Long-term Psychological and Cognitive Evaluation of Children Treated With Allogeneic Hematopoietic Stem Cell Transplantation for Immunodeficiency
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Standardized measure of intelligence quotient
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Years 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who underwent allogeneic transplantation for a primary immunodeficiency at Necker Hospital, regardless of the genetic diagnosis, and at least 2 years post Allogeneic hematopoietic stem cell transplantation.
- •Chronological age at the time of evaluation between 6 years and 8 years 11 months.
- •Holders of parental authority and children or adolescents or adults' patients informed and consenting to participate in the study
排除标准
- •Child transplanted for a condition other than the primary immunodeficiency or at a different center.
- •Presence of an associated acquired or genetic neurological condition, independent of the PID, likely to significantly impair cognitive development.
- •Severe uncorrected sensory impairment (auditory or visual) rendering the cognitive assessment uninterpretable.
- •Refusal to participate by the child or those with parental authority.
- •Child not fluent in French or not proficient enough in French to complete the cognitive tests and questionnaires.
- •Profound intellectual disability.
研究组 & 干预措施
Patients
Children between 6 and 8 years old who have received allogeneic hematopoietic stem cell transplantation for primary immunodeficiency for at least 2 years followed in the pediatric Immunohematology department of Necker Hospital (Assistance Publique-Hôpitaux de Paris).
干预措施: Neurocognitive, psychological, and psychosocial assessement (Other)
结局指标
主要结局
Standardized measure of intelligence quotient
时间窗: 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: * Verbal Comprehension Index (VCI) * Visual Spatial Index (VSI) * Fluid Reasoning Index (FRI) * Working Memory Index (WMI) * Processing Speed Index (PSI) Each index is standardized with a mean of 100 and a standard deviation (SD) of 15. Individual subtests have a mean score of 10 (SD 3). Higher scores indicate better cognitive performance. Interpretation of IQ scores: * \<70: Extremely low (clinically impaired) * 70-79: Borderline * 80-89: Low average * 90-109: Average * 110-119: High average * 120-129: Superior * ≥130: Very superior For subtest scaled scores: * 15-19: Very high * 12-14: High average * 9-11: Average * 7-8: Low average * 6: Borderline * 1-5: Impaired
次要结局
- Assessment of attention and executive functions(Time 0)
- Visual memory assessment and spatial organization(Time 0)
- Assessment of reading abilities(Time 0)
- Anxiety assessment(Time 0)
- Evaluation of behavioral functioning(Time 0)
- Assessment of autonomy and adaptability(Time 0)
- Assessment of socio-emotional skills(Time 0)
- Description of the experience of the illness and the transplant, and of the current psychological state(Time 0)
- Assessment of emotional and behavioral disorders(Time 0)
- Assessment of handwriting abilities(Time 0)