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Establishment of a framework for assessing mortality in persons with congenital hemophilia A and its application to an adverse event reporting database
dc.contributor.authorPipe, Steven W.
dc.contributor.authorKruse‐jarres, Rebecca
dc.contributor.authorMahlangu, Johnny N.
dc.contributor.authorPierce, Glenn F.
dc.contributor.authorPeyvandi, Flora
dc.contributor.authorKuebler, Peter
dc.contributor.authorDe Ford, Christian
dc.contributor.authorSanabria, Fabián
dc.contributor.authorKo, Richard H.
dc.contributor.authorChang, Tiffany
dc.contributor.authorHay, Charles R. M.
dc.date.accessioned2021-01-05T18:46:56Z
dc.date.availableWITHHELD_13_MONTHS
dc.date.available2021-01-05T18:46:56Z
dc.date.issued2021-01
dc.identifier.citationPipe, Steven W.; Kruse‐jarres, Rebecca ; Mahlangu, Johnny N.; Pierce, Glenn F.; Peyvandi, Flora; Kuebler, Peter; De Ford, Christian; Sanabria, Fabián ; Ko, Richard H.; Chang, Tiffany; Hay, Charles R. M. (2021). "Establishment of a framework for assessing mortality in persons with congenital hemophilia A and its application to an adverse event reporting database." Journal of Thrombosis and Haemostasis 19: 21-31.
dc.identifier.issn1538-7933
dc.identifier.issn1538-7836
dc.identifier.urihttps://hdl.handle.net/2027.42/163882
dc.description.abstractBackgroundDespite recent therapeutic advances, life expectancy in persons with congenital hemophilia A (PwcHA) remains below that of the non- HA population. As new therapies are introduced, a uniform approach to the assessment of mortality is required for comprehensive evaluation of risk- benefit profiles, timely identification of emerging safety signals, and comparisons between treatments.ObjectivesDevelop and test a framework for consistent reporting and analysis of mortality across past, current, and future therapies.Patients/MethodsWe identified known causes of mortality in PwcHA through literature review, analysis of the US Food and Drug Administration Adverse Event Reporting System (FAERS) database, and expert insights. Leading causes of death in general populations are those recognized by the Centers for Disease Control and Prevention and the World Health Organization. We developed an algorithm for assessing fatalities in PwcHA and used this to categorize FAERS data as a proof of concept.ResultsPwcHA share mortality causes with the non- HA population including cardiovascular disease, malignancy, infections, pulmonary disease, dementias, and trauma/suicide. Causes associated with HA include hemorrhage, thrombosis, human immunodeficiency virus, hepatitis C virus, and liver dysfunction. We propose an algorithm employing these classes to categorize fatalities and use it to classify FAERS fatality data between 01/01/2000 and 03/31/2020; the most common causes were hemorrhage (22.2%) and thrombosis (10.4%).ConclusionsA conceptual framework for examining mortality in PwcHA receiving any hemophilia therapy is proposed to analyze and interpret fatalities, enabling consistent and objective assessment. Application of the framework using FAERS data suggests a generally consistent pattern of reported mortality across HA treatments, supporting the utility of this unified approach.
dc.publisherWiley Periodicals, Inc.
dc.publisherRaven Press
dc.subject.otherdata analysis
dc.subject.othercause of death
dc.subject.otheralgorithms
dc.subject.otherhemophilia A
dc.subject.othermortality
dc.titleEstablishment of a framework for assessing mortality in persons with congenital hemophilia A and its application to an adverse event reporting database
dc.typeArticle
dc.rights.robotsIndexNoFollow
dc.subject.hlbsecondlevelInternal Medicine and Specialties
dc.subject.hlbtoplevelHealth Sciences
dc.description.peerreviewedPeer Reviewed
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/163882/1/jth15186_am.pdf
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/163882/2/jth15186.pdf
dc.identifier.doi10.1111/jth.15186
dc.identifier.sourceJournal of Thrombosis and Haemostasis
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dc.owningcollnameInterdisciplinary and Peer-Reviewed


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