Effect of intracranial bleeds on the neurocognitive, academic, behavioural and adaptive functioning of boys with haemophilia.

Brain insults are a risk factor for neuropsychological and academic deficits across several paediatric conditions. However, little is known about the specific effects of intracranial haemorrhage (ICH) in boys with haemophilia. The study compared neurocognitive, academic and socio-emotional/behavioural outcomes of boys with haemophilia with and without a history of ICH. Of 172 consecutive patients seen at a Pediatric Comprehensive Care Hemophila Centre, 18 had a history of ICH. Sixteen boys between the ages of 3 and 17 years were available for study and were matched to controls with haemophilia of the same age and disease severity and on the basis of maternal education. Groups were compared on neuropsychological and academic outcomes. Attention, socio-emotional function and executive skills were compared using data from parent questionnaires. Differences were found in intellectual function, visual-spatial skill, fine motor dexterity and particularly language-related skills, including vocabulary, word reading and applied math problem solving. Despite these group differences, outcomes were within the average range for most boys with ICH. No group differences were found in behavioural and socio-emotional functioning. Although ICH in haemophilia is not benign, it was not associated with significant cognitive and academic consequences for most boys. Early neuropsychological assessment may be indicated when there is a history of ICH. Investigation of age at ICH and quantitative measures of brain in relation to neurocognitive outcomes in larger groups of boys with ICH would be useful.

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.

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.

A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres.

A survey was conducted in 2002 to determine the pattern of factor prophylaxis use in boys or=45 weeks per year, was significantly higher for haemophilia A vs. haemophilia B cases (51% vs. 32%, P< 0.0001), and for boys with severe haemophilia A living in Canada vs. the USA (77% vs. 47%, P< 0.0001). Use of full-dose prophylaxis, defined as the infusion of 25-40 IU kg(-1) of factor VIII on alternate days (minimum three times per week) or 25-40 IU kg(-1) of factor IX twice weekly, was similar for boys