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Biomedical subjects

B M Feldman

Publications and source records attributed to B M Feldman.

At least 19 recordsLinked to original sources
PubMed2007

Definitions for haemophilia prophylaxis and its outcomes: the Canadian consensus study.

The creation of acceptable standard definitions for terms used in the care and assessment of haemophilia patients has become increasingly important, as a growing number of international clinical studies have been initiated. The Delphi approach has been used in health research to reach consensus in large groups and can be used to develop definitions by using several iterations of surveys eliciting opinions from specialists in the field. Three consecutive surveys were designed based on the Delphi approach and distributed to specialist physicians, nurses and physiotherapists in order to develop definitions for seven haemophilia terms: 'primary prophylaxis', 'secondary prophylaxis', 'target joint', 'joint bleed', 'significant soft-tissue bleed', 'superficial soft-tissue bleed' and 'mucosal bleed'. Suggestions were solicited, compiled into a subsequent survey and fed back to the group to rank-order the importance of each suggested component of the definition. Final definitions were created using the top-ranked suggestions and sent back to the experts for approval. Five of the seven terms were highly endorsed with greater than 90% agreement. Some differences in agreement were found when analysed by profession. Haemophilia terms were successfully defined using the Delphi approach. Further refinement from members of the international haemophilia community will ensure that comprehensive standard definitions can be used in multicentre studies in the future.

Canada↗
PubMed2006

Pilot study of fitness training and exercise testing in polyarticular childhood arthritis.

OBJECTIVE: To 1) assess the safety and feasibility of laboratory-based exercise testing in juvenile idiopathic arthritis (JIA), 2) test the safety and feasibility of a 3-month exercise program in JIA, 3) assess pain during exercise in JIA, 4) compare ratings of perceived effort (RPE) with heart rate (HR) achieved, and 5) estimate the training effect on metabolic efficiency of gait as measured by submaximal exercise testing. METHODS: Nine children with JIA were enrolled in a 12-week circuit training program involving pool, stationary bicycle, treadmill, and Fitball. They underwent formal exercise testing before and after the program, underwent a full joint assessment, were administered the Childhood Health Assessment Questionnaire and Juvenile Arthritis Functional Status Index, and were assessed for overall quality of life and health-related quality of life. A visual analog scale was used to assess pain during testing and training, and the Borg scale was used to measure RPE. RESULTS: Children with JIA were able to participate in exercise testing without any significant problems. Children with severe hip disease dropped out of the exercise program due to pain during the exercise sessions and worsened arthritis symptoms. Target HR was achieved and correlated with RPE in the bicycle and treadmill sessions. Submaximal exercise testing showed an improvement with a small to moderate effect size. CONCLUSION: This study suggests that it is safe, feasible, and acceptable for children with arthritis, in the absence of severe hip involvement, to participate in formal exercise testing and structured fitness programs.

Arthritis, Juvenile↗
PubMed2006

An international consensus survey of the diagnostic criteria for juvenile dermatomyositis (JDM).

OBJECTIVE: To develop revised criteria for the diagnosis of juvenile dermatomyositis (JDM) using an international consensus process. METHODS: An initial survey was circulated to members of the Network for JDM and the Paediatric Rheumatology International Trials Organisation (PRINTO). Each individual was asked to identify those criteria that were felt to be most helpful in the diagnosis of classical JDM. A second survey was derived from these results and used to rank these proposed criteria in order of their importance and usefulness in clinical practice. RESULTS: The first survey had a response rate of 49.8% (118 individuals) from 92 centres in 32 countries. All responders routinely used proximal muscle weakness and characteristic skin rash in the diagnosis of JDM, while 86.8% used elevated muscle enzymes. Muscle biopsy, magnetic resonance imaging (MRI) and changes on the electromyogram (EMG) were deemed important diagnostic criteria. Other criteria, including myositis-specific or -related antibodies, nailfold capillaroscopy, factor VIII-related antigen, muscle ultrasound, calcinosis and neopterin, were used by 35.3% of respondents. Seventy-eight respondents to the first survey (66%) responded to the second survey. Typical MRI and muscle biopsy changes were rated by all to be the most useful clinically relevant diagnostic criteria after proximal muscle weakness, characteristic skin rash and elevated muscle enzymes. These were followed by myopathic changes on EMG, calcinosis, dysphonia and nailfold capillaroscopy, which were ranked equally. CONCLUSION: This process identified nine criteria that clinicians felt to be helpful or important in the diagnosis of JDM. A further process of refinement and validation is necessary to agree an internationally acceptable, clinically usable set of diagnostic criteria.

Biomarkers↗
PubMed2006

Tumour necrosis factor alpha inhibitors in the treatment of childhood uveitis.

OBJECTIVE: To describe the efficacy of anti-TNF-alpha agents in the treatment of childhood uveitis. METHODS: We performed a retrospective chart review of all children with uveitis treated with TNF-alpha blockers at The Hospital for Sick Children, Toronto. RESULTS: Twenty-one children with uveitis were treated with the anti-TNF-alpha agents etanercept (11 patients) and infliximab (13 patients), resulting in 24 treatment courses. All patients had persistently active uveitis despite treatment with at least one standard immunosuppressive drug before the start of anti-TNF-alpha therapy. Six of 21 patients (29%) had idiopathic uveitis. In the other 15 patients, the underlying disease was juvenile idiopathic arthritis in 12 (57%), Behçet disease in two (9%) and sarcoidosis in one (5%). Response to etanercept treatment was good in 27%, moderate in 27% and poor in 45% of patients. Response to infliximab treatment was good in 38%, moderate in 54% and poor in 8% of patients. The difference in the percentage of patients with a moderate or good response was statistically significant (P = 0.0481). We also observed a lower rate of complications, such as new-onset or worsening glaucoma or cataract in the infliximab-treated group. CONCLUSION: Anti-TNF-alpha treatment was beneficial in a high percentage of patients with childhood uveitis refractory to standard immunosuppressive treatment. Infliximab resulted in better clinical responses with less ocular complications than etanercept.

Adolescent↗
PubMed2006

Predictors of disease course and remission in systemic juvenile idiopathic arthritis: significance of early clinical and laboratory features.

OBJECTIVE: To determine whether the disease course in systemic juvenile idiopathic arthritis (JIA) can be characterized as monophasic, polycyclic, or persistent, and to determine whether early clinical and laboratory characteristics can be used to predict the disease course and time to remission. METHODS: Forty-five children with systemic JIA diagnosed between 1996 and 2000 were followed up with a standardized data collection protocol, including data on clinical and laboratory features (mean followup 4.9 years). Disease was considered inactive if the clinical and laboratory features were normal. Three definitions of remission were applied to classify disease course. Predictors of disease course were evaluated using multiple logistic regression. Predictors of time to remission were evaluated using Cox proportional hazards regression. RESULTS: When applying a definition of remission requiring inactive disease while not receiving any medications for a period of 3 months, 42.2%, 6.7%, and 51.1% of the patients were classified as having monophasic, polycyclic, and persistent disease, respectively. Fever and active arthritis at 3 months (R2 = 0.42, area under the receiver operating characteristics curve [AUC] = 0.76) and an erythrocyte sedimentation rate (ESR) >26 mm/hour and corticosteroid use at 6 months (R2 = 0.49, AUC = 0.92) were predictive of a nonmonophasic course. Absence of active arthritis, an ESR of <26 mm/hour, and no requirement for corticosteroid therapy at 3 and 6 months were predictors of an earlier time to remission. CONCLUSION: The disease course in systemic JIA can be characterized as monophasic, polycyclic, or persistent using a definition of remission requiring 3 months of inactive disease while not receiving any therapy. Features at 3 and 6 months are predictive of the disease course and time to remission.

Adolescent↗
PubMed2006

Risk of new-onset uveitis in patients with juvenile idiopathic arthritis treated with anti-TNFalpha agents.

OBJECTIVE: To determine whether treatment with tumor necrosis factor alpha (TNFalpha)-blocking agents alters the incidence of new-onset uveitis in patients with juvenile idiopathic arthritis (JIA). STUDY DESIGN: Cohort study based on retrospective chart review. The charts of all 1109 patients with a diagnosis of JIA seen between January 1, 1996, and June 30, 2003, at our clinic were reviewed for diagnosis of uveitis and treatment with TNFalpha inhibitors. Cox regression analysis was performed with anti-TNFalpha treatment as a time-dependent covariate for risk of development of uveitis. RESULTS: We identified 70 patients treated with anti-TNFalpha without a prior diagnosis of uveitis. Two of these 70 patients (2.9%), both treated with etanercept, had development of new-onset uveitis during anti-TNFalpha therapy. One had juvenile psoriatic arthritis diagnosed 4.1 years before onset of uveitis. The other had extended oligoarticular JIA diagnosed 6.4 years before onset of uveitis. We found no statistically significant difference in the risk for development of uveitis between patients with or without anti-TNFalpha treatment. CONCLUSIONS: In our patients with JIA, anti-TNFalpha treatment did not alter the risk for development of new-onset uveitis. However, anti-TNFalpha therapy with etanercept did not prevent the development of uveitis in 2 patients.

Adolescent↗
PubMed2006

Recent developments in clinimetric instruments.

Assessment of impairment and function is essential in order to monitor joint status and evaluate therapeutic interventions in patients with haemophilia. The improvements in the treatment of haemophilia have required the development of more sensitive tools to detect the more minor dysfunctions that may now be apparent. This paper outlines some of the recent developments in this field. The Haemophilia Joint Health Score (HJHS) provides a systematic and robust measure of joint impairment. The MRI Scoring System has been designed to provide a comprehensive scoring system combining both progressive and additive scales. The Functional Independence Score for Haemophilia (FISH) has been developed to assess performance of functional activities and can be used in conjunction with the Haemophilia Activities List (HAL) which provides a self report measure of function. It is recommended that both measures are evaluated as these tools measure different constructs. Further refinement and testing of the psychometric properties of all of these tools is in progress. More widespread use of these tools will enable the sharing of data across the world so promoting best practice and ultimately enhancing patient care.

Activities of Daily Living↗
PubMed2006

Hemophilia joint health score reliability study.

UNLABELLED: Measurement of joint health is critically important when assessing children with haemophilia. Few measures exist; they lack sensitivity to small changes, don't account for normal development and were never formally validated. To address these concerns, the Hemophilia Joint Health Score (HJHS) was developed by modifying existing scores. OBJECTIVE: To test the inter-observer and test-retest reliability of the HJHS. METHODS: Using a fully factorial design, four physiotherapists (from Canada, the United States and Sweden) examined eight boys with severe haemophilia A on two consecutive days using the HJHS. The boys ranged in age from 4-12 years and presented with variable joint damage. Six index joints (elbows, knees and ankles) were assessed on 11 impairment items including swelling, flexion and extension loss and gait. Concordance was measured by the intra-class correlation co-efficient. RESULTS: Reliability of the HJHS was excellent with an inter-observer co-efficient of 0.83 and a test-retest of 0.89. CONCLUSION: This study is the first in a series to assess the psychometric properties of the HJHS, a promising new measure of joint health in boys with haemophilia.

Ankle Joint↗
PubMed2006

Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study.

BACKGROUND: Prophylactic treatment for severe hemophilia A is likely to be more effective than treatment when bleeding occurs, however, prophylaxis is costly. We studied an inception cohort of 25 boys using a tailored prophylaxis approach to see if clotting factor use could be reduced with acceptable outcomes. METHODS: Ten Canadian centers enrolled subjects in this 5-year study. Children were followed every 3 months at a comprehensive care hemophilia clinic. They were initially treated with once-weekly clotting factor; the frequency was escalated in a stepwise fashion if unacceptable bleeding occurred. Bleeding frequency, target joint development, physiotherapy and radiographic outcomes, as well as resource utilization, were determined prospectively. RESULTS: The median follow-up time was 4.1 years (total 96.9 person-years). The median time to escalate to twice-weekly therapy was 3.42 years (lower 95% confidence limit 2.05 years). Nine subjects developed target joints at a rate of 0.09 per person-year. There was an average of 1.2 joint bleeds per person-year. The cohort consumed on average 3656 IU kg(-1)year(-1) of factor (F) VIII. Ten subjects required central venous catheters (three while on study); no complications of these devices were seen. One subject developed a transient FVIII inhibitor. End-of-study joint examination scores--both clinically and radiographically--were normal or near-normal. CONCLUSIONS: Most boys with severe hemophilia A will probably have little bleeding and good joint function with tailored prophylaxis, while infusing less FVIII than usually required for traditional prophylaxis.

Canada↗
PubMed2005

Developing a disease activity tool for systemic-onset juvenile idiopathic arthritis by international consensus using the Delphi approach.

OBJECTIVES: The systemic form of juvenile idiopathic arthritis may present with many diverse symptoms, signs and laboratory abnormalities. Our aim was to elicit and pool items useful for developing a consensus disease activity measure for systemic arthritis in children, using an international pool of respondents. METHODS: We used a Delphi survey process in two steps. First we surveyed 187 paediatric rheumatologists and allied health professionals. We elicited 2607 items that, when combined with previously elicited items from parents/patients, could be pooled into 107 independent items. We then surveyed the paediatric rheumatologists to determine the frequency and importance of the 107 items. RESULTS: Our response rate was 83% to both surveys. We identified 29 items as being the most important and most frequently seen indicators of active disease. The most highly rated of these items were: presence of fever, presence of rash, elevated ESR, elevated CRP, requirement for increasing medications, abnormal physician global evaluation and presence of joints with active arthritis. CONCLUSIONS: Twenty-nine items are thought by medical practitioners to be most relevant in determining disease activity in systemic arthritis. As a next step, the measurement properties of these items will be tested to help develop a disease activity tool.

Algorithms↗
PubMed2005

A pilot study of ondansetron plus metopimazine vs. ondansetron monotherapy in children receiving highly emetogenic chemotherapy: a Bayesian randomized serial N-of-1 trials design.

GOALS OF WORK: Chemotherapy-induced nausea and vomiting is problematic in paediatric brain tumour treatment protocols which often discourage the use of corticosteroids as anti-emetics. The dopamine receptor antagonist, metopimazine, is an effective anti-emetic in combination with ondansetron in adults. The present study was designed to assess its efficacy in children with cancer, a group in which it has not been studied previously. PATIENTS AND METHODS: We conducted a series of randomized, multiple-crossover, double-blind, placebo-controlled N-of-1 trials comparing ondansetron/metopimazine with ondansetron monotherapy in children with brain tumours receiving highly emetogenic therapy and combined the individual results using Bayesian statistical modeling. MAIN RESULTS: Ten of twelve enrolled patients completed at least one chemotherapy cycle on study (median=2.5 cycles, range 1-11). Two patients were unable to complete any cycles, and a further three patients withdrew from the study prior to completing all cycles because of an inability to tolerate the taste of the study drug. Combination therapy increased the proportion of days during which patients had no emesis (overall odds ratio=1.52, 95% credible region=0.32-6.40, probability of odds ratio>1=72%), decreased the number of emetic episodes per day (overall rate ratio=0.67, 95% credible region=0.15-3.14, probability of rate ratio<1=75%) and decreased parents' ratings of their child's distress. The drug was more effective during the delayed chemotherapy phase than the acute phase. No adverse events were attributed to metopimazine. CONCLUSIONS: Based on this pilot study, we believe that the high likelihood that metopimazine is an effective adjunct to ondansetron monotherapy suggests that this combination therapy is worthy of further study in children receiving emetogenic chemotherapy.

Antiemetics↗
PubMed2005

What does "active disease" mean? patient and parent perceptions of disease activity in the systemic arthritis form of juvenile idiopathic arthritis (SO-JIA).

OBJECTIVE: The systemic onset form of juvenile idiopathic arthritis (SO-JIA) is a very serious chronic illness of childhood. At present, there is no specific tool to measure disease activity for SO-JIA. Our long-term goal is to develop a disease activity measure for SO-JIA using a consensus (Delphi) approach. In preparation for the development of this measure, we interviewed both patients and their parents. We sought to elicit specific items reflecting their perceptions of active disease that may be considered for inclusion in a disease activity measure for SO-JIA. METHODS: SO-JIA patients followed at The Hospital for Sick Children and their parents were chosen by purposive sampling. A trained research coordinator interviewed all participants using open-ended questions to elicit aspects of disease activity (defined as reversible manifestations of disease) of relevance to families. A list of these aspects was then generated and organized by categories to allow item reduction. RESULTS: Fourteen patients (eight females) with a mean age of 11.8 yr (mean disease duration 5.2 yr) and their parents were interviewed. A total of 292 items were generated, with an average of 11 items generated per interview. Arthralgia, ambulation difficulties, rash, decreased energy level and fever were the most common items mentioned by patients and their parents. Mood disturbances, decreased activity levels, arthritis severity and decreased school attendance were also common items identified as relevant aspects of disease activity. CONCLUSION: This study has allowed us to include patient and parent perspectives in preparation for developing a disease activity measure for SO-JIA. The resulting items will be added to future physician surveys in the further development and validation of a disease activity measure for SO-JIA.

Adolescent↗
PubMed2005

The effectiveness of treating juvenile dermatomyositis with methotrexate and aggressively tapered corticosteroids.

OBJECTIVE: Childhood dermatomyositis (DM) is often a chronic disease, lasting many years. It has traditionally been treated with long-term corticosteroid therapy; side effects are often seen. For more than a decade, methotrexate (MTX) has been safely used for the treatment of juvenile arthritis. Here, we report use of MTX as first-line therapy for DM, along with aggressively tapered corticosteroids, in an attempt to reduce treatment-related side effects. METHODS: We studied an inception cohort of 31 children with DM who were rigorously followed up in our myositis clinic, and compared them with a control group of 22 patients with incident cases of juvenile DM who received treatment just before we instituted a policy of first-line therapy with MTX. The mean starting dosage of MTX in the study group was 15 mg/m(2)/week. RESULTS: Both groups had similar improvement in strength and physical function; however, the median time during which patients in the study group received corticosteroids was 10 months, compared with 27 months for controls (P < 0.0001). As a result, the cumulative prednisone dose in the study group was approximately half that in the control group (7,574 mg versus 15,152 mg; P = 0.0006). The study group had greater height velocity during the first year of treatment and a smaller increase in the body mass index over the first 2 years. In the control group, the relative risk of cataracts developing was 1.95 (95% confidence interval 1.05-4.17). Side effects of MTX were rarely observed. CONCLUSION: Use of MTX in conjunction with an aggressively tapered course of prednisone may be as effective as traditional long-term corticosteroid therapy for children with DM, while decreasing the cumulative dose of corticosteroids.

Dermatomyositis↗
PubMed2005

Cost of severe haemophilia in Toronto.

Our objective was to determine costs and trends in treating boys with severe haemophilia A before our centre routinely used prophylaxis. One reviewer extracted data from patient charts to determine resource consumption for 17 boys with severe haemophilia A from 1978 to 1998 at Toronto's Hospital for Sick Children. Resources included factor concentrate, doctors and health care professionals (physiotherapists/social workers), tests (laboratory, radiological and diagnostic) and hospitalizations. Subgroup analysis on those patients infected with HIV and/or hepatitis were also performed. Costs in Canadian Dollars were taken from standard lists and discounted at 3%. Total average cost (range) 62 292 dollars (3339-121 738) per year patient(-1); the largest fraction, 59 910 dollars (3103-119 480), 96.2% was accounted for by factor VIII. Hospitalizations accounted for 1832 dollars (0-5217) per patient year(-1) including drugs, nursing care and stay. Doctor and health care professionals visits averaged 252 dollars (36-462) and 72 dollars (0-175) per patient year(-1), laboratory and other tests cost 201 dollars (22-377) and 26 dollars (2-60) per patient year(-1), respectively. The average number of bleeds was 12.9 (2.0-22.0) per patient year(-1), decreasing since 1977 by 0.68 per patient year(-1) (R(2) = 0.56). Hospitalizations averaged 0.22 (0-4) per patient year(-1), lasting 2.3 days. From 1984, hospitalizations decreased by 0.025 patient(-1) year(-1) (R(2) = 0.76). Concurrently, the average treatment costs increased by 5456 dollars patient(-1) year(-1) (R(2) = 0.81). Clotting factor concentrate cost per patient increased by 5521 dollars year(-1) (R(2) = 0.82). Patients with virally transmitted diseases had considerable higher costs. The cost per year was substantial. Costs increased with virally transmitted diseases. Number of bleeds and hospitalizations over the period of study decreased and costs increased because of factor use in secondary prophylaxis.

Adolescent↗
PubMed2004

Health-related quality of life (HRQL) scores reported from parents and their children with chronic illness differed depending on utility elicitation method.

OBJECTIVE: To describe the relationship between health-related quality of life (HRQL) as measured by utility when elicited from parents and their children with chronic illness. STUDY DESIGN AND SETTING: We enrolled families of children admitted for cancer chemotherapy and those attending outpatient rheumatology, hemophilia and bone marrow transplantation clinics. Children in grade 6 or higher were included. The child's HRQL was rated by parent and child using the Standard Gamble (SG), Visual Analogue Scale (VAS), Time Trade-Off (TTO), and Health Utilities Index Mark 2/3 (HUI2 and HUI3). RESULTS: 22 families were included. The mean parent SG was 0.92 +/- 0.09, which was similar to the mean SG elicited from their children of 0.92 +/- 0.10. The parent and child SG were moderately concordant (ICC=0.64, 95% CI=0.30, 0.83; P=.0005). In contrast, TTO scores were not concordant (ICC=0.14, 95% CI=-0.29, 0.53; P=.3), with parents (mean TTO=0.77 +/- 0.31) rating HRQL worse than children (mean TTO=0.92 +/- 0.11; P=.04). Similarly, the mean parent HUI2 of 0.82 +/- 0.22 was lower than the child HUI2 of 0.95 +/- 0.07; P=.02 and HUI2 were not concordant (ICC=0.11, 95% CI=-0.35, 0.53; P=.3) between parents and children. CONCLUSION: Parents and children rate HRQL similarly according to SG, but parents rate HRQL significantly worse using TTO and HUI2.

Adolescent↗
PubMed2004

Cost-utility analysis in evaluating prophylaxis in haemophilia.

Prophylaxis is an expensive form of management in haemophilia but has demonstrated many advantages with respect to decreasing joint bleeds and potentially preventing joint damage. The valuation of prophylaxis and how the costs and benefits of this intervention compare with other interventions in the management of haemophiliacs can be evaluated through cost-utility analysis (CUA). CUA is an economic method of analysis where the benefits of a healthcare intervention are expressed as an overall utility or preference, usually in the form of quality-adjusted life years (QALYs). This is a composite measure, which takes into consideration both an individual's lifespan and quality of life (QoL). The most difficult aspect of performing a CUA is the measurement of health-related QoL (HRQoL). Much work is ongoing into evaluating HRQoL in haemophiliacs. This paper addresses some of the ways in which this can be achieved and some of the problems with evaluating HRQoL. Ultimately CUA may provide a tool to allow societies to decide if prophylaxis is worth the cost and how the costs and benefits of prophylaxis compare to other healthcare interventions for other disease entities.

Child↗
PubMed2004

Physical therapy and imaging outcome measures in a haemophilia population treated with factor prophylaxis: current status and future directions.

Routine infusions of factor VIII to prevent bleeding, known as prophylaxis, and other intensive therapies are being more broadly applied to patients with haemophilia. These therapies differ widely in replacement product usage, cost, frequency of venous access and parental effort. In order to address residual issues relating to recommendations, implementation, and evaluations of prophylaxis therapy in persons with haemophila, a multinational working group was formed and called the International Prophylaxis Study Group (IPSG). The group was comprised of haemophilia treaters actively involved in studies of prophylaxis from North America and Europe. Two expert committees, the Physical Therapy (PT) Working Group and the Magnetic Resonance Imaging (MRI) Working Group were organized to critically assess existing tools for assessment of joint outcome. These two committees independently concluded that the WFH Physical Examination Scale (WFH PE Scale) and the WFH X-ray Scale (WFH XR Scale) were inadequately sensitive to detect early changes in joints. New scales were developed based on suggested modifications of the existing scales and called the Haemophilia Joint Health Score (HJHS) and the International MRI Scales. The new scales were piloted. Concordance was measured by the intra-class correlation coefficient of variation. Reliability of the HJHS was excellent with an inter-observer co-efficient of 0.83 and a test-retest value of 0.89. The MRI study was conducted using both Denver and European scoring approaches; inter-reader reliability using the two approaches was 0.88 and 0.87; test-retest reliability was 0.92 and 0.93. These new PT and MRI scales promise to improve outcome assessment in children on early preventive treatment regimens.

Hemophilia A↗