Rituximab and Abatacept Effectiveness in Differential Treatment of Interstitial Lymphocytic Lung Disease in Children With Primary Immunodeficiencies.

• Conditions: Interstitial Lymphocytic Lung Disease
• Interventions: Drug: Rituximab; Drug: Abatacept

• Registration Number: NCT04572620
• Lead Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Brief Summary

The rationale for this retrospective study is to evaluate the efficacy and safety of abatacept and rituximab treatment of ILLD in a cohort of pediatric patients with different forms of PID, who received one of the two therapy regimens predominantly based on the lesions histopathology.

Detailed Description

Primary immunodeficiencies (PID) represent a heterogeneous group of more than 400 inherited conditions with associated immune dysfunctions. Though severe recurrent/chronic infections are the main cause of mortality and morbidity in PID, immune dysregulation manifesting with oncological and autoimmune or autoinflammatory conditions involving various organs and systems have been the focus of research in the recent years.

The interstitial lymphocytic lung disease (ILLD) is one of the recently characterized non-malignant PID complications. Immune dysregulation in ILLD causes reactive bronchi-associated lymphoid tissue (BALT) hyperplasia that manifests in several pathomorphological forms: follicular bronchiolitis (FB), nodular lymphoid hyperplasia (NLH), and lymphocytic interstitial pneumonia (LIP). Treatment of ILLD patients with various immunosuppressive drugs leads to inconsistent results ranging from partial\\transient effect to no effect at all and has been often associated with adverse effects and an increase in infections' rate. Therefore there is a need for targeted therapy of ILLD. In small cohorts of adult PID patients rituximab in combination with azathioprine proved to be effective. Yet, the reports are scarce and there is currently no consensus on ILLD treatment, especially in children.

The study will collect and analyze information on the effectiveness and safety of ILLD monotherapy with rituximab or abatacept, chosen predominantly based on the pathomorphological characteristics of lymphoid infiltration, as well as genetic defects, in a cohort of pediatric patients with PID.

Study Timeline

• Status Verified: 2020-09
• Study Start: 2020-09-07
• Study First Submitted: 2020-09-21
• Study First Submitted QC: 2020-09-25
• Last Update Submitted: 2020-09-25
• Study First Posted: 2020-10-01
• Last Update Posted: 2020-10-01
• Primary Completion: 2021-09
• Study Completion: 2021-09

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

1. Histological and/or radiological diagnosis of ILLD.
2. Treatment with rituximab or abatacept for at least 12 months.
3. No signs of ongoing infectious pulmonary process at the start of the treatment.
4. Regular IG substitution with trough IgG levels > 5g/l.

Exclusion criteria are nonadherence to therapy.

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
group 1(rituximab)	Rituximab	-
group 2 (abatacept)	Abatacept	-

Primary Outcome Measures

Name	Time	Method
Dynamics of severity clinical and radiological symptoms	1 year of treatment	Dynamics of severity clinical and radiological symptoms of ILLD using scale score.

Secondary Outcome Measures

Name	Time	Method
Frequency of adverse events	from the time the first dose of study treatment was administered until 12 mo of treatment
quality of life of patients	before the time the first dose of study treatment was administered until 12 mo of treatment	The quality of life of patients before and after therapy

Trial Locations

Locations (1)

Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology; 🇷🇺 Moscow, Russian Federation