Treatment Protocol for Hemophagocytic Lymphohistiocytosis 2004

Karolinska University Hospital

Status and phase: Completed; Phase 3

Conditions: Hemophagocytic Lymphohistiocytosis

Treatments: Drug: Cyclosporin; Drug: Dexamethasone; Procedure: Intrathecal therapy; Procedure: Stem cell transplant; Drug: Etoposide

Study type: Interventional

Funder types: Other

Identifiers

NCT00426101

HLH-2004

Details and patient eligibility

About

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.

Full 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.

Enrollment: 368 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients who fulfil the diagnostic criteria of HLH.

Exclusion criteria

• Prior cytotoxic or cyclosporin treatment for HLH.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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368 participants in 1 patient group

Etoposide, Dexamethasone, Cyclosporin A plus IT MTX & Steroids

Experimental group

Description:

As compared to the HLH-94 treatment, the main changes are that 1. Cyclosporin A is administered from day 1 and 2. Intrathecal steroids are added to the intrathecal methotrexate. Drugs, dosage, frequency and duration are described in the paragraph "Interventions" below.

Treatment:

Drug: Etoposide

Procedure: Stem cell transplant

Drug: Cyclosporin

Drug: Dexamethasone

Procedure: Intrathecal therapy