drugset / Trial / NCT00426101
Treatment Protocol for Hemophagocytic Lymphohistiocytosis 2004
Phase 3
Completed
368 enrolled
Karolinska University Hospital
Baylor College of Medicine · collabChildren's Hospital Medical Center, Cincinnati · collabEhime University Graduate School of Medicine · collabGreat Ormond Street Hospital for Children NHS Foundation Trust · collabHospital JP Garrahan · collabHospital de Cruces · collabLeiden University Medical Center · collabMeyer Children's Hospital IRCCS · collabSt. Anna Kinderkrebsforschung · collabUniversitätsklinikum Hamburg-Eppendorf · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2004-01
- Primary completion
- 2011-12
- Completion
- 2017-12
Publications
- Background Henter JI, Horne A, Arico M, Egeler RM, Filipovich AH, Imashuku S, Ladisch S, McClain K, Webb D, Winiarski J, Janka G. HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 2007 Feb;48(2):124-31. doi: 10.1002/pbc.21039.
- Results Bergsten E, Horne A, Arico M, Astigarraga I, Egeler RM, Filipovich AH, Ishii E, Janka G, Ladisch S, Lehmberg K, McClain KL, Minkov M, Montgomery S, Nanduri V, Rosso D, Henter JI. Confirmed efficacy of etoposide and dexamethasone in HLH treatment: long-term results of the cooperative HLH-2004 study. Blood. 2017 Dec 21;130(25):2728-2738. doi: 10.1182/blood-2017-06-788349. Epub 2017 Sep 21.
- Bergsten E, Horne A, Hed Myrberg I, Arico M, Astigarraga I, Ishii E, Janka G, Ladisch S, Lehmberg K, McClain KL, Minkov M, Nanduri V, Rosso DA, Sieni E, Winiarski J, Henter JI. Stem cell transplantation for children with hemophagocytic lymphohistiocytosis: results from the HLH-2004 study. Blood Adv. 2020 Aug 11;4(15):3754-3766. doi: 10.1182/bloodadvances.2020002101.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Cyclosporine | Peptide | 6 mg/kg | — |
| Subject | Dexamethasone | Small molecule | 1.25 mg/m2 | — |
| Subject | Etoposide | Small molecule | 150 mg/m2 | Intravenous |