NCT07732361已完成不适用
A Secondary Real-world Data Study of Pediatric and Adult Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 307
- 试验地点
- 43
- 主要终点
- Overall Survival (OS)
研究概览
简要总结
The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and who were either complement inhibitor treatment naïve or had been treated with eculizumab.
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
- 4 Weeks 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be ≥ 28 days of age at the time of TMA diagnosis.
- •Body weight ≥ 5 kg at the time of TMA diagnosis.
- •Documented TMA on/after 01 Jan 2010 to 30 Jun 2024 with concurrent renal dysfunction with no documented alternative explanation ≤ 52 weeks from the HSCT.
- •Documentation of participant's vital status through 52 weeks after the date of TMA diagnosis.
- •Documentation of date and type of HSCT, date of TMA, absence of acute GVHD, and infection status at time of TMA diagnosis.
- •Documentation of at least 2 of the following: indication for most recent HSCT, serum creatinine at time of TMA diagnosis, presence of multiorgan dysfunction at time of TMA diagnosis.
- •Informed consent obtained if required by local regulations.
排除标准
- •Medical Conditions (Ongoing at the Time of TMA Diagnosis): TTP, ST-HUS, Immune-mediated hemolysis not due to TMA, DIC, Bone marrow/graft failure of HSCT, VOD (regardless of severity), HIV infection, Sepsis that required vasopressor support.
- •Prior/Concomitant Therapy: Received a complement inhibitor other than eculizumab from time of suspicion of HSCT-TMA diagnosis through 52 weeks post-HSCT-TMA diagnosis..
- •Other Exclusions: Participation in an investigational drug or device study for the treatment of TMA within 30 days prior to TMA diagnosis or during the 52 weeks following TMA diagnosis.
结局指标
主要结局
Overall Survival (OS)
时间窗: 52 weeks after TMA diagnosis
To assess OS through 52 weeks after TMA diagnosis.
次要结局
- Overall Survival (OS)(100 days and 26 weeks after TMA diagnosis)
- Non-relapse mortality(Through 100 days, 26 weeks, and 52 weeks after TMA diagnosis)
研究者
研究点 (43)
Loading locations...
相似试验
招募中
3 期
SIOP PNET 5 medulloblastoma2024-513724-42-00University Medical Center Hamburg-Eppendorf359
招募中
不适用
Pediatric-Inspired Regimen Combined With Venetoclax and Immunotherapy for Adult Ph-Negative Acute Lymphoblastic LeukemiaNCT07495631Institute of Hematology & Blood Diseases Hospital, China43
尚未招募
不适用
National Observatory of Children Hospitalized With BronchiolitisBronchiolitisNCT07485907Association Clinique Thérapeutique Infantile du val de Marne1,000
招募中
4 期
Safe, effective and cost-effective oxygen saturation targets for children and adolescents with respiratory distress: a randomized controlled trial.Lower respiratory tract infectionBronchiolitis2023-504817-56-00Spaarne Gasthuis560
尚未招募
不适用
Study of pneumonia in patients on ventilator life support in very sick children in Pediatric Intensive Care UnitCTRI/2025/03/082321300