Since 2017, the FIMATHO network has annually launched a call for projects intended to support any project related to its missions:

  • improvement of treatment for patients suffering from rare abdominal and thoracic diseases or malformations;
  • development of fundamental, translational and clinical research;
  • development of education, training and information.

This call for projects is intended for reference centres, research laboratories and patients associations who are partners of the network. The selection of the winning projects is based on criteria of relevance, quality and feasibility.

The winning projects chosen by the network’s scientific and management committees are presented during the annual FIMATHO event. The network’s project team accompanies the implementation and follow-up of these projects.

2020 winning projects

GRAAL: Short bowel syndrome and study of the absorption of antibiotics with good oral bioavailability

List of the disease(s) in question: Short bowel syndrome

Identification of the structure: CHRU of Nancy

Identification of the project manager: Niasha MICHOT

Project presentation: Short bowel syndrome (SBS) is defined by an extended resection of the small intestine leaving in place at least 200 cm of the post-duodenal intestine and causing severe malabsorption. For the most part, these patients thus present intestinal failure requiring parenteral support for water, electrolytes and/or macronutrients.

The bioavailability of an oral medication is one of the pharmacokinetic parameters that may determine the clinical effectiveness of treatment. In fact, a medication taken orally must be absorbed by the gastrointestinal tract in order to reach the blood stream and find its therapeutic targets. The absorption takes place at various levels of the gastrointestinal tract, from the stomach to the rectum depending on molecules. The resection of a part of the small intestine in patients with SBS seems to have a strong impact on this absorption stage. Finally, other criteria influence the absorption stage, such as the surface and the contact time with the gastrointestinal wall, blood flow, pH, and peristalsis. Therefore, these antibiotics may be subject to variable absorption in patients with SBS, thus modifying their bioavailability.

While oral antibiotherapy is not contraindicated in patients with a short bowel, this therapeutic option is rarely retained due to the absence of existing data on the absorption of these treatments.

The purpose of the study is to assess the bioavailability of five antibiotics (or combinations of antibiotics), i.e. amoxicillin, amoxicillin-clavulanic acid , ofloxacin, levofloxacin and sulfamethoxazole-trimethoprim, following the start of ingestion by mouth of an antibiotherapy initiated intravenously in patients with a short bowel and requiring curative antibiotherapy. The antibiotics chosen for this research were selected for their good oral bioavailability.

This pilot study may help obtain preliminary data on the feasibility of ingestion by mouth in this type of patient and develop a larger, multi-centric study in order to consolidate these first results.

Cooking workshop "Discovering textures and senses"

List of the disease(s) in question: Pathologies of the FIMATHO network for which children face the risk of developing oral feeding difficulties

Identification of the structure: AFAO association

Identification of the project manager: Laura GOUPIL and the entire team of AFAO volunteers

Project presentation: food is one of the first connections binding a mother to her child. For children suffering from an abdominal and thoracic malformation, which generally requires surgery just after birth, this bond is broken from the 1st feeding. Also, these children may be fed by tube for several years. This may lead to oral feeding problems.

One of the missions of AFAO is to accompany these children and their parents in order to (re)stimulate the pleasure of eating.

The objectives of the project are to:

  • Help with neurosensory development in small children and in children presenting oral feeding difficulties, to give them the pleasure of eating again - mainly by focusing on touch, taste and sight. It is about stimulating the sensory (and thus the relational) abilities of children to favour their growth and development;
  • Support families affected by oral feeding difficulties in their children by helping them meet each other;
  • Favour exchanges between these families and oral feeding specialists;
  • Inform families about oral feeding difficulties.

2019 winning projects

Empowerment of teens regarding self-care in parenteral nutrition

List of the disease(s) in question): All pathologies of the digestive system

Identification of the structure: La Vie par un Fil association

Identification of the project manager: Myriam LENNE

Project objectives: When a child is fed at home by parenteral nutrition, treatments are conducted by their parents, who have been trained at the hospital. As they grow up, teens may wish to become autonomous and thus need to learn to correctly and safely manage their parenteral nutrition treatments.

The La Vie Par Un Fil association, in collaboration with a specialised medical team, designed a training programme with the objectives of helping teen patients understand their disease and its treatment, managing it on a daily basis and acquiring the skills necessary for an overall and optimal treatment of their parenteral nutrition care.

Each training course will take place in three or four sessions during the school holidays in groups of five teens maximum. To mark the end of each training session, a two-day trip will be organised with patients and the medical team to finalise the increased autonomy of the teens, who will receive a personalised training booklet.

The purpose of this associative project will be to develop this type of training in all centres certified in home parenteral nutrition. The network’s support will help pay lodging expenses for patients and their parents during training sessions and during the organisation of the autonomy week-end.

Assessment of the respiratory impact of thoracic wall anomalies following a classic or minimally invasive surgical treatment for oesophageal atresia - RestriMIS

List of the disease(s) in question: Oesophageal atresia

Identification of the structure: CHU of Angers

Identification of the project manager: Françoise SCHMITT

Project objectives: Minimally invasive surgery is under evaluation in the surgical treatment of congenital oesophageal malformations. Its main principle is to limit the negative impact of the surgical path on morbidity and patient comfort, by using appropriate surgical instruments and videoscopic resources.

The priority objectives of the assessment of these new techniques are to have results at least equal or even superior to the classic path by posterolateral thoracotomy, and essentially defined by a number of complications fewer than or equal to classic techniques: re-fistula, stenosis and gastro-oesophageal reflux rates with regards to the cure for oesophageal atresia.

The main objective of this project is to assess, using a retrospective, non-interventional study, the existence of a restrictive pulmonary syndrome in patients with oesophageal atresia depending on the type of surgical path conducted (conventional or minimally invasive).

Assistance for the hospitalised child - accommodations for the family

List of the disease(s) in question: Oesophageal atresia

Identification of the structure: Association Française de l'Atrésie de l'OEsophage (AFAO, or French Oesophageal Atresia Association)

Identification of the project manager: ARMAND Viviane

Project objectives: Children suffering from rare abdominal and thoracic diseases are, in most cases, hospitalised from their birth in hospitals specialised in treating their pathologies, sometimes far from their families. In order to help parents stay close to their children, there exist family hosting or assistance structures in hospitals to find accommodations for them, but sometimes this is not enough.

AFAO proposes to establish, in collaboration with social services units in University hospitals, a "national lodging database" and to list available lodging options for families close to reference and knowledge centres of the network. If no other accommodation solution can be offered to a family to remain close to the child, AFAO may reimburse their transportation and lodging fees.

2018 winning projects

Influence of structural properties and interest of chemical and biological functionalisation of biomaterials on their biological integration in the host in the treatment of large-defect congenital diaphragmatic hernia

List of the disease(s) in question: Congenital diaphragmatic hernia

Identification of the structure: Inserm UMR_S 1121

Identification of the project manager: Isabelle TALON

Project objectives: Optimisation of tissue integration of prosthetics using functionalisation of biomaterials.

Functional evaluation of the foetal lung by MRI-BOLD imaging in congenital diaphragmatic and parietal malformations

List of the disease(s) in question: Diaphragmatic hernia, omphalocele, laparoschisis

Identification of the structure: Hôpital Necker-Enfants malades

Identification of the project manager: KHEN-DUNLOP Naziha

Project objectives: The purpose of this project is to assess the quality of response to the BOLD effect in foetuses with diaphragmatic hernias and abdominal wall malformations and to look for a correlation with post-natal respiratory evolution. There is always an impact on the lungs in diaphragmatic hernias; it is classic in omphalocele and especially in hepato-omphalocele and exceptional in laparoschisis. As this is an original exploratory study, there is no preliminary data available. 

If a correlation is found, the BOLD effect of the foetal lung may be considered as an early functional marker of post-natal pulmonary function. It may be used in addition to LHR during the pre-natal exam.

Oral feeding workshops on "Discovering textures and senses"

 

List of the disease(s) in question: Oesophageal atresia

Identification of the structure: Association Française de l'Atrésie de l'Oesophage (AFAO, or French Oesophageal Atresia Association)

Identification of the project manager: ARMAND Viviane

Project objectives: The purpose of this project is to improve the neurosensory development of infants and children by mainly focusing on touch and taste. It is about stimulating sensory (and also relational) abilities of children to favour their growth and development.

 

2017 winning projects

ProCDH: “Proteomic analysis of the amniotic liquid in case of diaphragmatic hernia: research on expression profiles for prognostic purposes"

List of the disease(s) in question: Diaphragmatic hernia

Identification of the structure: CHRU of Besançon

Identification of the project manager: AUBER Frédéric

Project objectives: Explore methods other than imaging to contribute to the evaluation of the prognosis in case of diaphragmatic hernia. In particular, we put forth the hypothesis that the proteomic analysis of the amniotic fluid of foetuses with diaphragmatic hernia may be likely to give prognostic information. 

The purpose of this study is therefore to identify, from a proteomic profile of the mother’s amniotic fluid in case of a foetus with diaphragmatic hernia, candidate markers for a death prognosis at two months in the infant.

First, this involves conducting an exploratory (and not interventional) study on a small sample (n=10) of the target population. This is a preliminary stage to see if results are encouraging, in which case a large-scale study may be envisaged on a biological collection to be put together (Biobanque projects supported by FIMATHO).

Interest of using invasive NAVA ventilation on breathing effort and synchronisation in newborns born at term suffering from congenital diaphragmatic hernia (CDH). "NAVA DIAPH” study

List of the disease(s) in question: Congenital diaphragmatic hernia

Identification of the structure: Hospices Civils de Lyon (University Hospital Centre of Lyon)

Identification of the project manager: DREYFUS Lélia

Project objectives: The main objective is to demonstrate that NAVA ventilation enables a reduction in breathing effort in newborns suffering from congenital diaphragmatic hernia, while maintaining diaphragm activity. 

Secondary objectives are to assess the impact of NAVA ventilation on: 

  • Patient - ventilator synchronisation;
  • Dynamic hyperinflation (intrinsic PEEP); 
  • Ventilation parameters: peak inhalation pressure, average pressure, tidal volume, Ti/Ttot ratio, FiO2;
  • Clinical parameters for newborns: respiratory frequency, cardiac frequency, arterial pressures;
  • Gas exchanges: measurement of transcutaneous pCO2, SaO2;
  • Comfort and pain scores in newborns.

Quality of life in therapeutic patient education

List of the disease(s) in question: Short bowel syndrome, with the possibility of subsequent extension to other rare diseases which are part of the FIMATHO network.

Identification of the structure: La Vie par un Fil association

Identification of the project manager: Marie-Astrid DE JOCAS

Project objectives: Help patients suffering from short bowel syndrome with an innovative and intuitive mobile digital therapeutic monitoring tool, used to acquire knowledge on relationships between quality of life and the treatment to acquire and maintain self-care skills and mobilise or acquire adaptation skills.

Develop understanding of the quality of life in therapeutic education and thus enable truly comprehensive treatment from a physician, in order to improve the overall quality of life for people suffering from short bowel syndrome and thus to preserve their life in society.