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Steven W Pipe

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Articles 108
Citations 2533
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Recent Articles
1.
Chowdary P, Carcao M, Kenet G, Pipe S
Lancet . 2025 Mar; 405(10480):736-750. PMID: 40023652
Haemophilia A and B are congenital X-linked bleeding disorders resulting from deficiencies in clotting factors VIII (haemophilia A) and IX (haemophilia B). Patients with severe deficiency, defined as having less...
2.
Escobar M, Hoffman M, Castaman G, Hermans C, Mahlangu J, Oldenburg J, et al.
Res Pract Thromb Haemost . 2025 Feb; 9(1):102670. PMID: 39990097
Management of bleeding in persons with hemophilia and inhibitors involves treatment with bypassing agents, including recombinant activated factor VII (rFVIIa). Two rFVIIa products are commercially approved for use in the...
3.
Miesbach W, Batty P, Chowdary P, Fong S, Kaczmarek R, Leebeek F, et al.
Res Pract Thromb Haemost . 2025 Feb; 9(1):102673. PMID: 39895992
Adeno-associated virus-based gene therapy for hemophilia has emerged as a revolutionary treatment option, offering potential correction of clotting factor deficiency through a single intravenous infusion of functional genes directed to...
4.
McKeown W, Hermans C, Unzu C, Kay M, Peyvandi F, Smith P, et al.
Haemophilia . 2025 Jan; PMID: 39868975
Introduction: Gene editing therapies offer the possibility of substantial improvement in treatment and quality of life for people with haemophilia (PWH) in a landscape of dynamic therapeutic advancement. Developing a...
5.
Hermans C, Coppens M, Ventriglia G, Ling G, Lehle M, Pipe S
Res Pract Thromb Haemost . 2025 Jan; 9(1):102648. PMID: 39810986
Congenital hemophilia A (HA) disease severity has traditionally been categorized according to intrinsic factor (F)VIII levels, with <1% of normal indicating severe HA, 1% to 5% moderate HA, and 6%...
6.
Nougier C, Pipe S, Pabinger I, Pouplard C, Schutgens R, Hermans C, et al.
J Thromb Haemost . 2025 Jan; PMID: 39798924
Background: Efanesoctocog is a B-domain-deleted, Fc-fusion factor (F)VIII linked to the D'D3 domain of von Willebrand factor and 2 XTEN polypeptides, designed for an ultra-extended half-life for prophylaxis in hemophilia...
7.
Quon D, Wang J, Wang M, Pepperell D, Park Y, Kenet G, et al.
Ther Adv Hematol . 2024 Dec; 15:20406207241304645. PMID: 39712873
Background: Hemophilia A is caused by coagulation factor VIII (FVIII) deficiency and increases bleeding risk during invasive procedures. Objectives: To investigate FVIII concentrate use and bleeding outcomes for invasive procedures...
8.
Leavitt A, Mahlangu J, Raheja P, Symington E, Quon D, Giermasz A, et al.
Res Pract Thromb Haemost . 2024 Dec; 8(8):102615. PMID: 39687929
Background: Valoctocogene roxaparvovec, an adeno-associated virus-mediated gene therapy for severe hemophilia A, enables endogenous factor (F)VIII expression and provides bleed protection. Objectives: Determine valoctocogene roxaparvovec durability, efficacy, and safety 4...
9.
Pipe M.D S, Pipe S, Lissitchkov T, Lissitchkov T, Georgiev P, Mangles S, et al.
Blood Adv . 2024 Dec; 9(5):1147-1158. PMID: 39642315
Fitusiran is an investigational small interfering RNA therapeutic that targets antithrombin (AT) to rebalance hemostasis in people with hemophilia. Here, we present the results of a completed phase 2 open-label...
10.
Hermans C, Valentino L, Thornburg C, Unzu C, Kay M, Peyvandi F, et al.
Haemophilia . 2024 Oct; 30(6):1272-1280. PMID: 39437171
Introduction: Despite the progress in gene editing platforms like CRISPR/Cas9 with the potential to transform the standard of care for haemophilia, the language used to explain and discuss gene editing...