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FältnamnIndikatorerMetadata
00005703naa a2200409 4500
001oai:lup.lub.lu.se:e3095320-26a2-44ac-9480-0bc1db8f948c
003SwePub
008240325| | |||||||||||000 ||eng|
0247 a https://lup.lub.lu.se/record/e3095320-26a2-44ac-9480-0bc1db8f948c2 URI
0247 a https://doi.org/10.1016/S2352-3026(24)00006-12 DOI
040 a (SwePub)lu
041 a engb eng
042 9 SwePub
072 7a art2 swepub-publicationtype
072 7a ref2 swepub-contenttype
1001 a Coppens, Michielu Academic Medical Center of University of Amsterdam (AMC)4 aut
2451 0a Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B) : 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
520 a Background: Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B treatment, was shown to be superior to treatment with continuous prophylactic factor IX in terms of bleeding protection 18 months after gene therapy in a phase 3 trial. We report post-hoc 24-month efficacy and safety data from this trial to evaluate the longer-term effects of etranacogene dezaparvovec in individuals with haemophilia B. Methods: The phase 3 HOPE-B trial enrolled males aged 18 years or older with inherited haemophilia B, classified as severe (plasma factor IX activity level <1%) or moderately severe (plasma factor IX activity level ≥1% and ≤2%), with a severe bleeding phenotype and who were on stable continuous factor IX prophylaxis. Participants were treated with a single infusion of etranacogene dezaparvovec (2 × 1013 genome copies per kg of bodyweight). The primary endpoint, reported previously, was non-inferiority of the annualised bleeding rate (ABR) during the 52 weeks following stable factor IX expression (defined as months 7–18 after treatment) versus an at least 6-month lead-in period in which participants received their usual continuous factor IX prophylaxis, and is updated here up to month 24. Additional, post-hoc efficacy analyses, including adjusted ABR, factor IX activity, participants within factor IX ranges, and factor IX use, and safety analyses were performed at 24 months after gene therapy. Data were analysed in the full analysis set, which comprised the 54 patients who received at least a partial dose of gene therapy. The trial is ongoing and is registered with ClinicalTrials.gov, number NCT03569891. Findings: The study began on June 27, 2018, and participants were treated between January, 2019, and March, 2020; the date of data cutoff was April 21, 2022. 54 adult males (40 White, two Asian, one Black or African American, 11 other or missing) received a single intravenous infusion of etranacogene dezaparvovec and were followed for a median of 26·51 months (IQR 24·54–27·99), after a lead-in period of 7·13 months (6·51–7·82). In the updated analysis comparing months 7–24 after gene therapy to the lead-in period, mean adjusted ABR significantly reduced from 4·18 to 1·51 (p=0·0002) for all bleeds and from 3·65 to 0·99 (p=0·0001) for factor IX-treated bleeds. During each 6-month period after gene therapy, at least 67% of participants experienced no bleeding (36 of 54 during months 0–6 and stable thereafter), compared with 14 (26%) of 54 during the lead-in period. 24 months after gene therapy, 1 (2%) participant had one-stage factor IX activity less than 5%, whereas 18 (33%) had factor IX activity more than 40% (non-haemophilia range), with mean factor IX activity stable and sustained at 36·7% (SD 19·0%). 52 (96%) of 54 participants expressed endogenous factor IX, remaining free of factor IX prophylaxis at month 24. No new safety concerns were identified and no treatment-related serious adverse events or treatment-related deaths occurred. The most common treatment-related adverse events were an increase in alanine aminotransferase (nine [17%] of 54 patients), headache (eight [15%]), influenza-like illness (seven [13%]), and an increase in aspartate aminotransferase (five [9%]). Interpretation: By providing durable disease correction throughout the 24 months after gene therapy, etranacogene dezaparvovec provides a safe and effective therapeutic option for patients with severe or moderately severe haemophilia B. Funding: uniQure and CSL Behring.
650 7a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Hematologi0 (SwePub)302022 hsv//swe
650 7a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Hematology0 (SwePub)302022 hsv//eng
7001 a Pipe, Steven W.u University of Michigan4 aut
7001 a Miesbach, Wolfgangu University Hospital Frankfurt4 aut
7001 a Astermark, Janu Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital4 aut0 (Swepub:lu)medf-jas
7001 a Recht, Michaelu Yale University4 aut
7001 a van der Valk, Paulu University Medical Center Utrecht4 aut
7001 a Ewenstein, Bruce4 aut
7001 a Pinachyan, Karen4 aut
7001 a Galante, Nicholas4 aut
7001 a Quellec, Sandra Le4 aut
7001 a Monahan, Paul E.4 aut
7001 a Leebeek, Frank W.G.u Erasmus University Medical Center4 aut
7102 a Academic Medical Center of University of Amsterdam (AMC)b University of Michigan4 org
7102 a HOPE-B Investigators
7730 t The Lancet Haematologyx 2352-3026
8564 u http://dx.doi.org/10.1016/S2352-3026(24)00006-1y FULLTEXT
8564 8u https://lup.lub.lu.se/record/e3095320-26a2-44ac-9480-0bc1db8f948c
8564 8u https://doi.org/10.1016/S2352-3026(24)00006-1

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