ArticleBlood advances2021
Transplant-associated thrombotic microangiopathy in pediatric patients: pre-HSCT risk stratification and prophylaxis.
Article in Blood advances, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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13 citing papers in PubMed, 23 citations in OpenAlex.
- Early Echocardiographic and Serum Biomarkers Predict Thrombotic Microangiopathy, Endotheliopathy, and Survival After Pediatric Hematopoietic Stem Cell Transplant.Transplantation and cellular therapy · 2026Article
- Complement-targeted therapies for transplant-associated thrombotic microangiopathy: recent advances.Frontiers in pharmacology · 2026Review
- Infection-associated sTNFR1 elevation predicts post-transplant thrombotic microangiopathy in severe aplastic anemia.Frontiers in immunology · 2026Article
- Detecting Endothelial Compromise Objectively through Dynamic EASIX scoring (DECODE) - predictive utility of EASIX and modified EASIX scores for endothelial complications post hematopoietic cell transplant.Frontiers in oncology · 2026Article
- Oxidative Stress Early After Hematopoietic Stem Cell Transplant.Transplantation and cellular therapy · 2025Article
- Complement activation and vascular complications after pediatric allogeneic hematopoietic stem cell transplantation.Scientific reports · 2025Article
- Age, GVHD prophylaxis, and timing matter in thrombotic microangiopathy after haematopoietic cell transplantation-A secondary CIBMTR analysis.British journal of haematology · 2024Article
- Outcomes of haploidentical bone marrow transplantation in infant acute leukemia: a single center experience.Bone marrow transplantation · 2024Article
- Comparison of upfront haploidentical hematopoietic stem cell transplantation and salvage haploidentical hematopoietic stem cell transplantation after immunosuppressive therapy in children with acquired severe aplastic anemia - a multicenter study.Frontiers in immunology · 2024Article
- Transplant-associated thrombotic microangiopathy in pediatrics: incidence, risk factors, therapeutic options, and outcome based on data from a single center.Frontiers in oncology · 2024Article
- Sickle cell disease is a risk factor for transplant-associated thrombotic microangiopathy in children.Blood advances · 2023Article
- Advancing therapy for neuroblastoma.Nature reviews. Clinical oncology · 2022Review
- Thrombotic Microangiopathy: Multi-Institutional Review of Pediatric Patients Who Underwent HSCT.Journal of personalized medicine · 2021Article
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Authors and funding
10 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Transplant-associated thrombotic microangiopathy (TA-TMA) is an endothelial injury syndrome that complicates hematopoietic stem cell transplant (HSCT). Morbidity and mortality from TA-TMA remain high, making prevention critical. We describe our retrospective single-center experience of TA-TMA after pediatric allogeneic HSCT and present a novel pre-HSCT risk-stratification system and prophylaxis regimen. From January 2012 through October 2019, 257 patients underwent 292 allogeneic HSCTs. Prospective risk stratification was introduced in December 2016. High-risk (HR) patients were treated with combination prophylaxis with eicosapentaenoic acid and N-acetylcysteine. The 1-year cumulative incidence of TA-TMA was 6.3% (95% confidence interval [CI], 3.2-9.4). Age ≥10 years, myeloablative conditioning with total body irradiation, HLA mismatch, diagnosis of severe aplastic anemia or malignancy, prior calcineurin inhibitor exposure, and recipient cytomegalovirus seropositivity were found to be pre-HSCT risk factors for development of TA-TMA. Before routine prophylaxis, TA-TMA rates were significantly different between the HR and standard-risk groups, at 28.2% (95% CI, 0-12.7) vs 3.2% (0.1-6.3), respectively (P < .001). After introduction of prophylaxis, the 1-year cumulative incidence of TA-TMA in the HR group decreased to 4.5% (95% CI, 0-13.1; P = .062, compared with the incidence before prophylaxis). Multicenter pediatric studies are needed to validate these risk criteria and to confirm the efficacy of the prophylactic regimen.
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