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Efficacy and safety of Privigen ® in patients with chronic inflammatory demyelinating polyneuropathy: results of a prospective, single‐arm, open‐label Phase III study (the PRIMA study)
Author(s) -
Léger JeanMarc,
De Bleecker Jan L.,
Sommer Claudia,
Robberecht Wim,
Saarela Mika,
Kamienowski Jerzy,
Stelmasiak Zbigniew,
Mielke Orell,
Tackenberg Björn,
Shebl Amgad,
Bauhofer Artur,
Zenker Othmar,
Merkies Ingemar S. J.
Publication year - 2013
Publication title -
journal of the peripheral nervous system
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1
H-Index - 67
eISSN - 1529-8027
pISSN - 1085-9489
DOI - 10.1111/jns5.12017
Subject(s) - medicine , chronic inflammatory demyelinating polyneuropathy , adverse effect , clinical endpoint , polyneuropathy , confidence interval , prospective cohort study , peripheral neuropathy , gastroenterology , open label , grip strength , antibody , surgery , clinical trial , immunology , diabetes mellitus , endocrinology
This prospective, multicenter, single‐arm, open‐label Phase III study aimed to evaluate the efficacy and safety of Privigen ® (10% liquid human intravenous immunoglobulin [ IVIG ], stabilized with l ‐proline) in patients with chronic inflammatory demyelinating polyneuropathy ( CIDP ). Patients received one induction dose of Privigen (2 g/kg body weight [bw]) and up to seven maintenance doses (1 g/kg bw) at 3‐week intervals. The primary efficacy endpoint was the responder rate at completion, defined as improvement of ≥1 point on the adjusted Inflammatory Neuropathy Cause and Treatment ( INCAT ) disability scale. The preset success criterion was the responder rate being ≥35%. Of the 31 screened patients, 28 patients were enrolled including 13 (46.4%) IVIG ‐pretreated patients. The overall responder rate at completion was 60.7% (95% confidence interval [ CI ]: 42.41%–76.43%). IVIG ‐pretreated patients demonstrated a higher responder rate than IVIG ‐naïve patients (76.9% vs. 46.7%). The median (25%–75% quantile) INCAT score improved from 3.5 (3.0–4.5) points at baseline to 2.5 (1.0–3.0) points at completion, as did the mean (standard deviation [ SD ]) maximum grip strength (66.7 [37.24] kPa vs. 80.9 [31.06] kPa ) and the median Medical Research Council sum score (67.0 [61.5–72.0] points vs. 75.5 [71.5–79.5] points). Of 108 adverse events ( AEs ; 0.417 AEs per infusion), 95 AEs (88.0%) were mild or moderate in intensity and resolved by the end of study. Two serious AEs of hemolysis were reported that resolved after discontinuation of treatment. Thus, Privigen provided efficacious and well‐tolerated induction and maintenance treatment in patients with CIDP .