Prevalence of Haemarthrosis and Clinical Impact on The Musculoskeletal System in People With Haemophilia in The United Kingdom; Evaluation of UK National Haemophilia Database and Haemtrack Patient Reported Data.

Abstract Introduction; Severe haemophilia is a rare x-linked recessive genetic disorder characterised by bleeding into soft tissue and joints. Haemarthrosis is the most common bleeding manifestation in haemophilia, leading to haemarthropathy. The ankle joint has been reported as the most common site of bleeding, but it is unclear whether the ankle is most affected in terms of joint health status. Aims; To determine the prevalence of joint bleeding and musculoskeletal health status at an individual joint level of children and adults with severe haemophilia A and B without a current inhibitor.Methods; Application was made to the National Haemophilia Database (NHD) with a request for the following; Haemtrack patient reported treatment record and Haemophilia Joint Health Scores (HJHS) in children (<18y) and adults (≥18y) with severe haemophilia A (HA) and B (HB) (FVIII/FIX, <0.01 iu/ml) without a current inhibitor. Data were collated and reported for 1st January to 31st December 2018.Results; 2238 cases were identified, of which 273 were Haemtrack compliant with contemporaneous HJHS. The median (IQR) age of children was 10 (6-13) and adults 40 (29-50) years. Haemarthrosis prevalence in HA/HB children was 33% and 47%, respectively and 60% and 42%, respectively, in adults. The most common haemarthrosis site in children was the knee in HA and ankle in HB. In adults, the incidence of haemarthrosis at the ankles and elbows was equal. Median (IQR) total HJHS in HA/HB children were 0 (0;0). In adults with HA/HB, HJHS were 18 (6; 31) and 11 (5; 24), respectively. In adults with HA/HB, mean (SD) ankle HJHS of 3.8 (4.1) and median 4.0 (0.0; 8.0) were higher than the knee (mean 2.9 (4.1) and median 1.0 (0.0; 5.0) and elbow (mean 3.3 (4.1) and median 1.0 (0.0; 7.0) joints.Conclusion; During 2018, NHD prevalence data of haemarthrosis indicate only two-thirds of children and one-third of adults from a UK cohort compliant with prophylaxis were bleed free. HJHS of zero in children suggests joint health status is either unaffected during childhood or undetected by HJHS. In adults, higher HJHS are reported for the ankles indicating worse joint health.

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P107 Prevalence of haemarthrosis and clinical impact on the musculoskeletal system in people with haemophilia in the United Kingdom: evaluation of UKHCDO and haemtrack patient reported data

Rheumatology ◽

10.1093/rheumatology/keaa111.105 ◽

2020 ◽

Vol 59 (Supplement_2) ◽

Author(s):

Richard A Wilkins ◽

Heidi J Siddle ◽

Graham Chapman ◽

Anthony J Redmond ◽

Hau Xiang ◽

...

Keyword(s):

United Kingdom ◽

Ankle Joint ◽

Health Research ◽

Clotting Factor ◽

Haemophilia A ◽

Severe Haemophilia ◽

Research Support ◽

Haemophilia B ◽

The United Kingdom ◽

Joint Health

Abstract Background Haemarthrosis whereby bleeding occurs within a joint is a significant feature of haemophilia. Despite the availability of prophylaxis clotting factor concentrates in the United Kingdom haemarthrosis is associated with worsening joint health and haemarthropathy in people with severe haemophilia. The ankle joint has been identified as the most affected joint followed by the knee and elbow. Whilst annual joint bleed rates (AJBR) are commonly reported in haemophilia research, bleed rates in individual affected joints and joint health status in paediatric and adult patients is yet to be established. Methods In 2018, paediatric (<18 years) and adult (≥18 years) patients with severe non-inhibitor haemophilia A and B registered with the United Kingdom Haemophilia Centres Doctors Organisation (UKHCDO) National haemophilia Database (NHD) were evaluated for haemarthrosis prevalence and associated joint health. Fully itemised Haemophilia Joint Health Scores (HJHS) were obtained from NHD records in combination with AJBR from Haemtrack (HT) compliant patients. Haemarthrosis prevalence and AJBR were reported as a total and for individual joints (ankles, knees and elbows). Results During 2018, 2233 individuals were identified; 273 reported ≥75% simultaneous HT compliance and electronic fully itemised HJHS data. The median (range) age of children is 10 (6-14) years and adults 40 (25-51) years. The joint bleed prevalence of haemophilia A and B in children is 33% and 47% respectively, and in adults 42% and 60% respectively. In children with haemophilia A (n = 80) the knee (data) was the most common site of bleeding. In haemophilia A adults (n = 157) the ankle and elbow were equally affected. In haemophilia B children (n = 17) and adults (n = 19) the elbow was the most prevalent site. Total HJHS scores in children with haemophilia A and B were 0.00 (1.00 SD) and 0.40 (0.90 SD) respectively. Total HJHS scores in adults with haemophilia A and B were 21.20 (16.80 SD) and 15.40 (15.10 SD) respectively. Mean HJHS scores itemised by joint were higher in adults compared with children. In children with haemophilia A and haemophilia B, mean (3.80) and median (4.00) scores for the ankle joint were higher than for the knee (2.90 and 1.00) and elbow joint (3.30 and 1.00). Conclusion Whilst there are limitations to this self-select subset of individuals with severe haemophilia, the prevalence of haemarthrosis is evenly distributed in all adult joints with a trend towards the knee in paediatrics. Despite prophylaxis 30% of children and 60% adults still reported bleeding over a 12-month period. Irrespective of the prevalence of joint bleeds in children with haemophilia the HJHS does not appear to be clinically sensitive enough to detect changes in joint health. Adults deemed adherent with prophylaxis and haemtrack still demonstrate worsening HJHS scores despite a low AJBR. Disclosures R.A. Wilkins Grants/research support; Funded by the National Institute for Health Research. H.J. Siddle Grants/research support; Funded by the National Institute for Health Research. G. Chapman None. A.J. Redmond None. H. Xiang None. M. Scott None. M. Richards None. L. Horn None. B. Palmer None. D. Stephensen None.

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Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates

Thrombosis and Haemostasis ◽

10.1160/th15-08-0637 ◽

2016 ◽

Vol 115 (05) ◽

pp. 931-938 ◽

Cited By ~ 20

Author(s):

Annelies Nijdam ◽

Wouter Foppen ◽

Piet de Kleijn ◽

Evelien P. Mauser-Bunschoten ◽

Goris Roosendaal ◽

...

Keyword(s):

Functional Limitations ◽

Objective Assessment ◽

Haemophilia A ◽

Severe Haemophilia ◽

On Demand ◽

Recommended Treatment ◽

Objective Monitoring ◽

Joint Health ◽

Observational Cohort

SummaryProphylaxis is the recommended treatment for children with severe haemophilia A, but whether prophylaxis should be continued in adulthood is still under debate. Previous studies with limited follow-up have suggested that some patients may be able to stop prophylaxis in adulthood, while maintaining good joint health. This single-centre observational cohort study examined patients with severe haemophilia A born 1970–1988 without inhibitor development, and assessed the long-term consequences of discontinuing prophylaxis. Patient-initiated changes in prophylaxis, including all switches to on-demand treatment lasting a minimum of two consecutive weeks, were recorded from the time self-infusion began until the last evaluation. Sixty-six patients were evaluated at a median age of 32.4 years: 26 of patients had stopped prophylaxis for a median of 10 years, 15 had interrupted prophylaxis and 59 had continued prophylaxis. Annual joint bleeding rate (AJBR), Haemophilia Joint Health Score (HJHS-2.1; 0–124 points), radiological Pettersson score (0–78 points) and Haemophilia Activities List score (HAL; 100–0 points) were compared between patients who stopped and patients who continued prophylaxis. Although self-reported bleeding rates and functional limitations were similar in both groups (AJBR: 1.5 vs 1.2 and HAL: 84 vs 84 for those who stopped and continued prophylaxis, respectively), objective assessment of joint status showed increased arthropathy after 10 years of on-demand treatment in patients who stopped prophylaxis compared with those who continued (HJHS: 23 vs. 14 and Pettersson: 16 vs 5, respectively; P< 0.01). These results support continuation of long-term prophylaxis in adults and demonstrate the need for objective monitoring of joint status.Trial registration: Dutch Trial Registry number 3098; UTN U1111–1121–7069.

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Impact of severe haemophilia A on patients' health status: results from the guardian ™ 1 clinical trial of turoctocog alfa (NovoEight ® )

Haemophilia ◽

10.1111/hae.12617 ◽

2015 ◽

Vol 21 (4) ◽

pp. 451-457 ◽

Cited By ~ 4

Author(s):

M. Ozelo ◽

P. Chowdary ◽

A. Regnault ◽

A. K. Busk

Keyword(s):

Clinical Trial ◽

Health Status ◽

Haemophilia A ◽

Severe Haemophilia ◽

The Guardian

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Utility of the Haemophilia Joint Health Score in study of episodically treated boys with severe haemophilia A and B in Lithuania

Haemophilia ◽

10.1111/j.1365-2516.2009.02178.x ◽

2010 ◽

Cited By ~ 3

Author(s):

S. SAULYTE TRAKYMIENE ◽

J. INGERSLEV ◽

L. RAGELIENE

Keyword(s):

Haemophilia A ◽

Severe Haemophilia ◽

Health Score ◽

Joint Health

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Wearable knee health system employing novel physiological biomarkers

Journal of Applied Physiology ◽

10.1152/japplphysiol.00366.2017 ◽

2018 ◽

Vol 124 (3) ◽

pp. 537-547 ◽

Cited By ~ 5

Author(s):

Omer T. Inan ◽

Daniel C. Whittingslow ◽

Caitlin N. Teague ◽

Sinan Hersek ◽

Maziyar Baran Pouyan ◽

...

Keyword(s):

Health Status ◽

Knee Injuries ◽

Health Assessment ◽

Machine Learning Algorithms ◽

Cross Sectional ◽

Quantitative Measurements ◽

Patient Reported ◽

Measured Time ◽

Joint Health ◽

Physical Examinations

Knee injuries and chronic disorders, such as arthritis, affect millions of Americans, leading to missed workdays and reduced quality of life. Currently, after an initial diagnosis, there are few quantitative technologies available to provide sensitive subclinical feedback to patients regarding improvements or setbacks to their knee health status; instead, most assessments are qualitative, relying on patient-reported symptoms, performance during functional tests, and physical examinations. Recent advances have been made with wearable technologies for assessing the health status of the knee (and potentially other joints) with the goal of facilitating personalized rehabilitation of injuries and care for chronic conditions. This review describes our progress in developing wearable sensing technologies that enable quantitative physiological measurements and interpretation of knee health status. Our sensing system enables longitudinal quantitative measurements of knee sounds, swelling, and activity context during clinical and field situations. Importantly, we leverage machine-learning algorithms to fuse the low-level signal and feature data of the measured time series waveforms into higher level metrics of joint health. This paper summarizes the engineering validation, baseline physiological experiments, and human subject studies—both cross-sectional and longitudinal—that demonstrate the efficacy of using such systems for robust knee joint health assessment. We envision our sensor system complementing and advancing present-day practices to reduce joint reinjury risk, to optimize rehabilitation recovery time for a quicker return to activity, and to reduce health care costs.

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