11. FEJEZET | HIVATKOZÁSOK
- Fischer K, Poonnoose P, Dunn AL, et al. Choosing outcome assessment tools in haemophilia care and research: a multidisciplinary perspective. Haemophilia. 2017;23(1):11-24.
- Wille N, Badia X, Bonsel G, et al. Development of the EQ- 5D-Y: a child-friendly version of the EQ-5D. Qual Life Res. 2010;19(6):875-886.
- Ravens-Sieberer U, Wille N, Badia X, et al. Feasibility, reliability, and validity of the EQ-5D-Y: results from a multinational study. Qual Life Res. 2010;19(6):887-897.
- Limperg PF, Terwee CB, Young NL, et al. Health-related quality of life questionnaires in individuals with haemophilia: a systematic review of their measurement properties. Haemophilia. 2017;23(4):497-510.
- Rentz A, Flood E, Altisent C, et al. Cross-cultural development and psychometric evaluation of a patient-reported health-related quality of life questionnaire for adults with haemophilia. H aemophilia. 2008;14(5):1023-1034.
- Skinner MW, Chai-Adisaksopha C, Curtis R, et al. The Patient Reported Outcomes, Burdens and Experiences (PROBE) project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia. Pilot Feasibility Stud. 2018;4:58.
- Patient Outcomes Research Group. Patient Reported Outcomes Burdens and Experiences (PROBE) study. PROBE website. https://probestudy.org/. Accessed November 6, 2019.
- World Federation of Hemophilia. World Bleeding Disorders Registry. World Federation of Hemophilia website. https://www.wfh.org/en/ our-work-research-data/world-bleeding-disorders-registry. Accessed January 15, 2020.
- Coffin D, Herr C, O’Hara J, et al. World bleeding disorders registry: the pilot study. Haemophilia. 2018;24(3):e113-e116.
- World Federation of Hemophilia. Compendium of Assessment Tools. World Federation of Hemophilia website. https://elearning. wfh.org/ resource/compendium-of-assessment-tools/. Accessed January 16, 2020.
- Blanchette VS, Key NS, Ljung LR, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014;12(11):1935-1939.
- Witkop M, Lambing A, Divine G, Kachalsky E, Rushlow D, Dinnen J. A national study of pain in the bleeding disorders community: a description of haemophilia pain. Haemophilia. 2012;18(3):e115-e119.
- Manco-Johnson MJ, Nuss R, Funk S, Murphy J. Joint evaluation instruments for children and adults with haemophilia. Haemophilia. 2000;6(6):649-657.
- Rambod M, Forsyth K, Sharif F, Khair K. Assessment and management of pain in children and adolescents with bleeding disorders: a cross-sectional study from three haemophilia centres. Haemophilia. 2016;22(1):65-71.
- van Genderen FR, Westers P, Heijnen L, et al. Measuring patients’ perceptions on their functional abilities: validation of the Haemophilia Activities List. Haemophilia. 2006;12(1):36-46.
- Kempton CL, Recht M, Neff A, et al. Impact of pain and functional impairment in US adults with haemophilia: patient-reported outcomes and musculoskeletal evaluation in the pain, functional impairment and quality of life (P-FiQ) study. Haemophilia. 2018;24(2):261-270.
- Witkop M, Neff A, Buckner TW, et al. Self-reported prevalence, description and management of pain in adults with haemophilia: methods, demographics and results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study. Haemophilia. 2017;23(4):556-565.
- Witkop M, Lambing A, Kachalsky E, Divine G, Rushlow D, Dinnen J. Assessment of acute and persistent pain management in patients with haemophilia. Haemophilia. 2011;17(4):612-619.
- Remor E, Arranz P, Quintana M, et al. Psychometric field study of the new haemophilia quality of life questionnaire for adults: the ‘Hemofilia-QoL’. Haemophilia. 2005;11(6):603-610.
- Gilbert MS. Prophylaxis: musculoskeletal evaluation. Semin Hematol. 1993;30(3 Suppl 2):3-6.
- Hilliard P, Funk S, Zourikian N, et al. Hemophilia joint health score reliability study. Haemophilia. 2006;12(5):518-525.
- Poonnoose PM, Srivastava A. Outcome assessment in hemophilia. In: Lee CA, Berntorp EE, Hoots WK, eds. Textbook of Hemophilia. 3rd ed. Hoboken, NJ: Blackwell Publishing Ltd; 2019:253-261.
- World Health Organization. International Classification of Functioning, Disability and Health (ICF). World Health Organization website. https://www.who.int/classifications/icf/en/. Accessed November 5, 2019.
- World Health Organization. Towards a Common Language for Functioning, Disability and Health: ICF. Geneva, Switzerland: World Health Organization, 2002. https://www.who.int/classifications/icf/icfbeginnersguide.pdf. Accessed January 15, 2020.
- Centers for Disease Control and Prevention. Health-Related Quality of Life (HRQOL). Centers for Disease Control and Prevention website. https://www.cdc.gov/hrqol/index.htm. Accessed November 18, 2019.
- Carcao M, Zunino L, Young NL, et al. Measuring the impact of changing from standard half-l ife (SHL) to extended half-life (EHL) FVIII prophylaxis on health-related quality of life (HRQoL) in boys with moderate/severe haemophilia A: lessons learned with the CHO-KLAT tool. Haemophilia. 2020;26(1):73-78.
- Feldman BM, Funk SM, Bergstrom BM, et al. Validation of a new pediatric joint scoring system from the International Hemophilia Prophylaxis Study Group: validity of the hemophilia joint health score. Arthritis Care Res (Hoboken). 2011;63(2):223-230.
- Gouw SC, Timmer MA, Srivastava A, et al. Measurement of joint health in persons with haemophilia: a systematic review of the measurement properties of haemophilia-specific instruments. Haemophilia. 2019;25(1):e1-e10.
- Pettersson H, Ahlberg A, Nilsson IM. A radiologic classification of hemophilic arthropathy. Clin Orthop Relat Res. 1980;149:153-159.
- Doria AS. State-of-the-art imaging techniques for the evaluation of haemophilic arthropathy: present and future. H aemophilia. 2010;16(Suppl 5):107-114.
- Chan MW, Leckie A, Xavier F, et al. A systematic review of MR imaging as a tool for evaluating haemophilic arthropathy in children. Haemophilia. 2013;19(6):e324-e334.
- Martinoli C, Della Casa Alberighi O, Di Minno G, et al. Development and definition of a simplified scanning procedure and scoring method for Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US). Thromb Haemost. 2013;109(6):1170-1179.
- Keshava SN, Gibikote SV, Mohanta A, et al. Ultrasound and magnetic resonance imaging of healthy paediatric ankles and knees: a baseline for comparison with haemophilic joints. Haemophilia. 2015;21(3):e210-e222.
- Kandagaddala M, Sundaramoorthy M, Keshava SN, et al. A new and simplified comprehensive ultrasound protocol of haemophilic joints: the Universal Simplified Ultrasound (US-US) protocol. Clin Radiol. 2019;74(11):;897 e899-897 e816.
- Volland LM, Zhou JY, Barnes RFW, et al. Development and reliability of the joint tissue activity and damage examination for quantitation of structural abnormalities by musculoskeletal ultrasound in hemophilic joints. J Ultrasound Med. 2019;38(6):1569-1581.
- Nguyen S, Lu X, Ma Y, Du J, Chang EY, von Drygalski A. Musculoskeletal ultrasound for intra-articular bleed detection: a highly sensitive imaging modality compared with conventional magnetic resonance imaging. J Thromb Haemost. 2018;16(3):490-499.
- Foppen W, van der Schaaf IC, Beek FJA, Mali W, Fischer K. Diagnostic accuracy of point-of-care ultrasound for evaluation of early blood- induced joint changes: comparison with MRI. Haemophilia. 2018;24(6):971-979.
- De la Corte-Rodriguez H, Rodriguez-Merchan EC, Alvarez-Roman MT, Martin-Salces M, Martinoli C, Jimenez-Yuste V. The value of HEAD- US system in detecting subclinical abnormalities in joints of patients with hemophilia. Expert Rev Hematol. 2018;11(3):253-261.
- Di Minno A, Spadarella G, Nardone A, et al. Attempting to remedy sub-optimal medication adherence in haemophilia: the rationale for repeated ultrasound visualisations of the patient’ s joint status. Blood Rev. 2019;33:106-116.
- Ceponis A, Wong-Sefidan I, Glass CS, von Drygalski A. Rapid musculoskeletal ultrasound for painful episodes in adult haemophilia patients. Haemophilia. 2013;19(5):790-798.
- Kidder W, Nguyen S, Larios J, Bergstrom J, Ceponis A, von Drygalski A. Point-of-care musculoskeletal ultrasound is critical for the diagnosis of hemarthroses, inflammation and soft tissue abnormalities in adult patients with painful haemophilic arthropathy. Haemophilia. 2015;21(4):530-537.
- International Prophylaxis Study Group. Hemophilia Joint Health Score (HJHS). World Federation of Hemophilia website. https://www1. wfh.org/docs/en/Publications/Assessment_Tools/HJHS_ Summary_Score.pdf. Accessed January 15, 2020.
- Lundin B, Manco-Johnson ML, Ignas DM, et al. An MRI scale for assessment of haemophilic arthropathy from the International Prophylaxis Study Group. Haemophilia. 2012;18(6):962-970.
- van Genderen FR, van Meeteren NL, van der Bom JG, et al. Functional consequences of haemophilia in adults: the development of the Haemophilia Activities List. Haemophilia. 2004;10(5):565-571.
- Timmer MA, Gouw SC, Feldman BM, et al. Measuring activities and participation in persons with haemophilia: a systematic review of commonly used instruments. Haemophilia. 2018;24(2):e33-e49.
- McLaughlin P, Morris R, Chowdary P. I nvestigating the relationship between the HJHS and HAL in routine clinical practice: a retrospective review. Haemophilia. 2018;24(6):988-994.
- Groen WG, van der Net J, Helders PJ, Fischer K. Development and preliminary testing of a Paediatric Version of the Haemophilia Activities List (pedhal). Haemophilia. 2010;16(2):281-289.
- Poonnoose PM, Thomas R, Keshava SN, et al. Psychometric analysis of the Functional Independence Score in Haemophilia (FISH). Haemophilia. 2007;13(5):620-626.
- Wharfe G, Buchner-Daley L, Gibson T, et al. The Jamaican Haemophilia Registry: describing the burden of disease. Haemophilia. 2018;24(4):e179-e186.
- Poonnoose PM, Manigandan C, Thomas R, et al. Functional Independence Score in Haemophilia: a new performance-based instrument to measure disability. Haemophilia. 2005;11(6):598-602.
- Padankatti SM, Macaden AS, Cherian SM, et al. A patient-prioritized ability assessment in haemophilia: the Canadian Occupational Performance Measure. Haemophilia. 2011;17(4):605-611.
- Tugwell P, Bombardier C, Buchanan WW, Goldsmith CH, Grace E, Hanna B. The MACTAR Patient Preference Disability Questionnaire–an individualized functional priority approach for assessing improvement in physical disability in clinical trials in rheumatoid arthritis. J Rheumatol. 1987;14(3):446-451.
- Zhou ZY, Wu J, Baker J, et al. Haemophilia utilization group study, Part Va (HUGS Va): design, methods and baseline data. Haemophilia. 2011;17(5):729-736.
- Eichler H, Schleicher C, Heine S, Graf N, von Mackensen S. Feasibility and results of a mobile haemophilia outpatient care pilot project. Hamostaseologie. 2018;38(3):129-140.
- Triemstra AH, Van der Ploeg HM, Smit C, Briet E, Ader HJ, Rosendaal FR. Well-being of haemophilia patients: a model for direct and indirect effects of medical parameters on the physical and psychosocial functioning. Soc Sci Med. 1998;47(5):581-593.
- Duncan N, Kronenberger W, Roberson C, Shapiro A. VERITAS-Pro: a new measure of adherence to prophylactic regimens in haemophilia. Haemophilia. 2010;16(2):247-255.
- Witkop ML, McLaughlin JM, Anderson TL, Munn JE, Lambing A, Tortella B. Predictors of non-adherence to prescribed prophylactic clotting-factor treatment regimens among adolescent and young adults with a bleeding disorder. Haemophilia. 2016;22(4):e245-e250.
- Globe DR, Curtis RG, Koerper MA. HUGS Steering Committee. Utilization of care in haemophilia: a resource-based method for cost analysis from the Haemophilia Utilization Group Study (HUGS). Haemophilia. 2004;10(Suppl 1):63-70.
- Cutter S, Molter D, Dunn S, et al. I mpact of mild to severe hemophilia on education and work by US men, women, and caregivers of children with hemophilia B: the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study. Eur J Haematol. 2017;98(Suppl 86):18-24.
- vandenBerg HM,Feldman BM,FischerK,BlanchetteV,Poonnoose P, Srivastava A. Assessments of outcome in haemophilia–what is the added value of QoL tools? Haemophilia. 2015;21(4):430-435.
- Ware JE. The SF36 Health Survey. In: Spilker B, ed. Quality of Life and Pharmacoeconomics in Clinical Trials. P hiladelphia, PA: Lippincott- Raven Publishers; 1996:337-345.
- Brazier J, Usherwood T, Harper R, Thomas K. Deriving a preferencebased single index from the UK SF-36 Health Survey. J Clin Epidemiol. 1998;51(11):1115-1128.
- Chai-Adisaksopha C, Skinner MW, Curtis R, et al. Exploring regional variations in the cross-cultural, international implementation of the Patient Reported Outcomes Burdens and Experience (PROBE) study. Haemophilia. 2019;25(3):365-372.
- Chai-Adisaksopha C, Skinner MW, Curtis R, et al. Test-retest properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire and its constituent domains. Haemophilia. 2019;25(1):75-83.
- Chai-Adisaksopha C, Skinner MW, Curtis R, et al. Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. BMJ Open. 2018;8(8):e021900.
- Young NL, Bradley CS, Blanchette V, et al. Development of a health-related quality of life measure for boys with haemophilia: the Canadian Haemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT). Haemophilia. 2004;10(Suppl 1):34-43.
- Remor E. Development and psychometric testing of the Hemophilia Well-being Index. Int J Behav Med. 2013;20(4):609-617.
- EuroQol Research Foundation. EQ-5D. EQ-5D website. https:// euroqol.org/. Accessed November 7, 2019.
- RAND Health Care. 36-Item Short Form Survey Instrument (SF-36). RAND Health Care website. https://www.rand.org/health-care/ surveys_tools/mos/36-item-short-form/survey-instrument.html. Accessed November 7, 2019.
- U.S. Department of Health and Human Services, FDA, CDER, CBER, CDRH. Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Silver Spring, MD, United States: U.S. Department of Health and Human Services, 2009. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-reported-outcome- measures-use-medical-product-development-suppo rt-labeling-claims. Accessed March 9, 2020.
- European Medicines Agency. Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man: the use of patient- reported outcome (PRO) measures in oncology studies. 2016. http://www.ema.europa.eu/en/documents/other/appen-dix-2-guideline- evaluation-anticancer-medicinal-products-man_en.pdf. Accessed May 20, 2020.
- HealthMeasures. PROMIS® (Patient-Reported Outcomes Measurement Information System). HealthMeasures website. https://www. healthmeasures.net/explore-measurement-systems/ promis. Accessed April 22, 2020.
- Recht M, Konkle BA, Jackson S, Neufeld EJ, Rockwood K, Pipe S. R ecognizing the need for personalization of haemophilia patient- reported outcomes in the prophylaxis era. Haemophilia. 2016;22(6):825-832.
- Beeton K, De Kleijn P, Hilliard P, et al. Recent developments in clini- metric instruments. Haemophilia. 2006;12(Suppl 3):102-107.
- Kempton CL, Wang M, Recht M, et al. Reliability of patient-reported outcome instruments in US adults with hemophilia: the Pain, Functional Impairment and Quality of life (P-FiQ) study. Patient Prefer Adherence. 2017;11:1603-1612.
- Porter ME, Larsson S, Lee TH. Standardizing patient outcomes measurement. N Engl J Med. 2016;374(6):504-506.
TOVÁBBI INFORMÁCIÓ
További kiegészítő információk találhatóak online a További információk részben.