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European initiative to improve knowledge, treatment and survival of haemophagocytic syndromes in children

Objective

Haemophagocytic lymphohistiocytosis (HLH) is a rare and life-threatening inherited immune disorder of early childhood characterised by an uncontrolled immune response. Three disease causing genes have been identified in about half of the patients. Diagnosis is difficult, pathophysiology poorly understood, and treatment unsatisfactory with about 40% of the children dying from treatment failure or toxicity. All European scientists active in research and treatment of HLH have joined in a consortium to enforce collaboration, combine the various expertises, and provide access to material and data. In vitro studies and several murine models for HLH and diseases predisposing to HLH, will be used to get more insight into the pathophysiology of the diverse genetic defects. Studies will address the role of triggers and antigen-presenting cells, the influence of cytokines in the initiation and propagation of the uncontrolled immune response, the interaction of immune effector cells and soluble factors, and the mechanism of cytotoxicity in lymphocytes and natural killer cells. Data about geno-phenotype will be combined to evaluate how known and unknown genetic defects reflect on the clinical course and immunophenotype of the patients. A diagnostic algorithm, incorporating knowledge from the known defects, will be developed and optimised in all countries to facilitate earlier and reliable diagnosis, and to separate patients with unknown defects or acquired HLH. The identification of risk factors for treatment failure and toxicity will be used to design improved treatment strategies. An experimental treatment will be tested in a murine model and will be the basis for a less toxic treatment in the future. Earlier diagnosis, better insight into the pathophysiology, and development of less toxic treatments are the steps towards a higher cure rate in HLH. In a rare disease as HLH, only combined efforts on an European-wide level will be able to achieve these goals.

Field of science

  • /medical and health sciences/basic medicine/physiology/pathophysiology

Call for proposal

FP7-HEALTH-2007-A
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF
Address
Martinistrasse 52
20251 Hamburg
Germany
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 546 987
Administrative Contact
Kai Lehmberg (Dr.)

Participants (8)

UNIVERSITA DEGLI STUDI DI BRESCIA
Italy
EU contribution
€ 346 200
Address
Piazza Mercato 15
25121 Brescia
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Raffaele Badolato (Prof.)
FORSCHUNGSZENTRUM BORSTEL
Germany
EU contribution
€ 548 213
Address
Parkallee 1-40
23845 Borstel
Activity type
Research Organisations
Administrative Contact
Susann Schrader (Ms.)
UNIVERSITAETSKLINIKUM FREIBURG
Germany
EU contribution
€ 486 920
Address
Hugstetter Strasse 49
79106 Freiburg
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Gerhard Henninger
THE CHANCELLOR MASTERS AND SCHOLARSOF THE UNIVERSITY OF CAMBRIDGE
United Kingdom
EU contribution
€ 72 170
Address
Trinity Lane The Old Schools
CB2 1TN Cambridge
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Dawn Barker (Ms.)
KAROLINSKA INSTITUTET
Sweden
EU contribution
€ 300 720
Address
Nobels Vag 5
17177 Stockholm
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Thomas Walstam (Mr.)
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 414 330
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
Administrative Contact
Annick Bertault (Ms.)
AZIENDA OSPEDALIERO-UNIVERSITARIA ANNA MEYER
Italy
EU contribution
€ 168 852
Address
Viale Pieraccini 24
50139 Firenze
Activity type
Research Organisations
Administrative Contact
Massimo Braganti (Dr.)
ISTITUTO GIANNINA GASLINI
Italy
EU contribution
€ 110 550
Address
Via Gerolamo Gaslini 5
16147 Genova
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Patrizia Fabrizi (Dr.)