ArticleBlood2012
Residual plasmatic activity of ADAMTS13 is correlated with phenotype severity in congenital thrombotic thrombocytopenic purpura.
Article in Blood, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.
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Who cites it
36 citing papers in PubMed, 121 citations in OpenAlex.
- The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: key findings at enrollment until 2017.Haematologica · 2019Trial
- Current standards and novel concepts in thrombotic thrombocytopenic purpura.Frontiers in medicine · 2026Review
- The landscape of ADAMTS13-related tests in thrombotic thrombocytopenic purpura: a review.Frontiers in medicine · 2026Review
- Rare onset of congenital thrombotic thrombocytopenic purpura during pregnancy: A novel ADAMTS13 gene variant.Transfusion · 2025Article
- Global prevalence of hereditary thrombotic thrombocytopenic purpura determined by genetic analysis.Blood advances · 2024Article
- Recombinant ADAMTS13: an effective rescue therapy for acute cTTP during pregnancy.Blood advances · 2024Article
- Novel mechanisms of action of emerging therapies of hereditary thrombotic thrombocytopenic purpura.Expert review of hematology · 2024Review
- A compound heterozygousFrontiers in medicine · 2024Article
- Hereditary Thrombotic Thrombocytopenic Purpura.Genes · 2023Review
- Frontiers in pathophysiology and management of thrombotic thrombocytopenic purpura.International journal of hematology · 2023Review
- Hypercoagulability and Inflammatory Markers in a Case of Congenital Thrombotic Thrombocytopenic Purpura Complicated by Fetal Demise.Journal of clinical medicine · 2022Article
- The Challenges in Diagnosis and Management of Acquired Thrombotic Thrombocytopenic Purpura: Consensus Report from Three Gulf Countries.Oman medical journal · 2022Review
- Assessing thrombogenesis and treatment response in congenital thrombotic thrombocytopenic purpura.EJHaem · 2021Article
- [Recurrent thrombocytopenia with hemolytic anemia in a boy aged 3 years].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2021Article
- Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management.Journal of clinical medicine · 2021Review
- Diagnosis of Hereditary TTP Caused by Homozygosity for a Rare Complex ADAMTS13 Allele After Salmonella Infection in a 43-Year-Old Asylum Seeker.Frontiers in medicine · 2021Article
- Article
- Clinical Features and Gene Mutation Analysis of Congenital Thrombotic Thrombocytopenic Purpura in Neonates.Frontiers in pediatrics · 2020Article
- Patient Experience with Congenital (Hereditary) Thrombotic Thrombocytopenic Purpura: A Conceptual Framework of Symptoms and Impacts.The patient · 2019Article
- Review
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17 authors at 7 institutions in 4 countries.
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Abstract
The quantification of residual plasmatic ADAMTS13 activity in congenital thrombotic thrombocytopenic purpura (TTP) patients is constrained by limitations in sensitivity and reproducibility of commonly used assays at low levels of ADAMTS13 activity, blunting efforts to establish genotype-phenotype correlations. In the present study, the residual plasmatic activity of ADAMTS13 was measured centrally by surface-enhanced laser desorption/ionization time-of-flight mass spectrometry (limit of detection = 0.5%) in 29 congenital TTP patients. The results were used to study correlations among ADAMTS13 genotype, residual plasmatic activity, and clinical phenotype severity. An ADAMTS13 activity above 0.5% was measured in 26 (90%) patients and lower levels of activity were associated with earlier age at first TTP episode requiring plasma infusion, more frequent recurrences, and prescription of fresh-frozen plasma prophylaxis. Receiver operating characteristic curve analysis showed that activity levels of less than 2.74% and 1.61% were discriminative of age at first TTP episode requiring plasma infusion < 18 years, annual rate of TTP episodes > 1, and use of prophylaxis. Mutations affecting the highly conserved N-terminal domains of the protein were associated with lower residual ADAMTS13 activity and a more severe phenotype in an allelic-dose dependent manner. The results of the present study show that residual ADAMTS13 activity is associated with the severity of clinical phenotype in congenital TTP and provide insights into genotype-phenotype correlations.
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