Rituximab and Abatacept Effectiveness in Differential Treatment of Interstitial Lymphocytic Lung Disease in Children With Primary Immunodeficiencies.

Interstitial Lymphocytic Lung Disease Clinical Trial

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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04572620
• Other study ID #: NCPHOI-2020-08
• Status: Recruiting
• Start date: September 7, 2020
• Est. completion date: September 2021

Study information

• Verified date: September 2020
• Source: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
• Contact: Anna Shcherbina, MD, PhD
• Phone: +7(495)2876570
• Email: shcher26[@]hotmail.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: September 2021
• Est. primary completion date: September 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Interstitial Lymphocytic Lung Disease
• Lung Diseases

Intervention

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.
• Abatacept
10 mg/kg i.v. every 2 weeks twice, then every 4 weeks for 12 months.

Locations

Country	Name	City	State
Russian Federation	Dmitry Rogachev National Research and Clinical Center for Pediatric Hematology, Oncology and Immunology	Moscow

Sponsors (1)

Lead Sponsor	Collaborator
Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Dynamics of severity clinical and radiological symptoms	Dynamics of severity clinical and radiological symptoms of ILLD using scale score.	before the time the first dose of study treatment was administered
Primary	Dynamics of severity clinical and radiological symptoms	Dynamics of severity clinical and radiological symptoms of ILLD using scale score.	3 months of treatment
Primary	Dynamics of severity clinical and radiological symptoms	Dynamics of severity clinical and radiological symptoms of ILLD using scale score.	6 months of treatment
Primary	Dynamics of severity clinical and radiological symptoms	Dynamics of severity clinical and radiological symptoms of ILLD using scale score.	1 year of treatment
Secondary	Frequency of adverse events		from the time the first dose of study treatment was administered until 12 mo of treatment
Secondary	quality of life of patients	The quality of life of patients before and after therapy	before the time the first dose of study treatment was administered until 12 mo of treatment