title: 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).
abstract: 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.
authors.affiliation: Reference Center for Rare Neuromuscular Diseases, Hôpital Pitié-Salpêtrière and University Paris VI, Paris, France. jean-marc.leger@psl.aphp.fr
authors.forename: Jean-Marc
authors.initials: JM
authors.lastname: Léger
authors.forename: Jan L
authors.initials: JL
authors.lastname: De Bleecker
authors.forename: Claudia
authors.initials: C
authors.lastname: Sommer
authors.forename: Wim
authors.initials: W
authors.lastname: Robberecht
authors.forename: Mika
authors.initials: M
authors.lastname: Saarela
authors.forename: Jerzy
authors.initials: J
authors.lastname: Kamienowski
authors.forename: Zbigniew
authors.initials: Z
authors.lastname: Stelmasiak
authors.forename: Orell
authors.initials: O
authors.lastname: Mielke
authors.forename: Björn
authors.initials: B
authors.lastname: Tackenberg
authors.forename: Amgad
authors.initials: A
authors.lastname: Shebl
authors.forename: Artur
authors.initials: A
authors.lastname: Bauhofer
authors.forename: Othmar
authors.initials: O
authors.lastname: Zenker
authors.forename: Ingemar S J
authors.initials: IS
authors.lastname: Merkies
mesh_terms: D000328:Adult; D000368:Aged; D005260:Female; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use*; D007155:Immunologic Factors / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D020277:Polyradiculoneuropathy, Chronic Inflammatory Demyelinating / Q000188:drug therapy*; D011446:Prospective Studies; D055815:Young Adult
chemical_list: D016756:Immunoglobulins, Intravenous; D007155:Immunologic Factors