AIMS: To determine immunogenicity, pharmacokinetics (PK), efficacy, safety and quality of life of prophylaxis with a polyethylene glycol (peg)-ylated FVIII (BAX 855) based on full-length recombinant FVIII (ADVATE) in paediatric previously treated patients (PTPs) with severe haemophilia A.
METHODS: PTPs <12 years without history of FVIII inhibitors received twice-weekly infusions of 50 ± 10 IU kg(-1) BAX 855 for ≥50 exposure days. Prophylactic dose increases to ≤80 IU kg(-1) were allowed under predefined conditions. PK was evaluated after single infusions of 60 ± 5 IU kg(-1) .
RESULTS: T1/2 and mean residence time were extended 1.3- to 1.5-fold compared to ADVATE (n = 31), depending on the analysis used. The point estimate for the mean annualized bleeding rate in 66 subjects receiving a median of 1.9 weekly infusions of 51.3 IU kg(-1) of BAX 855 each was 3.04 (median 2.0); 1.10 (median 0) for joint and 1.16 (median 0) for spontaneous bleeds. Overall, 38% of subjects had zero bleeds. No bleeds were severe. Haemostatic efficacy was rated excellent or good for 90% of bleeds; 91% were treated with one or two infusions. In 8/14 subjects all target joints resolved. No subject developed FVIII inhibitors or persistent binding antibodies that affected safety or efficacy. No adverse reactions occurred.
CONCLUSION: Twice-weekly prophylaxis with BAX 855 was safe and efficacious in paediatric PTPs with severe haemophilia A.
METHOD: A cross sectional study was carried out where caretakers of cleft lip and/or palate were asked to complete the translated Malay language version of Strength Difficulties Questionnaire. The hearing status of the children was analyzed based on recent pure tone audiometric and tympanogram results. The patients' age, gender, type of cleft pathology, age of palatal surgery and behavioural patterns were examined for their potential relationship with hearing status.
RESULTS: A total of 74 children (148 ears) aged between 7 and 17 years with cleft lip and/or palate were recruited. The result showed 37 ears (25.0%) had hearing loss with majority suffered from mild conductive hearing loss. There were 16 ears (10.8%) that had persistent middle ear effusion. Hearing improvement occurred when palatal repair was performed at the age of less than 1 year old. (p = 0.015) There was no significant relationship between patients' gender, age, type of cleft and history of myringotomy with their hearing status. In terms of behavioural patterns, 16.3% were abnormal for total behavioural score, 39.2% for peer problem and 17.6% for conduct problem. For prosocial behaviour, 16.3% were rated low and very low. There was fair correlation between age and hyperactivity problems (r = 0.44). Patients' gender, type of cleft pathology, had been teased apart and hearing status was found not related to behavioural problems.
CONCLUSION: Cleft lip and/or palate patients have a good longterm hearing outcome. Majority had normal hearing and if there is hearing impairment, it is only a mild loss. Early palatal repair surgery before the age of 1 year can significantly reduce the risk of hearing loss. Cleft lip and/or palate patients experienced peer problems. There was no significant correlation between behavioural difficulty and hearing status among school-aged children with cleft lip and palate.
METHODS: This was a cross-sectional study. A total of 327 secondary school students completed the Alcohol Use Disorder Identification Test and Children's Depression Inventory, and a sociodemographic questionnaire. Binary and multivariable logistic regression were used to examine the relationship between the different variables.
RESULTS: A total of 42.2% of adolescents had PAU. Urban adolescents had a higher prevalence compared with rural adolescents (OR, 1.612; 95% CI: 1.036-2.508 P = 0.034). Female adolescents (56.8%) and Bumiputera Sarawak adolescents (76.8%) comprised a large proportion of the respondents with PAU. Cultural norm (78.3%) and curiosity (68.1%) were the two main reasons for PAU. Only family history of alcohol use (OR, 2.273; 95% CI: 1.013-5.107; P = 0.047), ever consumed alcohol (OR, 57.585; 95% CI: 21.885-151.525; P
RESULTS: Out of 97 patients screened, only 7 were carriers for the 3.7 deletion and all patients were negative for the 4.2 deletion. The 3.7 deletion was found in Foor, Hawsa and Rezagat Sudanese tribes. In the carriers of the 3.7 deletion, Red Blood Cells and Haematocrit were significantly increased. The Red Blood Cells were 7.23 ± 0.78 × 1012/L in adult males and 7.21 ± 0.67 × 1012/L in adult females while in children were 5.07 ± 0.87 × 1012/L. The mean cell volume and mean cell haemoglobin were significantly decreased, but the mean cell haemoglobin concentration slightly decreased. Haemoglobin levels didn't revealed statistically significant decrease in adult males (11.7 ± 0.57 g/dL) and adult females (11.25 ± 0.64 g/dL), while in children were (11.6 ± 2.95 g/dL). Haemoglobin electrophoresis revealed two patients of the 3.7 and 4.2 negative were carriers for β-thalassemia. The study concluded that α3.7 deletion has frequency of 0.07 in Sudanese with hypochromasia and microcytosis.
METHODS: A prospective 6 wk review of all pediatric (< 18 y) attendees to an urban ED was done, with patient age, presenting complaints, diagnoses, time of arrival and disposition recorded.
RESULTS: Complete data were available on 1172 patients, with an age range of 4 d to 18 y (mean +/- SD 6.9 +/- 5.6 y); 43% were aged < or = 4 y. The main presenting complaints were injuries (26.9%), fever (24%) and breathing difficulties (16.6%). The most common diagnosis was minor trauma (24.2%), with soft-tissue injuries predominating (80.6%). The other diagnoses were asthma (12.6%), upper respiratory infections (12.1%), other infections (12.1%) and gastroenteritis (11.8%). Equal proportions of patients were seen throughout the day. 25% of patients were admitted. Young age (< 1 y); presence of past medical history, general practitioner referrals, diagnosis of bronchiolitis and pneumonia were significantly associated with risk of admission.
CONCLUSION: A wide spectrum of paediatric illnesses was seen in the ED, with an overrepresentation of young children. This supports the decision to have either a separate pediatric ED or paediatric residents on the staff. The training curricula should emphasize the management of pediatric trauma, infections and asthma. Alternatively, developing guidelines for the five most common presenting complaints would account for 82% of all attendees and could be directed towards all staff on the ED.