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Abstract
Discussion Forum (0)
Assessment timing and choice of outcome measure in determining treatment response in CIDP: a post-hoc analysis of the PRISM trial
Poster No: 1218

Presenter: Rabye Ouaja
Institution: University Hospitals Birmingham NHS Foundation Trust, U.K.

Introduction: Treatment response and its timing is variable in chronic inflammatory demyelinating polyneuropathy (CIDP). We here aimed to study this variability with multiple outcome measures.

Methods: We performed a post-hoc analysis of the PRISM trial, a 24-week prospective, multicentre, single-arm, open-label phase 3 study of IqYmune, a 10% intravenous immunoglobulin preparation, for CIDP. We ascertained timing of response with primary/secondary outcome measures.

Results: At 6 weeks post-treatment initiation, 13/40 subjects (32.5%) were defined as responders on the primary outcome measure, the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scale. This increased to 20/41 (48.8%) at 12 weeks and to 32/42 (76.2%) at 24 weeks. Use of minimal important difference (MID)-determined amelioration of the inflammatory Rasch-built Overall Disability Scale (I-RODS), or of the Medical Research Council Sum Score (MRCSS), or of dominant hand grip strength, in addition to the adjusted INCAT, offered a sensitivity of 41.7% in identifying adjusted INCAT non-responders at week 12 who subsequently responded at week 24. Specificity was of 60% versus INCAT non-responders at week 24. Consideration of amelioration of any amplitude on any secondary outcome measure offered a 75% sensitivity but only 30% specificity versus adjusted INCAT nonresponders at week 24.

Conclusions: Immunoglobulin treatment continuation may be justified for up to 24 weeks in CIDP. Additional outcome measures may help in early treatment stages to predict delayed response on the adjusted INCAT. However, their use is limited by high false-positive rates. More robust, reliable and relevant outcome measures are needed to detect early improvement in CIDP.

References: Yes
References 1: Clinical Trial J Peripher Nerv Syst. 2020 Dec;25(4):356-365. An international multicenter efficacy and safety study of IqYmune in initial and maintenance treatment of patients with chronic inflammatory demyelinating polyradiculoneuropathy: PRISM study.Eduardo Nobile-Orazio, Sonia Pujol, Fabrice Kasiborski, Rabye Ouaja, Gilles Della Corte, Robert Bonek, Dario Cocito, Angelo Schenone
References 2:
References 3:
References 4:

Grant Support:

Keywords: Chronic Inflammatory Demyelinating Polyneuropathy, , immunoglobulins, , outcome measures, , response to treatment
Assessment timing and choice of outcome measure in determining treatment response in CIDP: a post-hoc analysis of the PRISM trial
Poster No: 1218

Presenter: Rabye Ouaja
Institution: University Hospitals Birmingham NHS Foundation Trust, U.K.

Introduction: Treatment response and its timing is variable in chronic inflammatory demyelinating polyneuropathy (CIDP). We here aimed to study this variability with multiple outcome measures.

Methods: We performed a post-hoc analysis of the PRISM trial, a 24-week prospective, multicentre, single-arm, open-label phase 3 study of IqYmune, a 10% intravenous immunoglobulin preparation, for CIDP. We ascertained timing of response with primary/secondary outcome measures.

Results: At 6 weeks post-treatment initiation, 13/40 subjects (32.5%) were defined as responders on the primary outcome measure, the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scale. This increased to 20/41 (48.8%) at 12 weeks and to 32/42 (76.2%) at 24 weeks. Use of minimal important difference (MID)-determined amelioration of the inflammatory Rasch-built Overall Disability Scale (I-RODS), or of the Medical Research Council Sum Score (MRCSS), or of dominant hand grip strength, in addition to the adjusted INCAT, offered a sensitivity of 41.7% in identifying adjusted INCAT non-responders at week 12 who subsequently responded at week 24. Specificity was of 60% versus INCAT non-responders at week 24. Consideration of amelioration of any amplitude on any secondary outcome measure offered a 75% sensitivity but only 30% specificity versus adjusted INCAT nonresponders at week 24.

Conclusions: Immunoglobulin treatment continuation may be justified for up to 24 weeks in CIDP. Additional outcome measures may help in early treatment stages to predict delayed response on the adjusted INCAT. However, their use is limited by high false-positive rates. More robust, reliable and relevant outcome measures are needed to detect early improvement in CIDP.

References: Yes
References 1: Clinical Trial J Peripher Nerv Syst. 2020 Dec;25(4):356-365. An international multicenter efficacy and safety study of IqYmune in initial and maintenance treatment of patients with chronic inflammatory demyelinating polyradiculoneuropathy: PRISM study.Eduardo Nobile-Orazio, Sonia Pujol, Fabrice Kasiborski, Rabye Ouaja, Gilles Della Corte, Robert Bonek, Dario Cocito, Angelo Schenone
References 2:
References 3:
References 4:

Grant Support:

Keywords: Chronic Inflammatory Demyelinating Polyneuropathy, , immunoglobulins, , outcome measures, , response to treatment
Assessment timing and choice of outcome measure in determining treatment response in CIDP: a post-hoc analysis of the PRISM trial
Rabye Ouaja
Rabye Ouaja
PNS 2022 Annual Meeting eLibrary. Ouaja R. 04/14/2022; 356071; 1218
user
Rabye Ouaja
Abstract
Discussion Forum (0)
Assessment timing and choice of outcome measure in determining treatment response in CIDP: a post-hoc analysis of the PRISM trial
Poster No: 1218

Presenter: Rabye Ouaja
Institution: University Hospitals Birmingham NHS Foundation Trust, U.K.

Introduction: Treatment response and its timing is variable in chronic inflammatory demyelinating polyneuropathy (CIDP). We here aimed to study this variability with multiple outcome measures.

Methods: We performed a post-hoc analysis of the PRISM trial, a 24-week prospective, multicentre, single-arm, open-label phase 3 study of IqYmune, a 10% intravenous immunoglobulin preparation, for CIDP. We ascertained timing of response with primary/secondary outcome measures.

Results: At 6 weeks post-treatment initiation, 13/40 subjects (32.5%) were defined as responders on the primary outcome measure, the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scale. This increased to 20/41 (48.8%) at 12 weeks and to 32/42 (76.2%) at 24 weeks. Use of minimal important difference (MID)-determined amelioration of the inflammatory Rasch-built Overall Disability Scale (I-RODS), or of the Medical Research Council Sum Score (MRCSS), or of dominant hand grip strength, in addition to the adjusted INCAT, offered a sensitivity of 41.7% in identifying adjusted INCAT non-responders at week 12 who subsequently responded at week 24. Specificity was of 60% versus INCAT non-responders at week 24. Consideration of amelioration of any amplitude on any secondary outcome measure offered a 75% sensitivity but only 30% specificity versus adjusted INCAT nonresponders at week 24.

Conclusions: Immunoglobulin treatment continuation may be justified for up to 24 weeks in CIDP. Additional outcome measures may help in early treatment stages to predict delayed response on the adjusted INCAT. However, their use is limited by high false-positive rates. More robust, reliable and relevant outcome measures are needed to detect early improvement in CIDP.

References: Yes
References 1: Clinical Trial J Peripher Nerv Syst. 2020 Dec;25(4):356-365. An international multicenter efficacy and safety study of IqYmune in initial and maintenance treatment of patients with chronic inflammatory demyelinating polyradiculoneuropathy: PRISM study.Eduardo Nobile-Orazio, Sonia Pujol, Fabrice Kasiborski, Rabye Ouaja, Gilles Della Corte, Robert Bonek, Dario Cocito, Angelo Schenone
References 2:
References 3:
References 4:

Grant Support:

Keywords: Chronic Inflammatory Demyelinating Polyneuropathy, , immunoglobulins, , outcome measures, , response to treatment
Assessment timing and choice of outcome measure in determining treatment response in CIDP: a post-hoc analysis of the PRISM trial
Poster No: 1218

Presenter: Rabye Ouaja
Institution: University Hospitals Birmingham NHS Foundation Trust, U.K.

Introduction: Treatment response and its timing is variable in chronic inflammatory demyelinating polyneuropathy (CIDP). We here aimed to study this variability with multiple outcome measures.

Methods: We performed a post-hoc analysis of the PRISM trial, a 24-week prospective, multicentre, single-arm, open-label phase 3 study of IqYmune, a 10% intravenous immunoglobulin preparation, for CIDP. We ascertained timing of response with primary/secondary outcome measures.

Results: At 6 weeks post-treatment initiation, 13/40 subjects (32.5%) were defined as responders on the primary outcome measure, the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scale. This increased to 20/41 (48.8%) at 12 weeks and to 32/42 (76.2%) at 24 weeks. Use of minimal important difference (MID)-determined amelioration of the inflammatory Rasch-built Overall Disability Scale (I-RODS), or of the Medical Research Council Sum Score (MRCSS), or of dominant hand grip strength, in addition to the adjusted INCAT, offered a sensitivity of 41.7% in identifying adjusted INCAT non-responders at week 12 who subsequently responded at week 24. Specificity was of 60% versus INCAT non-responders at week 24. Consideration of amelioration of any amplitude on any secondary outcome measure offered a 75% sensitivity but only 30% specificity versus adjusted INCAT nonresponders at week 24.

Conclusions: Immunoglobulin treatment continuation may be justified for up to 24 weeks in CIDP. Additional outcome measures may help in early treatment stages to predict delayed response on the adjusted INCAT. However, their use is limited by high false-positive rates. More robust, reliable and relevant outcome measures are needed to detect early improvement in CIDP.

References: Yes
References 1: Clinical Trial J Peripher Nerv Syst. 2020 Dec;25(4):356-365. An international multicenter efficacy and safety study of IqYmune in initial and maintenance treatment of patients with chronic inflammatory demyelinating polyradiculoneuropathy: PRISM study.Eduardo Nobile-Orazio, Sonia Pujol, Fabrice Kasiborski, Rabye Ouaja, Gilles Della Corte, Robert Bonek, Dario Cocito, Angelo Schenone
References 2:
References 3:
References 4:

Grant Support:

Keywords: Chronic Inflammatory Demyelinating Polyneuropathy, , immunoglobulins, , outcome measures, , response to treatment

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