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

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Status: Unknown

Conditions: Interstitial Lymphocytic Lung Disease

Treatments: Drug: Rituximab; Drug: Abatacept

Study type: Observational

Funder types: Other

Identifiers

NCT04572620

NCPHOI-2020-08

Details and patient eligibility

About

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.

Full 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.

Enrollment: 30 estimated patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

30 participants in 2 patient groups

group 1(rituximab)

Treatment:

Drug: Rituximab

group 2 (abatacept)

Treatment:

Drug: Abatacept