PMID- 32445670
OWN - NLM
STAT- MEDLINE
DCOM- 20200925
LR  - 20200925
IS  - 1534-4436 (Electronic)
IS  - 1081-1206 (Linking)
VI  - 125
IP  - 3
DP  - 2020 Sep
TI  - Long-term safety and efficacy of subcutaneous C1-inhibitor in older patients with 
      hereditary angioedema.
PG  - 334-340.e1
LID - S1081-1206(20)30333-1 [pii]
LID - 10.1016/j.anai.2020.05.015 [doi]
AB  - BACKGROUND: Patients aged 65 years and older with hereditary angioedema (HAE) 
      owing to C1-inhibitor (C1-INH) deficiency may have an altered response to 
      treatment and are at higher risk for treatment-related adverse events (AEs) 
      because of comorbidities and polypharmacy. OBJECTIVE: To investigate the safety 
      and efficacy of subcutaneous C1 esterase inhibitor (C1-INH) in patients aged 65 
      years and older treated in an open-label extension of a phase 3 trial. METHODS: 
      Eligible patients (>/=4 attacks for more than 2 consecutive months) were randomized 
      to receive twice-weekly subcutaneous C1-INH with a dosage of 40 IU/kg or 60 IU/kg 
      for 52 to 140 weeks. Safety end points and efficacy outcomes were evaluated for 
      patients aged 65 years and above and younger than 65 years. RESULTS: Of the 126 
      patients treated, 10 were 65 years and older (mean age [range], 68 [65-72 
      years]). A total of 8 of 10 patients had multiple comorbidities, and 6 of these 
      10 patients were taking more than 5 non-HAE-related drugs concomitantly. AEs 
      occurring in more than 1 patient included injection site bruising (n = 2, 
      related), injection site pain (n = 2, related), urinary tract infection (n = 2, 
      unrelated), and diarrhea (n = 2, unrelated). No thromboembolic events or cases of 
      anaphylaxis were reported. Two patients aged 65 years and older experienced 
      unrelated serious AEs (dehydration and hypokalemia in 1 and pneumonia and an HAE 
      attack leading to hospitalization in another). A total of 6 of 9 evaluable 
      patients were responders, with a greater than or equal to 50% reduction in HAE 
      attacks vs prestudy; 6 of 10 patients had less than 1 attack over 4 weeks and 3 
      were attack-free (median attack rate, 0.52 attacks per month). CONCLUSION: 
      Subcutaneous C1-INH was well-tolerated and effective in the management of HAE in 
      patients aged 65 years and older with multiple comorbid conditions and 
      polypharmacy.
CI  - Copyright (c) 2020 The Authors. Published by Elsevier Inc. All rights reserved.
FAU - Bernstein, Jonathan A
AU  - Bernstein JA
AD  - Allergy Section, Division of Immunology, Department of Internal Medicine, 
      University of Cincinnati College of Medicine and Bernstein Clinical Research 
      Center, Cincinnati, Ohio. Electronic address: jonathan.bernstein@uc.edu.
FAU - Schwartz, Lawrence
AU  - Schwartz L
AD  - Department of Internal Medicine, Virginia Commonwealth University, Richmond, 
      Virginia.
FAU - Yang, William
AU  - Yang W
AD  - Ottawa Allergy Research Corporation and University of Ottawa Medical School, 
      Ottawa, Canada.
FAU - Baker, James
AU  - Baker J
AD  - Baker Allergy, Asthma, and Dermatology Research Center, Portland, Oregon.
FAU - Anderson, John
AU  - Anderson J
AD  - Clinical Research Center of Alabama, Birmingham, Alabama.
FAU - Farkas, Henriette
AU  - Farkas H
AD  - Hungarian Angioedema Reference Center, Third Department of Internal Medicine, 
      Semmelweis University, Budapest, Hungary.
FAU - Aygoren-Pursun, Emel
AU  - Aygoren-Pursun E
AD  - Klinikum der Johann Wolfgang-Goethe Universitat, Klinik fur Kinder- und 
      Jugendmedizin, Frankfurt, Germany.
FAU - Bygum, Anette
AU  - Bygum A
AD  - Hereditary Angioedema Centre Denmark, Department of Dermatology, and Allergy 
      Centre, Odense University Hospital, Odense, Denmark.
FAU - Jacobs, Iris
AU  - Jacobs I
AD  - CSL Behring, King of Prussia, Pennsylvania.
FAU - Feuersenger, Henrike
AU  - Feuersenger H
AD  - CSL Behring, Marburg, Germany.
FAU - Pragst, Ingo
AU  - Pragst I
AD  - CSL Behring, Marburg, Germany.
FAU - Riedl, Marc A
AU  - Riedl MA
AD  - University of California, San Diego School of Medicine, La Jolla, California.
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20200520
PL  - United States
TA  - Ann Allergy Asthma Immunol
JT  - Annals of allergy, asthma & immunology : official publication of the American 
      College of Allergy, Asthma, & Immunology
JID - 9503580
RN  - 0 (Complement C1 Inhibitor Protein)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Angioedemas, Hereditary/*drug therapy
MH  - Child
MH  - Complement C1 Inhibitor Protein/*adverse effects/*therapeutic use
MH  - Female
MH  - Humans
MH  - Male
MH  - Middle Aged
MH  - Treatment Outcome
MH  - Young Adult
EDAT- 2020/05/24 06:00
MHDA- 2020/09/26 06:00
CRDT- 2020/05/24 06:00
PHST- 2020/03/13 00:00 [received]
PHST- 2020/04/20 00:00 [revised]
PHST- 2020/05/12 00:00 [accepted]
PHST- 2020/05/24 06:00 [pubmed]
PHST- 2020/09/26 06:00 [medline]
PHST- 2020/05/24 06:00 [entrez]
AID - S1081-1206(20)30333-1 [pii]
AID - 10.1016/j.anai.2020.05.015 [doi]
PST - ppublish
SO  - Ann Allergy Asthma Immunol. 2020 Sep;125(3):334-340.e1. doi: 
      10.1016/j.anai.2020.05.015. Epub 2020 May 20.