Search: WFRF:(Castaman Giancarlo) >
Long-term efficacy ...
Long-term efficacy and safety of subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors
-
- Shapiro, Amy D. (author)
- Indiana Hemophilia and Thrombosis Center
-
- Angchaisuksiri, Pantep (author)
- Ramathibodi Hospital,Mahidol University
-
- Astermark, Jan (author)
- Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital
-
show more...
-
- Benson, Gary (author)
- Belfast City Hospital
-
- Castaman, Giancarlo (author)
- Careggi University Hospital
-
- Eichler, Hermann (author)
- Saarland University Hospital
-
- Jiménez-Yuste, Victor (author)
- University Hospital La Paz
-
- Kavakli, Kaan (author)
- Ege University
-
- Matsushita, Tadashi (author)
- Nagoya University Hospital
-
- Poulsen, Lone Hvitfeldt (author)
- Aarhus University
-
- Wheeler, Allison P. (author)
- Vanderbilt University Medical Center
-
- Young, Guy (author)
- University of Southern California
-
- Zupančić-Šalek, Silva (author)
- University of Zagreb,University Hospital Centre Zagreb,University of Osijek
-
- Oldenburg, Johannes (author)
- University Hospital Bonn
-
- Chowdary, Pratima (author)
- Royal Free London NHS Foundation Trust
-
show less...
-
(creator_code:org_t)
- 2022-06-08
- 2022
- English 11 s.
-
In: Blood Advances. - : American Society of Hematology. - 2473-9529 .- 2473-9537. ; 6:11, s. 3422-3432
- Related links:
-
http://dx.doi.org/10... (free)
-
show more...
-
https://lup.lub.lu.s...
-
https://doi.org/10.1...
-
show less...
Abstract
Subject headings
Close
- 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.
Subject headings
- 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)
Publication and Content Type
- art (subject category)
- ref (subject category)
Find in a library
To the university's database
- By the author/editor
-
Shapiro, Amy D.
-
Angchaisuksiri, ...
-
Astermark, Jan
-
Benson, Gary
-
Castaman, Gianca ...
-
Eichler, Hermann
-
show more...
-
Jiménez-Yuste, V ...
-
Kavakli, Kaan
-
Matsushita, Tada ...
-
Poulsen, Lone Hv ...
-
Wheeler, Allison ...
-
Young, Guy
-
Zupančić-Šalek, ...
-
Oldenburg, Johan ...
-
Chowdary, Pratim ...
-
show less...
- About the subject
-
- MEDICAL AND HEALTH SCIENCES
-
MEDICAL AND HEAL ...
-
and Clinical Medicin ...
-
and Cardiac and Card ...
-
- MEDICAL AND HEALTH SCIENCES
-
MEDICAL AND HEAL ...
-
and Clinical Medicin ...
-
and Hematology
- Articles in the publication
-
Blood Advances
- By the university
-
Lund University