Long Term Infant Follow-up in Trials Using a European Platform of Preterm Birth Cohorts
Background: The importance of RCTs
For scientifically sound and sustainable advances within life sciences, Randomised Controlled Trials (RCTs) have been used to compare the effects of drugs, medical techniques or other treatments and thereby generate new knowledge for improved development and procedures. Due to increasing complexities of interventions and costs of these methods, RCTs are rare in the neonatal demographic and thus need additional attention.
Follow-up investigations are essential to establish a more holistic perspective of the chosen interventions and to avoid a neglect of long-term consequences of medical interventions studied in RCTs.
*For an explanation of the terms marked in blue, please see the ‘Definitions’ section further down the page.
What is LIFT-UP Preterm?
LIFT-UP Preterm is a project lead by INSERM (French National Institute of Health and Medical Research), which aims to promote better health outcomes for very preterm populations through innovative methods. LIFT-UP Preterm is making use of the existing RECAP Preterm Cohort Platform, which is a European research data base developed to improve health, development, and quality of life of children and adults born very preterm or with a very low birth weight.
For more information on RECAP Preterm, visit EFCNI’s RECAP Preterm project page.
A total of 8 academic partners and 24 hospitals across 13 European countries are involved in LIFT-UP Preterm, which has a planned project duration of 4 years.
For more information about the project, please take a look at the flyer below.
Aims & objectives of the LIFT-UP Preterm project
The LIFT-UP Preterm project’s main objective is to create better health outcomes in individuals born very preterm by promoting RCTs in this population group. One major goal is to create an integrated data entry tool, with which parents can provide data on their child’s cognitive development via a parent-completed questionnaire. By using the RECAP Preterm data platform, its existing baseline data can be used to better analyse the newly collected follow-up measurements and thus provide essential information on long-term consequences of the initial interventions.
Other aspects of the LIFT-UP Preterm project focus on the analysis of data to allow for comparability between individual data sets and cohorts within the RECAP Preterm data platform.
An essential aspect of the LIFT-UP Preterm project is the TREOCAPA-long-term study (TREOCAPA-LT). This is a so-called ‘proof-of-concept’ study under the umbrella of the LIFT-UP Preterm project.
*For an explanation of the terms marked in blue, please see the ‘Definitions’ section further down the page.
Why TREOCAPA-LT?
The original TREOCAPA trial is investigating the effectiveness of preventive paracetamol use in preterm born babies within five days of life after birth for its purpose to close the patent ductus arteriosus (PDA). One of the main aspects of observation is the reduction of mortality and severe complications related to preterm birth during the newborn period.
The ductus arteriosus is the vessel, that communicates the aorta (the main and largest artery in the body that distributes blood enriched with oxygen to all parts of the body) and pulmonary artery (the artery carrying blood from the heart to the lungs where the blood is enriched with oxygen) when the baby is still in the womb. Normally, this vessel closes in the days following birth in newborns, but for a large proportion of preterm born babies, it remains open. This increases the risks of complications relating to lungs, brain or gut in preterm born babies.
Visit EFCNI’s TREOCAPA Website for more information on the original TREOCAPA trial.
Even early interventions can have long-term effects on a person’s health outcomes and thus it is essential to expand the area of observation to a more long-term perspective. The TREOCAPA-LT study aims to fill this gap after the conclusion of the original TREOCAPA trial.
The main objective of TREOCAPA-LT is to follow-up on the participants of the original TREOCAPA trial in order to investigate the safety and effectiveness of the intervention in children at two years of age. A special focus is laid on the potential development of neurological disorders or other cognitive irregularities.
The parents or caregivers whose children participated in the TREOCAPA trial, are asked to fill out a questionnaire once the child reaches 24 months of age. This questionnaire aims to determine any longer-term health effects of the paracetamol treatment on the child’s health and cognitive development, while considering the child’s gestational age at birth.
The inclusion of this new data set into the existing RECAP Preterm Cohort Platform allows for a more holistic perspective on the study population by providing a longer overall timeframe of study participation (through both TREOCAPA and TREOCAPA-LT) and thereby encourages a sustainable use of the collected information.
The role of EFCNI in LIFT-UP and TREOCAPA-LT
EFCNI has been involved in the RECAP Preterm project as well as in the initial TREOCAPA trial throughout its project duration and will continue to represent the voices of patients and parents in the TREOCAPA-LT study. Specifically, EFCNI will aid the project by spreading awareness of the project and create educational materials to make these novel methods and gained knowledge available to a broader audience.
Future materials may include:
- Factsheet on the importance of follow-up studies in life sciences
- Explanatory materials for parents showcasing the progress and results of the study
- Communication and dissemination activities
Definitions
A Randomised Controlled Trial (RCT) is a kind of study, which measures the effectiveness of a specific intervention. This can be done by testing a new medical treatment as compared to an existing one, a new medication compared to one that has been in use previously, or any other novel device or procedure.
The participants of a RCT are separated into two groups, one that receives the intervention and one that does not (often called “control group”). The participants of the study are randomly allocated to either group and neither the participants nor the study facilitators know, which group each person has been placed in. This is done to reduce or even eliminate the influence of any known or unknown factors, personal opinions or subconscious notions, which may otherwise affect the outcome of the trial.
RCTs are often considered the ‘gold standard’ of research, as it allows for the least amount of errors incurred by influencing factors. However, RCTs have relatively high costs of execution with regards to both time and money.
A cohort is a defined group of people who participate in a study. The term is used in many disciplines, such as medical, demographic, or statistical research and describes a group of individuals, who are often selected based on a shared characteristic. This defining characteristic could be their age, place of birth, or experience of a common event. In TREOCAPA-LT, the cohort is the entirety of participating children in this study.
A proof-of-concept study describes a research project which aims to showcase the efficacy of a specific new idea or approach.
In this context, TREOCAPA-LT is set to proof the benefits of using existing data for future research. Specifically, in the TREOCAPA-LT study, existing structures of the original TREOCAPA trials can be utilised to follow-up that same study population and investigate long-term effects of the intervention.
For more information on clinical trials in newborns and explanations on relating terminology, please have a look at our factsheet below:
Transparency statement
The TREOCAPA-LT study is funded through the MESSIDORE (Méthodologie des ESSais cliniques Innovants, Dispositifs, Outils et Recherches Exploitant les données de santé et biobanques — Innovative clinical trials methodology, Devices, tools and research using health data and biobanks) 2022 Strategic Program for Collaborative Health Research run by Inserm, the French National Institute of Health and Medical Research. Reference Inserm-MESSIDORE N°74.
