Clinical trial NCT03117010: Prospective Cohort for Adult Hemophagocytosis
Official title: A Prospective Cohort for Subjects With Adult Hemophagocytic Lymphohistiocytosis Like Syndrome
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hemophagocytic Lymphohistiocytoses
Keywords: Hemophagocytic lymphohistiocytosis
Lead sponsor: Samsung Medical Center
Sponsor class: OTHER
First posted: 2017-04-17
Enrollment: 81
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Subjects who have clinical and laboratory manifestations related with hemophagocytic lymphohistiocytosis.

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

Eligibility criteria: 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

Primary outcomes:
measure: Response
timeFrame: 28 days after the 1st treatment
Secondary outcomes:
measure: Progression-free survival
timeFrame: 3 years
measure: Overall survival
timeFrame: 3 years
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
Study officials:
affiliation: Samsung Medical Center
name: Seok Jin Kim, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Yoon SE, Eun Y, Huh K, Chung CR, Yoo IY, Cho J, Cho D, Ko YH, Park S, Kim WS, Kim SJ. A comprehensive analysis of adult patients with secondary hemophagocytic lymphohistiocytosis: a prospective cohort study. Ann Hematol. 2020 Sep;99(9):2095-2104. doi: 10.1007/s00277-020-04083-6. Epub 2020 May 21.