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Long-term efficacy ...
Long-term efficacy and safety of subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors
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- Shapiro, Amy D. (författare)
- Indiana Hemophilia and Thrombosis Center
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- Angchaisuksiri, Pantep (författare)
- Ramathibodi Hospital,Mahidol University
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- Astermark, Jan (författare)
- Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital
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- Benson, Gary (författare)
- Belfast City Hospital
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- Castaman, Giancarlo (författare)
- Careggi University Hospital
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- Eichler, Hermann (författare)
- Saarland University Hospital
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- Jiménez-Yuste, Victor (författare)
- University Hospital La Paz
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- Kavakli, Kaan (författare)
- Ege University
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- Matsushita, Tadashi (författare)
- Nagoya University Hospital
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- Poulsen, Lone Hvitfeldt (författare)
- Aarhus University
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- Wheeler, Allison P. (författare)
- Vanderbilt University Medical Center
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- Young, Guy (författare)
- University of Southern California
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- Zupančić-Šalek, Silva (författare)
- University of Zagreb,University Hospital Centre Zagreb,University of Osijek
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- Oldenburg, Johannes (författare)
- University Hospital Bonn
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- Chowdary, Pratima (författare)
- Royal Free London NHS Foundation Trust
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(creator_code:org_t)
- 2022-06-08
- 2022
- Engelska 11 s.
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Ingår i: Blood Advances. - : American Society of Hematology. - 2473-9529 .- 2473-9537. ; 6:11, s. 3422-3432
- Relaterad länk:
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http://dx.doi.org/10... (free)
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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Abstract
Ämnesord
Stäng
- Despite current therapies, there remains an unmet need for treatment for patients with hemophilia. The main parts of two phase 2 trials established clinical proof-of-concept for once-daily, subcutaneous concizumab prophylaxis in patients with hemophilia A/B with inhibitors (HAwI/HBwI; explorer4) and severe hemophilia A without inhibitors (HA; explorer5). Here, we present results from extension parts of these trials, included to evaluate longer term safety and efficacy. Both trials included main ($24 weeks) and extension (52-102 weeks) parts, with patients receiving concizumab 0.15 mg/kg with potential dose escalation to concizumab 0.20 or 0.25 mg/kg if they experienced $3 treated spontaneous bleeding episodes within 12 weeks. Endpoints included annualized bleeding rate (ABR), adverse events (AEs), and occurrence of antidrug antibodies. Thromboembolic events were AEs of special interest. Thirty-six patients with HA, 15 with HAwI, and 10 with HBwI were exposed to concizumab. Estimated ABRs during the main 1 extension parts at last dose level were 4.8 (95% confidence interval [CI], 3.2-7.2) and 6.4 (95% CI, 4.1-9.9) in explorer4 and explorer5, respectively (spontaneous ABRs were 1.8 [95% CI, 1.2-2.6] and 2.1 [95% CI, 1.3-3.3]). Most AEs were mild, with no deaths, events leading to withdrawal, or thromboembolic events. Anti-drug antibodies developed in 25% of patients and were low titer and transient, with no observed clinical effect in most cases. Results of the main 1 extension parts of these trials were consistent with results of the main parts. Ongoing phase 3 trials will further evaluate concizumab as a once-daily, subcutaneous treatment across hemophilia subtypes.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Kardiologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Cardiac and Cardiovascular Systems (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
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Till lärosätets databas
- Av författaren/redakt...
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Shapiro, Amy D.
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Angchaisuksiri, ...
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Astermark, Jan
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Benson, Gary
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Castaman, Gianca ...
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Eichler, Hermann
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Jiménez-Yuste, V ...
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Kavakli, Kaan
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Matsushita, Tada ...
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Poulsen, Lone Hv ...
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Wheeler, Allison ...
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Young, Guy
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Zupančić-Šalek, ...
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Oldenburg, Johan ...
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Chowdary, Pratim ...
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- Om ämnet
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Klinisk medicin
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och Kardiologi
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Klinisk medicin
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och Hematologi
- Artiklar i publikationen
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Blood Advances
- Av lärosätet
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Lunds universitet