Clinical trial NCT03786549: Pediatric -Adult Care Transition Program of Patients With Sickle Cell Disease
Official title: Impact of a Pediatric-adult Care Transition Program on the Health Status of Patients With Sickle Cell Disease - A Randomized Controlled Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Sickle Cell Disease
Keywords: sickle cell disease (SDC)
Pediatric-adult care transition
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2018-12-26
Enrollment: 196
Sex: ALL
Minimum age (years): 16
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: HEALTH_SERVICES_RESEARCH

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of sickle cell related severe complications leading to hospitalization
timeFrame: Within 24 months after transfer to the adult sector
Secondary outcomes:
measure: Frequency of emergency visits in the index hospital
timeFrame: Up to 2 years
measure: Frequency of emergency visits in the index hospital
timeFrame: Within 12 months after transfer to the adult sector
measure: Frequency of emergency visits in the index hospital
timeFrame: Within 12 and 24 months after transfer to the adult sector
measure: Medication Intake Survey-Asthma (MIS-A) questionnaire score
timeFrame: At inclusion
measure: Medication Rating Scale (MARS) questionnaire score
timeFrame: At inclusion
measure: MIS-A questionnaire score
timeFrame: Up to 2 years
measure: MARS questionnaire score
timeFrame: Up to 2 years
measure: MIS-A questionnaire score
timeFrame: within 12 months after transfer to the adult sector
measure: MARS questionnaire score
timeFrame: within 12 months after transfer to the adult sector
measure: MIS-A questionnaire score
timeFrame: within 24 months after transfer to the adult sector
measure: MARS questionnaire score
timeFrame: within 24 months after transfer to the adult sector
measure: Number of days absent at school
timeFrame: Up to 2 years
measure: Number of days absent at school
timeFrame: within 12 months after transfer to the adult sector
measure: Number of days absent at school
timeFrame: within 24 months after transfer to the adult sector
measure: World Health Organization Quality of Life (WHOQOL) questionnaire score
timeFrame: At inclusion
measure: WHOQOL questionnaire score
timeFrame: Up to 2 years
measure: WHOQOL questionnaire score
timeFrame: within 24 months after transfer to the adult sector
measure: EUropean Health Literacy questionnaire (HLS-EU-Q16) score
timeFrame: At inclusion
measure: HLS-EU-Q16 score
timeFrame: Up to 2 years
measure: HLS-EU-Q16 score
timeFrame: within 24 months after transfer to the adult sector
measure: Disease knowledge
timeFrame: At inclusion
measure: Disease knowledge
timeFrame: Up to 2 years
measure: Disease knowledge
timeFrame: within 24 months after transfer to the adult sector
measure: Patient activation measure-13 items questionnaire score
timeFrame: At inclusion
measure: Patient activation measure-13 items questionnaire score
timeFrame: Up to 2 years
measure: Patient activation measure-13 items questionnaire score
timeFrame: within 24 months after transfer to the adult sector
measure: Self efficacy specific instrument - sickle cell disease (SCD-SES) questionnaire score
timeFrame: At inclusion
measure: SCD-SES questionnaire score
timeFrame: Up to 2 years
measure: SCD-SES questionnaire score
timeFrame: within 24 months after transfer to the adult sector
measure: Transition readiness assessment questionnaire (TRAQ) questionnaire score
timeFrame: At inclusion
measure: TRAQ questionnaire score
timeFrame: Up to 2 years
measure: TRAQ questionnaire score
timeFrame: within 24 months after transfer to the adult sector
measure: cost effectiveness ratio
timeFrame: Up to 4 years
measure: number of pediatric-adult transition program sessions performed
timeFrame: Up to 4 years
measure: type of pediatric-adult transition program sessions performed
timeFrame: Up to 4 years
Other outcomes:
Locations:
city: Fort-de-France-La Martinique
country: France
facility: CHU de Fort de France
state: La Martinique
city: Créteil
country: France
facility: Centre Hospitalier Intercommunal de Creteil
city: Créteil
country: France
facility: Hôpital Mondor
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre
city: Lyon
country: France
facility: Hospices Civils de Lyon
city: Paris
country: France
facility: Hôpital Européen Georges Pompidou
city: Paris
country: France
facility: Hôpital Necker
city: Pontoise
country: France
facility: Centre Hospitalier de Pontoise
Study officials:
References:
citation: Hoegy D, Bleyzac N, Gauthier-Vasserot A, Cannas G, Denis A, Hot A, Bertrand Y, Occelli P, Touzet S, Dussart C, Janoly-Dumenil A; DREPADO study group. Impact of a paediatric-adult care transition programme on the health status of patients with sickle cell disease: study protocol for a randomised controlled trial (the DREPADO trial). Trials. 2020 Feb 10;21(1):152. doi: 10.1186/s13063-019-4009-9.