Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis

Sponsor

Swedish Orphan Biovitrum (Industry)

Overall Status

Completed

CT.gov ID

NCT06047210

Collaborator

Brigham and Women's Hospital (Other), Harvard Medical School (HMS and HSDM) (Other)

Enrollment

Location

34.5

Actual Duration (Months)

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This observational study is designed to provide a better understanding of the potential use of interferon gamma (IFNγ) and IFNγ-inducible chemokines as markers of HLH disease activity in adults, and the potential of IFNγ to represent a therapeutic target.

Condition or Disease	Intervention/Treatment	Phase
Hemophagocytic Lymphohistiocytoses	Other: Blood Draws Data Collection

Detailed Description

This is a observational study designed to determine the levels of pro-inflammatory markers in adult patients diagnosed with HLH and to assess the relationship between the biomarkers and disease activity in these patients during the HLH course. Inflammatory markers include IFNγ and IFNγ-induced chemokines C-X-C chemokine ligand 9 and C-X-C chemokine ligand 10.

In addition to the blood samples for the biomarker analysis, relevant information gathered by the treating physician will be collected in a data collection form. Whenever possible, collection of serum samples for biomarker analysis and relevant information should occur at HLH diagnosis, at regular time intervals during the treatment course (not more than once a week) up to resolution of HLH.

Blood samples will also be sent to the study sponsor (NovImmune S.A.) for cytokine testing. Before patient's samples are sent to the sponsor for testing, patient's name and any personal identifying information will be coded to protect participant's privacy.

Study Design

Study Type:

Observational

Actual Enrollment :

14 participants

Observational Model:

Case-Control

Time Perspective:

Other

Official Title:

An Observational, Multicenter Study to Evaluate Levels of Interferon Gamma (IFNγ) and Other Inflammatory Mediators in Adult Patients With Hemophagocytic Lymphohistiocytosis (A-HLH)

Actual Study Start Date :

Dec 14, 2016

Actual Primary Completion Date :

Oct 30, 2019

Actual Study Completion Date :

Oct 30, 2019

Arms and Interventions

Arm	Intervention/Treatment
A-HLH Patients Patients diagnosed with A-HLH. The diagnosis of A-HLH was established by the treating physician.	Other: Blood Draws Data Collection
Malignancy-Associated Hemophagocytic Lymphohistiocytosis (M-HLH) Patients Patients diagnosed with HLH in the context of a malignancy. The diagnosis of M-HLH was established by the treating physician.	Other: Blood Draws Data Collection

Arm

Intervention/Treatment

A-HLH Patients

Patients diagnosed with A-HLH. The diagnosis of A-HLH was established by the treating physician.

Other: Blood Draws Data Collection

Malignancy-Associated Hemophagocytic Lymphohistiocytosis (M-HLH) Patients

Patients diagnosed with HLH in the context of a malignancy. The diagnosis of M-HLH was established by the treating physician.

Other: Blood Draws Data Collection

Outcome Measures

Primary Outcome Measures

Serum Concentrations of Inflammatory Markers in Participants Diagnosed With HLH [11 months]
Serum samples were collected to determine the concentration of C-X-C chemokine ligand 10, C-X-C chemokine ligand 9, interferon gamma, interleukin 10, interleukin 1 beta, interleukin 6, neopterin, tumor necrosis factor alpha, Total human interferon gamma, soluble CD163 and soluble interleukin-2 receptor alpha.
Correlation Between Serum Concentrations of Inflammatory Markers and Markers of Disease Activity in Participants Diagnosed With HLH [11 months]
Serum samples were collected to determine the markers of disease activity as indicated by the following laboratory assessments: albumin, aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase, basophils, beta 2-microglobulin, total and conjugated bilirubin, blood urea nitrogen, serum creatinine, C-reactive protein, D-dimers, hemoglobin, hematocrit, red blood cells, white blood cells, and differential count, platelets, fasting triglycerides, ferritin, fibrinogen, lactate dehydrogenase, natural killer cell activity, prothrombin time and activated partial thromboplastin time, serum immunoglobulin G, Sodium.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patients >=18 years old diagnosed with active HLH as established by the treating physician.
HLH forms of unknown origin or secondary to infections or rheumatologic disorders.
Should the diagnosis of primary HLH or malignancy become apparent after inclusion, the data collected will be analyzed separately as additional cohorts.
The patient must have consented to the use of their clinical data for research purposes at the site.

Exclusion Criteria:

Patients with primary HLH (diagnosed by the presence of homozygous mutations in a known HLH causative gene) and secondary HLH due to malignancy are excluded.