Joint damage from bleeding episodes leads to physical or functional limitations in people with haemophilia. Various factors may influence the frequency and severity of joint damage. This study examined whether age, prophylaxis, history of high-titre inhibitors (HTI) and bleeding events influenced the Haemophilia Joint Health Score (HJHS) in children. Medical and physiotherapy notes of boys with severe haemophilia, aged 4-18 years, were reviewed to identify factors associated with increased HJHS. The HJHS of 83 boys (median age: 11) ranged from 0 to 25, with 44/83 (53%) having a score of zero. The median HJHS was 0 (mean 2.6). In the non-HTI group, the HJHS for boys on late prophylaxis was 2.68 times higher than those who started early and the HJHS was on average 10% higher for every additional recent bleed. In this group the odds of having a zero score fell by 30% for every year increase in age. Boys with a history of HTI had higher HJHS scores than the non-HTI group, and age, number of recent bleeds and tolerized status were positively associated with HJHS. The score rose on average by 28% for every year of age and by 76% for non-tolerized boys. This study provides further evidence supporting early prophylaxis use and the importance of immune tolerance therapy. The HJHS is a useful tool for identifying and tracking changes in joint health with respect to therapy or disease progression. With improvements in haemophilia treatment, the disproportionate number of zero scores will continue to make interpretation of the HJHS challenging.
Inter-physiotherapist discrepancies in routine HJHS hamper comparison of scores between treatment regimens. For multi-centre research, additional inter-observer standardization for HJHS scoring is needed.
Risks and consequences of ICH in IBD were highest within the neonatal period and first year of life. MDD after ICH was not reliably identified in early life and ongoing monitoring in the first decade of life will facilitate educational support or physical rehabilitation.
Background There is a lack of functional performance measures for children and young people with haemophilia (CYPwH) with associated control data from typically developing boys (TDB). The literature advocates development of a core set of outcome measures for different chronic conditions. As medical treatment improves, CYPwH are experiencing better outcomes; therefore, more challenging measures are required to monitor physical performance. Such testing is not performed routinely, due to practical and safety concerns. Aim Evaluate the feasibility, safety and acceptability of select outcome measures as part of a study protocol testing CYPwH; including myometry, 10 metre incremental shuttle walk test (10-m ISWT), iSTEP (an incremental step test, with data from TDB), and 1 week of accelerometry-wear at home. Methods Sixty-six boys aged 6–15 years with mild, moderate or severe haemophilia A or B (including inhibitors) attending routine clinics at Great Ormond Street Hospital were approached to participate. Descriptive statistics and content analysis were used to assess outcomes of feasibility, safety and acceptability, which included recruitment/retention rates, protocol completion within routine appointment timeframes, performance testing without serious adverse events/reactions (SAE/SARs), and acceptability to CYPwH of high-level performance measures. Results Outcomes were met: 43 boys completed testing at clinic review (Jan–Nov 2018) within a 10-month timeframe, retention was 95% at completion of protocol and no SAE/SARs were reported throughout testing. Conclusion Feasibility, safety and acceptability of the study protocol have been established in this population. Both high-level performance tests, iSTEP and 10-m ISWT, were an acceptable addition to boys’ routine clinic appointments and could be safe, acceptable choices of outcome measure as part of a core set of tests for CYPwH. Further investigation of the psychometric properties for the iSTEP is now justified, in order for it to be used as a standardised, validated, reliable outcome measure in clinical or research settings. Trial registration Retrospectively registered on September 3, 2019, on ClinicalTrials.gov (ID: NCT04076306).
BackgroundRepetitive joint bleeding leading to chronic synovitis and progressive arthropathy are the hallmarks of severe haemophilia and a major cause of disability in patients with this coagulopathy. Haemophilic synovitis is usually prevented by prophylactic clotting factor replacement and bypassing agents. However, a small number of patients have inhibitory antibodies to factor replacement and develop one or more target joints with chronic synovitis. Our aim was to set-up a novel radionuclide therapy service (our first at GOSH) using radiation synovectomy as an alternative treatment option to treat children with haemophilic synovitis.MethodsA multi-disciplinary group was formed to develop this service utilising a collaborative approach between haemophilia, physiotherapy and radiology. The intervention involves injecting Re186 into the joint capsule within interventional radiology to cause coagulation necrosis of the synovial tissue through β-irradiation. The λ-emission is then imaged using SPECT CT to assess the bio-distribution of the radio-colloid and detect any extra-articular radioactivity.ResultsThis service development was presented as a QIPP to the CCG. The approval for this therapy was based on its close alignment with efficiency and value strategies in delivering a personalised effective treatment for the patient with minimal inpatient stay, whilst significantly reducing the costs associated with prophylactic clotting factor.ConclusionsThis therapy should be most effective in grade I and II synovitis and it is anticipated that it will reduce the frequency of haemarthrosis and prevent progression to haemophiliac arthropathy. At present, 8 patients have been identified and accepted for this therapy under the referral criteria, with the service due to commence imminently. The intervention outcome parameters will be measured by; 1.Future joint bleed frequency, 2. Pre and post MR imaging to assess synovial thickness, 3. Joint pain scores, and 4.Physical objective and functional measures. Patient reported outcomes (PROMs) will be used to assess impact on quality of life.
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