Prospective Cohort for Adult Hemophagocytosis

Recruiting

• Conditions: Hemophagocytic Lymphohistiocytoses
• Interventions: Drug: Steroids; Drug: Etoposide

• Registration Number: NCT03117010
• Lead Sponsor: Samsung Medical Center

Brief Summary

This prospective study enrolls subjects who have clinical and laboratory manifestations related with hemophagocytic lymphohistiocytosis. The purpose of the study is to evaluate clinical and biological features of adult hemophagocytic lymphohistiocytosis. The enrolled subjects into this study will be evaluated according to the HLH (hemophagocytic lymphohistiocytosis)criteria and treated with systemic immunosuppressive therapy or chemotherapy. All subjects will be regularly monitored by physicians participating in this study.

Detailed Description

Study process is as follows.

1. Informed consent for subjects fulfilling the inclusion criteria.

2. Laboratory evaluation will be done for subjects according to the following diagnostic criteria.

* Diagnosis will be established if one of either 1 or 2 below is fulfilled

1. A molecular diagnosis consistent with HLH

2. Diagnostic criteria for HLH fulfilled (5 out of 8 criteria below)

* fever ≥ 38.5'C for ≥ 7 days

* splenomegaly ≥ 3 finger breadth below left subcostal margin

* cytopenias affecting ≥2 of 3 lineages in peripheral blood (Hemoglobin \< 9 g/L, Platelets \< 100 × 109/L, Absolute neutrophil count \< 1.0 × 109/L)

* Hypertriglyceridemia and/or hypofibrinogenemia: Fasting triglycerides ≥ 265 mg/dL, fibrinogen ≤ 1.5 g/L

* Hemophagocytosis in bone marrow or spleen or lymph node

* Low or absent NK-cell activity (according to local laboratory reference)

* Ferritin ≥ 500 mcg/L

* Soluble CD25 (sIL-2 receptor) ≥ 2,400 U/mL

Study Timeline

• Study Start: 2017-01-01
• Study First Submitted: 2017-04-12
• Study First Submitted QC: 2017-04-12
• Study First Posted: 2017-04-17
• Status Verified: 2023-11
• Last Update Submitted: 2024-08-06
• Last Update Posted: 2024-08-07
• Primary Completion: 2025-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 81

Inclusion Criteria

• Subjects should fulfill the following criteria

  1. Subjects should have at least one of the following problems

     1. Presence of hemophagocytosis in tissue or bone marrow
     2. Presence of at least 3 conditions among 8 conditions of HLH diagnostic criteria

  2. Age > 18 years

  3. Written informed consents

Exclusion Criteria

• Subjects cannot satisfy the inclusion criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Hemophagocytosis	Steroids	Subjects should fulfill the following criteria 1. Subjects should have at least one of the following problems 1. Presence of hemophagocytosis in tissue or bone marrow 2. Presence of at least 3 conditions among 8 conditions of HLH diagnostic criteria 2. Age \> 18 years 3. Written informed consents Subjects receive steroids and etoposide
Hemophagocytosis	Etoposide	Subjects should fulfill the following criteria 1. Subjects should have at least one of the following problems 1. Presence of hemophagocytosis in tissue or bone marrow 2. Presence of at least 3 conditions among 8 conditions of HLH diagnostic criteria 2. Age \> 18 years 3. Written informed consents Subjects receive steroids and etoposide

Primary Outcome Measures

Name	Time	Method
Response	28 days after the 1st treatment	Response to steroids and etoposide

Secondary Outcome Measures

Name	Time	Method
Progression-free survival	3 years	Time to date of progression or any kinds of death
Overall survival	3 years	Time to date of any kinds of death

Trial Locations

Locations (1)

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of