FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2529421796> ?p ?o ?g. }

• W2529421796 endingPage "e546" @default.
• W2529421796 startingPage "e537" @default.
• W2529421796 abstract "Background Thrombotic thrombocytopenic purpura is a rare thrombotic microangiopathy, related to a severe ADAMTS13 deficiency (a disintegrin and metalloprotease with thromboSpondin type 1 repeats, member 13; activity <10% of normal). Childhood-onset thrombotic thrombocytopenic purpura is very rare and initially often misdiagnosed, especially when ADAMTS13 deficiency is acquired (ie, not linked to inherited mutations of the ADAMTS13 gene). We aimed to investigate initial presentation, management, and outcome of acquired thrombotic thrombocytopenic purpura in children. Methods Between Jan 1, 2000, and Dec 31, 2015, we studied a cohort of patients with child-onset and adolescent-onset acquired thrombotic thrombocytopenic purpura included in the French national registry for thrombotic microangiopathies at presentation and during follow up. The inclusion criteria were: first episode before age 18 years; ADAMTS13 activity less than 10% of normal at presentation; positive anti-ADAMTS13 autoantibodies during an episode, or a recovery of ADAMTS13 activity in remission, or both. ADAMTS13 activity and anti-ADAMTS13 autoantibodies were investigated by a central laboratory, and medical records were extensively reviewed to collect clinical and biological features with a standardised form. This study is registered with ClinicalTrials.gov, number NCT00426686. Findings We enrolled 973 patients with childhood-onset thrombotic microangiopathy, of whom 74 had a severe ADAMTS13 deficiency (activity <10%) at presentation. 24 patients had an inherited thrombotic thrombocytopenic purpura also known as Upshaw-Schulman syndrome and five did not have follow-up data available, thus 45 children had acquired thrombotic thrombocytopenic purpura and were included in our database at presentation. 25 (56%) patients had idiopathic disease and 20 (44%) had miscellaneous associated clinical conditions. At diagnosis, median age was 13 years (IQR 7–16, range 4 months–17 years), with a sex ratio of 2·5 girls to 1 boy. Anti-ADAMTS13 autoantibodies were positive in 37 (82%) of 45 patients (24 [96%] of 25 idiopathic cases and 13 [65%] of 20 non-idiopathic cases). 39 (87%) of 45 patients were given plasma therapy and 21 (47%) received additional rituximab. Four (9%) children died after the first thrombotic thrombocytopenic purpura episode. Long-term follow up of the 41 survivors showed that ten (24%) patients relapsed and systemic lupus erythematosus occurred in two (5%) patients. Preemptive rituximab was used in seven (17%) of 41 patients with acquired thrombotic thrombocytopenic purpura. Interpretation Our study shows that child-onset and adolescent-onset acquired thrombotic thrombocytopenic purpura have specific clinical, biological and therapeutic features. Long-term follow-up is crucial to prevent relapses of the disease, to identify the occurrence of autoimmune disorders, and to evaluate consequences on social life. Child-onset and adolescent-onset acquired thrombotic thrombocytopenic purpura is a crucial diagnosis in the field of paediatric haematologic cytopenias because it is a life-threatening disease requiring a specific management. Funding Assistance Publique-Hôpitaux de Paris, France." @default.
• W2529421796 created "2016-10-14" @default.
• W2529421796 creator A5001037667 @default.
• W2529421796 creator A5014845281 @default.
• W2529421796 creator A5016762535 @default.
• W2529421796 creator A5017372134 @default.
• W2529421796 creator A5021213540 @default.
• W2529421796 creator A5040756049 @default.
• W2529421796 creator A5041549015 @default.
• W2529421796 creator A5047735374 @default.
• W2529421796 creator A5047925131 @default.
• W2529421796 creator A5050980807 @default.
• W2529421796 creator A5057650691 @default.
• W2529421796 creator A5071519850 @default.
• W2529421796 creator A5087152475 @default.
• W2529421796 creator A5088234627 @default.
• W2529421796 date "2016-11-01" @default.
• W2529421796 modified "2023-10-16" @default.
• W2529421796 title "Child-onset and adolescent-onset acquired thrombotic thrombocytopenic purpura with severe ADAMTS13 deficiency: a cohort study of the French national registry for thrombotic microangiopathy" @default.
• W2529421796 cites W1817345721 @default.
• W2529421796 cites W1840088320 @default.
• W2529421796 cites W1964569129 @default.
• W2529421796 cites W1980913602 @default.
• W2529421796 cites W1982003857 @default.
• W2529421796 cites W1994073450 @default.
• W2529421796 cites W1997822763 @default.
• W2529421796 cites W2004979737 @default.
• W2529421796 cites W2011967454 @default.
• W2529421796 cites W2020077683 @default.
• W2529421796 cites W2030337128 @default.
• W2529421796 cites W2038601456 @default.
• W2529421796 cites W2043315492 @default.
• W2529421796 cites W2049115072 @default.
• W2529421796 cites W2058437162 @default.
• W2529421796 cites W2061297221 @default.
• W2529421796 cites W2114572851 @default.
• W2529421796 cites W2114604545 @default.
• W2529421796 cites W2126092981 @default.
• W2529421796 cites W2137976895 @default.
• W2529421796 cites W2151503986 @default.
• W2529421796 cites W2163744485 @default.
• W2529421796 cites W2171710650 @default.
• W2529421796 cites W2176543664 @default.
• W2529421796 cites W2183705115 @default.
• W2529421796 cites W2186521452 @default.
• W2529421796 cites W2191807303 @default.
• W2529421796 cites W2201449395 @default.
• W2529421796 cites W2315229770 @default.
• W2529421796 cites W2340760838 @default.
• W2529421796 cites W2340762935 @default.
• W2529421796 cites W4230901927 @default.
• W2529421796 doi "https://doi.org/10.1016/s2352-3026(16)30125-9" @default.
• W2529421796 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/27720178" @default.
• W2529421796 hasPublicationYear "2016" @default.
• W2529421796 type Work @default.
• W2529421796 sameAs 2529421796 @default.
• W2529421796 citedByCount "48" @default.
• W2529421796 countsByYear W25294217962017 @default.
• W2529421796 countsByYear W25294217962018 @default.
• W2529421796 countsByYear W25294217962019 @default.
• W2529421796 countsByYear W25294217962020 @default.
• W2529421796 countsByYear W25294217962021 @default.
• W2529421796 countsByYear W25294217962022 @default.
• W2529421796 countsByYear W25294217962023 @default.
• W2529421796 crossrefType "journal-article" @default.
• W2529421796 hasAuthorship W2529421796A5001037667 @default.
• W2529421796 hasAuthorship W2529421796A5014845281 @default.
• W2529421796 hasAuthorship W2529421796A5016762535 @default.
• W2529421796 hasAuthorship W2529421796A5017372134 @default.
• W2529421796 hasAuthorship W2529421796A5021213540 @default.
• W2529421796 hasAuthorship W2529421796A5040756049 @default.
• W2529421796 hasAuthorship W2529421796A5041549015 @default.
• W2529421796 hasAuthorship W2529421796A5047735374 @default.
• W2529421796 hasAuthorship W2529421796A5047925131 @default.
• W2529421796 hasAuthorship W2529421796A5050980807 @default.
• W2529421796 hasAuthorship W2529421796A5057650691 @default.
• W2529421796 hasAuthorship W2529421796A5071519850 @default.
• W2529421796 hasAuthorship W2529421796A5087152475 @default.
• W2529421796 hasAuthorship W2529421796A5088234627 @default.
• W2529421796 hasConcept C126322002 @default.
• W2529421796 hasConcept C187212893 @default.
• W2529421796 hasConcept C18903297 @default.
• W2529421796 hasConcept C2777121669 @default.
• W2529421796 hasConcept C2777878052 @default.
• W2529421796 hasConcept C2778585876 @default.
• W2529421796 hasConcept C2779134260 @default.
• W2529421796 hasConcept C2780921031 @default.
• W2529421796 hasConcept C71924100 @default.
• W2529421796 hasConcept C72563966 @default.
• W2529421796 hasConcept C86803240 @default.
• W2529421796 hasConcept C89560881 @default.
• W2529421796 hasConceptScore W2529421796C126322002 @default.
• W2529421796 hasConceptScore W2529421796C187212893 @default.
• W2529421796 hasConceptScore W2529421796C18903297 @default.
• W2529421796 hasConceptScore W2529421796C2777121669 @default.
• W2529421796 hasConceptScore W2529421796C2777878052 @default.
• W2529421796 hasConceptScore W2529421796C2778585876 @default.
• W2529421796 hasConceptScore W2529421796C2779134260 @default.

• next