Long-term impact of joint bleeds in von Willebrand disease: a nested case-control study

Karin P M van Galen, Piet de Kleijn, Wouter Foppen, Jeroen Eikenboom, Karina Meijer, Roger E G Schutgens, Kathelijn Fischer, Marjon H Cnossen, Joke de Meris, Karin Fijnvandraat, Johanna G van der Bom, Britta A P Laros-van Gorkom, Frank W G Leebeek, Eveline P Mauser-Bunschoten, Win study group, Karin P M van Galen, Piet de Kleijn, Wouter Foppen, Jeroen Eikenboom, Karina Meijer, Roger E G Schutgens, Kathelijn Fischer, Marjon H Cnossen, Joke de Meris, Karin Fijnvandraat, Johanna G van der Bom, Britta A P Laros-van Gorkom, Frank W G Leebeek, Eveline P Mauser-Bunschoten, Win study group

Abstract

Patients with severe von Willebrand disease (VWD) may develop arthropathy after joint bleeds. Information on its prevalence and severity is limited. We aimed to assess the occurrence and severity of arthropathy in VWD and its impact on daily life. VWD patients with and without verified joint bleeds were matched for age, sex and Factor VIII level or von Willebrand Factor activity in a nested case-control study within the Willebrand in the Netherlands study. Assessments included the Hemophilia Joint Health Score (0-124), Pettersson score (0-13 per joint X-ray), Hemophilia Activity List score (0-100), joint pain (Visual Analog Scale 0-10), and the Impact on Participation and Autonomy questionnaire (0-20). Arthropathy was defined as a Hemophilia Joint Health Score of 10 or higher, or a Pettersson score over 3 of at least one joint. We included 48 patients with verified joint bleeds (cases) and 48 controls: 60% males, mean age 46 years (range 18-80), median von Willebrand Factor activity 5 versus 8 IU/dL and Factor VIII 24 versus 36 IU/dL. Arthropathy occurred in 40% of the cases versus 10% of the controls (P<0.01). The cases reported more functional limitations compared to the controls (median Hemophilia Activity List score: 88 vs. 100, P<0.01). Arthropathy was related to joint pain and less social participation (Visual Analog Scale>3: 13 of 19 vs. 3 of 28, P<0.01, and median score on the participation questionnaire 6.1 vs. 0.9, P<0.01). In conclusion, arthropathy occurs in 40% of VWD patients after joint bleeds and is associated with pain, radiological abnormalities, functional limitations, and less social participation (Dutch trial register: NTR4548).

Copyright© 2017 Ferrata Storti Foundation.

References

    1. Leebeek FW, Eikenboom JC. Von Willebrand’s Disease. N Engl J Med. 2016;375(21):2067–2080.
    1. de Wee EM, Sanders YV, Mauser-Bunschoten EP, et al. Determinants of bleeding phenotype in adult patients with moderate or severe von Willebrand disease. Thromb Haemost. 2012;108(4):683–692.
    1. Jansen NW, Roosendaal G, Lafeber FP. Understanding haemophilic arthropathy: an exploration of current open issues. Br J Haematol. 2008;143(5):632–640.
    1. van Galen KP, Sanders YV, Vojinovic U, et al. Joint bleeds in von Willebrand disease patients have significant impact on quality of life and joint integrity: a cross-sectional study. Haemophilia. 2015;21(3):e185–e192.
    1. van Galen KP, Mauser-Bunschoten EP, Leebeek FW. Hemophilic arthropathy in patients with von Willebrand disease. Blood Rev. 2012;26(6):261–266.
    1. Jette AM, Keysor JJ. Disability models: implications for arthritis exercise and physical activity interventions. Arthritis Rheum. 2003;49(1):114–120.
    1. Fischer K, Nijdam A, Holmstrom M, et al. Evaluating outcome of prophylaxis in haemophilia: objective and self-reported instruments should be combined. Haemophilia. 2016. February 8 [Epub ahead of print]
    1. de Wee EM, Leebeek FWG, Eikenboom JCJ. Diagnosis and Management of von Willebrand Disease in The Netherlands. Semin Thromb Hemost. 2011;37(5):480–487.
    1. Hilliard P, Funk S, Zourikian N, et al. Hemophilia joint health score reliability study. Haemophilia. 2006;12(5):518–525.
    1. Soucie JM, Wang C, Forsyth A, et al. Range of motion measurements: reference values and a database for comparison studies. Haemophilia. 2011;17(3):500–507.
    1. Fischer K, Steen CK, Petrini P, et al. Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s. Blood. 2013;122(7):1129–1136.
    1. van Galen KP, Timmer M, de Kleijn P, et al. Joint Assessment in Von Willebrand Disease: Validation of the Haemophilia Joint Health Score and Haemophilia Activities List. Thromb Haemost. 2017. May 11 [Epub ahead of print]
    1. Foppen W, van der Schaaf IC, Beek FJ, Verkooijen HM, Fischer K. Scoring haemophilic arthropathy on X-rays: improving inter- and intra-observer reliability and agreement using a consensus atlas. Eur Radiol. 2016;26(6):1963–1970.
    1. Foppen W, van der Schaaf IC, Beek FJ, Verkooijen HM, Fischer K. Scoring haemophilic arthropathy on X-rays: improving inter- and intra-observer reliability and agreement using a consensus atlas. Eur Radiol. 2016;26(6):1963–1970.
    1. 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.
    1. 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.
    1. Nijdam A, Foppen W, de Kleijn P, et al. Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates. Thromb Haemost. 2016;115(5):931–938.
    1. Cardol M, de Haan RJ, van den Bos GA, de Jong BA, de Groot IJ. The development of a handicap assessment questionnaire: the Impact on Participation and Autonomy (IPA). Clin Rehabil. 1999;13(5):411–419.
    1. van der Kloot WA, Oostendorp RA, van der Meij J, van den Heuvel J. [The Dutch version of the McGill pain questionnaire: a reliable pain questionnaire]. Ned Tijdschr Geneeskd. 1995;139(13):669–673.
    1. de Joode EW, van Meeteren NL, van den Berg HM, de Kleijn P, Helders PJ. Validity of health status measurement with the Dutch Arthritis Impact Measurement Scale 2 in individuals with severe haemophilia. Haemophilia. 2001;7(2):190–197.
    1. Feldman BM, Pullaneyagum E. Response to ‘Limits of agreement between raters are required for use of HJHS 2.1 in clinical studies’. Haemophilia. 2015; 21(1):e71.
    1. Fischer K, de Kleijn P. Using the Haemophilia Joint Health Score for assessment of teenagers and young adults: exploring reliability and validity. Haemophilia. 2013;19(6):944–950.
    1. den Uijl IE, Fischer K, van der Bom JG, Grobbee DE, Rosendaal FR, Plug I. Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels. Haemophilia. 2011;17(1):41–44.
    1. Oliveria SA, Felson DT, Reed JI, Cirillo PA, Walker AM. Incidence of symptomatic hand, hip, and knee osteoarthritis among patients in a health maintenance organization. Arthritis Rheum. 1995;38(8):1134–1141.
    1. 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.
    1. Kreuz W, Escuriola-Ettingshausen C, Funk M, Schmidt H, Kornhuber B. When should prophylactic treatment in patients with haemophilia A and B start?–The German experience. Haemophilia. 1998;4(4):413–417.
    1. Ling M, Heysen JP, Duncan EM, Rodgers SE, Lloyd JV. High incidence of ankle arthropathy in mild and moderate haemophilia A. Thromb Haemost. 2011;105(2):261–268.
    1. de Kleijn P, Gilbert M, Roosendaal G, Poonnose PM, Narayan PM, Tahir N. Functional recovery after bleeding episodes in haemophilia. Haemophilia. 2004;10 Suppl 4:157–160.
    1. Abshire TC, Federici AB, Alvarez MT, et al. Prophylaxis in severe forms of von Willebrand’s disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN). Haemophilia. 2013;19(1):76–81.

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