PMID- 35524357
OWN - NLM
STAT- MEDLINE
DCOM- 20220510
LR  - 20220510
IS  - 1539-6304 (Electronic)
IS  - 1088-5412 (Linking)
VI  - 43
IP  - 3
DP  - 2022 May 1
TI  - Prophylactic therapy with subcutaneous C1-inhibitor is associated with sustained 
      symptom control in patients with hereditary angioedema.
PG  - 202-208
LID - 10.2500/aap.2022.43.220016 [doi]
AB  - Background: Hereditary angioedema (HAE) due to C1-inhibitor (C1-INH) deficiency 
      is a rare genetic disorder characterized by disabling episodes of edema that 
      commonly affect the skin as well as the gastrointestinal tract and upper airway. 
      Prophylactic therapy can decrease the severity and number of attacks. Long-term 
      symptom control and rescue medication use were evaluated in patients with HAE who 
      received subcutaneous (SC) C1-INH enrolled in an open-label extension (OLE) of 
      the phase III COMPACT (Clinical Studies for Optimal Management of Preventing 
      Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy) trial, 
      including a subgroup analysis of patients treated for >/=12 months. Methods: The 
      OLE study evaluated patients >/= 6 years old who had had four or more attacks over 
      2 consecutive months before enrollment. Patients naive for C1-INH (SC) and 
      patients in the COMPACT rollover trial were included. The patients were 
      randomized to receive C1-INH (SC) 40 or 60 IU/kg twice weekly for 52 weeks. U.S. 
      patients were eligible to continue for up to 140 weeks. Results: A total of 63 
      patients were randomized to the U.S. Food and Drug Administration approved dose 
      of 60 IU/kg; 24 subjects were treated for at least 12 months. For the 63 
      subjects, the median (range) attacks per month were 0.09 (0.0-4.0) and per year 
      were 1.0 (0.0-48.0). Two-thirds of the patients used rescue medication fewer than 
      once per year. For the 24 patients with >/= 12 months of exposure, the median 
      (range) attacks per month and per year were 0.017 (0.000-2.4) and 0.199 
      (0.000-28.94), respectively. Of these patients, 12 (50%) were attack free 
      throughout the duration of the study, and 3 (12.5%) had fewer than one attack per 
      year. Conclusion: Prophylaxis with C1-INH (SC) provided sustained reductions in 
      attack frequency and decreased rescue medication use, with a substantial 
      proportion of patients being attack free.
FAU - Craig, Timothy
AU  - Craig T
AD  - From the Department of Medicine, Pediatrics and Biomedical Sciences, Pennsylvania 
      State University, Hershey, Pennsylvania.
FAU - Feuersenger, Henrike
AU  - Feuersenger H
AD  - Associate Director Biostatistics, CSL Behring Innovation, Marburg, Germany; and.
FAU - Pragst, Ingo
AU  - Pragst I
AD  - Associate Director Biostatistics, CSL Behring Innovation, Marburg, Germany; and.
FAU - Dang, John
AU  - Dang J
AD  - Global Clinical Scientist Lead, Clinical Research TA Immunology, CSL Behring, 
      King of Prussia, Pennsylvania.
LA  - eng
PT  - Journal Article
PT  - Randomized Controlled Trial
PL  - United States
TA  - Allergy Asthma Proc
JT  - Allergy and asthma proceedings
JID - 9603640
RN  - 0 (Complement C1 Inhibitor Protein)
SB  - IM
MH  - *Angioedemas, Hereditary/diagnosis/drug therapy/prevention & control
MH  - Child
MH  - Complement C1 Inhibitor Protein/therapeutic use
MH  - Hormone Replacement Therapy
MH  - Humans
MH  - Injections, Subcutaneous
MH  - Treatment Outcome
EDAT- 2022/05/08 06:00
MHDA- 2022/05/11 06:00
CRDT- 2022/05/07 00:33
PHST- 2022/05/07 00:33 [entrez]
PHST- 2022/05/08 06:00 [pubmed]
PHST- 2022/05/11 06:00 [medline]
AID - 10.2500/aap.2022.43.220016 [doi]
PST - ppublish
SO  - Allergy Asthma Proc. 2022 May 1;43(3):202-208. doi: 10.2500/aap.2022.43.220016.