Treatment Protocol for Hemophagocytic Lymphohistiocytosis 2004
|Hemophagocytic Lymphohistiocytosis||Drug: Dexamethasone Drug: Etoposide Drug: Cyclosporin Procedure: Intrathecal therapy Procedure: Stem cell transplant||Phase 3|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||HLH-2004 Treatment Protocol|
- Survival [ Time Frame: 1-year after diagnosis ]
- Late effects [ Time Frame: 5-years after diagnosis ]
|Study Start Date:||January 2004|
|Estimated Study Completion Date:||December 2017|
|Primary Completion Date:||December 2011 (Final data collection date for primary outcome measure)|
Experimental: Etoposide, Dexamethasone, Cyclosporin A plus IT MTX & Steroids
As compared to the HLH-94 treatment, the main changes are that
Drugs, dosage, frequency and duration are described in the paragraph "Interventions" below.
10 mg/m2 daily wk 1-2 5 mg/m2 daily wk 3-4 2.5 mg/m2 daily wk 5-6 1.25 mg/m2 daily wk 7 Steroids tapered wk 8
Pulses every 2nd wk, 10 mg/m2 for 3 days
150 mg/m2 iv twice/wk (wk 1-2) 150 mg/m2 iv once/wk (wk 3-8)
150 mg/m2 iv, every 2nd wk
Procedure: Intrathecal therapy
- Aim at around 200 microgram/L (trough value). Start: 6 mg/kg daily (divided in 2 daily doses) wk 1, if kidney function is normal.
- Aim for around 200 microgram/L. Monitor GFR.
Procedure: Stem cell transplant
If at 2 wks there are progressive neurological symptoms or if an abnormal CSF (cell count and protein) has not improved, then give 4 wkly intrathecal inj. Be aware that some pat may have increased intracranial pressure.
Methotrexate: <1 yr 6 mg, 1-2 yrs 8 mg, 2-3 yrs 10 mg, >3 yrs 12 mg. Prednisolone: <1 yr 4 mg, 1-2 yrs 6 mg, 2-3 yrs 8 mg, >3 yrs 10 mg.
The SCT procedure is up to the treating physician. However, a suggested regimen is provided.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00426101
|Childhood Cancer Research Unit, Karolinska Hospital|
|Stockholm, Sweden, S-171 76|
|Principal Investigator:||Jan-Inge Henter, MD, PhD||Karolinska Institutet|