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.

Neurofascin-155 IgG4 in chronic inflammatory demyelinating polyneuropathy

“…This was much below the percentage of responsive patients in our negative group (59%) or in reported studies (54%-63%). 1,[22][23][24] Similar observations were reported by a Spanish group, in which none of the 4 patients responded to IVIg. 3 In contrast, beneficial response was noted after plasma exchange in another study.…”

Immunotherapy in Peripheral Neuropathies

“…Moreover long-term therapy with IGIV-C, showed a maintained improvement in health-related quality of life in patients with CIDP [48]. The PRIMA study [49] confirmed these results with another IVIG 10 % brand.…”

Disease-modifying therapy in multiple sclerosis and chronic inflammatory demyelinating polyradiculoneuropathy: common and divergent current and future strategies

“…Another recent prospective, multi-centre, single-arm, open-label Phase III study [Privigen® Impact on Mobility and Autonomy (PRIMA) trial] evaluated the efficacy and safety of IVIG in 28 patients with CIDP [38]. Patients received one induction dose of IVIG (2 g/kg body weight) and up to seven maintenance doses (1 g/kg body weight) at 3-week intervals.…”