Efficacy and safety of IVIG in CIDP: Combined data of the PRIMA and PATH studies

on Behalf of the PRIMA Trial Investigators and the PATH Study Group

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

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)-naïve 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.].

Original languageEnglish (US)
Pages (from-to)48-55
Number of pages8
JournalJournal of the Peripheral Nervous System
Volume24
Issue number1
DOIs
StatePublished - Mar 1 2019

Fingerprint

Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Polyneuropathies
Intravenous Immunoglobulins
Safety
Drug-Related Side Effects and Adverse Reactions
Hand Strength
Hizentra
Biomedical Research
Double-Blind Method
Publications
Immunoglobulins

Keywords

  • CIDP
  • IVIG
  • PATH
  • PRIMA
  • efficacy

Cite this

Efficacy and safety of IVIG in CIDP : Combined data of the PRIMA and PATH studies. / on Behalf of the PRIMA Trial Investigators and the PATH Study Group.

In: Journal of the Peripheral Nervous System, Vol. 24, No. 1, 01.03.2019, p. 48-55.

Research output: Contribution to journalArticle

on Behalf of the PRIMA Trial Investigators and the PATH Study Group 2019, 'Efficacy and safety of IVIG in CIDP: Combined data of the PRIMA and PATH studies', Journal of the Peripheral Nervous System, vol. 24, no. 1, pp. 48-55. https://doi.org/10.1111/jns.12302
on Behalf of the PRIMA Trial Investigators and the PATH Study Group. / Efficacy and safety of IVIG in CIDP : Combined data of the PRIMA and PATH studies. In: Journal of the Peripheral Nervous System. 2019 ; Vol. 24, No. 1. pp. 48-55.
@article{11dcc34405044fc28e954f65634b25cf,
title = "Efficacy and safety of IVIG in CIDP: Combined data of the PRIMA and PATH studies",
abstract = "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)-na{\"i}ve 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.].",
keywords = "CIDP, IVIG, PATH, PRIMA, efficacy",
author = "{on Behalf of the PRIMA Trial Investigators and the PATH Study Group} and Merkies, {Ingemar S.J.} and {van Schaik}, {Ivo N.} and L{\'e}ger, {Jean Marc} and Vera Bril and {van Geloven}, Nan and Hartung, {Hans Peter} and Lewis, {Richard A.} and Gen Sobue and Lawo, {John Philip} and Durn, {Billie L.} and Cornblath, {David R.} and {De Bleecker}, {Jan L.} and Claudia Sommer and Wim Robberecht and Mika Saarela and Jerzy Kamienowski and Zbigniew Stelmasiak and Bj{\"o}rn Tackenberg and Orell Mielke and A. Sabet and K. George and L. Roberts and R. Carne and S. Blum and R. Henderson and {Van Damme}, P. and J. Demeestere and S. Larue and C. D'Amour and P. Kunc and M. Valis and J. Sussova and T. Kalous and R. Talab and M. Bednar and T. Toomsoo and I. Rubanovits and K. Gross-Paju and U. Sorro and M. Saarela and M. Auranen and J. Pouget and S. Attarian and Masson, {G. Le} and A. Wielanek-Bachelet and C. Desnuelle and E. Delmont and P. Clavelou and Allen, {Jeffrey A} and David Walk",
year = "2019",
month = "3",
day = "1",
doi = "10.1111/jns.12302",
language = "English (US)",
volume = "24",
pages = "48--55",
journal = "Journal of the Peripheral Nervous System",
issn = "1085-9489",
publisher = "Wiley-Blackwell",
number = "1",

}

TY - JOUR

T1 - Efficacy and safety of IVIG in CIDP

T2 - Combined data of the PRIMA and PATH studies

AU - on Behalf of the PRIMA Trial Investigators and the PATH Study Group

AU - Merkies, Ingemar S.J.

AU - van Schaik, Ivo N.

AU - Léger, Jean Marc

AU - Bril, Vera

AU - van Geloven, Nan

AU - Hartung, Hans Peter

AU - Lewis, Richard A.

AU - Sobue, Gen

AU - Lawo, John Philip

AU - Durn, Billie L.

AU - Cornblath, David R.

AU - De Bleecker, Jan L.

AU - Sommer, Claudia

AU - Robberecht, Wim

AU - Saarela, Mika

AU - Kamienowski, Jerzy

AU - Stelmasiak, Zbigniew

AU - Tackenberg, Björn

AU - Mielke, Orell

AU - Sabet, A.

AU - George, K.

AU - Roberts, L.

AU - Carne, R.

AU - Blum, S.

AU - Henderson, R.

AU - Van Damme, P.

AU - Demeestere, J.

AU - Larue, S.

AU - D'Amour, C.

AU - Kunc, P.

AU - Valis, M.

AU - Sussova, J.

AU - Kalous, T.

AU - Talab, R.

AU - Bednar, M.

AU - Toomsoo, T.

AU - Rubanovits, I.

AU - Gross-Paju, K.

AU - Sorro, U.

AU - Saarela, M.

AU - Auranen, M.

AU - Pouget, J.

AU - Attarian, S.

AU - Masson, G. Le

AU - Wielanek-Bachelet, A.

AU - Desnuelle, C.

AU - Delmont, E.

AU - Clavelou, P.

AU - Allen, Jeffrey A

AU - Walk, David

PY - 2019/3/1

Y1 - 2019/3/1

N2 - 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)-naïve 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.].

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)-naïve 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.].

KW - CIDP

KW - IVIG

KW - PATH

KW - PRIMA

KW - efficacy

UR - http://www.scopus.com/inward/record.url?scp=85061703237&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85061703237&partnerID=8YFLogxK

U2 - 10.1111/jns.12302

DO - 10.1111/jns.12302

M3 - Article

C2 - 30672091

AN - SCOPUS:85061703237

VL - 24

SP - 48

EP - 55

JO - Journal of the Peripheral Nervous System

JF - Journal of the Peripheral Nervous System

SN - 1085-9489

IS - 1

ER -