Thrombotic Microangiopathy (TMA) Associated With Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Adult Patients

Official title

Prospective Study of Thrombotic Microangiopathy (TMA) Associated With Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Adult Patients From Argentina

Overview

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).

Primary outcome measures

• Incidence of TMA in adult patients receiving allogeneic HSCT in specialized centers in Argentina [Time frame: 100 days post-HSCT (screening period)]

Secondary outcome measures (6)

• Overall Survival [Time frame: 3, 6, 9, 12 months (follow-up period)]
• Non-relapse Mortality [Time frame: 3, 6, 9, 12 months (follow-up period)]
• Proportion of patients achieving TMA response [Time frame: 3, 6, 9, 12 months (follow-up period)]
• Proportion of patients free of transfusion requirement [Time frame: 3, 6, 9, 12 months (follow-up period)]
• Proportion of patients with high-risk TMA, [Time frame: 3, 6, 9, 12 months (follow-up period)]
• Proportion of patients with multiple organ dysfunction [Time frame: 3, 6, 9, 12 months (follow-up period)]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Argentina · 1 center

• Instituto de Trasplantes de Alta Complejidad (ITAC) — Buenos Aires

Publications

• Schoettler ML, Carreras E, Cho B, Dandoy CE, Ho VT, Jodele S, Moissev I, Sanchez-Ortega I, Srivastava A, Atsuta Y, Carpenter P, Koreth J, Kroger N, Ljungman P, Page K, Popat U, Shaw BE, Sureda A, Soiffer R, Vasu S. Harmonizing Definitions for Diagnostic Criteria and Prognostic Assessment of Transplantation-Associated Thrombotic Microangiopathy: A Report on Behalf of the European Society for Blood PMID 36442770

Identifiers

NCT: NCT07059026 · ITAC-2024-01

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗