Phase 2
N=31
Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis
Hemophagocytic Lymphohistiocytosis
Bottom Line
View on ClinicalTrials.gov: NCT01104025 ↗Enrolled (actual)
31
Serious AEs
71.0%
Results posted
Oct 2020
Primary outcome: Primary: Overall Survival — 25 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- ATG, rabbit (Drug); Etoposide (Drug); Methotrexate (Drug); hydrocortisone (Drug); Dexamethasone (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- Nov 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Survival |
22 | — |
| SECONDARY Time to Response |
4 | — |
| SECONDARY Overall Survival |
22 | — |
| SECONDARY Number of Participants Who Experienced Reactivation |
4 | — |
| SECONDARY Overall Survival to Day +100 |
9 | — |
| SECONDARY Disease Status at BMT |
8 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT01104025). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.