The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: key findings at enrollment until 2017.
Journal
Haematologica
ISSN: 1592-8721
Titre abrégé: Haematologica
Pays: Italy
ID NLM: 0417435
Informations de publication
Date de publication:
10 2019
10 2019
Historique:
received:
16
01
2019
accepted:
20
02
2019
pubmed:
23
2
2019
medline:
8
7
2020
entrez:
23
2
2019
Statut:
ppublish
Résumé
Congenital thrombotic thrombocytopenic purpura is an autosomal recessive inherited disease with a clinically heterogeneous course and an incompletely understood genotype-phenotype correlation. In 2006, the Hereditary TTP Registry started recruitment for a study which aimed to improve the understanding of this ultra-rare disease. The objective of this study is to present characteristics of the cohort until the end of 2017 and to explore the relationship between overt disease onset and ADAMTS13 activity with emphasis on the recurring
Identifiants
pubmed: 30792199
pii: haematol.2019.216796
doi: 10.3324/haematol.2019.216796
pmc: PMC6886414
doi:
Substances chimiques
ADAMTS13 Protein
EC 3.4.24.87
ADAMTS13 protein, human
EC 3.4.24.87
Banques de données
ClinicalTrials.gov
['NCT01257269']
Types de publication
Clinical Trial
Journal Article
Multicenter Study
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
2107-2115Subventions
Organisme : NHLBI NIH HHS
ID : K01 HL135466
Pays : United States
Commentaires et corrections
Type : CommentIn
Informations de copyright
Copyright© 2019 Ferrata Storti Foundation.
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