Measuring patient-reported outcomes in haemophilia clinical research

Dennis Globe; N. L. Young; S. Von Mackensen; M. Bullinger; J. Wasserman; Victor S. Blanchette; Louis M. Aledort; Rolf Ljung; Marijke van den Berg; Brian M. Feldman; Alessandro Gringeri; Marilyn Manco-Johnson; Pia Petrini; Georges E. Rivard; Wolfgang Schramm

doi:10.1111/j.1365-2516.2008.01961.x

Measuring patient-reported outcomes in haemophilia clinical research

Dennis Globe, N. L. Young, S. Von Mackensen, M. Bullinger, J. Wasserman, Victor S. Blanchette (Contributor), Louis M. Aledort (Contributor), Rolf Ljung (Contributor), Marijke van den Berg (Contributor), Brian M. Feldman (Contributor), Alessandro Gringeri (Contributor), Marilyn Manco-Johnson (Contributor), Pia Petrini (Contributor), Georges E. Rivard (Contributor), Wolfgang Schramm (Contributor)

Research output: Contribution to journal › Review article › peer-review

Abstract

Patient-reported outcome (PRO) measures have been used to assess quality of life and health state preferences from the patient's perspective. However, they have not been fully utilized in haemophilia clinical practice and research. A series of meetings were convened to review and document the state of the art in PROs relevant to haemophilia. Experts developed a process for selection of measures and identified published measures of health-related quality of life (HRQoL) relevant to patients with haemophilia. These were synthesized and reviewed. Patient preference measures were also identified and reviewed. Although the majority of measures were developed for and validated in adults, several measures were identified for use in paediatric populations. This paper recommends an approach to the selection of PROs for application in haemophilia clinical research and practice and identifies several potential measures relevant for application in haemophilia clinical research and practice.

Original language	English
Pages (from-to)	843-852
Number of pages	10
Journal	Haemophilia
Volume	15
Issue number	4
DOIs	https://doi.org/10.1111/j.1365-2516.2008.01961.x
Publication status	Published - 2009 Jul 10

Free keywords

Factor VIII
Haemophilia
Health status
Primary prophylaxis
Quality of life
Utility

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Globe, D., Young, N. L., Von Mackensen, S., Bullinger, M., Wasserman, J., Blanchette, V. S., Aledort, L. M., Ljung, R., van den Berg, M., Feldman, B. M., Gringeri, A., Manco-Johnson, M., Petrini, P., Rivard, G. E., & Schramm, W. (2009). Measuring patient-reported outcomes in haemophilia clinical research. Haemophilia, 15(4), 843-852. https://doi.org/10.1111/j.1365-2516.2008.01961.x

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title = "Measuring patient-reported outcomes in haemophilia clinical research",

abstract = "Patient-reported outcome (PRO) measures have been used to assess quality of life and health state preferences from the patient's perspective. However, they have not been fully utilized in haemophilia clinical practice and research. A series of meetings were convened to review and document the state of the art in PROs relevant to haemophilia. Experts developed a process for selection of measures and identified published measures of health-related quality of life (HRQoL) relevant to patients with haemophilia. These were synthesized and reviewed. Patient preference measures were also identified and reviewed. Although the majority of measures were developed for and validated in adults, several measures were identified for use in paediatric populations. This paper recommends an approach to the selection of PROs for application in haemophilia clinical research and practice and identifies several potential measures relevant for application in haemophilia clinical research and practice.",

keywords = "Factor VIII, Haemophilia, Health status, Primary prophylaxis, Quality of life, Utility",

author = "Dennis Globe and Young, {N. L.} and {Von Mackensen}, S. and M. Bullinger and J. Wasserman and Blanchette, {Victor S.} and Aledort, {Louis M.} and Rolf Ljung and {van den Berg}, Marijke and Feldman, {Brian M.} and Alessandro Gringeri and Marilyn Manco-Johnson and Pia Petrini and Rivard, {Georges E.} and Wolfgang Schramm",

year = "2009",

month = jul,

day = "10",

doi = "10.1111/j.1365-2516.2008.01961.x",

language = "English",

volume = "15",

pages = "843--852",

journal = "Haemophilia",

issn = "1351-8216",

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number = "4",

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Globe, D, Young, NL, Von Mackensen, S, Bullinger, M, Wasserman, J, Blanchette, VS, Aledort, LM, Ljung, R, van den Berg, M, Feldman, BM, Gringeri, A, Manco-Johnson, M, Petrini, P, Rivard, GE & Schramm, W 2009, 'Measuring patient-reported outcomes in haemophilia clinical research', Haemophilia, vol. 15, no. 4, pp. 843-852. https://doi.org/10.1111/j.1365-2516.2008.01961.x

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AU - Globe, Dennis

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A2 - Ljung, Rolf

A2 - van den Berg, Marijke

A2 - Feldman, Brian M.

A2 - Gringeri, Alessandro

A2 - Manco-Johnson, Marilyn

A2 - Petrini, Pia

A2 - Rivard, Georges E.

A2 - Schramm, Wolfgang

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AB - Patient-reported outcome (PRO) measures have been used to assess quality of life and health state preferences from the patient's perspective. However, they have not been fully utilized in haemophilia clinical practice and research. A series of meetings were convened to review and document the state of the art in PROs relevant to haemophilia. Experts developed a process for selection of measures and identified published measures of health-related quality of life (HRQoL) relevant to patients with haemophilia. These were synthesized and reviewed. Patient preference measures were also identified and reviewed. Although the majority of measures were developed for and validated in adults, several measures were identified for use in paediatric populations. This paper recommends an approach to the selection of PROs for application in haemophilia clinical research and practice and identifies several potential measures relevant for application in haemophilia clinical research and practice.

KW - Factor VIII

KW - Haemophilia

KW - Health status

KW - Primary prophylaxis

KW - Quality of life

KW - Utility

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DO - 10.1111/j.1365-2516.2008.01961.x

M3 - Review article

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JO - Haemophilia

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Measuring patient-reported outcomes in haemophilia clinical research

Abstract

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