Hôpital Necker-Enfants Malades
Paris, 75015, France
NCT Number: NCT07766304
Primary immunodeficiencies (PIDs) are a large group of genetic diseases of the immune system with highly variable clinical presentations. Allogeneic hematopoietic stem cell transplantation (HSCT) is one of the treatments offered to some patients with PIDs. It is a curative but particularly demanding treatment, potentially life-threatening, requiring several months of hospitalization, prolonged limitations in social interactions for the patient, and impacting the entire family unit. The short-term complications of HSCT are numerous and well-known. However, few studies describe the long-term psychological and cognitive complications of HSCT, particularly in the context of PIDs. The few published studies in children concern patients transplanted for hematological malignancies, a context very different from that of primary immunodeficiencies.
This study is a pilot research project focusing on the multidimensional assessment of the neurocognitive, psychological, and psychosocial functioning of children between 6 and 8 years old who have received allogeneic hematopoietic stem cell transplantation for primary immunodeficiency for at least 2 years. These stringent criteria aim to limit biases related to the diversity of ages at which care is provided.
Trial opening soon.
Get Notified6 year–8 year
All sexes
Observational
Paris, 75015, France
Primary immunodeficiencies (PIDs) are a large group of genetic diseases of the immune system with highly variable clinical presentations. Allogeneic hematopoietic stem cell transplantation (HSCT) is one of the treatments offered to some patients with PIDs. It is a curative but particularly demanding treatment, potentially life-threatening, requiring several months of hospitalization, prolonged limitations in social interactions for the patient, and impacting the entire family unit. The short-term complications of HSCT are numerous and well-known. However, few studies describe the long-term psychological and cognitive complications of HSCT, particularly in the context of PIDs. The few published studies in children concern patients transplanted for hematological malignancies, a context very different from that of primary immunodeficiencies.
This study is a pilot research project focusing on the multidimensional assessment of the neurocognitive, psychological, and psychosocial functioning of children between 6 and 8 years old who have received allogeneic hematopoietic stem cell transplantation for primary immunodeficiency for at least 2 years. These stringent criteria aim to limit biases related to the diversity of ages at which care is provided.
The study will take place within the pediatric immuno-hematology department of Necker-Enfants Malades Hospital (Assistance Publique-Hôpitaux de Paris) during a day hospital visit as part of routine follow-up.
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
Time frame: Time 0
Assessment of global intellectual functioning using the Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition (WPPSI-IV) or the Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V), according to the participant's age.
The Full-Scale IQ is derived from five cognitive indices:
Higher scores indicate better cognitive performance.
Interpretation of IQ scores:
For subtest scaled scores:
Time frame: Time 0
Assessment of sustained attention, selective attention and executive attention using the Test of Everyday Attention for Children (TEA-Ch).
The assessment includes the following subtests:
Interpretation:
Time frame: Time 0
Assessment of visuospatial construction and visual memory using the Rey-Osterrieth Complex Figure Test.
The following measures are analyzed:
Time frame: Time 0
Reading abilities are assessed using the Alouette-R Reading Test.
Outcome measures include:
Alouette-R
Time frame: Time 0
Assessment of anxiety using the Revised Children's Manifest Anxiety Scale (R-CMAS).
The questionnaire evaluates:
Interpretation:
Time frame: Time 0
Assessment of behavioral functioning using the Conners 3rd Edition (Conners-3).
The questionnaire evaluates:
Interpretation:
Time frame: Time 0
Assessment of adaptive functioning using the Vineland Adaptive Behavior Scales, Second Edition (Vineland-II).
The assessment evaluates adaptive functioning across three domains:
Interpretation of v-scale scores:
Time frame: Time 0
Assessment of socio-emotional competencies using the Social Skills Improvement System, Social-Emotional Learning Edition (SSIS-SEL).
The assessment evaluates five core domains:
Time frame: Time 0
Qualitative analysis of the semi-structured interview with the child and then with the family, focused on the experience of the illness and the transplant and on the current psychological state.
Time frame: Time 0
Assessment of emotional and behavioral functioning using the Child Behavior Checklist (CBCL).
The following syndrome scales and composite scores are evaluated:
Results are expressed as standardized T-scores. Higher T-scores indicate greater emotional or behavioral difficulties.
Interpretation:
Time frame: Time 0
Handwriting abilities are assessed using the BHK Handwriting Test.
Outcome measures include:
Contact information is provided by the study sponsor or research team.
Assistance Publique - Hôpitaux de Paris
Other
Acronym: PSY-DIP
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View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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