Prospective Evaluation of Transplant Associated Thrombotic Microangiopathy (TA-TMA) Markers in a High-risk Cohort of Adults Undergoing Allogeneic Stem Cell Transplantation (SCT)
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
- 1
- 主要终点
- Primary Objectives
研究概览
简要总结
The goal of this observational study is to learn about the incidence of Transplant Associated Thrombotich Microangiopathy (TA-TMA), which is a known but underreported complication of Allogeneic Stem Cell Transplant (SCT). The main question it aims to answer is:
What is the incidence of TA-TMA in adults undergoing SCT? How does TA-TMA diagnosis impact survival and other outcomes? Patients undergoing SCT will be eligible for this study, which will consist of collection of biological samples and standard clinical follow up.
详细描述
This will be a prospective non-interventional study that will include patients undergoing allogeneic stem cell transplant (SCT) deemed to be at high risk for developing Transplant Associated Thrombotic Microangiopathy (TA-TMA). All patients will have prospective biospecimens collected per study schedule starting prior to SCT. Biospecimens will be collected for use in translational testing and sent to Viracor for commercial testing. Results of commercial testing will be made available to treating physicians. Diagnosis of TA-TMA will be made based on clinical and laboratory values according to existing consensus guidelines, however, therapeutic decisions will be left up to the treating clinical team. Patients will be followed up for clinical outcomes.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Inclusion Criteria
- •Adult male or female, age ≥18
- •Undergoing allogeneic SCT at UAB for any indication and any donor source.
- •For patients in cohort 1: Patients deemed high-risk for developing high-risk TA-TMA as stated by any of the following criteria*:
- •BMI > 35[42]
- •Mismatched donor (either Haploidentical or Mismatched Unrelated donor)[18, 29]
- •Non-Malignant Transplant Indication (Severe Aplastic Anemia or Sickle Cell Disease)[18, 43]
- •Acute Lymphoblastic Leukemia (any kind)[18, 31]
- •African American, Hispanic, Asian or Native American Ethnicity [44]
- •Myeloablative Conditioning Regimen[18]
- •Pre-Existing Renal Disease** (defined as any of the following: 24 Hr Creatinine Clearance <60, baseline serum creatinine > 1.2, 24 hr proteinuria >150mg or random spot urine Protein Creatinine ratio > 150mg/g)[20, 45]
- •TBI-containing conditioning regimen >400cGy [46]
- •Prior Autologous or Allogeneic SCT[45]
- •*Some of these criteria have been adapted from pediatric literature due to a higher number of publications in that setting and in cases where the adult data is lacking or contradictory.
- •**Proteinuria thresholds are obtained from KDIGO guidelines and include moderate and severe levels of proteinuria[47].
- •Females of childbearing potential must have a negative urine or serum pregnancy test prior to enrollment.
- •For patients in cohort 2: Patients who develop GVHD with any of the following characteristics and were not included in cohort 1
- •Grade III-IV aGVHD, or SR-aGVHD of any grade, whichever occurs first
- •Moderate to Severe cGVHD or SR-cGVHD of any grade, whichever occurs first
- •For patients in cohort 3: Patients included in either cohort 1 or 2 who are diagnosed with TA-TMA and receive eculizumab
排除标准
- •Exclusion Criteria
- •Any other active malignancy requiring treatment or with expected survival ≤1 year.
- •Patients with psychiatric illness or social situations that would limit compliance with the study requirements.
研究组 & 干预措施
Cohort 3
Patients included in either cohort 1 or 2 who are diagnosed with TA-TMA and receive eculizumab
Cohort 2
Patients who develop GVHD with any of the following characteristics and were not included in cohort 1
- Grade III-IV aGVHD, or SR-aGVHD of any grade, whichever occurs first
- Moderate to Severe cGVHD or SR-cGVHD of any grade, whichever occurs first
Cohort 1
Main Cohort of patients who fulfill Inclusion Criteria
结局指标
主要结局
Primary Objectives
时间窗: From enrollment until 1 year post SCT
The primary objective of the study is to report the rates of high-risk TA-TMA in a cohort for high-risk patients undergoing allogeneic SCT. High-risk TA-TMA will be defined as per the ASTCT Harmonization Criteria which will include the TMA Harmonization panel consensus recommended diagnostic criteria and any of the high-risk TA-TMA features (Schoettler et al. TCT March 2023).
Co-Primary Objective
时间窗: From enrollment until 1 year post SCT
To evaluate rates of Non-Relapse Mortality (NRM) in patients with and without TA-TMA. NRM is defined as the rate of death from any cause in patients who do not exhibit any sign of relapse or progression of their underlying hematologic malignancy
次要结局
- Secondary Objectives(From enrollment until 1 year post SCT)
- Secondary Objective(From enrollment until 1 year post SCT)
研究者
Manuel Ricardo Espinoza-Gutarra
Assistant Professor
University of Alabama at Birmingham