title: An international consortium proposal of uniform response criteria for myelodysplastic/myeloproliferative neoplasms (MDS/MPN) in adults.
abstract: Myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN) are hematologically diverse stem cell malignancies sharing phenotypic features of both myelodysplastic syndromes and myeloproliferative neoplasms. There are currently no standard treatment recommendations for most adult patients with MDS/MPN. To optimize efforts to improve the management and disease outcomes, it is essential to identify meaningful clinical and biologic end points 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 comprising laboratory and clinical experts in MDS/MPN was established involving 3 independent academic MDS/MPN workshops (March 2013, December 2013, and June 2014). These recommendations are the result of this collaborative project sponsored by the MDS Foundation.
authors.affiliation: Vanderbilt-Ingram Cancer Center/Vanderbilt University Medical Center, TN;
authors.forename: Michael R
authors.identifier: http://orcid.org/0000-0003-3763-5504
authors.initials: MR
authors.lastname: Savona
authors.affiliation: University of Pavia and Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;
authors.forename: Luca
authors.initials: L
authors.lastname: Malcovati
authors.affiliation: H. Lee Moffitt Cancer Center, Tampa, FL;
authors.forename: Rami
authors.initials: R
authors.lastname: Komrokji
authors.affiliation: Cleveland Clinic Taussig Cancer Institute, Cleveland, OH;
authors.forename: Ramon V
authors.initials: RV
authors.lastname: Tiu
authors.affiliation: Tufts University Medical Center, Boston, MA;
authors.forename: Tariq I
authors.initials: TI
authors.lastname: Mughal
authors.affiliation: Weill Cornell Medical College, New York, NY;
authors.forename: Attilio
authors.initials: A
authors.lastname: Orazi
authors.affiliation: Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris, Université Paris Diderot, Paris, France;
authors.forename: Jean-Jacques
authors.initials: JJ
authors.lastname: Kiladjian
authors.affiliation: H. Lee Moffitt Cancer Center, Tampa, FL;
authors.forename: Eric
authors.initials: E
authors.lastname: Padron
authors.affiliation: Institut Gustave Roussy, Villejuif, France;
authors.forename: Eric
authors.initials: E
authors.lastname: Solary
authors.affiliation: Mayo Clinic Cancer Center, Scottsdale, AZ;
authors.forename: Raoul
authors.initials: R
authors.lastname: Tibes
authors.affiliation: Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris, Université Paris Diderot, Paris, France;
authors.forename: Raphael
authors.initials: R
authors.lastname: Itzykson
authors.affiliation: University of Pavia and Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;
authors.forename: Mario
authors.initials: M
authors.lastname: Cazzola
authors.affiliation: Mayo Clinic Cancer Center, Scottsdale, AZ;
authors.forename: Ruben
authors.initials: R
authors.lastname: Mesa
authors.affiliation: Cleveland Clinic Taussig Cancer Institute, Cleveland, OH;
authors.forename: Jaroslaw
authors.initials: J
authors.lastname: Maciejewski
authors.affiliation: Hôpital Saint-Louis, Assistance Publique - Hôpitaux de Paris, Université Paris Diderot, Paris, France;
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
authors.affiliation: MD Anderson Cancer Center, Houston, TX;
authors.forename: Guillermo
authors.initials: G
authors.lastname: Garcia-Manero
authors.affiliation: Cleveland Clinic Taussig Cancer Institute, Cleveland, OH;
authors.forename: Aaron
authors.initials: A
authors.lastname: Gerds
authors.affiliation: Hospital Universitario y Politecnico La Fe, Valencia, Spain;
authors.forename: Guillermo
authors.initials: G
authors.lastname: Sanz
authors.affiliation: University of Freiburg, Germany;
authors.forename: Charlotte M
authors.initials: CM
authors.lastname: Niemeyer
authors.affiliation: The August Pi i Sunyer Biomedical Research Institute, University of Barcelona, Barcelona, Spain;
authors.forename: Francisco
authors.initials: F
authors.lastname: Cervantes
authors.affiliation: University of Düsseldorf, Düsseldorf, Germany; and.
authors.forename: Ulrich
authors.initials: U
authors.lastname: Germing
authors.affiliation: University of Southampton and Wessex Regional Genetics Laboratory, Salisbury, United Kingdom.
authors.forename: Nicholas C P
authors.initials: NC
authors.lastname: Cross
authors.affiliation: H. Lee Moffitt Cancer Center, Tampa, FL;
authors.forename: Alan F
authors.initials: AF
authors.lastname: List
mesh_terms: D000465:Algorithms; D049109:Cell Proliferation; D002986:Clinical Trials as Topic; D018450:Disease Progression; D019337:Hematologic Neoplasms / Q000175:diagnosis* / Q000628:therapy*; D006405:Hematology / Q000592:standards*; D006801:Humans; D007391:International Cooperation; D008495:Medical Oncology / Q000592:standards*; D009154:Mutation; D009190:Myelodysplastic Syndromes / Q000175:diagnosis* / Q000628:therapy*; D009196:Myeloproliferative Disorders / Q000175:diagnosis* / Q000628:therapy*; D010641:Phenotype; D017410:Practice Guidelines as Topic; D011795:Surveys and Questionnaires; D016896:Treatment Outcome