Efficacy and Tolerance of Therapeutic Apheresis in Paediatric Neurology: a French Multicenter Study

Efficacy and Tolerance of Therapeutic Apheresis in Paediatric Neurology: a Retrospective French Multicenter Study

After developing in pediatric hematology and nephrology, therapeutic apheresis is increasingly used in pediatric neurology despite a sparse level of evidence.

There are a few retrospective series with a small number of patients, concerning mainly autoimmune diseases (encephalitis, myasthenia gravis, polyradiculoneuritis).

The objective of this work is to study therapeutic apheresis (including plasma exchange and immunoadsorption) among french neuropediatric tertiary centers and to prove that this treatment modality is effective and well tolerated in pediatric neurology diseases.

Study Overview

• Status: Completed
• Conditions: Apheresis Related Complication
• Intervention / Treatment: Other: Efficacy of apheresis

Detailed Description

Multicenter medical data collection :

• Montpellier
• Toulouse
• Bordeaux
• Marseille
• Nancy
• Paris Kremlin Bicêtre
• Paris Robert Debré
• Paris Necker
• Paris Trousseau
• Lyon
• Besançon
• Rennes
• Reims
• Strasbourg
• La Réunion
• Nantes
• Tours

• Study Type: Observational
• Enrollment (Actual): 200

Contacts and Locations

Study Locations

• France
  • Montpellier, France, 34295
    • UH Montpellier

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Children under 18, Supported in a participating CHU between 2014 and 2019 and Having benefited from therapeutic apheresis (plasma exchanges and immunoadsorption) in a neuropediatric indication

Description

Inclusion criteria:

• Children under 18.
• Supported in a participating CHU between 2014 and 2019.
• Having benefited from therapeutic apheresis (plasma exchanges and immunoadsorption) in a neuropediatric indication

Exclusion criteria:

- Family or patient refusal to participate

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
plasma exchange; All patients underwent plasma exchanges between 2014 and 2019 for a neuropediatric pathology	Other: Efficacy of apheresis; Efficacy of apheresis
immunoadsorption; All patients who underwent immunoadsorptions between 2014 and 2019 for a neuropediatric pathology	Other: Efficacy of apheresis; Efficacy of apheresis

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
global neurological efficacy of therapeutic apheresis as assessed	lobal neurological efficacy of therapeutic apheresis as assessed by modified rankin score Modifed Rankin score, ranging from 0 (no symptoms) to 6 (death)	through study completion, an average of 2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of therapeutic apheresis adverse events as assessed	tIncidence of therapeutic apheresis adverse events as assessed by low blood tension rate, transfusion rate, infection rate, hypocalcaemia rate, thrombosis rate, allergy rate, anemia rate, hemostasis disorders rate).	through study completion, an average of 2 years
Incidence of adjuvant treatments	Incidence of adjuvant treatments (corticosteroid therapy, immunoglobulins, immunosuppressants),	through study completion, an average of 2 years
Incidence of hospitalization in intensive care	Incidence of hospitalization in intensive care, intubation rate	through study completion, an average of 2 years
Description of the therapeutic apharesis modalities	number of procedures	through study completion, an average of 2 years
Description of the therapeutic apharesis modalities	exchanged volume (in plasma volume percentage) for each procedure	through study completion, an average of 2 years
Description of the therapeutic apharesis modalities	type of separation et number of participants with ttt-related advesre events	through study completion, an average of 2 years
Description of the therapeutic apharesis modalities	type of replacement solution (isotonic saline, albumin, fresh frozen plasma)	through study completion, an average of 2 years
Number of participitants with treatment-related adverse events	rate of arterial hypotension (< age standards and and requiring isotonic saline bolus, for each patient and for procedure)	during procedures
Number of participitants with treatment-related adverse events	rate of hypocalcemia (ionized calcium <1,1 mmol/L, for each patient and for each procedure)	during procedures
Number of participitants with treatment-related adverse events	rate of hypoalbuminemiafor for each patient and for each procedure)	during procedures
Number of participitants with treatment-related adverse events	rate of thrombosis (confirmed by a CT scan or ultrasound, for each patient)	during procedures
Number of participitants with treatment-related adverse events	rate of infections related catheter (for each patient)	during procedures
Number of participitants with treatment-related adverse events	rate of allergics reactions (for each patient and for each procedure)	during procedures
Number of participitants with treatment-related adverse events	rate of anemia (Hb < 7,0 g/dL, for each patient)	during procedures
Number of participitants with treatment-related adverse events	rate of transfusion (for each patient)	during procedures
Number of participitants with treatment-related adverse events	rate of haemostasis disorder (platelets < 100 <G/L ; TQ < 40 % ; fibrinogen < 1 G/L ; (for each patient and for each procedure)	during procedures
Number of participitants with treatment-related adverse events	rate of death	during procedures

Collaborators and Investigators

• Sponsor: University Hospital, Montpellier

Study record dates

Study Major Dates

• Study Start (ACTUAL): February 15, 2021
• Primary Completion (ACTUAL): December 1, 2021
• Study Completion (ACTUAL): December 31, 2021

Study Registration Dates

• First Submitted: February 16, 2021
• First Submitted That Met QC Criteria: April 13, 2021
• First Posted (ACTUAL): April 15, 2021

Study Record Updates

• Last Update Posted (ACTUAL): April 12, 2022
• Last Update Submitted That Met QC Criteria: April 11, 2022
• Last Verified: April 1, 2022

More Information

Terms related to this study

• Keywords: Children; immunoadsorption; therapeutic exchange; neuropediatric
• Other Study ID Numbers: RECHMPL21_0084

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: NC

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No