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

NCT07059026 | Recruiting

• 1 other identifier: ITAC-2024-01
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

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.

Conditions

Thrombotic Microangiopathy

Keywords

Thromobotic microangiopathy; Allogeneic Hematopoietic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• Incidence of TMA in adult patients receiving allogeneic HSCT in specialized centers in Argentina

  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.

  100 days post-HSCT (screening period)

Secondary Outcomes (6)

• 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)

Study Arms (1)

HSCT

Adults with Allogeneic Hematopoietic Stem Cell Transplantation

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All adult patients with Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) will be candidates for this study

You may qualify if:

• 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.

You may not qualify if:

• Participation in an interventional treatment study of any therapy for TMA.

Sponsors & Collaborators

• ITAC (Instituto de Trasplantes y Alta Complejidad): lead

Study Sites (1)

Instituto de Trasplantes de Alta Complejidad (ITAC)

Buenos Aires, C1425EGH, Argentina

RECRUITING

Related Publications (1)

• 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 and Marrow Transplantation, American Society for Transplantation and Cellular Therapy, Asia-Pacific Blood and Marrow Transplantation Group, and Center for International Blood and Marrow Transplant Research. Transplant Cell Ther. 2023 Mar;29(3):151-163. doi: 10.1016/j.jtct.2022.11.015. Epub 2022 Nov 25.

  PMID: 36442770 BACKGROUND

MeSH Terms

Conditions

Thrombotic Microangiopathies

Condition Hierarchy (Ancestors)

Thrombocytopenia; Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Central Study Contacts

Maria Belen Rosales Ostriz, MD

CONTACT

+54 9 11 5047-6401; belenrosalesostriz@gmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 12 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 1, 2025
• First Posted: July 10, 2025
• Study Start: December 3, 2024
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: July 14, 2025

Record last verified: 2025-07

Locations

AR (1)