Prospective Study of Thrombotic Microangiopathy (TMA) Associated With Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Adult Patients From Argentina
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Enrollment
- 200
- Locations
- 1
- Primary Endpoint
- Incidence of TMA in adult patients receiving allogeneic HSCT in specialized centers in Argentina
Study Overview
Brief Summary
Thrombotic Microangiopathy (TMA) Associated with Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) is a serious complication that is associated with increased morbidity, related to multiple organ failure, with increased mortality in transplant patients. The incidence and evolution of TMA, especially in the adult population, is unclear due to the lack of early systematic screening and clear criteria for its diagnosis. For this reason, we designed this protocol to study the incidence and evolution of TMA Associated with allogeneic HSCT in adult patients from Argentina.
Detailed Description
This is a prospective multicenter non-interventional observational study that will include sites that perform routine screening using harmonized definitions and diagnostic criteria during the first 100 days post-HSCT (screening period).
All patients entering the study (HSCT accessible population), regardless of suspicion or diagnosis of TMA, will be followed every 3 months up to 12 months after transplant to evaluate secondary outcomes (follow-up period).
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Adult patients (≥ 18 years) undergoing allogeneic HSCT in specialized centers that, as part of their usual follow-up protocol, carry out basic screening (laboratory/clinical) for TMA and in which patients consent (within the general transplant consent), to have their data used in observational studies.
- •Patients with ≥ 3/laboratory/clinical diagnostic markers of TMA in two consecutive assessments within 14 days, namely: 1-Elevated Schistocytes in peripheral blood; 2-LDH above the upper normal limit; 3-De novo thrombocytopenia or requirement for platelet transfusion; 4-De novo anemia or requirement for red blood cell transfusion; 5-High blood pressure (≥140/90); 6-Protein/creatinine ratio > 1mg/mg or proteinuria ≥ 30mg/dl in a random sample).
- •Patients with suspected/diagnosed TMA who have signed the specific consent for the study.
Exclusion Criteria
- •Participation in an interventional treatment study of any therapy for TMA.
Outcomes
Primary Outcomes
Incidence of TMA in adult patients receiving allogeneic HSCT in specialized centers in Argentina
Time Frame: 100 days post-HSCT (screening period)
The incidence of TMA will be assessed by the presence of ≥ 4/diagnostic laboratory/clinical markers present in 2 successive assessments within 14 days or if TMA is identified histologically by tissue biopsy up to day 100 post-transplant. Laboratory/clinical diagnostic markers (Harmonizing Definitions for Diagnostic Criteria and Prognostic Assessment of Transplantation-Associated published by Schoettler et al Transplant Cell Ther. 2023) are: 1-Elevated schistocytes in peripheral blood; 2-LDH above the upper limit of normal; 3-De novo thrombocytopenia or requirement for platelet transfusion; 4-De novo anemia or requirement for red blood cell transfusion; 5-High blood pressure (≥140/90); 6-Protein/creatinine ratio \> 1 mg/mg or proteinuria ≥ 30 mg/dl in a random sample; 7-sC5b-9 above the upper limit of normal.
Secondary Outcomes
- Overall Survival(3, 6, 9, 12 months (follow-up period))
- Non-relapse Mortality(3, 6, 9, 12 months (follow-up period))
- Proportion of patients achieving TMA response(3, 6, 9, 12 months (follow-up period))
- Proportion of patients free of transfusion requirement(3, 6, 9, 12 months (follow-up period))
- Proportion of patients with high-risk TMA,(3, 6, 9, 12 months (follow-up period))
- Proportion of patients with multiple organ dysfunction(3, 6, 9, 12 months (follow-up period))