Subcutaneous C1-Esterase Inhibitor [C1-INH(SC)] to Prevent Hereditary Angioedema (HAE) Attacks: Subject and Investigator Assessments from the Compact Trial

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY(2017)

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Abstract
The COMPACT trial established the safety and efficacy of routine prevention with C1-INH(SC) in subjects with HAE. This analysis presents subject- and investigator-reported assessments of treatment effectiveness. Subjects (n=90) were randomized to 1 of 4 treatment sequences: twice-weekly C1-INH(SC) (40 or 60 IU/kg) for 16 weeks, preceded or followed by placebo for 16 weeks. Assessments using the Treatment Satisfaction Questionnaire for Medication (TSQM), the Investigator’s Global Assessment of Response to Therapy (IGART), and the Subject’s Global Assessment of Response to Therapy (SGART) were conducted after 14 weeks of C1-INH(SC) and after 14 weeks of placebo. A post hoc comparison of the mean (within-subject) difference in TSQM scores was performed. The percentage of subjects with responses of none, poor, fair, good, or excellent was determined using the IGART/SGART. C1-INH(SC) (40 and 60 IU/kg, combined) had a larger effect on TSQM Effectiveness compared with placebo (mean difference [95% CI], 37.07 [24.86, 49.28]). For both the IGART and the SGART, the percentages of subjects with a “good or excellent” response to therapy were higher during treatment with C1-INH(SC) than placebo. For the IGART, 80.0% (72/90) of subjects on C1-INH(SC) (40 and 60 IU/kg, combined) received a rating of “good or excellent” versus 12.2% (11/90) on placebo. For the SGART, 75.6% (68/90) of subjects on C1-INH(SC) (40 and 60 IU/kg, combined) indicated a rating of “good or excellent” versus 23.3% (21/90) on placebo. These findings provide further evidence that C1-INH(SC) is an effective attack prevention option for the management of HAE.
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C1 Inhibitor Deficiency
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