See Appendix for complete list of Study group members.
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)
Version of Record online: 19 JUN 2013
© 2013 The Authors. Journal of the Peripheral Nervous System published by Wiley Periodicals, Inc. on behalf of Peripheral Nerve Society.
This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
Journal of the Peripheral Nervous System
Volume 18, Issue 2, pages 130–140, June 2013
How to Cite
Léger, J.-M., De Bleecker, J. L., Sommer, C., Robberecht, W., Saarela, M., Kamienowski, J., Stelmasiak, Z., Mielke, O., Tackenberg, B., Shebl, A., Bauhofer, A., Zenker, O., Merkies, I. S. J. and on behalf of the PRIMA study investigators (2013), 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). Journal of the Peripheral Nervous System, 18: 130–140. doi: 10.1111/jns5.12017
- Issue online: 19 JUN 2013
- Version of Record online: 19 JUN 2013
- Accepted manuscript online: 15 MAY 2013 11:49AM EST
- Manuscript Accepted: 9 APR 2013
- Manuscript Revised: 2 APR 2013
- Manuscript Received: 9 NOV 2012
- CSL Behring AG
- chronic inflammatory demyelinating polyneuropathy;
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.