Clinical response to rhC1-INH in one out of six patients
Six patients were included in the study, four females and two males.
C1-INH function and C4 levels were normal in all patients. At the start
of the study, the mean age was 41 years and the median disease duration
was 8 years. Frequency of AE attacks varied from 2.6 attacks to 15.8
attacks per month in the six months prior to the study. Other baseline
characteristics are presented in table 1. None of the patients had been
treated with long term immunosuppressive treatment (methotrexate,
azathioprine, mycophenolic acid, cyclosporine), Icatibant or omalizumab
before the study.
In patient 1, the attack frequency was reduced by 84% (6.3-fold) during
the treatment period compared to that during the observation period (3
versus 19 attacks, respectively; Figure 1). One of the three attacks in
this patient during the second treatment month occurred 7 days after the
last rhC1-INH administration when the patient had missed a treatment
visit. The AAS28 scores of this patient decreased 8-fold during
treatment months compared to the observation period with an accumulated
AAS score of 29 in the two treatment months versus 233 in the two
observational months (table 2). AE related quality of life (AE-QoL)
scores improved, from 26 (small effect on AE-QoL) during the observation
period to 12,5 (no effect on AE-QoL) during treatment.
None of the other patients (patients 2 to 6) showed a clinical response
to rhC1-INH either measured as attack frequency or accumulated AAS28
scores. No improvement of AE-QoL during treatment was observed
(Supplementary table 2). Patient 3 showed a trend of a higher AAS score
during the treatment period. Patients 4, 5 and 6 had lower AAS scores in
the second treatment and observation periods compared to those during
the first treatment and observation period.