Health-related out-of-pocket expenses for children living with rare diseases - tuberous sclerosis and mitochondrial disorders: A prospective pilot study in Australian families.

AIM: We aimed to describe health-related out-of-pocket (OOP) expenses incurred by Australian families living with children with chronic and complex diseases. METHODS: A prospective pilot study of OOP expenses in families with children with tuberous sclerosis (TS) or mitochondrial disorders (MD) in 2016-2017. An initial survey assessed the family's financial situation, child's health functioning and estimated previous 6 months' and lifetime OOP expenses. Thereafter, families completed a survey each month for 6 months, prospectively tracking OOP expenses. RESULTS: Initial surveys were completed by 13 families with 15 children; median age 7 years (range: 1-12); 5 with MD, 10 with TS. All families reported OOP expenses: 38% paid $2000 per annum, more than double the annual per-capita OOP costs reported for Australia by the Organisation for Economic Co-operation and Development. Eight families estimated $5000-$25 000 in OOP expenses over their child's lifetime and 62% of mothers reduced or stopped work due to caring responsibilities. Eleven families paid annual private health insurance premiums of $2000-$5122, but 72% said this was poor value-for-money. Prospective tracking by eight families (9 children) identified the median OOP expenditure was $863 (range $55-$1398) per family for 6 months. OOP spending was associated with visits to allied health professionals, non-prescription medicines, special foods, supplements and disposable items. Eight families paid for 91 prescription medications over 6 months. CONCLUSION: All families caring for children with TS or MD reported OOP expenses. A larger study is needed to explore the affordability of health care for children living with a broader range of chronic diseases.

Ten Years of National Seasonal Surveillance for Severe Complications of Influenza in Australian Children.

BACKGROUND: Severe complications of influenza in children are uncommon but may result in admission to hospital or an intensive care unit (ICU) and death. METHODS: Active prospective surveillance using the Australian Paediatric Surveillance Unit with monthly reporting by pediatricians of national demographic and clinical data on children with <15 years of age hospitalized with severe complications of laboratory-confirmed influenza during ten influenza seasons 2008-2017. RESULTS: Of 722 children notified, 613 had laboratory-confirmed influenza and at least one severe complication. Most (60%) were <5 years of age; 10% were <6 months, hence ineligible for vaccination. Almost half of all cases were admitted to ICU and 30 died. Most children were previously healthy: 40.3% had at least one underlying medical condition. Sixty-five different severe complications were reported; pneumonia was the most common, occurring in over half of all cases. Influenza A accounted for 68.6% hospitalizations; however, influenza B was more often associated with acute renal failure (P = 0.014), rhabdomyolysis (P = 0.019), myocarditis (P = 0.015), pericarditis (P = 0.013), and cardiomyopathy (P = 0.035). Children who died were more likely to be older (5-14 years), have underlying medical conditions, be admitted to ICU, and have encephalitis, acute renal failure, or myocarditis. Only 36.1% of all children reported received antiviral medications, and 8.5% were known to be vaccinated for seasonal influenza. CONCLUSIONS: Severe influenza complications cause morbidity and mortality in children, which may increase if coinfection with COVID-19 occurs in the 2020 season and beyond. Increased vaccination rates, even in healthy children, early diagnosis and timely antiviral treatment are needed to reduce severe complications and death.

Implementation of e-mental health for depression and anxiety: A critical scoping review.

The aim of this review was to scope the growth and development of implementation research of e-mental healthcare programs for anxiety and depression, the research and evaluation tools used, and the specific implementation processes and outcomes examined. A search of four electronic databases (MEDLINE, EMBASE, PsycINFO, and CINAHL) was conducted from January 2000 to January 2019. Of 33 studies identified, most (n = 28) were published in the last five years. Only 10 used an implementation framework to guide implementation or evaluation. Most studies reported on acceptability (n = 28), appropriateness (n = 23), and feasibility (n = 17). Less commonly reported implementation outcomes were fidelity (n = 10) and adoption (n = 7); with penetration (n = 4), sustainability (n = 3), and implementation cost (n = 2) being studied rarely. Of the 21 studies that used surveys to study implementation outcomes, less than half used a previously published survey (n = 9). More rigorous implementation studies, underpinned by strong theory and real-world understanding, are urgently needed.

Paediatric Active Enhanced Disease Surveillance inaugural annual report, 2014.

INTRODUCTION: The Paediatric Active Enhanced Disease Surveillance (PAEDS) network is a hospital-based active surveillance system employing prospective case ascertainment of selected uncommon vaccine preventable diseases and potential adverse events following immunisation (AEFI). PAEDS enhances other Australian surveillance systems by providing prospective detailed clinical and laboratory data for the same child. METHODS: Specialist surveillance nurses screen hospital admissions, emergency department records, laboratory and other data, to prospectively identify hospitalised children aged under 15 years in 5 paediatric tertiary referral hospitals in New South Wales, Victoria, South Australia, Western Australia and Queensland. Standardised protocols and case definitions are used across all sites. Conditions under surveillance include vaccine preventable diseases: acute flaccid paralysis, varicella, pandemic and seasonal influenza and pertussis, and potential AEFIs: febrile seizures and intussusception. PAEDS also conducts surveillance for acute childhood encephalitis. RESULTS: Since August 2007, PAEDS has recruited a total of 6,227 hospitalised cases in total, for all conditions. From January to December 2014, there were 1,220 cases recruited across all conditions. Key outcomes include: enhanced acute flaccid paralysis surveillance to reach World Health Organization targets; supporting varicella and influenza vaccination in children; confirmation of a known low risk of febrile seizures following the 1st dose of measles-mumps-rubella vaccine but no increased risk of febrile seizures after measles-mumps-rubella-varicella vaccine, and a slightly increased risk of developing intussusception 1-7 days after rotavirus vaccination in infants aged less than 3 months. Acute childhood encephalitis data facilitated rapid investigation and response to the enterovirus 71 outbreak in 2013-2014. CONCLUSIONS: PAEDS provides unique policy-relevant data. This is the first of planned PAEDS annual reports to Communicable Diseases Intelligence.

Australian Paediatric Surveillance Unit annual report, 2013.

This report provides an update on the surveillance conducted by the Australian Paediatric Surveillance Unit (APSU) during the period January to December 2013. The APSU facilitates national active surveillance of uncommon diseases of childhood including selected communicable diseases. This report includes data on the following conditions: acute flaccid paralysis (AFP), congenital cytomegalovirus (cCMV), congenital rubella, perinatal exposure to HIV and paediatric HIV infection, neonatal herpes simplex virus (HSV), congenital varicella, neonatal varicella, severe complications of varicella and juvenile onset recurrent respiratory papillomatosis (JoRRP). Surveillance of severe complications of influenza was undertaken during the influenza season (July to September 2013).

Influenza infection in infants aged <6 months during the H1N1-09 pandemic: a hospital-based case series.

AIMS: To document risk factors, clinical features and outcomes in infants <6 months old admitted with laboratory-confirmed influenza to The Children's Hospital at Westmead during the H1N1-09 pandemic. METHODS: Prospective, hospital-based case series of infants admitted June-September 2009, identified by the Paediatric Active Enhanced Disease Surveillance system and supplemented by telephone interview post-discharge. RESULTS: Thirty-two infants <6 months old had influenza A: 18 H1N1-09, 11 H3N2 and three unknown subtypes. After discharge, 28 (88%) families were telephoned and provided additional information. Documented risk factors included close contact with young children (46%), living with a smoker (36%), intensive or special care at birth (25%), pre-existing illness (16%) and preterm birth (14%). The number of persons per household was double the state average. Only 14% of mothers were vaccinated against seasonal influenza. Infants commonly presented with cough (69%), coryza (69%), lethargy (38%), fever (31%), dyspnoea (31%) and vomiting (28%). Complications included pneumonia (22%), and bacterial (9%) and viral (6%) co-infection. Five infants (15%) required admission to intensive care, and one was mechanically ventilated. Sixteen (57%) had ongoing respiratory problems, and six (21%) presented to the Emergency Department within 6 months of discharge. CONCLUSIONS: These novel data are clinically important. Rates of influenza in infants may be reduced by vaccinating close contacts and minimising exposure to infected contacts and cigarette smoke.

Australian families living with rare disease: experiences of diagnosis, health services use and needs for psychosocial support.

BACKGROUND: Families of children living with a rare disease report significant health and social burden, however, few studies have systematically examined family needs by using validated tools to assess the scope and extent of this burden. Our aim was to develop a comprehensive survey to assess health, psychosocial and financial impacts on Australian families caring for a child with a rare disease. METHODS: We developed a self-administered survey for parents/carers incorporating pre-validated tools. The survey included questions about experiences of diagnosis, health services use and needs, needs for peer and financial supports. Forty-seven families attending the state-wide Genetic Metabolic Disorders Service at the Children's Hospital at Westmead, Sydney were invited to participate. RESULTS: Of 46 families who received the survey, 30 (65%) completed it. Most (93%) found the survey acceptable and relevant (91%). Patients were 1-17 years old, 14 (47%) male, and 12 (40%) non-Caucasian. Eighteen (60%) had a lysosomal storage disease and 12(40%) had a mitochondrial disorder. Eleven (38%) saw 3-5 doctors and four (14%) saw 6-10 doctors before receiving the correct diagnosis; 43% felt diagnosis was delayed. Four (13%) were dissatisfied with the way diagnosis was given, due to insensitive style of communication, inadequate information and psychological support. Psychosocial impact was moderate to high for 90% of families and the level of impact was not dependent on the level of health functioning of the child. Twenty-six (87%) wanted, but only 13(43%) received, information about peer-support groups. The 30 children accounted for 168 visits to general practitioners and 260 visits to specialist doctors; 21 (70%) children had at least one admission to hospital, including one who had 16 admissions in the previous 12 months. Most families (77%) received financial assistance but 52% believed this was insufficient. Families benefited from a specialised multi-disciplinary clinic but called for patient-held electronic medical records. CONCLUSIONS: Australian families caring for children with genetic metabolic disorders are adversely impacted by delays in diagnosis, lack of easy access to peer support groups and lack of psychological support. Further research is needed to estimate economic impact and to analyse health service delivery models for children with rare diseases in Australia.

A national prospective surveillance study of acute rheumatic fever in Australian children.

BACKGROUND: Acute rheumatic fever (ARF) is an important cause of heart disease in Indigenous people of northern and central Australia. However, little is known about ARF in children across all Australian population groups. This national prospective study was conducted to determine patterns of disease, and populations and regions at highest risk. METHODS: The Australian Paediatric Surveillance Unit surveillance model was used to collect data on children with ARF across Australia. Children up to 15 years of age were included if they had an ARF episode diagnosed between October 1, 2007 and December 31, 2010 that met the case definition. RESULTS: ARF was identified in 151 children: 131 Indigenous Australians, 10 non-Indigenous Australians, 8 Pacific Islanders and 1 African (1 unknown). Common presenting features were joint symptoms, fever and carditis. Sydenham chorea was reported in 19% of children. Aseptic monoarthritis was a major manifestation in 19% of high-risk children. Seven non-Indigenous Australian children presented with classic, highly specific features compared with 23% of high-risk children, suggesting that subtle presentations of ARF are being missed in non-Indigenous children. Recent sore throat was reported in 33% of cases, including 25% of remote Indigenous children. There were delays in presentation to care and referral to higher-level care across urban/rural and remote areas. CONCLUSIONS: ARF may be more common than previously thought among low-risk children. These data should prompt an awareness of ARF diagnosis and management across all regions, including strategies for primary prevention. There should be renewed emphasis on treatment of sore throat in high-risk groups.

Novel inpatient surveillance in tertiary paediatric hospitals in New South Wales illustrates impact of first-wave pandemic influenza A H1N1 (2009) and informs future health service planning.

AIM: To document the impact of pandemic influenza A H1N1 (2009) in New South Wales (NSW) children's hospitals. METHODS: A novel surveillance system, Paediatric Active Enhanced Disease Surveillance (PAEDS), identified hospitalised children <15 years with laboratory-proven influenza (1 June-30 September 2009) in the three children's hospitals in NSW: Children's Hospital at Westmead (CHW), Sydney Children's Hospital, John Hunter Children's Hospital. Clinical characteristics, management and complications were documented, and at CHW comparison made with 2007 data. RESULTS: The 324 children identified represented 1802 hospital bed-days and 230 PICU bed-days. Most (73.1%) children had H1N1, one had an oseltamivir-resistant isolate. Median age was 2.5 years: 65% were <5 years. Although 80.9% had cough, 8.0% had no respiratory symptoms. Complications occurred in 34.6%, of whom 56% were previously healthy. Only 50% received antivirals. Forty children (12.3%) were admitted to PICU: one child with H1N1 died. At CHW, comparison between 2009 and 2007 showed nearly twice the total number of admissions (226 vs. 122) and PICU admissions (22 vs. 13), but no deaths either year. Vomiting was more frequent in 2009 than 2007 (38.5% vs. 13.1%; P = 0.0001) as were neurological complications (11.4% vs. 2.4%; P = 0.0027) but length of hospital and PICU stay were similar. CONCLUSIONS: PAEDS is a valuable surveillance tool that documented the impact of the H1N1 (2009) pandemic in NSW children's hospitals. High numbers of complications, often in previously well children, suggest an important role for early diagnosis, antiviral therapy and influenza vaccination. Observed regional differences identify areas potentially at greater risk in a subsequent wave.

What do paediatricians think of the Australian Paediatric Surveillance Unit?

AIM: To explore clinicians' perceptions of the value, usefulness and limitations of the Australian Paediatric Surveillance Unit (APSU) and obtain direct feedback regarding the surveillance mechanism and suggestions for improvement. METHODS: Anonymous postal survey of Australian paediatricians (n = 1260) in 2007. RESULTS: Of 1260 clinicians surveyed 818 (65%) responded, a similar proportion from all states/territories and specialties. Over half had participated in surveillance for >10 years. The majority (95%), believe APSU research is valuable, for generating knowledge (81%), identifying research needs (78%), facilitating collaborative research (75%), supporting education and advocacy (74%), guiding clinical practice (70%), informing future policy (70%) and evaluating current policy (68%). Of 458 respondents who had ever reported a case (90%) had no objection to providing de-identified clinical information; and about 75% said questionnaires were easy to complete; however, one third said clinical information requested was not always readily available. Most (680, 83%) respondents believed their contribution to the APSU was appropriately acknowledged and 20% said they had personally benefited from participation. The majority (90%) were willing to report immediately by email/fax/phone in an epidemiological emergency. Lack of time and resources was the most common limitation to participation identified by clinicians: some suggested on-line reporting would improve the ease and timeliness of reporting. Clinicians also suggested better use of the APSU website to disseminate results. CONCLUSION: Clinicians acknowledged the APSU as valuable. Improving communication with clinicians, ensuring that information requested in questionnaires is relevant and available, and developing a secure web-based reporting system are future APSU priorities.

The burden of childhood influenza in a tertiary paediatric setting.

Influenza is usually considered a mild winter-time illness but can be associated with a range of serious complications. We undertook a retrospective medical record review to study the impact of admissions of children with laboratory-confirmed influenza to The Children's Hospital at Westmead, Sydney, during 2007. One hundred and twenty-two children were identified, representing 530 hospital admission days. There was no clearly documented evidence of influenza vaccination for any patient eligible for vaccination. Fever (97.5%) and cough (69.7%) were the most frequent manifestations. Admissions occurred almost entirely between June and September with a peak in July (n=61, 50%). Two-thirds of the children were aged less than 2 years (median 1.5 years). Most (61.5%) had an underlying chronic medical disorder. Lumbar puncture was performed in 28 (23%) children, mostly infants aged less than 3 months (n=18). Antibiotics were commonly prescribed (67.2%), but use of available influenza-specific antiviral agents was uncommon (13.1%). The nosocomial infection rate was 9.8% and the clinical staff vaccination rate was low (less than 30%). Pneumonia was the most common complication (12.3%). No influenza-related deaths occurred. Influenza in young children poses a significant burden to health care services, tertiary admissions representing the tip-of-the-iceberg. Vaccination rates are inappropriately low in both eligible patients and hospital clinical staff. Early 'point of care' testing, use of influenza-specific antiviral agents, and extension of current vaccination schedules to include all children aged six to 23 months could considerably reduce over-investigation, unnecessary use of antibiotics and the health care impact of influenza.

Evaluation of a national resource to identify and study rare diseases: the Australian Paediatric Surveillance Unit.

AIMS: To evaluate the Australian Paediatric Surveillance Unit (APSU). METHODS: We used criteria recommended by the Centres for Disease Control and Prevention (CDC) for evaluating surveillance systems and reviewed productivity, response rates, completeness of the mailing list and impacts of APSU studies. Anonymous evaluation questionnaires were sent to 1260 reporting clinicians, 42 researchers and 86 public health professionals to seek their feedback as users of the APSU. RESULTS: APSU provides national epidemiological and clinical data about rare childhood conditions that inform public health policy and clinical practice. Between 2000 and 2007, APSU data were disseminated in 106 journal articles, 207 scientific presentations and 85 media items. Of paediatricians and paediatric sub-specialists actively practicing in Australia and listed as Fellows of the Royal Australasian College of Physicians, 92% participate in APSU surveillance. An average 96% of monthly report cards were returned per annum since 2000. Sensitivity of case ascertainment was difficult to calculate for many conditions because alternative sources of ascertainment do not exist. Evaluation questionnaires were returned by 818 (65%) reporting clinicians, who believe APSU is valuable for generating knowledge 665 (81%), identifying research priorities 635 (78%), guiding clinical practice 572 (70%) and informing public health policy 575 (70%). Similar responses were received from researchers and public health professionals. CONCLUSIONS: The APSU fulfils its objectives and meets relevant CDC criteria for usefulness, simplicity, acceptability and representativeness, sensitivity and timeliness of data quality. However, stability is threatened by lack of continuing core funding. APSU is highly productive and valued by child health clinicians, researchers and public health professionals.

Burden of eating disorders in 5-13-year-old children in Australia.

OBJECTIVE: To collect nationally representative epidemiological data on early-onset eating disorders (EOEDs) in children. DESIGN: Prospective, active surveillance using the Australian Paediatric Surveillance Unit with key informant design. SETTING: Child health specialists in Australia (July 2002 to June 2005). PATIENTS: Incident cases of EOEDs in children aged 5-13 years. MAIN OUTCOME MEASURES: Disease rates, demographic characteristics, clinical features and complications, hospitalisation, psychological comorbidity, and concordance of clinical features with Diagnostic and statistical manual of mental disorders, fourth edition (DSM-IV) criteria. RESULTS: We identified 101 children aged 5-13 years with EOEDs (median age, 12.2 years; range, 5.5-13.9 years), of whom one in four were boys. Most were hospitalised (78%), and the mean duration of hospitalisation was 24.7 days (range, 1-75 days). More than 70% of inpatients were admitted to specialised eating disorder units in paediatric teaching hospitals. Among inpatients, 37% met DSM-IV diagnostic criteria for anorexia nervosa; although 61% had life-threatening complications of malnutrition, only 51% met weight criteria. Psychological symptoms were similar to those in adults with anorexia nervosa: 67% of inpatients met both psychological diagnostic criteria for anorexia nervosa (fear of weight gain/fatness and misperception of body shape). Of 19 postmenarchal girls, 18 had secondary amenorrhoea. Nasogastric feeding was used in 58% of inpatients, and 34% received psychotropic medications. CONCLUSIONS: This is the first prospective national study of EOEDs. It demonstrates the limitations of applying DSM-IV diagnostic criteria for anorexia nervosa to young children; the high proportion of boys affected by EOEDs; and the significant psychological comorbidity and high frequency of hospitalisation associated with EOEDs. Potentially life-threatening medical complications are common at presentation, suggesting possible missed diagnoses and a need for education of health professionals. The study underlines the severity of EOEDs and the need for joint medical and psychiatric specialist management.

Perinatal exposure to HIV among children born in Australia, 1982-2006.

OBJECTIVE: To describe the pattern of perinatal HIV exposure and outcomes among children born in Australia, 1982-2006. DESIGN AND SETTING: National surveillance for perinatal HIV exposure. PARTICIPANTS: Women with HIV infection and their perinatally exposed children. MAIN OUTCOME MEASURES: Trends in the age-standardised rate of perinatal exposure, uptake of interventions by women with an antenatal HIV diagnosis, and rate of mother-to-child transmission. RESULTS: Between 1982 and 2006, there were 354 reported cases of perinatal HIV exposure among children born in Australia. The age-standardised rate of perinatal exposure per 100,000 live births increased from 2.3 (1982-1986) to 5.1 (1991-1998), 9.9 (1999-2002) and 8.3 (2003-2006). Among children whose mother was diagnosed antenatally, the mother-to-child transmission rate declined significantly, from 25% (4/16; 95% CI, 7%-52%) in 1987-1990 to 5% (4/82; 95% CI, 1%-12%) in 2003-2006 (P < 0.001). The rate declined from 8% (4/51; 95% CI, 2%-19%) in 1987-1998 to 1% (2/151; 95% CI, 0.2%-5%) in 1999-2006 among children whose mother used at least two interventions. Mother-to-child transmission remained high among children born to women diagnosed postnatally (39/87, 45%; 95% CI, 34%-56%) and to women diagnosed antenatally who used no interventions (7/15, 47%; 95% CI, 21%-73%). CONCLUSION: The increasing rate of perinatal exposure and the decreasing rate of mother-to-child transmission among children whose mothers' HIV infection was diagnosed antenatally were temporally associated with use of interventions for minimising mother-to-child transmission. Mother-to-child transmission remained high when the mother's HIV infection was not known during pregnancy.

Enhanced surveillance for serious complications of influenza in children: role of the Australian Paediatric Surveillance Unit.

Influenza contributes significantly to disease burden among children aged less than five years. Existing influenza surveillance systems do not provide detailed data on clinical presentation, management, vaccination status, risk factors and complications in hospitalised children, or link such data with laboratory results. Following a number of child deaths due to influenza in 2007, the Australian Government Department of Health and Ageing approached the Australian Paediatric Surveillance Unit (APSU) to examine the feasibility of enhancing APSU surveillance to identify children hospitalised with severe complications of influenza. Active, national, weekly surveillance was conducted during September 2007 with reporting by 1,256 Australian paediatricians working in hospitals and outpatient settings. The weekly report card return rate was 93%; detailed clinical data were provided on 88% of all notified cases and 15 children met the case criteria for severe complications of influenza. Admission to hospital occurred within 48 hours of onset of symptoms in over half of the children, of whom 13 had influenza A and two had influenza B, confirmed mostly by polymerase chain reaction on nasopharyngeal aspirate. Serious complications included pneumonia, presumed viral (67%), secondary bacterial infection, shock, cardiomyopathy, myocarditis and hypoglycaemia. No child aged six months or older had been vaccinated against influenza, including three children with underlying chronic conditions. No eligible child received an antiviral agent for influenza. Length of hospital stay ranged from 2 to 34 days; four children were admitted to a Paediatric Intensive Care Unit and one was ventilated. This study demonstrates the feasibility of using the established APSU mechanism for enhanced emergency surveillance during disease outbreaks, emergence or importation.

Indigenous child health: urgent need for improved data to underpin better health outcomes.

Accurate data about Indigenous child health is vital to enable us to understand its current state, to acknowledge achievements, and to determine how to reduce inequalities between Indigenous and non-Indigenous children. We have identified a paucity of national, or nationally representative, data relating to Indigenous child health outcomes, and significant deficiencies in available data. A coordinated national approach will help address current data limitations, including lack of identification of Indigenous status, lack of currency, and lack of information about specific health disorders affecting Indigenous children. To ensure that health data collected are relevant and useful, Indigenous communities must have a role in data collection and management.