Author:  J. Dobke, Last modification: 2016/05/09 https://kinderkrebsinfo.de/doi/e57553

HLH-2004 HLH-2004, Treatment Protocol of the Second International HLH Study 2004
Disease Hempohagocytic Lymphohistiocytosis
Type International, multicentre, prospective therapy optimazation trial
Problem / Objectives

1. Optimazation of the treatment of patients < 18 years with genetic HLH, or severe secondary HLH (non-genetic), througt the intenisification of the induction therapy.
2. Standardization of the therapy, with the aim to collect data on the success of the SCT concerning the donor type, the desease activity before transplantation and the neurological long term side effects.
3. Improved understanding of pathophysiology in HLH by biological studies on genetics and cytotoxicity in affected patients, including genotype-phenotype studies and the prognostic value of NK-cells-activity subtyping.

Inclusion Criteria

All newly diagnosed patients who meet the following criteria are eligible to be fully enrolled and followed in the study:

-patients who fulfill the diagnostic criteria of HLH
-Age <18 years at onset of therapy
-No prior cytotoxic or cyclosoprin A treatment for HLH

Patients with HLH starting the HLH 2004 protocoll who do not fulfill the diagnostic criteria or >0=18 years will also be registered in the study but will be studied separately.

Status Beginn: Januar 2004
Principal Investigator Jan-Inge Henter MD, PhD/ Prof. Dr. Gritta Janka
E-Mail janka@uke.uni-hamburg.de

Study chairman
Jan-Inge Henter MD, PhD
Child Cancer Research unit
Department of Pediatrics
Karolinska Hospital, Q6:05
S-171 76 Stockholm, Sweden
Tel. +46 -8 5177 1098
Fax +46-8 5177 3184

National coordinator

Prof. Dr. med. Gritta Janka Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik f. Päd. Hämatologie u. Onkologie Martinistraße 52 20246 Hamburg Telefon +49 (40) 42803 4369 Fax +49 (40) 42803 4601 janka@uke.uni-hamburg.de

Weitere Informationen The immunomodulating treatment with Dexamethasone, Etoposide and Cyclosporine A (CSA) serves to achieve a clinicaly stable resolution of the desease and to cure by SCT. To reduce mortality before SCT, Cyclosporine A is already given before SCT in contrary to the HLH 1994 trial.
The HLH 2004 study is designed for the primary inherited desease FHL, but may be beneficial in patients with secondary HLH as well.
At the start of the treatment in many children the specific determination of the desease is not possible. If the desease is severe and persistant, or reactivating, treatment according on HLH 2004 is suggested initially for 8 weeks.
In secondary HLH therapy can be finished if the desease has a complete resolution.
If the desease after induction therapy is still severe and resistant, or reactivating, continuation therapy and SCT is suggested.