Introduction: Self-completed measures of physical function and quality of life are increasingly being used in clinical practice yet little is known about how these measures correlate with joint scores in boys with severe haemophilia. In addition, it is not known whether currently used measures of functional outcome correlate with quality of life measures, which measure of physical function is most accurate and whether these measures are acceptable to a well treated contemporary cohort of boys. Methods: The Study Of physical Function In adolescenTs with haemophilia (SO-FIT) is a multicenter, randomized cross-over study designed to answer these questions, and to determine whether these self-reported measures are completed more fully and frequently if made available on mobile devices rather than with conventional pen and paper questionnaires. The study is being run by haemophilia nurse specialists and physiotherapists in UK haemophilia centres and will recruit 120 boys with severe haemophilia A or B, with or without inhibitors aged 8-16 years. Results:The SO-FIT study is now underway, and is expected to complete by the end of 2014.
If the inline PDF is not rendering correctly, you can download the PDF file here.
1. Manco-Johnson MJ Abshire TC Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
2. Liesner RJ Khair K Hann IM. The impact of prophyactic treatment on children with severe haemophilia. Br J Haematol 1996; 92: 973-8.
3. Srivastava A Brewer AK Mauser-Bunschoten EP et al. Guidelines for the management of hemophilia. Haemophilia 2013; 19(1): e1-47.
4. Richards M Williams M Chalmers E et al. A United Kingdom Haemophilia Centre Doctors' Organization guideline approved by the British Committee for Standards in Haematology: guideline on the use of prophylactic factor VIII concentrate in children and adults with severe haemophilia A. Br J Haematol 2010; 149: 498-507.
5. UKHDCO. Annual report 2012 & bleeding disorder statistics for the financial year 2011/12. A report from the UKHCDO and NHD. Available at http://firstname.lastname@example.org.
6. Feldman BM Funk S 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 (HJHS). Arthritis Care & Research 2011; 63: 223-30.
7. Bladen M Main E Hubert N et al. Factors affecting the Haemophilia Joint Health Score in children with severe haemophilia. Haemophilia 2013; 19: 626-31.
8. Hilliard P Funk S Zourikian N. Hemophilia joint health score reliability study. Haemophilia 2006; 12: 518-25.
9. Doward LC McKenna SP Defining Patient-Reported Outcomes. Value in Health 2004; 7(S1): S4-S8.
10. 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: 281-9.
11. Groen W Van Der Net J Lacatusu AM Serban M et al. Functional limitations in Romanian children with haemophilia; further testing of psychometric properties of the Paediatric Haemophilia Activities List. Haemophilia 2103; 19: e116-125
12. von Mackensen S Czepa D Herbsleb M Hilberg T. Development and validation of a new questionnaire for the assessment of subjective physical performance in adult patients with haemophilia--the HEP-Test-Q. Haemophilia 2010; 16: 170-8.
13. von Mackensen S Bullinger M; Haemo-QoL Group. Development and testing of an instrument to assess the Quality of Life of Children with Haemophilia in Europe (Haemo-QoL).Haemophilia 2004; 10 Suppl 1: 17-25.
14. Pollak E Mühlan H von Mackensen S Bullinger M; HAEMO-QOL GROUP. The Haemo-QoL Index: developing a short measure for health-related quality of life assessment in children and adolescents with haemophilia. Haemophilia 2006; 12: 384-92.
15. Department of Health. Equity and Excellence: Liberating the NHS. DoH July 2010.
16. Tinetti ME Basch E. Patients’ responsibility to participate in decision-making and research. JAMA 2013; 309: 2331-2.