SwePub
Sök i LIBRIS databas

  Extended search

onr:"swepub:oai:lup.lub.lu.se:9523cf94-85cb-478e-b63b-08992dc1c4aa"
 

Search: onr:"swepub:oai:lup.lub.lu.se:9523cf94-85cb-478e-b63b-08992dc1c4aa" > Gene Therapy with E...

  • 1 of 1
  • Previous record
  • Next record
  •    To hitlist
  • Pipe, Steven W.University of Michigan (author)

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

  • Article/chapterEnglish2023

Publisher, publication year, extent ...

  • 2023
  • 13 s.

Numbers

  • LIBRIS-ID:oai:lup.lub.lu.se:9523cf94-85cb-478e-b63b-08992dc1c4aa
  • https://lup.lub.lu.se/record/9523cf94-85cb-478e-b63b-08992dc1c4aaURI
  • https://doi.org/10.1056/NEJMoa2211644DOI

Supplementary language notes

  • Language:English
  • Summary in:English

Part of subdatabase

Classification

  • Subject category:art swepub-publicationtype
  • Subject category:ref swepub-contenttype

Notes

  • Background: Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby protecting against bleeding without burdensome factor IX replacement. Methods: In this open-label, phase 3 study, after a lead-in period (≥6 months) of factor IX prophylaxis, we administered one infusion of adeno-associated virus 5 (AAV5) vector expressing the Padua factor IX variant (etranacogene dezaparvovec; 2×1013 genome copies per kilogram of body weight) to 54 men with hemophilia B (factor IX activity ≤2% of the normal value) regardless of preexisting AAV5 neutralizing antibodies. The primary end point was the annualized bleeding rate, evaluated in a noninferiority analysis comparing the rate during months 7 through 18 after etranacogene dezaparvovec treatment with the rate during the lead-in period. Noninferiority of etranacogene dezaparvovec was defined as an upper limit of the two-sided 95% Wald confidence interval of the annualized bleeding rate ratio that was less than the noninferiority margin of 1.8. Superiority, additional efficacy measures, and safety were also assessed. Results: The annualized bleeding rate decreased from 4.19 (95% confidence interval [CI], 3.22 to 5.45) during the lead-in period to 1.51 (95% CI, 0.81 to 2.82) during months 7 through 18 after treatment, for a rate ratio of 0.36 (95% Wald CI, 0.20 to 0.64; P<0.001), demonstrating noninferiority and superiority of etranacogene dezaparvovec as compared with factor IX prophylaxis. Factor IX activity had increased from baseline by a least-squares mean of 36.2 percentage points (95% CI, 31.4 to 41.0) at 6 months and 34.3 percentage points (95% CI, 29.5 to 39.1) at 18 months after treatment, and usage of factor IX concentrate decreased by a mean of 248,825 IU per year per participant in the post-treatment period (P<0.001 for all three comparisons). Benefits and safety were observed in participants with predose AAV5 neutralizing antibody titers of less than 700. No treatment-related serious adverse events occurred. Conclusions: Etranacogene dezaparvovec gene therapy was superior to prophylactic factor IX with respect to the annualized bleeding rate, and it had a favorable safety profile.

Subject headings and genre

Added entries (persons, corporate bodies, meetings, titles ...)

  • Leebeek, Frank W.G.Erasmus University Medical Center (author)
  • Recht, MichaelYale University (author)
  • Key, Nigel S.University of North Carolina (author)
  • Castaman, GiancarloCareggi University Hospital (author)
  • Miesbach, WolfgangUniversity Hospital Frankfurt (author)
  • Lattimore, SusanYale University (author)
  • Peerlinck, KathelijneUniversity Hospitals Leuven (author)
  • Van Der Valk, PaulUniversity Medical Center Utrecht (author)
  • Coppens, MichielAcademic Medical Center of University of Amsterdam (AMC) (author)
  • Kampmann, PeterCopenhagen University Hospital (author)
  • Meijer, KarinaUniversity Medical Center Groningen (author)
  • O'connell, Niamh (author)
  • Pasi, K. JohnQueen Mary University (author)
  • Hart, Daniel P.Royal London Hospital,Queen Mary University (author)
  • Kazmi, RashidUniversity Hospital Southampton (author)
  • Astermark, JanLund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital(Swepub:lu)medf-jas (author)
  • Hermans, Cedric R.J.R.Catholic University of Louvain,Saint-Luc University Hospital (author)
  • Klamroth, RobertUniversity Hospital Bonn,Vivantes hospital im Friedrichshain (author)
  • Lemons, RichardUniversity of Utah (author)
  • Visweshwar, NathanUniversity of South Florida (author)
  • Von Drygalski, Annette (author)
  • Young, GuyUniversity of Southern California (author)
  • Crary, Shelley E.University of Arkansas for Medical Sciences (author)
  • Escobar, MiguelUniversity of Texas Health Science Center at Houston (author)
  • Gomez, Esteban (author)
  • Kruse-Jarres, RebeccaUniversity of Washington (author)
  • Quon, Doris V. (author)
  • Symington, EmilyAddenbrooke's Hospital (author)
  • Wang, MichaelUniversity of Colorado (author)
  • Wheeler, Allison P.Vanderbilt University Medical Center (author)
  • Gut, Robert (author)
  • Liu, Ying P.UniQure Biopharma (author)
  • Dolmetsch, Ricardo E. (author)
  • Cooper, David L. (author)
  • Li, Yanyan (author)
  • Goldstein, Brahm (author)
  • Monahan, Paul E. (author)
  • University of MichiganErasmus University Medical Center (creator_code:org_t)

Related titles

  • In:New England Journal of Medicine388:8, s. 706-7180028-4793

Internet link

Find in a library

To the university's database

  • 1 of 1
  • Previous record
  • Next record
  •    To hitlist

Kungliga biblioteket hanterar dina personuppgifter i enlighet med EU:s dataskyddsförordning (2018), GDPR. Läs mer om hur det funkar här.
Så här hanterar KB dina uppgifter vid användning av denna tjänst.

 
pil uppåt Close

Copy and save the link in order to return to this view