Rituximab and Abatacept Effectiveness in Differential Treatment of Interstitial Lymphocytic Lung Disease in Children With Primary Immunodeficiencies.
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|ClinicalTrials.gov Identifier: NCT04572620|
Recruitment Status : Recruiting
First Posted : October 1, 2020
Last Update Posted : October 1, 2020
|Condition or disease||Intervention/treatment|
|Interstitial Lymphocytic Lung Disease||Drug: Rituximab Drug: Abatacept|
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 Type :||Observational|
|Estimated Enrollment :||30 participants|
|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.|
|Actual Study Start Date :||September 7, 2020|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||September 2021|
375 mg/m2 as 4 weekly consecutive i.v. infusions with subsequent infusions of 375 mg/m2 performed every 3 months for 12 months.
|group 2 (abatacept)||
10 mg/kg i.v. every 2 weeks twice, then every 4 weeks for 12 months.
- Dynamics of severity clinical and radiological symptoms [ Time Frame: before the time the first dose of study treatment was administered ]Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
- Dynamics of severity clinical and radiological symptoms [ Time Frame: 3 months of treatment ]Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
- Dynamics of severity clinical and radiological symptoms [ Time Frame: 6 months of treatment ]Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
- Dynamics of severity clinical and radiological symptoms [ Time Frame: 1 year of treatment ]Dynamics of severity clinical and radiological symptoms of ILLD using scale score.
- 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 [ Time Frame: 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04572620
|Contact: Anna Shcherbina, MD, PhD||+7(495)2876570 ext email@example.com|
|Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology||Recruiting|
|Moscow, Russian Federation, 117198|
|Contact: Anna Shcherbina, MD, PhD +7(495)2876570 ext 6299 firstname.lastname@example.org|