Prune belly syndrome: a 31 year experience at the Chris Hani Baragwanath Academic Hospital, South Africa

A research report submitted to the Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, in partial fulfilment of the requirements for the degree of Master of Medicine in Paediatrics Johannesburg, 2017Background: PBS is a rare congenital disorder with a triad of signs: absent abdominal wall musculature, urinary tract malformations, cryptorchidism. Objectives: Describe the patient profile, management, and outcome of patients attending the Paediatric Nephrology clinic at Chris Hani Baragwanath Academic Hospital. Methods: Retrospective descriptive record review. Results: 44 patient files were analysed. Median duration of follow up: 24 months (1.7–130). Median age at presentation was 5.5 days (1–730). Associated conditions included: urological 48%, orthopaedic 11%, congenital heart disease 7%, gastrointestinal 4.5%. Medical management included prophylactic antibiotics and intermittent bladder catheterisation. Surgical management included abdominoplasty with orchidopexy (3 years) and circumcision (7.7 years). Patients over the age of 2 years had a better overall outcome. There was a high default rate of 57%. Conclusion: Patient profile and management is comparable to other reported series. Deterioration in renal function to ESRD was observed less in this review possibly due to the high default rate.DH201

Prune belly syndrome: a South African perspective

Background: Prune belly syndrome (PBS) is a rare congenital disorder with a triad of signs: absent abdominal wall musculature, urinary tract malformations, and cryptorchidism. The aims of this study were to describe the epidemiology, renal function, management and outcome of patients with PBS at the Chris Hani Baragwanath Academic Hospital, Johannesburg, South Africa.Methods: A retrospective record review was conducted of patients with PBS referred to the paediatric nephrology unit from 1984 to 2014.Results: A total of 44 patient records were analysed. Most of the patients presented as neonates (n = 35, 80%). The accompanying congenital malformations included urological problems (21, 48%), orthopaedic abnormalities (5, 11%), congenital heart disease (3, 7%) and gastrointestinal abnormalities (2, 4%). The medical management included the use of prophylactic antibiotics and intermittent bladder catheterisation. Surgical management included abdominoplasty with orchidopexy and circumcision. At the last clinic visit, severe reductions in renal function were noted in 11 (58%) of the patients under the age of 2 years, whereas normal renal function was present in 14 (56%) of the patients over 2 years of age. The default rate was 57%; 6 (14%) patients were transferred to an adult clinic and 2 (4%) patients died.Conclusions: The patient profiles and the management recorded are comparable with other reported series. There are differences in the frequencies of congenital malformations and the rates of surgical intervention. The renal outcome for children older than two years was favourable compared to younger children. Progression to end-stage renal disease was observed infrequently, but this may be underestimated in view of the high default rate

Detecting the harmonics of oscillations with time-variable frequencies

A method is introduced for the spectral analysis of complex noisy signals containing several frequency components. It enables components that are independent to be distinguished from the harmonics of nonsinusoidal oscillatory processes of lower frequency. The method is based on mutual information and surrogate testing combined with the wavelet transform, and it is applicable to relatively short time series containing frequencies that are time variable. Where the fundamental frequency and harmonics of a process can be identified, the characteristic shape of the corresponding oscillation can be determined, enabling adaptive filtering to remove other components and nonoscillatory noise from the signal. Thus the total bandwidth of the signal can be correctly partitioned and the power associated with each component then can be quantified more accurately. The method is first demonstrated on numerical examples. It is then used to identify the higher harmonics of oscillations in human skin blood flow, both spontaneous and associated with periodic iontophoresis of a vasodilatory agent. The method should be equally relevant to all situations where signals of comparable complexity are encountered, including applications in astrophysics, engineering, and electrical circuits, as well as in other areas of physiology and biology

A Programmatic Approach to Patient Blood Management – reducing transfusions and improving patient outcomes

In July 2008, the Western Australia (WA) Department of Health embarked on a landmark 5-year project to implement a sustainable comprehensive health-system-wide Patient Blood Management Program. Fundamentally, it was a quality and safety initiative, which also had profound resource and economic implications. Unsustainable escalating direct and indirect costs of blood, potentially severe blood shortages due to changing population dynamics, donor deferrals, loss of altruism, wide variations in transfusion practice and growing knowledge of transfusion limitations and adverse outcomes necessitate a paradigm shift in the management of anemia and blood loss. The concept of patient-focused blood management is proving to be an effective force for change. This approach has now evolved to embrace comprehensive hospital-wide Patient Blood Management Programs. These programs show significant reductions in blood utilisation, reduced costs while achieving similar or improved patient outcomes. The WA Program is achieving these outcomes across a health jurisdiction in a sustained manner

Long-term healthcare utilisation, costs and quality of life after invasive group B Streptococcus disease: a cohort study in five low-income and middle-income countries

Introduction There are no published data on the long-term impact of invasive group B Streptococcus disease (iGBS) on economic costs or health-related quality of life (HRQoL) in low-income and middle-income countries. We assessed the impact of iGBS on healthcare utilisation, costs and HRQoL in Argentina, India, Kenya, Mozambique and South Africa. Methods Inpatient and outpatient visits, out-of-pocket (OOP) healthcare payments in the 12 months before study enrolment, and health-state utility of children and caregivers (using the EuroQol 5-Dimensions-3-Level) were collected from iGBS survivors and an unexposed cohort matched on site, age at recruitment and sex. We used logistic or Poisson regression for analysing healthcare utilisation and zero-inflated gamma regression models for family and health system costs. For HRQoL, we used a zero-inflated beta model of disutility pooled data. Results 161 iGBS-exposed and 439 unexposed children and young adults (age 1–20) were included in the analysis. Compared with unexposed participants, iGBS was associated with increased odds of any healthcare utilisation in India (adjusted OR 11.2, 95% CI 2.9 to 43.1) and Mozambique (6.8, 95% CI 2.2 to 21.1) and more frequent healthcare visits (adjusted incidence rate ratio (IRR) for India 1.7 (95% CI 1.4 to 2.2) and for Mozambique 6.0 (95% CI 3.2 to 11.2)). iGBS was also associated with more frequent days in inpatient care in India (adjusted IRR 4.0 (95% CI 2.3 to 6.8) and Kenya 6.4 (95% CI 2.9 to 14.3)). OOP payments were higher in the iGBS cohort in India (adjusted mean: Int$682.22 (95$364.28 to Int$1000.16) vs Int$133.95 (95% CI Int$72.83 to Int$195.06)) and Argentina (Int$244.86 (95$47.38 to Int$442.33) vs Int$52.38 (95% CI Int$−1.39 to Int$106.1)). For all remaining sites, differences were in the same direction but not statistically significant for almost all outcomes. Health-state disutility was higher in iGBS survivors (0.08, 0.04–0.13 vs 0.06, 0.02–0.10). Conclusion The iGBS health and economic burden may persist for years after acute disease. Larger studies are needed for more robust estimates to inform the cost-effectiveness of iGBS prevention

Group B streptococcus infection during pregnancy and infancy: estimates of regional and global burden

Background: Group B streptococcus (GBS) colonisation during pregnancy can lead to invasive GBS disease (iGBS) in infants, including meningitis or sepsis, with a high mortality risk. Other outcomes include stillbirths, maternal infections, and prematurity. There are data gaps, notably regarding neurodevelopmental impairment (NDI), especially after iGBS sepsis, which have limited previous global estimates. In this study, we aimed to address this gap using newly available multicountry datasets. Methods: We collated and meta-analysed summary data, primarily identified in a series of systematic reviews published in 2017 but also from recent studies on NDI and stillbirths, using Bayesian hierarchical models, and estimated the burden for 183 countries in 2020 regarding: maternal GBS colonisation, iGBS cases and deaths in infants younger than 3 months, children surviving iGBS affected by NDI, and maternal iGBS cases. We analysed the proportion of stillbirths with GBS and applied this to the UN-estimated stillbirth risk per country. Excess preterm births associated with maternal GBS colonisation were calculated using meta-analysis and national preterm birth rates. Findings: Data from the seven systematic reviews, published in 2017, that informed the previous burden estimation (a total of 515 data points) were combined with new data (17 data points) from large multicountry studies on neurodevelopmental impairment (two studies) and stillbirths (one study). A posterior median of 19·7 million (95% posterior interval 17·9–21·9) pregnant women were estimated to have rectovaginal colonisation with GBS in 2020. 231800 (114 100–455000) early-onset and 162 200 (70200–394 400) late-onset infant iGBS cases were estimated to have occurred. In an analysis assuming a higher case fatality rate in the absence of a skilled birth attendant, 91 900 (44800–187 800) iGBS infant deaths were estimated; in an analysis without this assumption, 58300 (26 500–125800) infant deaths from iGBS were estimated. 37100 children who recovered from iGBS (14600–96200) were predicted to develop moderate or severe NDI. 40500 (21500–66 200) maternal iGBS cases and 46200 (20 300–111300) GBS stillbirths were predicted in 2020. GBS colonisation was also estimated to be potentially associated with considerable numbers of preterm births. Interpretation: Our analysis provides a comprehensive assessment of the pregnancy-related GBS burden. The Bayesian approach enabled coherent propagation of uncertainty, which is considerable, notably regarding GBS-associated preterm births. Our findings on both the acute and long-term consequences of iGBS have public health implications for understanding the value of investment in maternal GBS immunisation and other preventive strategies

Quantifying the Acute Care Costs of Neonatal Bacterial Sepsis and Meningitis in Mozambique and South Africa.

BACKGROUND: Sepsis and meningitis are among the leading causes of neonatal deaths in sub-Saharan Africa (SSA). Neonatal sepsis caused ~400 000 deaths globally in 2015, half occurring in Africa. Despite this, there are few published data on the acute costs of neonatal sepsis or meningitis, with none in SSA. METHODS: We enrolled neonates admitted to 2 hospitals in South Africa and Mozambique between 16 April 2020 and 1 April 2021. In South Africa all cases were microbiologically confirmed, but in Mozambique both clinically suspected and microbiologically confirmed cases were included. Data were collected on healthcare resource use and length of stay, along with information on household expenditure and caregiving. We used unit costs of healthcare resources in local currencies to estimate healthcare provider costs per patient and costs per household. Results were converted to 2019 international dollars (I$). RESULTS: We enrolled 11 neonates in Mozambique and 18 neonates in South Africa. Mean length of stay was 10 days (median, 9 [interquartile range {IQR}, 4-14) and 16 days (median, 15 [IQR, 13-18]), respectively. In Mozambique we estimated mean household costs of I$49.62 (median, 10.19 [IQR, 5.10-95.12]) and hospitalization costs of I$307.58 (median, 275.12 [IQR, 149.43-386.12]). In South Africa these costs were I$52.31 (median, 30.82 [IQR, 19.25-73.08]) and I$684.06 (median, 653.62 [IQR, 543.33-827.53]), respectively. CONCLUSIONS: We found substantial costs associated with acute neonatal bacterial (all-cause) sepsis and meningitis in SSA. Our estimates will inform economic evaluations of interventions to prevent neonatal invasive bacterial infections

Quantifying long-term health and economic outcomes for survivors of group B Streptococcus invasive disease in infancy: protocol of a multi-country study in Argentina, India, Kenya, Mozambique and South Africa.

Sepsis and meningitis due to invasive group B Streptococcus (iGBS) disease during early infancy is a leading cause of child mortality. Recent systematic estimates of the worldwide burden of GBS suggested that there are 319,000 cases of infant iGBS disease each year, and an estimated 147,000 stillbirths and young-infant deaths, with the highest burden occurring in Sub-Saharan Africa.  The following priority data gaps were highlighted: (1) long-term outcome data after infant iGBS, including mild disability, to calculate quality-adjusted life years (QALYs) or disability-adjusted life years (DALYs) and (2) economic burden for iGBS survivors and their families. Geographic data gaps were also noted with few studies from low- and middle- income countries (LMIC), where the GBS burden is estimated to be the highest. In this paper we present the protocol for a multi-country matched cohort study designed to estimate the risk of long-term neurodevelopmental impairment (NDI), socioemotional behaviors, and economic outcomes for children who survive invasive GBS disease in Argentina, India, Kenya, Mozambique, and South Africa. Children will be identified from health demographic surveillance systems, hospital records, and among participants of previous epidemiological studies. The children will be aged between 18 months to 17 years. A tablet-based custom-designed application will be used to capture data from direct assessment of the child and interviews with the main caregiver. In addition, a parallel sub-study will prospectively measure the acute costs of hospitalization due to neonatal sepsis or meningitis, irrespective of underlying etiology. In summary, these data are necessary to characterize the consequences of iGBS disease and enable the advancement of effective strategies for survivors to reach their developmental and economic potential. In particular, our study will inform the development of a full public health value proposition on maternal GBS immunization that is being coordinated by the World Health Organization

A randomised trial and economic evaluation of the effect of response mode on response rate, response bias, and item non-response in a survey of doctors

<p>Abstract</p> <p>Background</p> <p>Surveys of doctors are an important data collection method in health services research. Ways to improve response rates, minimise survey response bias and item non-response, within a given budget, have not previously been addressed in the same study. The aim of this paper is to compare the effects and costs of three different modes of survey administration in a national survey of doctors.</p> <p>Methods</p> <p>A stratified random sample of 4.9% (2,702/54,160) of doctors undertaking clinical practice was drawn from a national directory of all doctors in Australia. Stratification was by four doctor types: general practitioners, specialists, specialists-in-training, and hospital non-specialists, and by six rural/remote categories. A three-arm parallel trial design with equal randomisation across arms was used. Doctors were randomly allocated to: online questionnaire (902); simultaneous mixed mode (a paper questionnaire and login details sent together) (900); or, sequential mixed mode (online followed by a paper questionnaire with the reminder) (900). Analysis was by intention to treat, as within each primary mode, doctors could choose either paper or online. Primary outcome measures were response rate, survey response bias, item non-response, and cost.</p> <p>Results</p> <p>The online mode had a response rate 12.95%, followed by the simultaneous mixed mode with 19.7%, and the sequential mixed mode with 20.7%. After adjusting for observed differences between the groups, the online mode had a 7 percentage point lower response rate compared to the simultaneous mixed mode, and a 7.7 percentage point lower response rate compared to sequential mixed mode. The difference in response rate between the sequential and simultaneous modes was not statistically significant. Both mixed modes showed evidence of response bias, whilst the characteristics of online respondents were similar to the population. However, the online mode had a higher rate of item non-response compared to both mixed modes. The total cost of the online survey was 38% lower than simultaneous mixed mode and 22% lower than sequential mixed mode. The cost of the sequential mixed mode was 14% lower than simultaneous mixed mode. Compared to the online mode, the sequential mixed mode was the most cost-effective, although exhibiting some evidence of response bias.</p> <p>Conclusions</p> <p>Decisions on which survey mode to use depend on response rates, response bias, item non-response and costs. The sequential mixed mode appears to be the most cost-effective mode of survey administration for surveys of the population of doctors, if one is prepared to accept a degree of response bias. Online surveys are not yet suitable to be used exclusively for surveys of the doctor population.</p