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

Официальное название

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

Обзор

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.

Подробное описание

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).

Первичные конечные точки

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

Вторичные конечные точки (6)

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

Критерии участия

Критерии включения

• 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.

Критерии исключения

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Аргентина · 1 центр

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

Публикации

• 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

Идентификаторы

NCT: NCT07059026 · ITAC-2024-01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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