Detail
Článek
Článek online
FT
Medvik - BMČ
  • Je něco špatně v tomto záznamu ?

Becoming attack-free further improves health-related quality of life in patients with hereditary angioedema receiving garadacimab

P. Staubach, TJ. Craig, T. Fukuda, E. Aygoren-Pursun, R. Hakl, J. Braverman, JP. Lawo, M. Pollen, C. Nenci, PH. Li, H. Farkas

. 2025 ; 46 (3) : 200-208. [pub] 20250501

Jazyk angličtina Země Spojené státy americké

Typ dokumentu časopisecké články, klinické zkoušky, fáze III, randomizované kontrolované studie, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc25015826

Background: Hereditary angioedema (HAE) is associated with substantial health-related quality of life (HRQoL) impairments. Complete disease control and life normalization are key treatment goals. In previous studies, garadacimab prevented HAE attacks with a favorable safety profile and HRQoL improvements. Objective: HRQoL was evaluated in patients with HAE receiving garadacimab stratified by attack-free status. Methods: In the pivotal phase III study (NCT04656418), 39 patients received garadacimab 200 mg subcutaneously once monthly and 25 volume-matched placebo. In the phase III open-label extension (OLE), 90 patients in the garadacimab-naive group (received placebo in previous studies or newly enrolled) and 71 patients in the previous garadacimab exposure group (received garadacimab in previous studies) received garadacimab (NCT04739059). Patients ages ≥ 18 years completed the Angioedema Quality of Life (AE-QoL) questionnaire in both studies; scores were evaluated post hoc by attack-free status. Results: In the pivotal phase III and phase III OLE studies, 62% and 60% of patients, respectively, were attack-free. In the pivotal phase III study, the mean AE-QoL total score improved with garadacimab, from 38.8 (day 1) to 6.6 (month 6) for attack-free patients (n = 19) and to 18.4 for patients with one or more attacks (n = 14) versus a change in mean AE-QoL total score from 43.7 to 40.5 with placebo (n = 20). In the phase III OLE study, the mean AE-QoL total score for patients who were garadacimab naive decreased from 46.2 (day 1) to 8.6 (month 12) for attack-free patients (n = 34) and from 54.5 to 23.5 for patients with one or more attacks (n = 30). For the previous garadacimab exposure group, AE-QoL improvements were maintained from previous studies, regardless of attack-free status. Conclusion: Garadacimab was associated with HRQoL improvement versus run-in in all groups. After garadacimab exposure in previous studies, improvements were maintained in the phase III OLE study. Attack-free patients had the greatest HRQoL improvements, bringing them closer to complete disease control and life normalization.Clinical trials NCT04656418, NCT04739059, www.clinicaltrials.gov.

Citace poskytuje Crossref.org

000      
00000naa a2200000 a 4500
001      
bmc25015826
003      
CZ-PrNML
005      
20250731091242.0
007      
ta
008      
250708s2025 xxu f 000 0|eng||
009      
AR
024    7_
$a 10.2500/aap.2025.46.250026 $2 doi
035    __
$a (PubMed)40380356
040    __
$a ABA008 $b cze $d ABA008 $e AACR2
041    0_
$a eng
044    __
$a xxu
100    1_
$a Staubach, Petra $u From the Department of Dermatology and Allergy, University Medical Center Mainz, Johannes Gutenberg University, Mainz, Germany
245    10
$a Becoming attack-free further improves health-related quality of life in patients with hereditary angioedema receiving garadacimab / $c P. Staubach, TJ. Craig, T. Fukuda, E. Aygoren-Pursun, R. Hakl, J. Braverman, JP. Lawo, M. Pollen, C. Nenci, PH. Li, H. Farkas
520    9_
$a Background: Hereditary angioedema (HAE) is associated with substantial health-related quality of life (HRQoL) impairments. Complete disease control and life normalization are key treatment goals. In previous studies, garadacimab prevented HAE attacks with a favorable safety profile and HRQoL improvements. Objective: HRQoL was evaluated in patients with HAE receiving garadacimab stratified by attack-free status. Methods: In the pivotal phase III study (NCT04656418), 39 patients received garadacimab 200 mg subcutaneously once monthly and 25 volume-matched placebo. In the phase III open-label extension (OLE), 90 patients in the garadacimab-naive group (received placebo in previous studies or newly enrolled) and 71 patients in the previous garadacimab exposure group (received garadacimab in previous studies) received garadacimab (NCT04739059). Patients ages ≥ 18 years completed the Angioedema Quality of Life (AE-QoL) questionnaire in both studies; scores were evaluated post hoc by attack-free status. Results: In the pivotal phase III and phase III OLE studies, 62% and 60% of patients, respectively, were attack-free. In the pivotal phase III study, the mean AE-QoL total score improved with garadacimab, from 38.8 (day 1) to 6.6 (month 6) for attack-free patients (n = 19) and to 18.4 for patients with one or more attacks (n = 14) versus a change in mean AE-QoL total score from 43.7 to 40.5 with placebo (n = 20). In the phase III OLE study, the mean AE-QoL total score for patients who were garadacimab naive decreased from 46.2 (day 1) to 8.6 (month 12) for attack-free patients (n = 34) and from 54.5 to 23.5 for patients with one or more attacks (n = 30). For the previous garadacimab exposure group, AE-QoL improvements were maintained from previous studies, regardless of attack-free status. Conclusion: Garadacimab was associated with HRQoL improvement versus run-in in all groups. After garadacimab exposure in previous studies, improvements were maintained in the phase III OLE study. Attack-free patients had the greatest HRQoL improvements, bringing them closer to complete disease control and life normalization.Clinical trials NCT04656418, NCT04739059, <ext-link xmlns:xlink="http://www.w3.org/1999/xlink" ext-link-type="uri" xlink:href="http://www.clinicaltrials.gov">www.clinicaltrials.gov</ext-link>.
650    _2
$a lidé $7 D006801
650    12
$a kvalita života $7 D011788
650    12
$a hereditární angioedémy $x farmakoterapie $x diagnóza $7 D054179
650    _2
$a ženské pohlaví $7 D005260
650    _2
$a mužské pohlaví $7 D008297
650    _2
$a lidé středního věku $7 D008875
650    _2
$a dospělí $7 D000328
650    12
$a humanizované monoklonální protilátky $x terapeutické užití $x škodlivé účinky $7 D061067
650    _2
$a výsledek terapie $7 D016896
650    _2
$a průzkumy a dotazníky $7 D011795
650    _2
$a senioři $7 D000368
650    _2
$a mladý dospělý $7 D055815
655    _2
$a časopisecké články $7 D016428
655    _2
$a klinické zkoušky, fáze III $7 D017428
655    _2
$a randomizované kontrolované studie $7 D016449
655    _2
$a multicentrická studie $7 D016448
700    1_
$a Craig, Timothy J $u Allergy, Asthma and Immunology, Department of Medicine, Pediatrics, Obstetrics and Gynecology, Maternal-Fetal Medicine, and Biomedical Sciences, Pennsylvania State University, Hershey, Pennsylvania
700    1_
$a Fukuda, Tomoo $u Department of Dermatology, Saitama Medical Center, Saitama Medical University, Saitama, Japan
700    1_
$a Aygoren-Pursun, Emel $u Department for Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt, Germany
700    1_
$a Hakl, Roman $u Department of Clinical Immunology and Allergology, St. Anne's University Hospital in Brno, Brno, Czech Republic
700    1_
$a Braverman, Julia $u CSL Behring, King of Prussia, Pennsylvania
700    1_
$a Lawo, John-Philip $u CSL Innovation GmbH, Marburg, Germany
700    1_
$a Pollen, Maressa $u CSL Behring, King of Prussia, Pennsylvania
700    1_
$a Nenci, Chiara $u CSL Behring AG, Bern, Switzerland
700    1_
$a Li, Philip H $u Division of Rheumatology and Clinical Immunology, Department of Medicine, Queen Mary Hospital, University of Hong Kong, Hong Kong; and
700    1_
$a Farkas, Henriette $u Department of Internal Medicine and Hematology, Hungarian Angioedema Center of Reference and Excellence, Semmelweis University, Budapest, Hungary
773    0_
$w MED00005058 $t Allergy and asthma proceedings $x 1539-6304 $g Roč. 46, č. 3 (2025), s. 200-208
856    41
$u https://pubmed.ncbi.nlm.nih.gov/40380356 $y Pubmed
910    __
$a ABA008 $b sig $c sign $y - $z 0
990    __
$a 20250708 $b ABA008
991    __
$a 20250731091236 $b ABA008
999    __
$a ok $b bmc $g 2366569 $s 1252951
BAS    __
$a 3
BAS    __
$a PreBMC-MEDLINE
BMC    __
$a 2025 $b 46 $c 3 $d 200-208 $e 20250501 $i 1539-6304 $m Allergy and asthma proceedings $n Allergy Asthma Proc $x MED00005058
LZP    __
$a Pubmed-20250708

Najít záznam

Citační ukazatele

Možnosti archivace