An international consortium proposal of uniform response criteria for myelodysplastic/myeloproliferative neoplasms (MDS/MPN) in adults
Cet article présente les recommandations d'un groupe d'experts internationaux sur la définition de critères de jugement de la réponse thérapeutique dans les essais cliniques de nouveaux traitements des patients atteints d'un syndrome myélodysplasique ou d'une néoplasie myéloproliférative
MDS/MPN are hematologically diverse stem cell malignancies sharing phenotypic features of both myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN). Currently there are no standard treatment recommendations for most adult patients with MDS/MPN. In order to optimize efforts to improve the management and disease outcomes it is essential to identify meaningful clinical and biologic endpoints, and standardized response criteria for clinical trials. The dual dysplastic and proliferative features in these stem cell malignancies define their uniqueness and challenges. We propose response assessment guidelines to harmonize future clinical trials with the principal objective of establishing suitable treatment algorithms. An international panel comprised of laboratory and clinical experts in MDS/MPN was established involving three independent academic MDS/MPN workshops (March 2013, Dec 2013, and June 2014). These recommendations are the result of this collaborative project sponsored by the MDS Foundation.
Blood , résumé, 2014