ArticleBlood2016
The genetic fingerprint of susceptibility for transplant-associated thrombotic microangiopathy.
Article in Blood, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03384693 (A Pilot Trial of Using Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious Thrombotic Microangiopathy in High-Risk Hematopoietic Stem Cell Transplant Patients), which is not on this map. Cited by 104 papers, 4 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Pilot Trial of Using Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious Thrombotic Microangiopathy in High-Risk Hematopoietic Stem Cell Transplant Patients
Who cites it
104 citing papers in PubMed, 4 syntheses or guidelines pooled it, 182 citations in OpenAlex.
- Guideline
- A systematic review of diagnostic, prognostic, and risk blood and urine biomarkers of transplant-associated thrombotic microangiopathy.Frontiers in immunology · 2022Pooled it
- Clinical implications of HLA locus mismatching in unrelated donor hematopoietic cell transplantation: a meta-analysis.Oncotarget · 2017Pooled it
- Recommendations for diagnosis and treatment of Atypical Hemolytic Uremic Syndrome (aHUS): an expert consensus statement from the Rare Diseases Committee of the Brazilian Society of Nephrology (COMDORA-SBN).Jornal brasileiro de nefrologiaGuideline
- Complement-mediated thrombotic microangiopathy associated with lupus nephritis.Blood advances · 2018Trial
- Rapid genomic analysis for early identification of complement abnormalities in adults with transplant-associated thrombotic microangiopathy.Hematology, transfusion and cell therapy · 2026Article
- Lipid dysregulation after hematopoietic stem cell transplant.Haematologica · 2026Article
- Outcomes of Terminal Complement Blockade in Adults with High-Risk Transplant-Associated Thrombotic Microangiopathy: A Comparative Analysis.Transplantation and cellular therapy · 2026Article
- Proteomic analysis of pericardial effusions in hematopoietic stem cell transplant recipients.Haematologica · 2026Article
- KLF4 overexpression protects against complementmediated endothelial injury in transplant-associated thrombotic microangiopathy.Haematologica · 2026Article
- Complement-targeted therapies for transplant-associated thrombotic microangiopathy: recent advances.Frontiers in pharmacology · 2026Review
- Complement-related genetic analysis for Japanese children with transplant-associated thrombotic microangiopathy.Frontiers in pediatrics · 2026Article
- Narsoplimab Results in Excellent Survival in Adults and Children With Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy (TA-TMA).American journal of hematology · 2025Article
- Complement activation in secondary thrombotic microangiopathies.Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association · 2025Review
- How I Treat: Genetic Testing in Atypical Hemolytic Uremic Syndrome.Clinical journal of the American Society of Nephrology : CJASN · 2025Article
- An ASTCT, CIBMTR, EBMT, and APBMT Consensus Statement Defining Response Criteria for Hematopoietic Cell Transplantation Associated Thrombotic Microangiopathy (TA-TMA) Directed Therapy.Transplantation and cellular therapy · 2025Article
- Genetic Susceptibility in Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease: A Case-Control Study.International journal of molecular sciences · 2025Article
- Article
- Clinical Outcomes and Treatment Strategies of Adult Transplant-Associated Thrombotic Microangiopathy: External Validation of Harmonizing Definitions and High-Risk Criteria.American journal of hematology · 2025Article
- Ten tips for managing complement-mediated thrombotic microangiopathies (formerly atypical hemolytic uremic syndrome): narrative review.BMC nephrology · 2025Review
44 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
11 authors at 4 institutions in 1 country.
Funding
Abstract
Transplant-associated thrombotic microangiopathy (TA-TMA) occurs frequently after hematopoietic stem cell transplantation (HSCT) and can lead to significant morbidity and mortality. There are no data addressing individual susceptibility to TA-TMA. We performed a hypothesis-driven analysis of 17 candidate genes known to play a role in complement activation as part of a prospective study of TMA in HSCT recipients. We examined the functional significance of gene variants by using gene expression profiling. Among 77 patients undergoing genetic testing, 34 had TMA. Sixty-five percent of patients with TMA had genetic variants in at least one gene compared with 9% of patients without TMA (P < .0001). Gene variants were increased in patients of all races with TMA, but nonwhites had more variants than whites (2.5 [range, 0-7] vs 0 [range, 0-2]; P < .0001). Variants in ≥3 genes were identified only in nonwhites with TMA and were associated with high mortality (71%). RNA sequencing analysis of pretransplantation samples showed upregulation of multiple complement pathways in patients with TMA who had gene variants, including variants predicted as possibly benign by computer algorithm, compared with those without TMA and without gene variants. Our data reveal important differences in genetic susceptibility to HSCT-associated TMA based on recipient genotype. These data will allow prospective risk assessment and intervention to prevent TMA in highly susceptible transplant recipients. Our findings may explain, at least in part, racial disparities previously reported in transplant recipients and may guide treatment strategies to improve outcomes.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.