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出境医 / 临床实验 / Study of Electrical Prognostic Factors of Response to Intravenous Immunoglobulin Treatment in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (PRONELEC PIDC)

Study of Electrical Prognostic Factors of Response to Intravenous Immunoglobulin Treatment in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (PRONELEC PIDC)

Study Description
Brief Summary:

Chronic inflammatory demyelinating polyradiculoneuropathy (PIDC) is an acquired dysimmune polyneuropathy whose prevalence is estimated to be between 1 to 4 per 100,000 depending on the study, with an incidence of less than 1 per 100,000 per year. The clinical presentation of PIDC is heterogeneous, characterized by a symmetrical lesion predominating generally on large fibers, the most myelinated, responsible for ataxia and a motor deficit in the foreground. In the typical form, patients describe a proximal and distal motor or sensory deficit associated with isflexia that signifies a peripheral neurogenic syndrome.

The physiopathological hypothesis is that of an inflammation responsible for demyelinating nerve fibers, which results in electroneuromyogram (ENMG) by conduction abnormalities and histologically when a neuromuscular biopsy is performed by segmental demyelination.

Given the heterogeneity of the clinical presentation, electrical diagnostic criteria are proposed by the European Federation of Neurological Society in order to classify IPDCs into three categories: certain, probable and possible. In the absence of sufficient criteria to make the diagnosis of IPDC, it is also possible to use criteria of support, using a paraclinical report including the presence of an increase in protein (hyperproteinorachie) without cells for cerebrospinal fluid analysis, visualization of radicular inflammation on imaging (MRI of lumbar and / or brachial plexus), proximal peripheral involvement with somatosensory evoked potentials.

Therapeutically, a joint management between rehabilitation and the introduction of a background treatment allows the clinical improvement of certain patients. To date, the treatments proposed in first intention are corticosteroids, intravenous immunoglobulins (IVIg) and plasma exchanges. In fact, the efficacy of intravenous immunoglobulins has been widely shown by controlled and randomized therapeutic trials. Efficacy studies of IVIg in the literature are most often based on an assessment of clinical response after 24 weeks, but in clinical practice the response to treatment and continuation of treatment is often evaluated after 3 courses of treatment with the help of a clinical evaluation and the realization of an electroneuromyogram. These are administered in day hospitalization or traditional hospitalization, every four weeks, to patients whose diagnosis of PIDC has been established by electroneuromyogram according to the EFNS criteria.

Clinical prognostic factors for good response to IVIG therapy have been described in previous studies, including subacute disease, symmetrical involvement, and absence of amyotrophy.

In order to optimize the management of IPDCs, it is important to identify patients who respond to IVIg. Thus, the objective of our study is to look at the electroneuromyogram, the presence of electrical predictors of good response to treatment with IVIg.


Condition or disease
Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Study Design
Layout table for study information
Study Type : Observational
Actual Enrollment : 42 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Study of Electrical Prognostic Factors of Response to Intravenous Immunoglobulin Treatment in Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Actual Study Start Date : July 17, 2019
Actual Primary Completion Date : October 31, 2020
Estimated Study Completion Date : March 31, 2021
Arms and Interventions
Outcome Measures
Primary Outcome Measures :
  1. Identify electromyographic predictors of treatment response [ Time Frame: Week 24 ]
    This corresponds to highlight, in the data of the electroneuromyogram performed before the initiation of treatment with IVIg, differences between the responder groups or not treatment [decrease of at least 1 point in the ONLS score], i.e three courses of IVIG.


Secondary Outcome Measures :
  1. study of the demographic characteristics of patients followed for IPDC and treated with IVIg [ Time Frame: Week 24 ]
    This corresponds to percentage of patients with a certain / probable or possible IPDC.

  2. Clinical evidence of response to IVIG treatment [ Time Frame: Week 24 ]
    comparison of the results of the complementary explorations in the patients answering or not to the treatment


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with IPDC, hospitalized in a traditional hospital ward or week hospital or day hospital, who received at least 3 courses of IVIG under IPDC between January 2014 and March 2019
Criteria

Inclusion Criteria:

  • Patients whose age is ≥ 18 years
  • Patients with IPDC
  • Patients hospitalized in a traditional hospital ward or week hospital or day hospital
  • Patients who received at least 3 courses of IVIG under IPDC between January 2014 and March 2019
  • Francophone patients

Exclusion Criteria:

  • Patients under guardianship or curatorship
  • Patients deprived of their liberty
  • Patients who oppose the use of their data for this research
Contacts and Locations

Locations
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France
Groupe Hospitalier Paris Saint-Joseph
Paris, France, 75014
Sponsors and Collaborators
Groupe Hospitalier Paris Saint Joseph
Investigators
Layout table for investigator information
Principal Investigator: Pauline REACH, MD Groupe Hospitalier Paris Saint Joseph
Tracking Information
First Submitted Date May 24, 2019
First Posted Date May 30, 2019
Last Update Posted Date February 4, 2021
Actual Study Start Date July 17, 2019
Actual Primary Completion Date October 31, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 28, 2019)
Identify electromyographic predictors of treatment response [ Time Frame: Week 24 ]
This corresponds to highlight, in the data of the electroneuromyogram performed before the initiation of treatment with IVIg, differences between the responder groups or not treatment [decrease of at least 1 point in the ONLS score], i.e three courses of IVIG.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: May 28, 2019)
  • study of the demographic characteristics of patients followed for IPDC and treated with IVIg [ Time Frame: Week 24 ]
    This corresponds to percentage of patients with a certain / probable or possible IPDC.
  • Clinical evidence of response to IVIG treatment [ Time Frame: Week 24 ]
    comparison of the results of the complementary explorations in the patients answering or not to the treatment
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Study of Electrical Prognostic Factors of Response to Intravenous Immunoglobulin Treatment in Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Official Title Study of Electrical Prognostic Factors of Response to Intravenous Immunoglobulin Treatment in Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Brief Summary

Chronic inflammatory demyelinating polyradiculoneuropathy (PIDC) is an acquired dysimmune polyneuropathy whose prevalence is estimated to be between 1 to 4 per 100,000 depending on the study, with an incidence of less than 1 per 100,000 per year. The clinical presentation of PIDC is heterogeneous, characterized by a symmetrical lesion predominating generally on large fibers, the most myelinated, responsible for ataxia and a motor deficit in the foreground. In the typical form, patients describe a proximal and distal motor or sensory deficit associated with isflexia that signifies a peripheral neurogenic syndrome.

The physiopathological hypothesis is that of an inflammation responsible for demyelinating nerve fibers, which results in electroneuromyogram (ENMG) by conduction abnormalities and histologically when a neuromuscular biopsy is performed by segmental demyelination.

Given the heterogeneity of the clinical presentation, electrical diagnostic criteria are proposed by the European Federation of Neurological Society in order to classify IPDCs into three categories: certain, probable and possible. In the absence of sufficient criteria to make the diagnosis of IPDC, it is also possible to use criteria of support, using a paraclinical report including the presence of an increase in protein (hyperproteinorachie) without cells for cerebrospinal fluid analysis, visualization of radicular inflammation on imaging (MRI of lumbar and / or brachial plexus), proximal peripheral involvement with somatosensory evoked potentials.

Therapeutically, a joint management between rehabilitation and the introduction of a background treatment allows the clinical improvement of certain patients. To date, the treatments proposed in first intention are corticosteroids, intravenous immunoglobulins (IVIg) and plasma exchanges. In fact, the efficacy of intravenous immunoglobulins has been widely shown by controlled and randomized therapeutic trials. Efficacy studies of IVIg in the literature are most often based on an assessment of clinical response after 24 weeks, but in clinical practice the response to treatment and continuation of treatment is often evaluated after 3 courses of treatment with the help of a clinical evaluation and the realization of an electroneuromyogram. These are administered in day hospitalization or traditional hospitalization, every four weeks, to patients whose diagnosis of PIDC has been established by electroneuromyogram according to the EFNS criteria.

Clinical prognostic factors for good response to IVIG therapy have been described in previous studies, including subacute disease, symmetrical involvement, and absence of amyotrophy.

In order to optimize the management of IPDCs, it is important to identify patients who respond to IVIg. Thus, the objective of our study is to look at the electroneuromyogram, the presence of electrical predictors of good response to treatment with IVIg.

Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Retrospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients with IPDC, hospitalized in a traditional hospital ward or week hospital or day hospital, who received at least 3 courses of IVIG under IPDC between January 2014 and March 2019
Condition Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: August 5, 2019)
42
Original Estimated Enrollment
 (submitted: May 28, 2019)
40
Estimated Study Completion Date March 31, 2021
Actual Primary Completion Date October 31, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Patients whose age is ≥ 18 years
  • Patients with IPDC
  • Patients hospitalized in a traditional hospital ward or week hospital or day hospital
  • Patients who received at least 3 courses of IVIG under IPDC between January 2014 and March 2019
  • Francophone patients

Exclusion Criteria:

  • Patients under guardianship or curatorship
  • Patients deprived of their liberty
  • Patients who oppose the use of their data for this research
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries France
Removed Location Countries  
 
Administrative Information
NCT Number NCT03967899
Other Study ID Numbers PRONELEC
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Groupe Hospitalier Paris Saint Joseph
Study Sponsor Groupe Hospitalier Paris Saint Joseph
Collaborators Not Provided
Investigators
Principal Investigator: Pauline REACH, MD Groupe Hospitalier Paris Saint Joseph
PRS Account Groupe Hospitalier Paris Saint Joseph
Verification Date February 2021