Health-Related Quality of Life and Clinical Utilization in Pediatric and Young Adult Patients With Hereditary Hemorrhagic Telangiectasia
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- PedsQL Total Score
研究概览
简要总结
This observational study evaluates health-related quality of life (HR-QoL) in pediatric and young adult patients aged 2-25 years with Hereditary Hemorrhagic Telangiectasia (HHT). Eligible participants are patients receiving care at Cincinnati Children's Hospital Medical Center and / or their caregivers. Participants will complete validated quality-of-life questionnaires assessing physical, emotional, social, and disease-specific functioning over the past 30 days. A paired retrospective chart review will assess disease severity and clinical utilization, including procedures and imaging studies. The primary objective is to describe mean QoL scores for this population. Secondary objectives include evaluating associations between QoL scores, disease severity, and clinical utilization.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 2 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •• Patient aged 2-25 years with a confirmed (either genetic or clinical) diagnosis of definite HHT
- •Parent or legal guardian willing and able to complete the caregiver survey for patients aged 2-
- •For patients >18, willing and able to complete the patient survey.
- •Receipt of care through CCHMC (at least one visit with genetic counselor or director of HHT Center)
- •Ability to complete survey in English
- •For self-report: patient age greater than or equal to 8 years old that assent to survey.
排除标准
- •Patients that are older than 25 years old.
- •Patients that are younger than 2 years old.
- •Individuals without definite HHT diagnosis.
结局指标
主要结局
PedsQL Total Score
时间窗: 30 days
Mean total health-related quality of life score measured using the Pediatric Quality of Life Inventory (PedsQL). Scores range from 0-100, with lower scores indicating worse quality of life.
HHT-QOL
时间窗: past 30 days
Mean score ranging from 0-16 assessing HHT-related symptom burden and functional impact. Higher scores indicate worse impairment.
次要结局
- Epistaxis Severity Score(past 30 days)
- HHT Severity Score(lifetime)
- Clinical Utilization(lifetime)
研究者
Ashley Nelson
Sponsor-Investigator
Children's Hospital Medical Center, Cincinnati