Objective
Repeated hemarthrosis in hemophilia causes arthropathy with pain and dysfunction. The Hemophilia Joint Health Score (HJHS) was developed to be more sensitive for detecting arthropathy than ...the World Federation of Hemophilia (WFH) physical examination scale, especially for children and those using factor prophylaxis. The HJHS has been shown to be highly reliable. We compared its validity and sensitivity to the WFH scale.
Methods
We studied 226 boys with mild, moderate, and severe hemophilia at 5 centers. The HJHS was scored by trained physiotherapists. Study physicians at each site blindly determined individual and total joint scores using a series of visual analog scales.
Results
The mean age was 10.8 years. Sixty‐eight percent were severe (93% of whom were treated with prophylaxis), 15% were moderate (24% treated with prophylaxis), and 17% were mild (3% treated with prophylaxis). The HJHS correlated moderately with the physician total joint score (rs = 0.42, P < 0.0001) and with overall arthropathy impact (rs = 0.42, P < 0.0001). The HJHS was 97% more efficient than the WFH at differentiating severe from mild and moderate hemophilia. The HJHS was 74% more efficient than the WFH at differentiating subjects treated with prophylaxis from those treated on demand. We identified items on the HJHS that may be redundant or rarely endorsed and could be removed from future versions.
Conclusion
Both the HJHS and WFH showed evidence of strong construct validity. The HJHS is somewhat more sensitive for mild arthropathy; its use should be considered for studies of children receiving prophylaxis.
Outcome measures in Haemophilia: Beyond ABR (Annualized Bleeding Rate) Manco‐Johnson, Marilyn J.; Warren, Beth Boulden; Buckner, Tyler W. ...
Haemophilia : the official journal of the World Federation of Hemophilia,
February 2021, 2021-Feb, 2021-02-00, 20210201, Letnik:
27, Številka:
S3
Journal Article
Recenzirano
Odprti dostop
Options for management of haemophilia are increasing rapidly with completely novel therapeutic approaches that cannot be compared using traditional factor assays. In addition, as prophylaxis regimens ...have improved, bleeding rates have decreased, and consequently, it is difficult to show an impact of novel therapies on rates of spontaneous bleeding. There is currently an urgent need for a panel of outcome measures to compare therapies that are dissimilar in many essential ways. Conventional objective outcome measures including joint physical examination and joint imaging continue to hold a central importance. Factor assays are essential for evaluation of products derived from native factor genes, but are not applicable to some extended half‐life factors or non‐factor bypassing agents. Global assays including thrombin generation and chromogenic assays of factor X activation are under investigation for their usefulness in haemophilia assessment. Bleeding rate is a conventional subjective patient‐reported outcome that, while decreasing in frequency, is indispensable as an outcome given that the primary manifestation of haemophilia is bleeding. Other patient‐reported outcomes such as pain intensity and interference, health‐related quality of life and activities and participation are increasingly important to distinguish superior outcomes in comparative trials. This review of outcome measures for haemophilia presents examples of existing outcome measures with an emphasis on their strengths and limitations.
Background
The Colorado Adult Joint Assessment Scale (CAJAS) is designed to assess joint health in adults with hemophilia. The CAJAS comprises nine items (swelling, muscle atrophy, axial deformity, ...crepitus, range of motion, contracture, instability, strength, gait) and assesses six joints.
Objective
To assess CAJAS content validity and psychometric properties.
Patients/Methods
Data were obtained from the Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment (SPINART) study and a separate CAJAS validation study. CAJAS assessments in SPINART were performed by physical therapists (PTs) from the United States, Romania, Bulgaria, and Argentina. In the validation study, content validity was assessed from interviews with six PTs at three US hemophilia centers; cultural equivalence was assessed with seven non‐US PTs from SPINART. Reliability data were collected from 30 subjects at four US centers. Test‐retest reliability was evaluated by having the same PT perform CAJAS examinations at two visits, 7‐10 days apart. Inter‐rater reliability was assessed by comparing CAJAS scores of two different PTs performing separate examinations of the same patient several hours apart at the same visit. Psychometric properties were assessed using SPINART and validation study data.
Results
The CAJAS demonstrated good content validity. Test‐retest reliability was high (intraclass correlation coefficient, 0.98), as was inter‐rater reliability (intraclass correlation coefficient, 0.88). Internal consistency reliability was strong (α = .90). The CAJAS demonstrated good convergent/divergent validity, known‐groups validity, and ability to detect change.
Conclusions
The CAJAS is a valid and reliable measure of joint health in adults with moderate‐severe hemophilia and is appropriate for use in clinical practice.
Introduction
The Hemophilia Joint Health Score (HJHS) was developed to detect early changes in joint health in children and adolescents with haemophilia. The HJHS is considered by some to be too time ...consuming for clinical use and this may limit broad adoption.
Aim
This study was a first step to develop a shorter and/or more convenient version of the HJHS for the measurement of joint function in children and young adults with haemophilia, by combining real‐life data and expert opinion.
Methods
A cross‐sectional multicenter secondary analysis on pooled data of published studies using the HJHS (0‐124, optimum score 0) in persons with haemophilia A/B aged 4‐30 was performed. Least informative items, scoring options and/or joints were identified. An expert group of 19 international multidisciplinary experts evaluated the results and voted on suggestions for adaptations in a structured meeting (consensus set at ≥ 80%).
Results
Original data on 499 persons with haemophilia from 7 studies were evaluated. Median age was 15.0 years range 4.0‐29.9, 83.2% had severe haemophilia and 61.5% received prophylaxis. Median (IQR) HJHS total was 6.0 (1.0‐17.0). The items 'duration swelling' and 'crepitus' were identified as clinically less informative and appointed as candidates for reduction.
Conclusion
Analysis of 499 children and young adults with haemophilia showed that the HJHS is able to discriminate between children and adults and different treatment regimens. Reduction of the items 'duration swelling' and 'crepitus' resulted in the HJHSshort, which had the same discriminative ability. Additional steps are needed to achieve a substantially shorter HJHS assessment.
Background and Objectives
National guidelines for gastrointestinal (GI) cancers offer surveillance algorithms to facilitate detection of recurrent disease, yet adherence rates are unknown. We sought ...to characterize postoperative surveillance patterns for veterans with GI cancer at a tertiary care Veterans Affairs Hospital.
Methods
A single‐center retrospective cohort study identified patients who underwent surgical resection for colorectal, gastroesophageal or hepatopancreaticobiliary malignancy from 2010‐2016. We calculated the annual rate of cancer‐directed clinic visits and abdominal imaging and used National Comprehensive Cancer Network guidelines as a benchmark by which to assess adequate surveillance.
Results
Ninety‐seven patients met inclusion criteria. Median surveillance time was 1203 days. Overall, 44% of patients had insufficient surveillance. Specifically, 11% received no postoperative imaging and 7% had no cancer‐directed clinic visits. An additional 30% received less than recommended surveillance imaging and 12% attended fewer than recommended clinic visits. By disease site, insufficient imaging was most common for patients with hepatopancreaticobiliary cancer (63%), while inadequate clinic follow‐up was highest for colorectal cancer (24%).
Conclusion
A significant proportion of veterans with GI cancer received either inadequate postoperative surveillance based on national guidelines. This deficiency represents an opportunity for improvement through targeted efforts, including telemedicine and education of patients and providers.
The Joint Outcome Study (JOS), a randomized controlled trial, demonstrated that children with severe hemophilia A (HA) initiating prophylactic factor VIII (FVIII) prior to age 2.5 years had reduced ...joint damage at age 6 years compared with those treated with episodic FVIII for bleeding. The Joint Outcome Continuation Study (JOS-C) evaluated early vs delayed prophylaxis effects on long-term joint health, following JOS participants to age 18 years in an observational, partially retrospective study. Index joint magnetic resonance imaging (MRI) scores of osteochondral (OC) damage (primary outcome), joint physical examination scores, and annualized rates of joint/other bleeding episodes (secondary outcomes) were collected. Thirty-seven of 65 JOS participants enrolled in JOS-C, including 15 randomized to prophylaxis at mean age 1.3 years (“early prophylaxis”); 18 initially randomized to episodic treatment, starting “delayed prophylaxis” at mean age 7.5 years; and 4 with high-titer inhibitors. At JOS-C exit, MRI OC damage was found in 77% of those on delayed and 35% of those on early prophylaxis for an odds ratio of OC damage, in the delayed vs early prophylaxis group, of 6.3 (95% confidence interval, 1.3, 29.9; P = .02). Annualized bleeding rates were higher with delayed prophylaxis (mean plus or minus standard deviation, 10.6 ± 6.6 vs 3.5 ± 2.1; P < .001), including when only comparing time periods on prophylaxis (6.2 ± 5.3 vs 3.3 ± 1.9; P < .05). In severe HA, early initiation of prophylaxis provided continued protection against joint damage throughout childhood compared with delayed initiation, but early prophylaxis was not sufficient to fully prevent damage. This trial was registered at www.clinicaltrials.gov as #NCT01000844.
•Joint outcomes in hemophilia are better in young adults if prophylaxis is started before age 2.5 years compared with after age 6 years.•Standard FVIII prophylaxis is insufficient to fully protect joints from damage through adolescence in severe HA.
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Social and behavioural factors are critical to the emergence, spread and containment of human disease, and are key determinants of the course, duration and outcomes of disease outbreaks. Recent ...epidemics of Ebola in West Africa and coronavirus disease 2019 (COVID-19) globally have reinforced the importance of developing infectious disease models that better integrate social and behavioural dynamics and theories. Meanwhile, the growth in capacity, coordination and prioritization of social science research and of risk communication and community engagement (RCCE) practice within the current pandemic response provides an opportunity for collaboration among epidemiological modellers, social scientists and RCCE practitioners towards a mutually beneficial research and practice agenda. Here, we provide a review of the current modelling methodologies and describe the challenges and opportunities for integrating them with social science research and RCCE practice. Finally, we set out an agenda for advancing transdisciplinary collaboration for integrated disease modelling and for more robust policy and practice for reducing disease transmission.
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