OBJECTIVES: To review and assess the efficacy of currently available treatment options for preventing and managing advanced liver disease in children and adults with cystic fibrosis.
SEARCH METHODS: We searched the Cochrane Cystic Fibrosis Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. Date of last search: 19 November 2019. We also searched the reference lists of relevant articles and reviews and online trials registries. Date of last search: 01 January 2020.
SELECTION CRITERIA: Any published and unpublished randomised controlled trials and quasi-randomised controlled trials of advanced liver disease in cystic fibrosis with cirrhosis or liver failure, portal hypertension or variceal bleeding (or both).
DATA COLLECTION AND ANALYSIS: Authors independently examined titles and abstracts to identify potentially relevant trials, but none were eligible for inclusion in this review.
MAIN RESULTS: A comprehensive search of the literature did not identify any published eligible randomised controlled trials.
AUTHORS' CONCLUSIONS: In order to develop the best source of evidence, there is a need to undertake randomised controlled trials of interventions for preventing and managing advanced liver disease in adults and children with cystic fibrosis.
METHODS: This retrospective population-based analysis estimated crude and standardized incidences of VLD and NLD in twelve hospitals in Brazil (n = 3), Mexico (n = 3), and Malaysia (n = 6) over a 1-year period before the introduction of the tetravalent dengue vaccine. Catchment areas were estimated using publicly available population census information and administrative data. The denominator population for incidence rates was calculated, and sensitivity analyses assessed the impact of important assumptions.
RESULTS: Total cases adjudicated as definite VLD were 5, 57, and 56 in Brazil, Mexico, and Malaysia, respectively. Total cases adjudicated as definite NLD were 103, 29, and 26 in Brazil, Mexico, and Malaysia, respectively. Crude incidence rates of cases adjudicated as definite VLD in Brazil, Mexico, and Malaysia were 1.17, 2.60, and 1.48 per 100,000 person-years, respectively. Crude incidence rates of cases adjudicated as definite NLD in Brazil, Mexico, and Malaysia were 4.45, 1.32, and 0.69 per 100,000 person-years, respectively.
CONCLUSIONS: Background incidence estimates of VLD and NLD obtained in Mexico, Brazil, and Malaysia could provide context for cases occurring after the introduction of the tetravalent dengue vaccine.
SUMMARY OF BACKGROUND DATA: A previous systematic review identified 115 outcomes in 60 trials and systematic reviews evaluating treatments for children with appendicitis, suggesting the need for a COS.
METHODS: The development process consisted of 4 phases: (1) an updated systematic review identifying all previously reported outcomes, (2) a 2-stage international Delphi study in which parents with their children and surgeons rated these outcomes for inclusion in the COS, (3) focus groups with young people to identify missing outcomes, and (4) international expert meetings to ratify the final COS.
RESULTS: The systematic review identified 129 outcomes which were mapped to 43 unique outcome terms for the Delphi survey. The first-round included 137 parents (8 countries) and 245 surgeons (10 countries), the second-round response rates were 61% and 85% respectively, with 10 outcomes emerging with consensus. After 2 young peoples' focus groups, 2 additional outcomes were added to the final COS (12): mortality, bowel obstruction, intraabdominal abscess, recurrent appendicitis, complicated appendicitis, return to baseline health, readmission, reoperation, unplanned appendectomy, adverse events related to treatment, major and minor complications.
CONCLUSION: An evidence-informed COS based on international consensus, including patients and parents has been developed. This COS is recommended for all future studies evaluating treatment ofsimple appendicitis in children, to reduce heterogeneity between studies and facilitate data synthesis and evidence-based decision-making.
AIM: These post hoc analyses investigated the bleeding patterns in target joints.
METHODS: Patients randomized to 40 or 10 IU kg(-1) once weekly prophylaxis who had at least one target joint were included. Baseline demographics and disease-specific data were collected. Bleeding patterns were assessed, and an International Society on Thrombosis and Haemostasis (ISTH) definition of target joints was used.
RESULTS: A total of 67% and 8% of patients in the 40 and 10 IU kg(-1) arm, respectively, did not experience target joint bleeds during the paradigm(™) 2 trial. Twenty-four target joints were recorded in each prophylaxis arm at baseline. During the paradigm(™) 2 trial, no bleeds were reported in 17 (71%) and 7 (29%) target joints in the 40 and 10 IU kg(-1) arms respectively. All target joint bleeds in the 40 IU kg(-1) once weekly prophylaxis arm were controlled with a single injection of 40 IU kg(-1) nonacog beta pegol. By the latest ISTH definition, 90% and 58% of target joints in the 40 and 10 IU kg(-1) arms, respectively, were no longer considered target joints at the end of the paradigm(™) 2 trial. At the end of the paradigm(™) 4 extension trial, all target joints in the 40 IU kg(-1) arm were no longer considered target joints.
CONCLUSION: Routine prophylaxis with 40 IU kg(-1) once weekly nonacog beta pegol has the potential for effective management of target joint bleeds in haemophilia B patients.
DESIGN: Death-related data were retrospectively and prospectively assessed in a longitudinal regional cohort study.
METHODS: Children under routine HIV care at sites in Cambodia, India, Indonesia, Malaysia, Thailand, and Vietnam between 2008 and 2017 were followed. Causes of death were reported and then independently and centrally reviewed. Predictors were compared using competing risks survival regression analyses.
RESULTS: Among 5918 children, 5523 (93%; 52% male) had ever been on combination antiretroviral therapy. Of 371 (6.3%) deaths, 312 (84%) occurred in those with a history of combination antiretroviral therapy (crude all-cause mortality 9.6 per 1000 person-years; total follow-up time 32 361 person-years). In this group, median age at death was 7.0 (2.9-13) years; median CD4 cell count was 73 (16-325) cells/μl. The most common underlying causes of death were pneumonia due to unspecified pathogens (17%), tuberculosis (16%), sepsis (8.0%), and AIDS (6.7%); 12% of causes were unknown. These clinical diagnoses were further grouped into AIDS-related infections (22%) and noninfections (5.8%), and non-AIDS-related infections (47%) and noninfections (11%); with 12% unknown, 2.2% not reviewed. Higher CD4 cell count and better weight-for-age z-score were protective against death.
CONCLUSION: Our standardized cause of death assessment provides robust data to inform regional resource allocation for pediatric diagnostic evaluations and prioritization of clinical interventions, and highlight the continued importance of opportunistic and nonopportunistic infections as causes of death in our cohort.
PATIENTS AND METHODS: Adult men with hypospadias repair in childhood were recruited (n = 55; aged 19.9 [IQR 19.2-22.1]). Coping styles were determined with the Utrecht Coping List (UCL) and results compared with a reference group of male students (n = 55, age 20-30 years, no medical history). Sub analysis of coping styles of the hypospadias groups was done based on three items: severity of hypospadias, time of last hypospadias surgery and occurrence of postoperative complications.
RESULTS: Compared to the reference groups, patients with hypospadias had higher scores on Avoidance (P
DATA SOURCES: A systematic search using relevant keywords was conducted in PubMed-Medline, EMBASE, PsycINFO, CINAHL, Scopus, and The Cochrane Library.
INCLUSION CRITERIA: Randomized controlled trials (RCTs) that compared technology-based interventions with inactive controls in the treatment of moderate to severe dental anxiety were included.
RESULTS: A total of seven RCTs were included in the review. These studies investigated the effectiveness of video modeling, computerized cognitive behavioral therapy, virtual reality exposure therapy, and distraction with music and audiovisual video material. Six studies examining video modeling, computerized cognitive behavioral therapy, virtual reality exposure therapy, and distraction (audiovisual) showed significantly greater reductions in dental anxiety than inactive controls in both children and adults. None of the included studies followed Consolidated Standards of Reporting Trials guidelines completely or reported sufficient data, thereby precluding a possible meta-analysis. Four out of seven included studies were assessed to be at high risk of bias.
CONCLUSIONS: A limited number of studies supported the effectiveness of technology-based interventions in the treatment of dental anxiety in children and adults.
CLINICAL SIGNIFICANCE: The quality of the methods of studies on the effects of technology-based interventions allows only limited inferences on the effects of these interventions. However, within the limitations of the systematic review, the results converge to suggest that technology-based interventions may be useful as an adjunct to standard dental care. High-quality RCTs are needed to determine the (relative) effectiveness of these interventions.
PROSPERO REGISTRATION NUMBER: CRD42017064810.
METHODS: This cross-sectional study in a tertiary hospital in Kuala Lumpur, Malaysia involved parents of children with asthma. Parents of children without asthma were the control group. Eleven validated video clips showing wheeze, stridor, transmitted noises, snoring or normal breathing were shown to the parents. Parents were asked, in English or Malay, "What do you call the sound this child is making?" and "Where do you think the sound is coming from?"
RESULTS: Two hundred parents participated in this study: 100 had children with asthma while 100 did not. Most (71.5 %) answered in Malay. Only 38.5 % of parents correctly labelled wheeze. Parents were significantly better at locating than labelling wheeze (OR 2.4, 95 % CI 1.64-3.73). Parents with asthmatic children were not better at labelling wheeze than those without asthma (OR1.04, 95 % CI 0.59-1.84). Answering in English (OR 3.4, 95 % CI 1.69-7.14) and having older children with asthma (OR 9.09, 95 % CI 3.13-26.32) were associated with correct labelling of wheeze. Other sounds were mislabelled as wheeze by 16.5 % of respondents.
CONCLUSION: Parental labelling of wheeze was inaccurate especially in the Malay language. Parents were better at identifying the origin of wheeze rather than labelling it. Physicians should be wary about parental reporting of wheeze as it may be inaccurate.
OBJECTIVES: To determine the HRQoL and developmental outcome of children on HMV.
METHODS: This cross-sectional study used the TNO-AZL Preschool children's Quality Of Life (TAPQOL; <5 years old) and Health Utilities Index (HUI) 2/3 (≥5 years old) to assess the quality of life and the Schedule of Growing Skills-II to assess development. Instruments were used on children currently or previously on HMV (≥3 months) and compared with age and sex-matched controls.
RESULTS: Sixty-five patients and 130 controls were recruited. Patients' median (interquartile range) age was 3.12 (1.65, 5.81) years. Patients had significantly lower TAPQOL scores in the domains of lung, liveliness, positive mood, social functioning, motor functioning, and communication, and lower HUI 2/3 scores in hearing, sensation, pain, speech, mobility, ambulatory, dexterity, and self-care domains. The developmental outcome of patients was poorer in all domains. However, patients had fewer behavioral problems. Those with respiratory tract disease and without comorbidities had better HRQoL and developmental scores. Having a parent as the primary caregiver was associated with better speech and language skills.
CONCLUSIONS: HRQoL and the developmental outcome are lower in children on HMV compared to controls. Children with respiratory tract disease and without comorbidities have a better outcome. Parents play a crucial role in the acquisition of speech.
METHODOLOGY: This prospective cohort study involved children 1 month to 5-years-old admitted with an LRTI. Children with asthma were excluded. Patients were reviewed at 1-, 6-, and 12-months post-hospital discharge. The parent cough-specific quality of life, the depression, anxiety, and stress scale questionnaire and cough diary for 1 month, were administered. Outcomes reviewed were number of unscheduled healthcare visits, respiratory symptoms and final respiratory diagnosis at 6 and/or 12 month-review by pediatric pulmonologists.
RESULTS: Three hundred patients with a mean ± SD age of 14 ± 15 months old were recruited. After 1 month, 239 (79.7%) returned: 28.5% (n = 68/239) had sought medical advice and 18% (n = 43/239) had cough at clinic review. Children who received antibiotics in hospital had significantly lower total cough scores (P = .005) as per the cough diary. After 1 year, 26% (n = 78/300) had a respiratory problem, predominantly preschool wheezing phenotype (n = 64/78, 82.1%). Three children had bronchiectasis or bronchiolitis obliterans. The parent cough-specific quality of life (PCQOL) was significantly lower in children with respiratory sequelae (P