B, Kampman DH, Bakkum L, Brands MM, et al.
Navigating the outcome maze: a scoping review of outcomes and instruments in clinical trials
in genetic neurodevelopmental disorders and intellectual disability. Therapeutic Advances in
Rare Disease. 2024;5:26330040241245721.
35. 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. 2023:1-11.
36. Kirkham JJ, Gorst S, Altman DG, Blazeby JM, Clarke M, Tunis S, et al. Core outcome Set-
STAndardised protocol items: the COS-STAP statement. Trials. 2019;20:1-7.
37. Wilson IB, Cleary PD. Linking clinical variables with health-related quality of life. A conceptual
model of patient outcomes. JAMA. 1995;273.
38. 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.
39. 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.
40. de Vet HCW, Terwee CB, Mokkink LB, Knol DL. Measurement in Medicine: A Practical Guide:
Cambridge University Press; 2011.
72











































   72   73   74   75   76