Use of Eculizumab in Pediatric Patients with High-Risk Transplantation-Associated Thrombotic Microangiopathy: Outcomes and Risk Factors Associated with Response and Survival. A Retrospective Study on Behalf of the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC)

被引：7

作者：

Carabante, Maria Isabel Benitez ^{[1
]}

Bueno, David ^{[2
]}

Garcia, Laura Alonso ^{[1
]}

Torija, Ivan Lopez ^{[3
]}

Marsal, Julia ^{[4
]}

Navarro, Jose Maria Fernandez ^{[5
]}

Oficialdegui, Maria Luz Uria ^{[1
]}

Panesso, Melissa ^{[1
]}

Molina, Blanca ^{[6
]}

Bieler, Cristina Belendez ^{[7
]}

Palomo, Pilar ^{[8
]}

Martinez, Antonio Perez ^{[2
]}

Diaz-de-Heredia, Cristina ^{[1
]}

机构：

[1] Hosp Univ Vall dHebron, Vall dHebron Inst Recerca VHIR, Div Pediat Hematol & Oncol, Barcelona 08035, Spain

[2] Hosp Infantil Univ La Paz, Dept Pediat Hematol & Oncol, idiPAZ Res Inst, Madrid, Spain

[3] Hosp Santa Creu & Sant Pau, Dept Pediat Hematol & Oncol, Barcelona, Spain

[4] Hosp St Joan Deu, Dept Pediat Hematol & Oncol, Barcelona, Spain

[5] Hosp Univ & Politecn La Fe, Dept Pediat Hematol & Oncol, Valencia, Spain

[6] Hosp Infantil Univ Nino Jesus, Dept Pediat Hematol & Oncol, Madrid, Spain

[7] Hosp Gen Univ Gregorio Maranon, Dept Pediat Hematol & Oncol, Madrid, Spain

[8] Hosp Univ Cent Asturias, Dept Pediat Hematol & Oncol, Oviedo, Spain

来源：

TRANSPLANTATION AND CELLULAR THERAPY | 2024年 / 30卷 / 06期

关键词：

Hematopoietic stem cell transplantation (HSCT); Transplantation-associated thrombotic microangiopathy (TA-TMA); Eculizumab; Children; VERSUS-HOST-DISEASE; COMPLEMENT BLOCKADE; DIAGNOSTIC-CRITERIA; EUROPEAN-SOCIETY; CLINICAL-TRIALS; BLOOD; PATHOPHYSIOLOGY; SUSCEPTIBILITY; CHILDREN;

D O I：

10.1016/j.jtct.2024.03.019

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

ABS T R A C T Transplantation-associated thrombotic microangiopathy (TA-TMA) is associated with high morbidity and mortality. Although survival has improved significantly with the introduction of eculizumab, the need for improvement remains, especially in high-risk patients. This study aimed to describe the results obtained with eculizumab in a pediatric cohort with the attempt to define which risk factors could determine the response to treatment. We designed a national multicenter retrospective study of children treated with eculizumab for high-risk TA-TMA. The study cohort comprised 29 patients who had undergone a first (n = 28) or second (n = 1) allogeneic hematopoietic stem cell transplantation (HSCT) for malignant (n = 17) or nonmalignant (n = 12) disease. The median time from HSCT to TA-TMA diagnosis was 154 days (interquartile range [IQR], 103 to 263

引用

页码：601.e1 / 601.e13

页数：13

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