Treatment Protocol for Hemophagocytic Lymphohistiocytosis 2004

HLH-2004 Treatment Protocol

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00426101

Enrollment

368

Registered

2007-01-24

Start date

2004-01-31

Completion date

2017-12-31

Last updated

2018-07-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophagocytic Lymphohistiocytosis

Keywords

Hemophagocytic lymphohistiocytosis

Brief summary

Without therapy HLH is often fatal, and often rapidly fatal. The treatment protocol HLH-94 has improved survival markedly as compared to the survival earlier. We now aim to improve survival further.

Detailed description

The most dangerous period after HLH diagnosis is the first 2 months. In HLH-2004 we provide additional therapy during this period as compared to in HLH-94.

Interventions

DRUGDexamethasone

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 If continuation: Pulses every 2nd wk, 10 mg/m2 for 3 days

DRUGEtoposide

150 mg/m2 iv twice/wk (wk 1-2) 150 mg/m2 iv once/wk (wk 3-8) If continuation: 150 mg/m2 iv, every 2nd wk

DRUGCyclosporin

WK 1-8: \- 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. If continuation: \- Aim for around 200 microgram/L. Monitor GFR.

PROCEDUREIntrathecal therapy

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.

PROCEDUREStem cell transplant

The SCT procedure is up to the treating physician. However, a suggested regimen is provided.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 18 Years

Healthy volunteers

Inclusion criteria

* Patients who fulfil the diagnostic criteria of HLH.

Exclusion criteria

* Prior cytotoxic or cyclosporin treatment for HLH.

Design outcomes

Primary

Measure	Time frame
Survival	1-year after diagnosis

Secondary

Measure	Time frame
Late effects	5-years after diagnosis

Countries

Sweden

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 3, 2026