Clinical trial NCT03666767: Management and Outcomes of Congenital Anomalies in Low-, Middle- and High-Income Countries
Official title: Management and Outcomes of Congenital Anomalies in Low-, Middle- and High-Income Countries: A Multi-Centre, International, Prospective Cohort Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Oesophageal Atresia
Congenital Diaphragmatic Hernia
Intestinal Atresia
Gastroschisis
Exomphalos
Anorectal Malformation
Hirschsprung Disease
Lead sponsor: King's College London
Sponsor class: OTHER
First posted: 2018-09-12
Enrollment: 3850
Sex: ALL
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Neonates, infants and children up to 16-years of age presenting for the first time with one of the seven congenital anomalies in the study to institutions in low-, middle- and high-income countries globally.

All institutions that provide surgical care for the patients being studied can contribute data. Participating institutions will be recruited through convenience sampling with snowballing.

Brief summary: This study is a multi-centre, international, prospective cohort study of congenital anomalies to compare outcomes between LMICs and high-income countries (HICs) globally.

Detailed description: Background: Congenital anomalies have risen to become the 5th leading cause of death in children under 5-years of age globally, yet limited literature exists, particularly from low- and middle-income countries (LMICs) where most of these deaths occur.

Aim: To undertake a multi-centre prospective cohort study of congenital anomalies to compare outcomes between LMICs and high-income countries (HICs) globally.

Methods: The Global PaedSurg Research Collaboration will be established consisting of children's surgical care providers from around the world to participate in the study; collaborators will be co-authors of resulting presentations and publication(s). Data will be collected on patients presenting primarily with seven congenital anomalies (oesophageal atresia, congenital diaphragmatic hernia, intestinal atresia, gastroschisis, exomphalos, anorectal malformation and Hirschsprung's disease) for a minimum of one month between Oct 2018 - April 2019. Anonymous data will be collected on patient demographics, clinical status, interventions and outcome. Data will be captured using the secure, online data collection tool REDCap.

The primary outcome will be all-cause in-hospital mortality and the secondary outcomes will be occurrence of post-operative complications. Chi-squared analysis will be used to compare mortality between LMICs and HICs. Multilevel, multivariate logistic regression analysis will be undertaken to identify patient level and hospital level factors affecting outcomes with adjustment for confounding factors. P\<0.05 will be deemed significant. Study approval will be sought from all participating centres. Funding has been granted by the Wellcome Trust.

Outcomes: The study aims to be the first large-scale, geographically comprehensive, multi-centre prospective cohort study of a selection of common congenital anomalies to define current management and outcomes globally. Results will be used to aid advocacy and global health prioritisation and inform future interventional studies aimed at improving outcomes.

Eligibility criteria: Inclusion Criteria:

* Any neonate, infant or child under the age of 16-years, presenting for the first time, with one of the study conditions can be included in the study.
* Children who have NOT previously received any surgery for their condition.
* Children who have received basic resuscitative and supportive care for their condition at a different healthcare facility and then been transferred to the study centre.
* Patients presenting primarily with one of the study conditions who receive palliative care or no care must be included within the study to reflect true outcomes.

Exclusion Criteria:

* Any neonate, infant or child with one of the study conditions who has previously received surgery (including a stoma) for their condition
* If they have recently received surgery for their condition, were discharged and then represented with a complication of the surgery during the study period they should NOT be included in the study.

Primary outcomes:
measure: All-cause, in-hospital mortality
timeFrame: Mortality whilst in hospital during primary admission, up to a maximum of 30-days following primary intervention or 30-days following presentation for those who do not receive an intervention and are still in hospital.
Secondary outcomes:
measure: Surgical site-infection
timeFrame: Occurring within 30-days of primary intervention
measure: Wound dehiscence
timeFrame: Occurring within 30-days of primary intervention
measure: Need for re-intervention
timeFrame: Occurring within 30-days of primary intervention
measure: Condition specific complications
timeFrame: Occurring within 30-days of primary intervention
measure: Condition specific outcome variables
timeFrame: Occurring within 30-days of primary intervention
measure: Length of hospital stay
timeFrame: Maximum 30-days post-intervention or following presentation for those who do not receive an intervention
measure: 30-day post primary intervention mortality
timeFrame: Death within 30-days of primary intervention or 30-days of presentation in those who do not receive an intervention
measure: Ventilation requirement
timeFrame: Occurring within 30-days of primary intervention or admission for those not receiving an intervention
measure: Time to first enteral feed
timeFrame: Occurring within 30-days of primary intervention or admission for those not receiving an intervention
measure: Time to full enteral feeds
timeFrame: Occurring within 30-days of primary intervention or admission for those not receiving an intervention
measure: Parenteral nutrition requirement
timeFrame: Occurring within 30-days of primary intervention or admission for those not receiving an intervention
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: King's College London
Study officials:
affiliation: King's College London
name: Naomi J Wright, MBChB BSc MRCS DCH MSc
role: PRINCIPAL_INVESTIGATOR
References:
citation: Wright NJ; Global PaedSurg Research Collaboration. Management and outcomes of gastrointestinal congenital anomalies in low, middle and high income countries: protocol for a multicentre, international, prospective cohort study. BMJ Open. 2019 Sep 3;9(8):e030452. doi: 10.1136/bmjopen-2019-030452.