Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis

Status: Recruiting
Phase: Phase 2
Diagnosis: Pediatric Histiocytosis
NCT ID: NCT01104025 (View complete trial on ClinicalTrials.gov)
DFCI Protocol ID: 11-243


Despite good progress during the last decade, hemophagocytic lymphohistiocytosis (HLH) remains difficult to treat. Two different treatment regimens have been used successfully. The first one, a treatment regimen based on two drugs called etoposide and dexamethasone, has been used worldwide. The second regimen, based on two drugs called Anti-thymocyte globulin (ATG) and prednisone, has been used mostly at one hospital in Paris, for over 15 years. With either regimen, about three quarters of treated children survive the most difficult time, the first two months after diagnosis. These two different regimens appear to work somewhat differently, and we suspect that combining them may give better results than either regimen alone. We are conducting this clinical trial to test the combination of ATG, dexamethasone, and etoposide for the treatment of HLH. The purpose of this research study is to find out what effects (good and bad) this drug combination has on you and your HLH.


Conducting Institutions:
Children's Hospital Boston, Dana-Farber Cancer Institute

Overall PI:
Barbara Degar, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

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 study. - 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
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