Primary ciliary dyskinesia (PCD) is an inherited autosomal-recessive disorder of motile cilia characterised by chronic lung disease, rhinosinusitis, hearing impairment and subfertility. Nasal symptoms and respiratory distress usually start soon after birth, and by adulthood bronchiectasis is invariable. Organ laterality defects, usually situs inversus, occur in ∼50% of cases. The estimated prevalence of PCD is up to ∼1 per 10 000 births, but it is more common in populations where consanguinity is common. This review examines who to refer for diagnostic testing. It describes the limitations surrounding diagnosis using currently available techniques and considers whether recent advances to genotype patients with PCD will lead to genetic testing and screening to aid diagnosis in the near future. It discusses the challenges of monitoring and treating respiratory and ENT disease in children with PCD.

Aim To describe longitudinal development of self‐care and its relationship to manual ability in children with cerebral palsy (CP) aged 18 months to 5 years across all functional abilities. Method This was a prospective longitudinal population‐based study of 290 children with CP (178 [61%] males, 112 [39%] females). Self‐care was assessed using the Pediatric Evaluation of Disability Inventory (PEDI). At 60 months (n=242), children were classified using the Manual Ability Classification System (MACS); 113 in level I (47%), 61 in MACS level II (25%), 24 in MACS level III (10%), 14 in MACS level IV (6%), and 30 in MACS level V (12%). Measures were taken at 18 months, 24 months, 30 months, 36 months, 48 months, and 60 months of age. Longitudinal analyses were performed using mixed‐effects linear regression models. Results Self‐care development achieved by 60 months was negatively associated with the severity of manual ability impairment. Distinct self‐care developmental trajectories were found with estimated changes in PEDI self‐care scaled scores per month: 0.61 for MACS level I, 0.46 for MACS levels II, 0.31 for MACS level III, 0.16 for MACS level IV, and 0.03 for MACS level V. Children classified in MACS level V had the lowest level of self‐care skills at 18 months and showed no progress in self‐care development. Interpretation This study reports rate of self‐care development in preschool children with CP. Self‐care performance was highest in children with greatest manual ability. Clinicians may use rates of change to predict or monitor self‐care performance. PEDI trajectories inform goal setting in discussions with families regarding expected levels of independence in self‐care. What this paper adds Distinct self‐care developmental trajectories in children with cerebral palsy were found according to Manual Ability Classification System (MACS) levels. Children in MACS levels IV and V with epilepsy did not show any significant change in self‐care. Children in MACS levels IV and V without epilepsy demonstrated small yet significant gains in self‐care performance.

The somatostatin analogs (SSAs) lanreotide Autogel/Depot and octreotide long-acting release are used to treat acromegaly and neuroendocrine tumors. The present study evaluated opinions on SSA injection devices, including a recently approved lanreotide new device (lanreotide-ND), among nurses in Europe and the USA. Nurses injecting SSAs for at least three patients per year (n = 77) were interviewed regarding SSA devices. Device attributes were rated via questionnaire; nurses were then timed administering test injections with lanreotide-ND and octreotide long-acting release. The most important delivery system attributes were easy/convenient preparation and injection (ranked in the top five by 70% of nurses), low clogging risk (58%), and high product efficacy (55%). Compared with the octreotide long-acting release device, lanreotide-ND scored higher on 15/16 attributes, had shorter mean preparation and administration time (329 versus 66 seconds, respectively; P ≤ 0.01) and a higher overall preference score (70 versus 114, respectively; P ≤ 0.01). The five most important lanreotide-ND attributes were: prefilled device, confidence a full dose was delivered, low clogging risk, easy/convenient preparation and injection, and fast administration. These device features could lead to improvements in clinical practice and benefit patients/caregivers who administer SSAs at home.

Tonsillectomy is one of the most frequently performed surgical procedures; however, pain management remains challenging. Procedure-specific efficacy as well as specific risks of treatment options should guide selection of pain management protocols based on evidence and should optimise analgesia without harm. The aims of this systematic review were to evaluate the available literature and develop recommendations for optimal pain management after tonsillectomy. A systematic review utilising preferred reporting items for systematic reviews and meta-analysis guidelines with procedure-specific postoperative pain management (PROSPECT) methodology was undertaken. Randomised controlled trials published in the English language up to November 2019 assessing postoperative pain using analgesic, anaesthetic or surgical interventions were identified. Out of the 719 potentially eligible studies identified, 226 randomised controlled trials met the inclusion criteria, excluding the studies examining surgical techniques. Pre-operative and intra-operative interventions that improved postoperative pain were paracetamol; non-steroidal anti-inflammatory drugs; intravenous dexamethasone; ketamine (only assessed in children); gabapentinoids; dexmedetomidine; honey; and acupuncture. Inconsistent evidence was found for local anaesthetic infiltration; antibiotics; and magnesium sulphate. Limited evidence was found for clonidine. The analgesic regimen for tonsillectomy should include paracetamol; non-steroidal anti-inflammatory drugs; and intravenous dexamethasone, with opioids as rescue analgesics. Analgesic adjuncts such as intra-operative and postoperative acupuncture as well as postoperative honey are also recommended. Ketamine (only for children); dexmedetomidine; or gabapentinoids may be considered when some of the first-line analgesics are contra-indicated. Further randomised controlled trials are required to define risk and combination of drugs most effective for postoperative pain relief after tonsillectomy.

ObjectivesCerebral palsy (CP) remains the world’s most common childhood physical disability with total annual costs of care and lost well-being of $A3.87b. The PREDICT-CP (NHMRC 1077257 Partnership Project: Comprehensive surveillance to PREDICT outcomes for school age children with CP) study will investigate the influence of brain structure, body composition, dietary intake, oropharyngeal function, habitual physical activity, musculoskeletal development (hip status, bone health) and muscle performance on motor attainment, cognition, executive function, communication, participation, quality of life and related health resource use costs. The PREDICT-CP cohort provides further follow-up at 8–12 years of two overlapping preschool-age cohorts examined from 1.5 to 5 years (NHMRC 465128 motor and brain development; NHMRC 569605 growth, nutrition and physical activity).Methods and analysesThis population-based cohort study undertakes state-wide surveillance of 245 children with CP born in Queensland (birth years 2006–2009). Children will be classified for Gross Motor Function Classification System; Manual Ability Classification System, Communication Function Classification System and Eating and Drinking Ability Classification System. Outcomes include gross motor function, musculoskeletal development (hip displacement, spasticity, muscle contracture), upper limb function, communication difficulties, oropharyngeal dysphagia, dietary intake and body composition, participation, parent-reported and child-reported quality of life and medical and allied health resource use. These detailed phenotypical data will be compared with brain macrostructure and microstructure using 3 Tesla MRI (3T MRI). Relationships between brain lesion severity and outcomes will be analysed using multilevel mixed-effects models.Ethics and disseminationThe PREDICT-CP protocol is a prospectively registered and ethically accepted study protocol. The study combines data at 1.5–5 then 8–12 years of direct clinical assessment to enable prediction of outcomes and healthcare needs essential for tailoring interventions (eg, rehabilitation, orthopaedic surgery and nutritional supplements) and the projected healthcare utilisation.Trial registration numberACTRN: 12616001488493