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NCT Number: NCT07059026

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

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.

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Key information

Conditions

Thrombotic Microangiopathy Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Thrombocytopenia Thrombotic Microangiopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Instituto de Trasplantes de Alta Complejidad (ITAC)

Buenos Aires, C1425EGH, Argentina

Location status: Recruiting

Location contact

Maria Belen Rosales Ostriz, MD

CONTACT

[email protected]

+54 9 11 5047-6401

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. 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

  1. Overall Survival

    Time frame: 3, 6, 9, 12 months (follow-up period)

    Proportion of TMA patients alive at each evaluation time frame

  2. Non-relapse Mortality

    Time frame: 3, 6, 9, 12 months (follow-up period)

    Proportion of TMA patients that have died (death from any cause with exception of death due to progression or recurrence of the underlying disease) at each evaluation time frame

  3. Proportion of patients achieving TMA response

    Time frame: 3, 6, 9, 12 months (follow-up period)

    TMA response is defined through the presence of response criteria (platelet count ≥ 50,000/mm3, lactate dehydrogenase < 1.5 upper limit of normal, absence of schistocytes (if present at baseline) and increase in eGFR ≥ 30% from baseline or interruption of dialysis for patients on dialysis treatment at baseline), at each evaluation time frame

  4. Proportion of patients free of transfusion requirement

    Time frame: 3, 6, 9, 12 months (follow-up period)

    Assessed through the non-requirement of transfusion of red blood cells or platelets indicated for the management of TMA for ≥ 28 days, at each evaluation time frame (Transfusions required for causes other than TMA will not be considered within the evaluation of this endpoint)

  5. Proportion of patients with high-risk TMA,

    Time frame: 3, 6, 9, 12 months (follow-up period)

    Assessed using the following criteria: LDH > 2 upper limit of normal; Protein/creatinine ratio > 1 mg/mg or proteinuria ≥ 30 mg/dl in a random sample; Any multi-organ dysfunction except grade 1 acute kidney injury; sC5b-9 above the upper limit of normal; Grade II-IV acute graft-versus-host disease, systemic viral or bacterial infection, evaluated at any time up to 12 months after HSCT

  6. Proportion of patients with multiple organ dysfunction

    Time frame: 3, 6, 9, 12 months (follow-up period)

    Assessed through specific definitions (Schoettler et al Transplant Cell Ther. 2023)

Study contacts

Contact information is provided by the study sponsor or research team.

Maria Belen Rosales Ostriz, MD

CONTACT

[email protected]

+54 9 11 5047-6401

Sponsors and collaborators

Lead sponsor

ITAC (Instituto de Trasplantes y Alta Complejidad)

Other

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Jul 10, 2025
Registry last updated
Jul 14, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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