Clinical trial NCT04572620: Rituximab and Abatacept Effectiveness in Differential Treatment of Interstitial Lymphocytic Lung Disease in Children With Primary Immunodeficiencies.
Official title: Retrospective Chart Review of Children With Primary Immunodeficiencies (PID) Who Received Targeted Therapy of Interstitial Lymphocytic Lung Disease (ILLD) With Abatacept or Rituximab.
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Interstitial Lymphocytic Lung Disease
Keywords: interstitial lymphocytic lung disease
primary immunodeficiency
children
treatment
rituximab
abatacept
Lead sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Sponsor class: OTHER
First posted: 2020-10-01
Enrollment: 30
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric patients with interstitial lymphocytic lung disease treated in Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russia

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.

Eligibility 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.

Primary outcomes:
measure: Dynamics of severity clinical and radiological symptoms
timeFrame: before the time the first dose of study treatment was administered
measure: Dynamics of severity clinical and radiological symptoms
timeFrame: 3 months of treatment
measure: Dynamics of severity clinical and radiological symptoms
timeFrame: 6 months of treatment
measure: Dynamics of severity clinical and radiological symptoms
timeFrame: 1 year of treatment
Secondary outcomes:
measure: Frequency of adverse events
timeFrame: from the time the first dose of study treatment was administered until 12 mo of treatment
measure: quality of life of patients
timeFrame: before the time the first dose of study treatment was administered until 12 mo of treatment
Other outcomes:
Locations:
city: Moscow
country: Russian Federation
facility: Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology
Study officials:
References: