Pediatric -Adult Care Transition Program of Patients With Sickle Cell Disease

Study Overview
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Brief Summary
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Background The pediatric-adult care transition is a risk-disrupting time for patients with chronic disease. This care transition takes place during adolescence; a period of psychological upheavals and adaptations of family roles. During this period, medication adherence is non-optimal and absenteeism at medical appointments is high. Sickle cell disease (SCD) is the first genetic disease detected in France. It is chronic disease characterized by frequent painful vaso-occlusive crises (VOC) requiring emergency hospitalization when they are severe. Other serious complications are acute chest syndromes (ACS) and stroke. In order to improve the health status of teenagers with sickle cell disease, it is necessary to anticipate this care transition and to involve the pediatric and adult sectors. The biopsychosocial health approach and the Social-Ecological Model of Adolescent and Young Adult Readiness to Transition (SMART) describe a care transition integrating bioclinical and psychosocial factors such as integration of the patient's family, education on disease and therapeutics, psychological management of pain and medico-social orientation. The pediatric-adult transition program proposed is based on this biopsychosocial approach. It aims to improve the health status of adolescents with SCD, their quality of life and the use of health care service. Objective of the study To assess the impact of a pediatric-adult transition program on the incidence of sickle-cell-related complications leading to hospitalization on 24-months after transfer to the adult sector. The evaluation focuses on severe complications leading to hospitalization, such as VOC, ACS, and stroke. Study design Multicenter Open-label individual Randomized Controlled Trial Population : Patients aged at least 16 years old with sickle cell disease, and their parents (or legal representatives Number of subject : 196 patients (98 patients by arm) The study will last 24 months Expected results For patients and families Better health and quality of life for patients is expected, including better use of medical care after the transition program. It is also expected a better experience of the pediatric-adult care transition and indirectly a better experience of intrafamilial relations. For health professionals This project is expected to provide solutions to improve the pediatric-adult care transition of patients with chronic disease. Indeed, the methodological quality of the study will make it possible to evaluate the efficiency of the proposed program, to possibly adapt it and test it to other chronic diseases presenting the same care transition problematic. In terms of public health SCD mainly affects populations of sub-Saharan origin, with low visibility and high social vulnerability. By focusing on this population, this project will reduce the social inequalities in health, experienced by patients with SCD and their families. By improving the health, quality of life and care of patients with SCD, this project is expected to decrease the cost of the pediatric-adult care transition period.

Official Title
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Impact of a Pediatric-adult Care Transition Program on the Health Status of Patients With Sickle Cell Disease - A Randomized Controlled Trial

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Other: pediatric-adult care transition program


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: For patients : Age: 16-17 years, With major sickle cell syndrome, defined by hemoglobinopathy of homozygosity SS, or double heterozygosity SC or Sβ-thalassemia, Benefiting from social insurance of the type Affection of long duration (ALD). For family members : Included children's parents or legal representatives, Accepting to participate in the study and having signed the informed consent. Exclusion Criteria: Presenting a cognitive or psychiatric disorder known and major that may hinder interventions or evaluation, the judgment of the investigator, and / or having a family history with this type of disorders, Cured of SCD by an allograft of hematopoietic stem cells.

Ages Eligible for Study
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Minimum Age: 16 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Health Services Research
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| No Intervention: Control<br>Patients included in this arm wil have usual follow-up. | |
| Experimental: Care transitional program<br>Patients included in this arm will get a care transitional program. Three structured axes of multidisciplinary interventions are added to the usual follow-up for the patients drawn in this interventional arm. Those axes integrate the bioclinical medical care and include the parents of the adolescent Three axes are : Educative, family (patient and parent), at home Psychological, with the patient individually Medico-social orientation, group of patients | Other: pediatric-adult care transition program<br>* Three structured axes of multidisciplinary interventions are added to the usual follow-up for the patients drawn in this interventional arm. Those axes integrate the bioclinical medical care and include the parents of the adolescent Three axes are : Educative, family (patient and parent), at home Psychological, with the patient individually Medico-social orientation, group of patients<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of sickle cell related severe complications leading to hospitalization | Number of hospital admission or emergency visit in the index hospital | Within 24 months after transfer to the adult sector | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of emergency visits in the index hospital | Frequency of emergency visits in the index hospital Within inclusion and transfer to the adult sector | Up to 2 years | 
| Frequency of emergency visits in the index hospital |  | Within 12 months after transfer to the adult sector | 
| Frequency of emergency visits in the index hospital |  | Within 12 and 24 months after transfer to the adult sector | 
| Medication Intake Survey-Asthma (MIS-A) questionnaire score | Medication adherence evaluation at inclusion | At inclusion | 
| Medication Rating Scale (MARS) questionnaire score | Medication adherence evaluation at inclusion | At inclusion | 
| MIS-A questionnaire score | Medication adherence evaluation At transfer to the adult sector | Up to 2 years | 
| MARS questionnaire score | Medication adherence evaluation At transfer to the adult sector | Up to 2 years | 
| MIS-A questionnaire score | Medication adherence evaluation within 12 months after transfer to the adult sector | within 12 months after transfer to the adult sector | 
| MARS questionnaire score | This score will allow medication adherence evaluation within 12 months after transfer to the adult sector | within 12 months after transfer to the adult sector | 
| MIS-A questionnaire score | Medication adherence evaluation within 24 months after transfer to the adult sector | within 24 months after transfer to the adult sector | 
| MARS questionnaire score | Medication adherence evaluation within 24 months after transfer to the adult sector | within 24 months after transfer to the adult sector | 
| Number of days absent at school | Scholarly Absenteeism evaluation At transfer to the adult sector | Up to 2 years | 
| Number of days absent at school | Scholarly Absenteeism evaluation within 12 months after transfer to the adult sector | within 12 months after transfer to the adult sector | 
| Number of days absent at school | Scholarly Absenteeism evaluation within 24 months after transfer to the adult sector | within 24 months after transfer to the adult sector | 
| World Health Organization Quality of Life (WHOQOL) questionnaire score | Quality of Life evaluation At inclusion | At inclusion | 
| WHOQOL questionnaire score | Quality of Life evaluation At transfer to the adult sector | Up to 2 years | 
| WHOQOL questionnaire score | Quality of Life evaluation within 24 months after transfer to the adult sector | within 24 months after transfer to the adult sector | 
| EUropean Health Literacy questionnaire (HLS-EU-Q16) score | This will allow Health Literacy evaluation | At inclusion | 
| HLS-EU-Q16 score | Health Literacy evaluation At transfer to the adult sector | Up to 2 years | 
| HLS-EU-Q16 score | Health Literacy evaluation within 24 months after transfer to the adult sector | within 24 months after transfer to the adult sector | 
| Disease knowledge | Questionnaire developed for this study | At inclusion | 
| Disease knowledge | Questionnaire developed for this study | Up to 2 years | 
| Disease knowledge | Questionnaire developed for this study | within 24 months after transfer to the adult sector | 
| Patient activation measure-13 items questionnaire score | Patient activation At inclusion | At inclusion | 
| Patient activation measure-13 items questionnaire score | Patient activation At transfer to the adult sector | Up to 2 years | 
| Patient activation measure-13 items questionnaire score | Patient activation within 24 months after transfer to the adult sector | within 24 months after transfer to the adult sector | 
| Self efficacy specific instrument - sickle cell disease (SCD-SES) questionnaire score | Self efficacy evaluation at inclusion | At inclusion | 
| SCD-SES questionnaire score | Self efficacy evaluation At transfer to the adult sector | Up to 2 years | 
| SCD-SES questionnaire score | Self efficacy evaluation within 24 months after transfer to the adult sector | within 24 months after transfer to the adult sector | 
| Transition readiness assessment questionnaire (TRAQ) questionnaire score | Transition readiness evaluation at inclusion | At inclusion | 
| TRAQ questionnaire score | Transition readiness evaluation At transfer to the adult sector | Up to 2 years | 
| TRAQ questionnaire score | Transition readiness evaluation within 24 months after transfer to the adult sector | within 24 months after transfer to the adult sector | 
| cost effectiveness ratio | Cost analysis at the end of the study | Up to 4 years | 
| number of pediatric-adult transition program sessions performed | number of interventions performed per patient and date of implementation At the end of the study | Up to 4 years | 
| type of pediatric-adult transition program sessions performed | type of interventions performed per patient and date of implementation At the end of the study | Up to 4 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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sickle cell disease (SDC), Pediatric-adult care transition