Annual incidence and severity of acute episodes in hereditary thrombotic thrombocytopenic purpura
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00023736%3A_____%2F21%3A00013254" target="_blank" >RIV/00023736:_____/21:00013254 - isvavai.cz</a>
Alternative codes found
RIV/00843989:_____/21:E0109149
Result on the web
<a href="https://doi.org/10.1182/blood.2020009801" target="_blank" >https://doi.org/10.1182/blood.2020009801</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1182/blood.2020009801" target="_blank" >10.1182/blood.2020009801</a>
Alternative languages
Result language
angličtina
Original language name
Annual incidence and severity of acute episodes in hereditary thrombotic thrombocytopenic purpura
Original language description
This study reports prospective data on 87 patients from the Hereditary TTP Registry (clinicaltrials.gov #NCT01257269) for survival, frequency, and severity of acute episodes from enrollment until December 2019. The 87 patients, followed up for a median of 4.2 years (range, 0.01-15 years), had a median age at overt disease onset and at clinical diagnosis of 4.6 years and 18 years (range, 0.0-70 years for both), respectively. Forty-three patients received regular plasma prophylaxis, whereas 22 did not, and treatment changed over time or was unknown in the remaining 22. Forty-three patients experienced 131 acute episodes, of which 91 (69%) occurred in patients receiving regular prophylaxis. This resulted in an annual incidence of acute episodes of 0.36 (95% confidence interval [CI], 0.29-0.44) with regular plasma treatment and of 0.41 (95% CI, 0.30-0.56) without regular plasma treatment. More than one-third of acute episodes (n = 51) were documented in children <10 years of age at enrollment and were often triggered by infections. Their annual incidence of acute episodes was significantly higher than in patients aged >40 years (1.18 [95% CI, 0.88-1. 55] vs 0.14 [95% CI, 0.08-0.23]). The prophylactic plasma infusion regimens used were insufficient to prevent acute episodes in many patients. Such regimens are burdensome, and caregivers, patients, and their guardians are reluctant to start regular plasma infusions, from which children particularly would benefit.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30205 - Hematology
Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2021
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Blood
ISSN
0006-4971
e-ISSN
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Volume of the periodical
137
Issue of the periodical within the volume
25
Country of publishing house
US - UNITED STATES
Number of pages
13
Pages from-to
3563-3575
UT code for WoS article
000669372800015
EID of the result in the Scopus database
2-s2.0-85106725094