Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set

被引：27

作者：

van Balen, Erna C. ^{[1
]}

O'Mahony, Brian ^{[2
]}

Cnossen, Marjon H. ^{[3
]}

Dolan, Gerard ^{[4
]}

Blanchette, Victor S. ^{[5
]}

Fischer, Kathelijn ^{[6
]}

Gue, Deborah ^{[7
]}

O'Hara, Jamie ^{[8
]}

Iorio, Alfonso ^{[9
,10
]}

Jackson, Shannon ^{[7
]}

Konkle, Barbara A. ^{[11
,12
]}

Nugent, Diane J. ^{[13
,14
]}

Coffin, Donna ^{[15
]}

Skinner, Mark W. ^{[16
,17
]}

Smit, Cees ^{[1
]}

Srivastava, Alok ^{[18
]}

van Eenennaam, Fred ^{[19
,20
]}

van der Bom, Johanna G. ^{[1
,21
]}

Gouw, Samantha C. ^{[1
,22
]}

机构：

[1] Leiden Univ, Dept Clin Epidemiol, Med Ctr, Leiden, Netherlands

[2] Trinity Coll Dublin, Irish Haemophilia Soc, Dublin, Ireland

[3] Erasmus MC, Dept Pediat Hematol, Sophia Childrens Hosp, Rotterdam, Netherlands

[4] St Thomas Hosp, Ctr Haemostasis & Thrombosis, London, England

[5] Univ Toronto, Hosp Sick Children, Div Hematol Oncol, Dept Pediat, Toronto, ON, Canada

[6] Univ Med Ctr Utrecht, Dept Hematol, Va Creveldklin, Utrecht, Netherlands

[7] St Pauls Hosp, Adult Div, British Columbia Prov Bleeding Disorders Program, Vancouver, BC, Canada

[8] Univ Chester, Fac Hlth & Social Care, Chester, Cheshire, England

[9] McMaster Univ, Dept Med, McMaster Bayer Endowed Res Chair Clin Epidemiol C, Hamilton, ON, Canada

[10] McMaster Univ, McMaster Bayer Endowed Res Chair Clin Epidemiol C, Dept Hlth Res Methods Evidence & Impact, Hamilton, ON, Canada

[11] Bloodworks Northwest, Seattle, WA USA

[12] Univ Washington, Dept Med, Seattle, WA USA

[13] Univ Calif Irvine, Childrens Hosp, Irvine, CA USA

[14] Ctr Inherited Blood Disorders, Orange, CA USA

[15] World Federat Haemophilia, Montreal, PQ, Canada

[16] Natl Hemophilia Fdn, New York, NY USA

[17] Inst Policy Adv Ltd, Washington, DC USA

[18] Christian Med Coll & Hosp, Dept Haematol, Vellore, Tamil Nadu, India

[19] Decis Grp, Amsterdam, Netherlands

[20] Erasmus Univ, Erasmus Sch Accounting & Assurance, Rotterdam, Netherlands

[21] Sanquin LUMC, Ctr Clin Transfus Res, Leiden, Netherlands

[22] Univ Amsterdam, Amsterdam Univ Med Ctr, Emma Childrens Hosp, Dept Pediat Hematol, Amsterdam, Netherlands

来源：

RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS | 2021年 / 5卷 / 04期

关键词：

delivery of health care; health care; hemophilia A; hemophilia B; outcome assessment; patient-reported outcome measures; DEFINITIONS;

D O I：

10.1002/rth2.12488

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Background: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia. Methods: A systematic literature search was performed to identify possible health outcomes and risk adjustment variables. Persons with hemophilia and multidisciplinary health care providers were involved in an iterative nominal consensus process to select the most important health outcomes and risk adjustment variables for persons with hemophilia. Recommendations were made for outcome measurement instruments. Results: Persons with hemophilia were defined as all men and women with an X-linked inherited bleeding disorder caused by a deficiency of coagulation factor VIII or IX with plasma activity levels <40 IU/dL. We recommend collecting the following 10 health outcomes at least annually, if applicable: (i) cure, (ii) impact of disease on life expectancy, (iii) ability to engage in normal daily activities, (iv) severe bleeding episodes, (v) number of days lost from school or work, (vi) chronic pain, (vii) disease and treatment complications, (viii) sustainability of physical functioning, (ix) social functioning, and (x) mental health. Validated clinical as well as patient-reported outcome measurement instruments were endorsed. Demographic factors, baseline clinical factors, and treatment factors were identified as risk-adjustment variables. Conclusion: A consensus-based international set of health outcomes relevant to all persons with hemophilia, and corresponding measurement instruments, was identified for use in clinical care to facilitate harmonized longitudinal monitoring and comparison of outcomes.

引用

页数：13

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