An observational study in Interstitial Lymphocytic Lung Disease, sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology. Status unknown at 1 site in Russian Federation. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2020-10-01.
Sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology · Observational
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.
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.
3,303 studies on the registry are indexed under Lung Diseases; 355 are open to participants now.
This study's planned enrollment of 30 is below the median of 157 across 929 observational studies indexed under Lung Diseases.
Browse Lung Diseases studies →Federal Research Institute of Pediatric Hematology, Oncology and Immunology is the lead sponsor of 55 studies on the registry; 17 are open to participants now.
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Pediatric patients with interstitial lymphocytic lung disease treated in Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russia
Exclusion criteria are nonadherence to therapy.
Drug: Rituximab
Drug: Abatacept
375 mg/m2 as 4 weekly consecutive i.v. infusions with subsequent infusions of 375 mg/m2 performed every 3 months for 12 months.
10 mg/kg i.v. every 2 weeks twice, then every 4 weeks for 12 months.
Dynamics of severity clinical and radiological symptoms
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Time frame: before the time the first dose of study treatment was administered
Dynamics of severity clinical and radiological symptoms
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Time frame: 3 months of treatment
Dynamics of severity clinical and radiological symptoms
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Time frame: 6 months of treatment
Dynamics of severity clinical and radiological symptoms
Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
Time frame: 1 year of treatment
Frequency of adverse events
Time frame: from the time the first dose of study treatment was administered until 12 mo of treatment
quality of life of patients
The quality of life of patients before and after therapy
Time frame: before the time the first dose of study treatment was administered until 12 mo of treatment
This study is status unknown, as verified in Sep 2020. You cannot join it, but the record below documents what was studied.
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Federal Research Institute of Pediatric Hematology, Oncology and Immunology