Interstitial Lymphocytic Lung Disease
Conditions
Keywords
interstitial lymphocytic lung disease, primary immunodeficiency, children, treatment, rituximab, abatacept
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.
Interventions
DRUGRituximab
375 mg/m2 as 4 weekly consecutive i.v. infusions with subsequent infusions of 375 mg/m2 performed every 3 months for 12 months.
DRUGAbatacept
10 mg/kg i.v. every 2 weeks twice, then every 4 weeks for 12 months.
Sponsors
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Dynamics of severity clinical and radiological symptoms | before the time the first dose of study treatment was administered | Dynamics of severity clinical and radiological symptoms of ILLD using scale score. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
Russia
Contacts
Primary ContactAnna Shcherbina, MD, PhD
Outcome results
None listed