Bensarsa, Sandra
[UCL]
VERSAVAU, Julia
[UCL]
Jacobs, Vanessa
[UCL]
Sokal, Etienne
[UCL]
INTRODUCTION Due to several legal, financial and ethical constraints, the setting up of clinical trials for a child population has provoked during many years debates on their objectives, effectiveness and limitations, while recognizing at the same time their necessity given the lack of availability of therapeutic options adapted to the pediatric domain. With the objective of obtaining marketing authorizations of new treatments for pediatric populations, the Authorities in charge of health agreed therefore on basic principles for the development of these trials; principally on safety and effectiveness of their conduct. AIM The aim of this thesis is two folds; (1) study and identify the effects of long-term experimental clinical trials on children from a medical, ethical, economic and psychosocial point of view and (2) assess the opinion of the families whose children were included in those studies. METHODS This thesis is based firstly on the comparative analysis of 6 different clinical studies on the development of hepatitis B and C therapeutic options conducted worldwide and proposed to 20 patients treated in the pediatric gastroenterology and hepatology unit of Saint-Luc University Clinics. This analysis will help us primarily to determine if, comparing to a larger scale, those clinical studies offered a successful outcome for our patients, and more generally to what extend proposing an experimental clinical trial to the children has helped them medically. This analysis is completed by a collection of questionnaires proposed to the parents of these children, gathering their feelings and concerns regarding these experiences from a prospective point of view. The survey developed in this thesis is composed of four parts: detailed understanding of the aims of the clinical trial proposed to the child, and both parents’ and child’s opinion on the quality of the documents received ; quality of the supervision during the experience ; potential impact on parents professional activities and children scholar schedule ; global opinion and parents’ views aiming at improving the clinical trials experiments. RESULTS For the retrospective analysis of this thesis, the experience has been positive for many of the children who accepted to participate to those studies (20 cases out of which 50 to 100% for the hepatitis C vs 25 to 100% for the hepatitis B trials who did the primary endpoint) with better control of the disease’s progression (lower viral charge, regression of hepatic lesions), good tolerability to the products and an increased probability of healing profile (seroconversion has been detected for 5 patients suffering from hepatitis B). As regards the prospective aspect of this study, 50% of parents agreed to complete the questionnaire proposed. The results are that 83.3% declared having been well informed, 100% considered the documents to be clear for adults and 83.3% estimated that their child received a ludic and appropriate document for its age. 50% confirmed that their child beneficiated from a better medical care because he was included in a clinical trial, while the other 50% considered that it didn’t make any difference. 83.6 to 66.66% estimated that the child’s clinical trial didn’t have a significant impact on their daily lives but commented that adjustments were required. Finally, 33.3% declared that this experiment had an impact on their child’s scholar activities. All in all, 100% expressed global satisfaction with these clinical trials. CONCLUSIONS This analysis allowed to conclude that as far as globally spread chronic diseases are concerned, these clinical trials represent an opportunity for therapeutic accessibility to new products not yet available to children or too expensive for them in long term use. For a large proportion of the patients that have participated to these experiments, the access to the treatment has improved their clinical condition and in particular the progression of the disease. These clinical studies have also provided future hope and an improved long-term prognosis for these children. They have stimulated a feeling of satisfaction for the parents who expressed comfort to the idea that their child has new chances for recovery and in the meantime, a possible guaranteed follow-up. However, these studies are costly. They request a daily adaptation for both the patients and their families for the application and follow up of the procedures required.


Bibliographic reference |
Bensarsa, Sandra ; VERSAVAU, Julia ; Jacobs, Vanessa ; Sokal, Etienne. Role/impact of pediatric clinical studies in the pediatric gastroenterology and hepatology department of Saint-Luc University Clinics.46ème congrès annuel de la Société Belge de Pédiatrie (Bruxelles, Belgique, du 08/03/2018 au 09/03/2018). |
Permanent URL |
http://hdl.handle.net/2078.1/196246 |