PMID- 30672091 OWN - NLM STAT- MEDLINE DCOM- 20200604 LR - 20210109 IS - 1529-8027 (Electronic) IS - 1085-9489 (Print) IS - 1085-9489 (Linking) VI - 24 IP - 1 DP - 2019 Mar TI - Efficacy and safety of IVIG in CIDP: Combined data of the PRIMA and PATH studies. PG - 48-55 LID - 10.1111/jns.12302 [doi] AB - Intravenous immunoglobulin (IVIG) is a potential therapy for chronic inflammatory demyelinating polyneuropathy (CIDP). To investigate the efficacy and safety of the IVIG IgPro10 (Privigen) for treatment of CIDP, results from Privigen Impact on Mobility and Autonomy (PRIMA), a prospective, open-label, single-arm study of IVIG in immunoglobulin (Ig)-naive or IVIG pre-treated subjects (NCT01184846, n = 28) and Polyneuropathy And Treatment with Hizentra (PATH), a double-blind, randomized study including an open-label, single-arm IVIG phase in IVIG pre-treated subjects (NCT01545076, IVIG restabilization phase n = 207) were analyzed separately and together (n = 235). Efficacy assessments included change in adjusted inflammatory neuropathy cause and treatment (INCAT) score, grip strength and Medical Research Council (MRC) sum score. Adverse drug reactions (ADRs) and ADRs/infusion were recorded. Adjusted INCAT response rate was 60.7% in all PRIMA subjects at Week 25 (76.9% in IVIG pre-treated subjects) and 72.9% in PATH. In the pooled cohort (n = 235), INCAT response rate was 71.5%; median time to INCAT improvement was 4.3 weeks. No clear demographic differences were noticed between early (responding before Week 7, n = 148) and late responders (n = 21). In the pooled cohort, median change from baseline to last observation was -1.0 (interquartile range -2.0; 0.0) point for INCAT score; +8.0 (0.0; 20.0) kPa for maximum grip strength; +3.0 (1.0; 7.0) points for MRC sum score. In the pooled cohort, 271 ADRs were reported in 105 subjects (44.7%), a rate of 0.144 ADRs per infusion. This analysis confirms the efficacy and safety of IgPro10, a recently FDA-approved IVIG for CIDP, in a population of mainly pre-treated subjects with CIDP [Correction added on 14 March 2019 after first online publication: the INCAT response rate has been corrected.]. CI - (c) 2019 The Authors. Journal of the Peripheral Nervous System published by Wiley Periodicals, Inc. on behalf of Peripheral Nerve Society. FAU - Merkies, Ingemar S J AU - Merkies ISJ AD - Department of Neurology, Maastricht University Medical Center, Maastricht, The Netherlands. AD - Department of Neurology, St Elisabeth Hospital, Willemstad, Curacao. FAU - van Schaik, Ivo N AU - van Schaik IN AD - Department of Neurology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, The Netherlands. FAU - Leger, Jean-Marc AU - Leger JM AD - National Referral Center for Rare Neuromuscular Diseases, Hopital Pitie-Salpetriere and University Paris VI, Paris, France. FAU - Bril, Vera AU - Bril V AD - Ellen and Martin Prosserman Centre for Neuromuscular Diseases, Division of Neurology, Department of Medicine, University Health Network, University of Toronto, Toronto, Canada. AD - Institute for Research and Medical Consultations, Imam Abdulrahman Bin Faisal University, Dammam, Saudi Arabia. FAU - van Geloven, Nan AU - van Geloven N AD - Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands. FAU - Hartung, Hans-Peter AU - Hartung HP AD - Department of Neurology, UKD and Center for Neurology and Neuropsychiatry, LVR Klinikum, Medical Faculty, Heinrich Heine University, Dusseldorf, Germany. FAU - Lewis, Richard A AU - Lewis RA AD - Department of Neurology, Cedars-Sinai Medical Center, Los Angeles, California. FAU - Sobue, Gen AU - Sobue G AD - Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya, Japan. FAU - Lawo, John-Philip AU - Lawo JP AD - CSL Behring, Marburg, Germany, and King of Prussia, Pennsylvania. FAU - Durn, Billie L AU - Durn BL AD - CSL Behring, Marburg, Germany, and King of Prussia, Pennsylvania. FAU - Cornblath, David R AU - Cornblath DR AD - Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland. FAU - De Bleecker, Jan L AU - De Bleecker JL AD - Department of Neurology, AZ St-Lucas, Ghent, Belgium. FAU - Sommer, Claudia AU - Sommer C AD - Department of Neurology, Universitatsklinikum Wurzburg, Wurzburg, Germany. FAU - Robberecht, Wim AU - Robberecht W AD - Department of Neurosciences, UZ Leuven, Leuven, Belgium. FAU - Saarela, Mika AU - Saarela M AD - Department of Neurology, Helsinki University Central Hospital, Helsinki, Finland. FAU - Kamienowski, Jerzy AU - Kamienowski J AD - Dolnoslaski Szpital Specjalistyczny, Wroclaw, Poland. FAU - Stelmasiak, Zbigniew AU - Stelmasiak Z AD - Department of Neurology, Samodzielny Publiczny Szpital Kliniczny, Lublin, Poland. FAU - Tackenberg, Bjorn AU - Tackenberg B AD - Department of Neurology, Philipps University, Marburg, Germany. FAU - Mielke, Orell AU - Mielke O AD - CSL Behring, Marburg, Germany, and King of Prussia, Pennsylvania. CN - PRIMA Trial Investigators and the PATH Study Group LA - eng PT - Journal Article PT - Randomized Controlled Trial PT - Research Support, Non-U.S. Gov't DEP - 20190215 PL - United States TA - J Peripher Nerv Syst JT - Journal of the peripheral nervous system : JPNS JID - 9704532 RN - 0 (Immunoglobulins, Intravenous) RN - 0 (Immunologic Factors) SB - IM MH - Adult MH - Aged MH - Aged, 80 and over MH - Double-Blind Method MH - Europe MH - Female MH - Humans MH - Immunoglobulins, Intravenous/administration & dosage/*pharmacology MH - Immunologic Factors/administration & dosage/*pharmacology MH - Male MH - Middle Aged MH - *Outcome Assessment, Health Care MH - Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/*drug therapy MH - Prospective Studies MH - Young Adult PMC - PMC6594229 OTO - NOTNLM OT - CIDP OT - IVIG OT - PATH OT - PRIMA OT - efficacy FIR - Sabet, A IR - Sabet A FIR - George, K IR - George K FIR - Roberts, L IR - Roberts L FIR - Carne, R IR - Carne R FIR - Blum, S IR - Blum S FIR - Henderson, R IR - Henderson R FIR - Van Damme, P IR - Van Damme P FIR - Demeestere, J IR - Demeestere J FIR - Larue, S IR - Larue S FIR - D'Amour, C IR - D'Amour C FIR - Kunc, P IR - Kunc P FIR - Valis, M IR - Valis M FIR - Sussova, J IR - Sussova J FIR - Kalous, T IR - Kalous T FIR - Talab, R IR - Talab R FIR - Bednar, M IR - Bednar M FIR - Toomsoo, T IR - Toomsoo T FIR - Rubanovits, I IR - Rubanovits I FIR - Gross-Paju, K IR - Gross-Paju K FIR - Sorro, U IR - Sorro U FIR - Saarela, M IR - Saarela M FIR - Auranen, M IR - Auranen M FIR - Pouget, J IR - Pouget J FIR - Attarian, S IR - Attarian S FIR - Masson, G Le IR - Masson GL FIR - Wielanek-Bachelet, A IR - Wielanek-Bachelet A FIR - Desnuelle, C IR - Desnuelle C FIR - Delmont, E IR - Delmont E FIR - Clavelou, P IR - Clavelou P FIR - Aufauvre, D IR - Aufauvre D FIR - Schmidt, J IR - Schmidt J FIR - Zschuentzsch, J IR - Zschuentzsch J FIR - Sommer, C IR - Sommer C FIR - Kramer, D IR - Kramer D FIR - Hoffmann, O IR - Hoffmann O FIR - Goerlitz, C IR - Goerlitz C FIR - Haas, J IR - Haas J FIR - Chatzopoulos, M IR - Chatzopoulos M FIR - Yoon, R IR - Yoon R FIR - Gold, R IR - Gold R FIR - Berlit, P IR - Berlit P FIR - Jaspert-Grehl, A IR - Jaspert-Grehl A FIR - Liebetanz, D IR - Liebetanz D FIR - Kutschenko, A IR - Kutschenko A FIR - Stangel, M IR - Stangel M FIR - Trebst, C IR - Trebst C FIR - Baum, P IR - Baum P FIR - Bergh, F IR - Bergh F FIR - Klehmet, J IR - Klehmet J FIR - Meisel, A IR - Meisel A FIR - Klostermann, F IR - Klostermann F FIR - Oechtering, J IR - Oechtering J FIR - Lehmann, H IR - Lehmann H FIR - Schroeter, M IR - Schroeter M FIR - Hagenacker, T IR - Hagenacker T FIR - Mueller, D IR - Mueller D FIR - Sperfeld, A IR - Sperfeld A FIR - Bethke, F IR - Bethke F FIR - Drory, V IR - Drory V FIR - Algom, A IR - Algom A FIR - Yarnitsky, D IR - Yarnitsky D FIR - Murinson, B IR - Murinson B FIR - Di Muzio, A IR - Di Muzio A FIR - Ciccocioppo, F IR - Ciccocioppo F FIR - Sorbi, S IR - Sorbi S FIR - Mata, S IR - Mata S FIR - Schenone, A IR - Schenone A FIR - Grandis, M IR - Grandis M FIR - Lauria, G IR - Lauria G FIR - Cazzato, D IR - Cazzato D FIR - Antonini, G IR - Antonini G FIR - Morino, S IR - Morino S FIR - Cocito, D IR - Cocito D FIR - Zibetti, M IR - Zibetti M FIR - Yokota, T IR - Yokota T FIR - Ohkubo, T IR - Ohkubo T FIR - Kanda, T IR - Kanda T FIR - Kawai, M IR - Kawai M FIR - Kaida, K IR - Kaida K FIR - Onoue, H IR - Onoue H FIR - Kuwabara, S IR - Kuwabara S FIR - Mori, M IR - Mori M FIR - Iijima, M IR - Iijima M FIR - Ohyama, K IR - Ohyama K FIR - Baba, M IR - Baba M FIR - Tomiyama, M IR - Tomiyama M FIR - Nishiyama, K IR - Nishiyama K FIR - Akutsu, T IR - Akutsu T FIR - Yokoyama, K IR - Yokoyama K FIR - Kanai, K IR - Kanai K FIR - van Schaik, I N IR - van Schaik IN FIR - Eftimov, F IR - Eftimov F FIR - Notermans, N C IR - Notermans NC FIR - Visser, N IR - Visser N FIR - Faber, C IR - Faber C FIR - Hoeijmakers, J IR - Hoeijmakers J FIR - Rejdak, K IR - Rejdak K FIR - Chyrchel-Paszkiewicz, U IR - Chyrchel-Paszkiewicz U FIR - Casanovas Pons, C IR - Casanovas Pons C FIR - Antonia, M IR - Antonia M FIR - Gamez, J IR - Gamez J FIR - Salvado, M IR - Salvado M FIR - Infante, C Marquez IR - Infante CM FIR - Benitez, S IR - Benitez S FIR - Lunn, M IR - Lunn M FIR - Morrow, J IR - Morrow J FIR - Gosal, D IR - Gosal D FIR - Lavin, T IR - Lavin T FIR - Melamed, I IR - Melamed I FIR - Testori, A IR - Testori A FIR - Ajroud-Driss, S IR - Ajroud-Driss S FIR - Menichella, D IR - Menichella D FIR - Simpson, E IR - Simpson E FIR - Lai, E Chi-Ho IR - Lai EC FIR - Dimachkie, M IR - Dimachkie M FIR - Barohn, R J IR - Barohn RJ FIR - Beydoun, S IR - Beydoun S FIR - Johl, H IR - Johl H FIR - Lange, D IR - Lange D FIR - Shtilbans, A IR - Shtilbans A FIR - Muley, S IR - Muley S FIR - Ladha, S IR - Ladha S FIR - Freimer, M IR - Freimer M FIR - Kissel, J IR - Kissel J FIR - Latov, N IR - Latov N FIR - Chin, R IR - Chin R FIR - Ubogu, E IR - Ubogu E FIR - Mumfrey, S IR - Mumfrey S FIR - Rao, T IR - Rao T FIR - MacDonald, P IR - MacDonald P FIR - Sharma, K IR - Sharma K FIR - Gonzalez, G IR - Gonzalez G FIR - Allen, J IR - Allen J FIR - Walk, D IR - Walk D FIR - Hobson-Webb, L IR - Hobson-Webb L FIR - Gable, K IR - Gable K FIR - De Bleecker, J L IR - De Bleecker JL FIR - Robberecht, W IR - Robberecht W FIR - Saarela, M IR - Saarela M FIR - Franques, J IR - Franques J FIR - Leger, J-M IR - Leger JM FIR - Morales, R Juntas IR - Morales RJ FIR - Sommer, C IR - Sommer C FIR - Nguento, A IR - Nguento A FIR - Schmidt, J IR - Schmidt J FIR - Schrey, Ch IR - Schrey C FIR - Kamienowski, J IR - Kamienowski J FIR - Stelmasiak, Z IR - Stelmasiak Z FIR - Zwolinska, G IR - Zwolinska G EDAT- 2019/01/24 06:00 MHDA- 2020/06/05 06:00 PMCR- 2019/06/26 CRDT- 2019/01/24 06:00 PHST- 2018/10/10 00:00 [received] PHST- 2018/11/15 00:00 [revised] PHST- 2019/01/12 00:00 [accepted] PHST- 2019/01/24 06:00 [pubmed] PHST- 2020/06/05 06:00 [medline] PHST- 2019/01/24 06:00 [entrez] PHST- 2019/06/26 00:00 [pmc-release] AID - JNS12302 [pii] AID - 10.1111/jns.12302 [doi] PST - ppublish SO - J Peripher Nerv Syst. 2019 Mar;24(1):48-55. doi: 10.1111/jns.12302. Epub 2019 Feb 15.