Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis

• Status: Completed
• Trial end date: 2016-04-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Treatments: BB 1101; Cortisol succinate; Dexamethasone; Dexamethasone 21-phosphate; Dexamethasone acetate; Etoposide; Etoposide phosphate; Hydrocortisone; Hydrocortisone 17-butyrate 21-propionate; Hydrocortisone acetate; Methotrexate; Thymoglobulin

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