Effect of Exchange Transfusions on Cognitive Efficiency in Patients With Sickle Cell Disease
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 85
- 试验地点
- 6
- 主要终点
- Information Processing Speed
研究概览
简要总结
DREPA-COG is an observational study evaluating the effect of exchange transfusions on cognitive function in adults with severe sickle cell disease (SS or Sβ0). Information processing speed is assessed at three time points during the transfusion cycle using the Symbol Digit Modalities Test (SDMT) and additional validated neuropsychological measures. This minimal-risk, fully remote study aims to identify processing speed as a reproducible marker for clinical monitoring and future therapeutic trials.
详细描述
DREPA-COG is a non-interventional, multicenter observational study investigating the effect of exchange transfusions on cognitive performance in adults with severe sickle cell disease (SS or Sβ0). Cognitive complications, including slowed information processing and executive dysfunction, are frequent and disabling in this population. Exchange transfusion, a standard therapy replacing sickled red blood cells with donor cells, may positively influence cognition, but systematic evidence is lacking.
The study evaluates information processing speed as a potential neurological marker across the transfusion cycle. Participants complete three brief neuropsychological assessments remotely by videoconference: before the transfusion, one week after, and prior to the next transfusion. The Symbol Digit Modalities Test (SDMT) is the primary measure, complemented by validated assessments of global cognition, attention, memory, fatigue, mood, and pain.
Primary analysis will use mixed-effects models to assess changes in processing speed over time, adjusting for demographic and clinical covariates. This minimal-risk, fully remote study requires no additional hospital visits and aims to establish processing speed as a reproducible and clinically relevant outcome measure in sickle cell disease.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults (≥18 years) with severe sickle cell disease (SS or Sβ0).
- •Enrolled in a regular monthly transfusion exchange program.
- •Not institutionalized.
- •No known dementia.
- •No severe aphasia.
- •Affiliated with or beneficiary of a social security system.
- •Exclusion criteria:
- •Insufficient mastery of spoken French.
- •Severe comorbidities preventing short-term follow-up.
- •Known psychiatric disorders.
- •Participation in another clinical study with ongoing exclusion periods.
- •Lack of adequate computer equipment (minimum 11-inch screen and internet connection).
- •Vulnerable patients under legal protection (guardianship or curatorship).
排除标准
- 未提供
结局指标
主要结局
Information Processing Speed
时间窗: within 48 hours before the next scheduled transfusion exchange
Primary outcome measure: Information processing speed, assessed using the oral version of the Symbol Digit Modality Test (SDMT), as a marker of cerebral involvement in adult patients with sickle cell disease.
次要结局
- Global Cognitive Function - Montreal Cognitive Assessment (Telephone version, T-MoCA)(Time Point 1 (Baseline): within 48 hours before the scheduled exchange transfusion. Time Point 2 (Post-transfusion): 7 to 9 days after the exchange transfusion. Time Point 3 (End of cycle): within 48 hours before the next scheduled exchange transfusion)
- Fatigue Severity Score (FSS)(Time Point 1 (Baseline): within 48 hours before the scheduled exchange transfusion. Time Point 2 (Post-transfusion): 7 to 9 days after the exchange transfusion. Time Point 3 (End of cycle): within 48 hours before the next scheduled exchange transfusion)
- Anxiety and Depression Symptoms (Hospital Anxiety and Depression Scale, HADS(Time Point 1 (Baseline): within 48 hours before the scheduled exchange transfusion. Time Point 2 (Post-transfusion): 7 to 9 days after the exchange transfusion. Time Point 3 (End of cycle): within 48 hours before the next scheduled exchange transfusion)
- Pain Intensity (Numeric Rating Scale, NRS)(Time Point 1 (Baseline): within 48 hours before the scheduled exchange transfusion. Time Point 2 (Post-transfusion): 7 to 9 days after the exchange transfusion. Time Point 3 (End of cycle): within 48 hours before the next scheduled exchange transfusion)
- Change in Information Processing Speed Measured by the Symbol Digit Modalities Test (SDMT)(Within 48 hours before exchange transfusion 7-9 days after exchange transfusion Within 48 hours before the next scheduled exchange transfusion)
- Working Memory - WAIS-IV Digit Span Subtest(Time Point 1 (Baseline): within 48 hours before the scheduled exchange transfusion. Time Point 2 (Post-transfusion): 7 to 9 days after the exchange transfusion. Time Point 3 (End of cycle): within 48 hours before the next scheduled exchange transfusion)