Essey-les-nancy, France

The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis

Myasthenia gravis (MG) is an autoimmune neuromuscular disease characterised by fatigable muscle weakness due to autoantibodies targeting components of the neuromuscular junction. Deficits are measured using validated clinical scales such as the Quantitative Myasthenia Gravis score (QMG) QMG, Myasthenia Gravis Composite Score (MCS), Myasthenia Gravis Activities of Daily Living score (MG-ADL), etc., Unfortunately, this type of assessment might require expensive material, time, and personnel training, which make MG assessment difficult for clinicians in routine care. Research on the matter has highlighted the importance of Patient-Reported Outcomes (PROs), composite measures of disease severity and quality-of-life measurements. This has led to the development of a number of tools for the self-assessment of PROs with validated questionnaires. However, there is no tool that overcomes the constraints of standard tests such as the QMG while allowing for the collection of objective data and PROs between visits. ME&MG is a standalone software (digital solution) running on patients smartphones, connected to a web portal for physicians. It is intended to be used as an unsupervised digital self-assessment tool for the monitoring of disabilities in patients living with MG. ME&MG contains digital active tests for the assessment of ptosis, breathing, dysarthria, upper- and lower-limb (arms and legs) weakness, treatment follow-up, and validated e-questionnaires related to daily activities, pain, fatigue, sleep, and depression disorders. The objectives of this study are to validate the accuracy, reliability and reproducibility of the unsupervised at-home self-assessment of symptoms on the patient's smartphone with ME&MG versus the standard in-clinic testing, as well as to evaluate the safety of the solution, its usability and satisfaction.

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Statistical analyses will be independently performed by the Biostatistics Department of University of Lille under the responsibility of Julien Labreuche. Data will be analyzed using the SAS software (SAS Institute Inc, Cary, NC, USA). A detailed statistical analysis plan will be written and finalized prior to the database lock. Baseline characteristics will be described for each group. Quantitative variables will be expressed as mean (standard deviation) or median (interquartile range) for non-Gaussian distribution. Qualitative variables will be expressed as frequencies and percentages. Normality of distribution will be assessed graphically and using the Shapiro-Wilk test. All applicable statistical tests will be 2-sided and no correction for multiple comparisons will be applied; all secondary objectives will be considered as exploratory and results were reported with only effect size estimates with their confidence intervals (CIs). All CIs presented will be 95%CI and 2-sided. The final report will be written, based on the CONSORT statement recommendations.

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A Multicentre French Prospective Study of Children With Food Protein Induced Enterocolitis Syndrome in Its Acute Form

Food protein induced enterocolitis syndrome (FPIES), is a non-IgE mediated food allergy (FA) which seems to expand, and occurring in infancy. Prevalence of FPIES is unknown. In 2011, Katz published cumulative incidence of cow 'milk FPIES of 3 per 1000 new-borns, from prospective birth cohort in Israel. The offending food depend on the country, probably in relation to eating habits. Cow's milk (CM) is most commonly incriminated and can lead to a chronic digestive disease or in its acute form with potentially life-threatening vomiting/diarrhoea/dehydration, confusing with anaphylaxis. Rice and oat in US, or fish and egg in France are the solid food most often implicated. This disease is usually unknown by clinicians. Its diagnostic is based on clinical history, and differential diagnosis elimination. In 2017, an international workgroup of American Academy of Allergy, Asthma and Immunology published clinical criteria to specify the diagnosis and management. According to this last definition (JACI 2017), patient have to meet the major criterion and at least 3 minor criteria. Major criterion is vomiting in the 1- to 4-h period after ingestion of the suspect food and absence of classic IgE-mediated allergic skin or respiratory symptoms. Minor criteria are : 1. A second (or more) episode of repetitive vomiting after eating the same suspect food, 2. Repetitive vomiting episode 1-4 h after eating a different food 3. Extreme lethargy with any suspected reaction 4. Marked pallor with any suspected reaction 5. Need for emergency department visit with any suspected reaction 6. Need for intravenous fluid support with any suspected reaction 7. Diarrhea in 24 h (usually 5-10 h) 8. Hypotension 9. Hypothermia Skin prick test et IgE antibody are negative except atypical FPIES. Acute management begins with clinical evaluation, then administer normal saline bolus quickly. Parenteral ondansetron can be used to stop vomiting. Nutritional management implicate elimination of the offending foods. Only the oral food challenge in hospital can be done to determine resolution of FPIES after a long time of no symptom. The age of tolerance, depend of the food. The average age of acquiring tolerance for cow's milk changes in the literature, around 8-10 months in Korea, around 1 year in Israel, around 5 years in the United States. There is no data in France on the recovery age of CM-FPIES. However, there is a lack of information in literature for describe the evolution and atypical phenotypes. In addition, no prospective French series has been published to date. Our work is a national prospective study, which will collect news cases of acute FPIES diagnosis in sixteen French centres. Main objective: To determine the rate of acquisition of tolerance by food at 1 year, 2 years, 3 years post inclusion. Secondary objectives: - Description of a population of children with newly diagnosed FPIES. 2. Describe the rate of patients with FPIES progressing to IgE sensitization whatever the food at 1 year, 2 years, 3 years post inclusion. 3. Determine per food the rate of FPIES patients evolving towards IgE sensitization at 1 year, 2 years, 3 years post inclusion. 4. Describe the rate of patients with FPIES progressing to clinical symptoms of IgE-mediated allergy, whatever the food, at 1 year, 2 years, 3 years post inclusion. 5. Determine, by food, the rate of FPIES evolving towards clinical symptoms of IgE-mediated allergy at 1 year, 2 years, 3 years post inclusion. 6. Describe the rate of patients with multiple FPIES at each time point of the study. 7. Describe at each time the rates of patients with personal atopic comorbidities. The inclusion period will last three years, and the follow up of each patient will last three years. Allergologist will see the patient at inclusion visit, then one time a year. If the patient does not acquire tolerance, an oral food challenge (OFC) in hospital will lead to answer. The aim of our work will help allergologist to manage FPIES children, with French specificities in offending food, and tolerance.