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An Open Label Phase II Pilot Study of Hybrid ImmunoTherapy(ATG/Dexamethasone/Etoposide) for Hemophagocytic LymphoHistiocytosis:HIT-HLH

Phase 2
18 Years
Open (Enrolling)
Hemophagocytic Lymphohistiocytosis

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Trial Information

An Open Label Phase II Pilot Study of Hybrid ImmunoTherapy(ATG/Dexamethasone/Etoposide) for Hemophagocytic LymphoHistiocytosis:HIT-HLH

Hemophagocytic lymphohistiocytosis (HLH) is a rare immunological disorder first recognized
almost 70 years ago.(1) Genetic and animal studies have indicated that the familial form of
HLH is clearly due to a deficiency of cytotoxic killing. Patients with HLH present with a
potentially fatal syndrome of 'hyperimmunity.' These patients have severe inflammation,
associated with cytopenias and variably severe bone marrow, liver, or CNS damage. Tissue
damage and mortality appear to be due to hypercytokinemia related to persistent immune
hyperactivation. An animal model of HLH and correlative human studies all suggest that
excessive and abnormal activation of T cells drives the pathophysiology of this disorder,
and that suppressing this excessive activation is critical for successful therapy of HLH. It
is believed a combination of the two proven induction regimens for hemophagocytic
lymphohistiocytosis (HLH) (anti-thymocyte globulin (ATG)- and etoposide-based) will result
in response rates and overall survival rates at eight weeks which are comparable or better
than the current standard of care (induction therapy per the HLH-94 protocol).

Inclusion Criteria:

- diagnosis of hemophagocytic lymphohistiocytosis

- Patients <18 years of age

- The patient must have active disease at the time of enrollment

- Patient's legal guardians must sign an Institutional Review Board approved consent
form indicating their awareness of the investigational nature and the risks of this

- Eligible subjects must be enrolled with the protocol coordinating center

Exclusion Criteria:

- Recent treatment, within 3 months, with another therapeutic regimen for HLH

- Known active malignancy

- Known rheumatologic diagnosis which may be the underlying cause of HLH

- Pregnancy (as determined by serum or urine test) or active breast feeding

- Failure to provide signed informed consent

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Complete Response Rate

Outcome Description:

To determine the complete response rate and overall survival at 8 weeks after an ATG/Dexamethasone/Etoposide based induction regimen for patients with hemophagocytic lymphohistiocytosis

Outcome Time Frame:

8 Weeks

Safety Issue:


Principal Investigator

Michael Jordan, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Hospital Medical Center, Cincinnati


United States: Institutional Review Board

Study ID:




Start Date:

April 2010

Completion Date:

April 2018

Related Keywords:

  • Hemophagocytic Lymphohistiocytosis
  • hemophagocytic lymphohistiocytosis
  • hybrid immunotherapy
  • dexamethasone
  • Etoposide
  • ATG,rabbit
  • Lymphohistiocytosis, Hemophagocytic



Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania  19104
Phoenix Children's HospitalPhoenix, Arizona  85016-7710
Cincinnati Children's Hospital Medical CenterCincinnati, Ohio  45229-3039
Tulane University Medical CenterNew Orleans, Louisiana  70112
Children's Hospital BostonBoston, Massachusetts  02115
University of California, San Francisco Department of PediatricsSan Francisco, California  
Florida All Children's HospitalSt. Petersburg, Florida  
Texas Children's Cancer Center/Baylor College of MedicineHouston, Texas