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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
Sponsor:
Information provided by (Responsible Party):
Federal Research Institute of Pediatric Hematology, Oncology and Immunology

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.

Condition or disease Intervention/treatment
Interstitial Lymphocytic Lung Disease Drug: Rituximab Drug: Abatacept

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.

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Study Type : Observational
Estimated Enrollment : 30 participants
Observational Model: Cohort
Time Perspective: Retrospective
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


Group/Cohort Intervention/treatment
group 1(rituximab) Drug: Rituximab
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) Drug: Abatacept
10 mg/kg i.v. every 2 weeks twice, then every 4 weeks for 12 months.




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.


Secondary Outcome Measures :
  1. Frequency of adverse events [ Time Frame: from the time the first dose of study treatment was administered until 12 mo of treatment ]
  2. 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



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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
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.


Information from the National Library of Medicine

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


Contacts
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Contact: Anna Shcherbina, MD, PhD +7(495)2876570 ext 6299 shcher26@hotmail.com

Locations
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Russian Federation
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    shcher26@hotmail.com   
Sponsors and Collaborators
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
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Responsible Party: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
ClinicalTrials.gov Identifier: NCT04572620    
Other Study ID Numbers: NCPHOI-2020-08
First Posted: October 1, 2020    Key Record Dates
Last Update Posted: October 1, 2020
Last Verified: September 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Federal Research Institute of Pediatric Hematology, Oncology and Immunology:
interstitial lymphocytic lung disease
primary immunodeficiency
children
treatment
rituximab
abatacept
Additional relevant MeSH terms:
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Lung Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Respiratory Tract Diseases
Rituximab
Abatacept
Antineoplastic Agents, Immunological
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Immunosuppressive Agents