Most of this information is not available in the studies performe

Most of this information is not available in the studies performed see more to date. Regarding future investigations in the area of inhibitor development, EHTSB recommends that the studies be carried out on well characterized, large cohorts of severe (clotting activity <1%), infusion-naïve PUPs with consecutive enrolment. The only exception to this recommendation is the evaluation of immunogenicity of new factor concentrates which, according to the EMEA guidelines, should first be carried out in PTPs. Potentially confounding factors should be addressed and

genetic factors taken into account. Validated assays (e.g. Nijmegen) for inhibitor analysis should preferably be performed in a central laboratory with a pre-defined cut-off value and, in a case where an inhibitor is detected, confirmed with another test within the shortest possible interval. Patients who develop an inhibitor should be classified by clear criteria as high responders (≥5 BU), low responders (<5 BU) and whether the inhibitor is transient (disappearing within 3 months without a change in treatment regimen, or disappearing) or not. Enzyme linked immune sorbent assay (ELISA) should also be performed to detect all antibodies produced against the deficient factor. Well-conducted studies will contribute to our understanding of the pathophysiology AZD1208 of inhibitor development, thereby enabling

the use of treatment approaches with the potential to minimize inhibitor development in patients with haemophilia. The EHTSB is a collaborative independent network of European haemophilia centres sponsored by an unrestricted grant from Baxter. C. Altisent, Barcelona, Spain; J. Astermark, Malmö, Ribose-5-phosphate isomerase Sweden; A. Batorova, Bratislava, Slovakia; P. de Moerloose, Geneva, Switzerland; G. Dolan, Nottingham, UK; K. Fijnvandraat, Amsterdam, The Netherlands; K. Fischer, Utrecht, The Netherlands; A. Gringeri, Milan, Italy; C. Hermans, Brussels, Belgium; P. A. Holme, Oslo, Norway; K. Holstein, Hamburg, Germany; M. João

Diniz, Lisbon, Portugal; A. Karafoulidou, Athens, Greece; R. Klamroth, Berlin, Germany; T. Lambert, Paris, France; R. Lassila, Helsinki, Finland; G. Lavigne-Lissalde, Nîmes, France; F. Lopéz, La Coruňa, Spain; R. Pérez, Seville, Spain; M. Richards, Leeds, UK; A. Rocino, Naples, Italy; M. Schiavoni, Bari, Italy; M. von Depka, Hannover, Germany; J. Windyga, Warsaw, Poland. Dr Astermark has received research funds from Baxter, Bayer, Wyeth, Octapharma, CSL Behring and Grifols. He has also received honoraria for organising education sessions, for speaking at scientific meetings or for consultancy services from Baxter, Bayer, Wyeth, Octapharma, CSL Behring, Novo Nordisk, and Biovitrum. Dr Batorova has received honoraria for organizing educational session and speaking at scientific meetings from Bayer, Octapharma, Novo Nordisk, and consultancy fees from Baxter.

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