PMID- 23121116
OWN - NLM
STAT- MEDLINE
DCOM- 20130510
LR  - 20221012
IS  - 1398-9995 (Electronic)
IS  - 0105-4538 (Linking)
VI  - 68
IP  - 1
DP  - 2013 Jan
TI  - Recombinant human C1 inhibitor for the prophylaxis of hereditary angioedema 
      attacks: a pilot study.
PG  - 118-24
LID - 10.1111/all.12060 [doi]
AB  - BACKGROUND: Hereditary angioedema (HAE) is a disease characterized by recurrent 
      tissue swelling affecting various body locations. Recent literature shows that 
      patients with frequent attacks may benefit from long-term prophylaxis. This study 
      evaluated the safety and prophylactic effect of weekly administrations of 
      recombinant C1INH (rhC1INH). METHODS: Patients with a history of HAE attacks 
      occurring >/=every 2 weeks received a once weekly administration of 50 U/kg 
      rhC1INH. Hereditary angioedema attack history was collected at screening. 
      Breakthrough attacks during the study were recorded at each visit. Following a 
      2-week run-in period, HAE patients received 8 weekly rhC1INH administrations and 
      were followed-up for an additional 6 weeks. Efficacy was evaluated by comparing 
      the HAE attack incidence during the treatment period to the historical attacks 
      over the previous 2 years. Safety evaluation was based on clinical laboratory and 
      adverse events (AEs) reports. RESULTS: The 25 participants reported a mean of 
      0.9 attacks/week over the past 2 years. The mean breakthrough attack rate during 
      the treatment period was 0.4 attacks/week (95% CI 0.28-0.56). A total of 30 
      treatment-emergent-AEs were reported in 13 patients, all mild to moderate. One 
      patient died from a laryngeal attack 25 days after last study drug 
      administration. The only possible drug related AEs reported were dry mouth, 
      dizziness and anxiety in one patient and hypotension in another. There were no 
      allergic AEs and no neutralizing antibodies observed. CONCLUSIONS: Weekly 
      administrations of 50 U/kg rhC1INH appeared to reduce the frequency of HAE 
      attacks and were generally safe and well tolerated.
CI  - (c) 2012 John Wiley & Sons A/S.
FAU - Reshef, A
AU  - Reshef A
AD  - Allergy, Clinical Immunology & Angioedema Unit, Chaim Sheba Medical Center, 52651 
      Tel Hashomer, Israel. areshef@sheba.health.gov.il
FAU - Moldovan, D
AU  - Moldovan D
FAU - Obtulowicz, K
AU  - Obtulowicz K
FAU - Leibovich, I
AU  - Leibovich I
FAU - Mihaly, E
AU  - Mihaly E
FAU - Visscher, S
AU  - Visscher S
FAU - Relan, A
AU  - Relan A
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20121105
PL  - Denmark
TA  - Allergy
JT  - Allergy
JID - 7804028
RN  - 0 (Complement C1 Inhibitor Protein)
RN  - 0 (Recombinant Proteins)
SB  - IM
CIN - Allergy. 2013 Sep;68(9):1207-9. PMID: 24074153
CIN - Allergy. 2013 Sep;68(9):1208-9. PMID: 24215081
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Complement C1 Inhibitor Protein/administration & dosage/adverse 
      effects/pharmacokinetics/*therapeutic use
MH  - Female
MH  - Hereditary Angioedema Types I and II/diagnosis/*prevention & control
MH  - Humans
MH  - Male
MH  - Middle Aged
MH  - Pilot Projects
MH  - Recombinant Proteins/administration & dosage/adverse 
      effects/pharmacokinetics/*therapeutic use
MH  - Treatment Outcome
MH  - Young Adult
EDAT- 2012/11/06 06:00
MHDA- 2013/05/11 06:00
CRDT- 2012/11/06 06:00
PHST- 2012/09/25 00:00 [accepted]
PHST- 2012/11/06 06:00 [entrez]
PHST- 2012/11/06 06:00 [pubmed]
PHST- 2013/05/11 06:00 [medline]
AID - 10.1111/all.12060 [doi]
PST - ppublish
SO  - Allergy. 2013 Jan;68(1):118-24. doi: 10.1111/all.12060. Epub 2012 Nov 5.