Adults with severe or moderately severe haemophilia B receiving etranacogene dezaparvovec in the HOPE-B phase 3 trial experience a stable increase in mean Factor IX activity levels and durable haemostatic protection after 24 months’ follow-up

Pipe, W S; Leebeek, WG F; Recht, Michael; Key, S N; Lattimore, Susan; Castaman, Giancarlo; Coppens, Magali; Cooper, D; Gut, Robert; Slawka, Sergio; Verweij, Stephanie; Dolmetsch, Ricardo; Li, Y; Monahan, PE; Miesbach, Wolfgang

doi:10.1055/s-0042-1760523

Cited by 2 publications

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“…In the HOPE-B study, 54 adult males with haemophilia B were treated with etranacogene dezaparvovec following a ≥ 6-month lead-in period with FIX prophylaxis [ 7 ]. Annualised bleeding rate reduced from 4.19 during the lead-in period to 1.51 post-treatment (Months 7–18) and was sustained at Month 24; 96.3% of study participants stopped and remained free from prophylactic FIX therapy over 24 months post-treatment [ 7 , 8 ]. At Months 6 and 24, mean FIX activity levels increased to 39.0 IU/dL and 36.7 IU/dL, respectively [ 7 , 8 ].…”

Section: Introductionmentioning

confidence: 99%

“…Annualised bleeding rate reduced from 4.19 during the lead-in period to 1.51 post-treatment (Months 7–18) and was sustained at Month 24; 96.3% of study participants stopped and remained free from prophylactic FIX therapy over 24 months post-treatment [ 7 , 8 ]. At Months 6 and 24, mean FIX activity levels increased to 39.0 IU/dL and 36.7 IU/dL, respectively [ 7 , 8 ]. Positive outcomes were also reported from the Phase 3 BENEGENE-2 trial investigating fidanacogene elaparvovec in adults with haemophilia B, with relatively stable FIX activity levels observed at Month 24 in the 22/45 patients for which data were reported; mean FIX activity level using the one-stage SynthASil assay was 25.0% in these patients [ 9 ].…”

Section: Introductionmentioning

confidence: 99%

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A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient

Miesbach,

Mulders,

Breederveld

et al. 2024

Orphanet J Rare Dis

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Background Adeno-associated virus (AAV)-based gene therapy for haemophilia has advanced substantially in the last 13 years; recently, three products have received approvals from regulatory authorities. Although the impact on quality of life seems promising, some limitations remain, such as the presence of pre-existing anti-AAV neutralising antibodies and the occurrence of hepatotoxicity. This review follows the CSL Behring-sponsored symposium at the 27th Congress of the European Hematology Association (EHA) 2022 that examined the haemophilia gene therapy process from a 360-degree multidisciplinary perspective. Here, the faculty (haematologist, nurse and haemophilia patient) summarised their own viewpoints from the symposium, with the aim of highlighting the key considerations required to engage with gene therapy effectively, for both patients and providers, as well as the importance of multidisciplinary collaboration, including with industry. Results When considering these new therapies, patients face a complex decision-making process, which includes whether gene therapy is right for them at their current stage of life. The authors agreed that collaboration and tailored education across the multidisciplinary team (including patients and their carers/families), starting early in the process and continuing throughout the long-term follow-up period, is key for the success of gene therapy. Additionally, patient expectations, which may surround eligibility, follow-up requirements and treatment outcomes, should be continually explored. During these ongoing discussions, transparent communication of the unknown factors, such as anticipated clotting factor levels, long-term factor expression and safety, and psychological changes, is critical. To ensure efficiency and comprehensiveness, clearly‑defined protocols should outline the whole process, which should include the recording and management of long-term effects. Conclusion In order to engage effectively, both patients and providers should be familiar with these key considerations prior to their involvement with the haemophilia gene therapy process. The future after the approval of haemophilia gene therapies remains to be seen and real-world evidence is eagerly awaited.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient

Miesbach,

Mulders,

Breederveld

et al. 2024

Orphanet J Rare Dis

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Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review

Ay,

Frenzel,

Pinachyan

et al. 2023

Haemophilia

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IntroductionWith recent approval of the first two gene therapies for haemophilia A and B, educational materials about AAV‐based gene therapy are needed by the haemophilia community for a better understanding of this novel therapeutic approach and helping healthcare providers and patients making personalized choices amongst an increasing array of therapeutic options.AimTo provide a comprehensive summary of the whole process of AAV‐based gene therapy from basic principles to clinical implementation through an illustrated review.MethodsThe authors, with expertise in and knowledge about gene therapy for haemophilia A and B, reviewed relevant articles from PubMed database and translated them into illustrations.ResultsThe review is divided into eight illustrated sections providing an overview of gene therapy for haemophilia A and B from haemophilia basics and current treatment landscape, principles of the AAV‐based liver‐directed gene therapy, through exploring the efficacy and safety results of published phase III clinical trials, current and future challenges, to implementation in clinical practice, including the hub and spoke models and the patient journey.ConclusionThis illustrated review educates healthcare professionals on AAV‐based gene therapy for haemophilia A and B enabling them to further educate their peers and their patients.

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Adults with severe or moderately severe haemophilia B receiving etranacogene dezaparvovec in the HOPE-B phase 3 trial experience a stable increase in mean Factor IX activity levels and durable haemostatic protection after 24 months’ follow-up

Cited by 2 publications

References 0 publications

A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient

A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient

Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review

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