title: Chronic myelomonocytic leukemia in younger patients: molecular and cytogenetic predictors of survival and treatment outcome.
abstract: In patients with chronic myelomonocytic leukemia (CMML), age>65 years is an adverse prognostic factor. Our objective in the current study was to examine risk factors for survival and treatment outcome in 261 'young' adults with CMML, as defined by age ⩽65 years. In multivariable analysis, lower HB (P=0.01), higher circulating blast % (P=0.002), ASXL1 (P=0.0007) and SRSF2 mutations (P=0.008) and Mayo-French cytogenetic stratification (P=0.04) negatively impacted survival. Similarly, leukemia-free survival was independently affected by higher circulating blast % (P<0.0001), higher bone marrow blast % (P=0.0007) and the presence of circulating immature myeloid cells (P=0.0002). Seventy-five (29%) patients received hypomethylating agents (HMA), with the median number of cycles being 5, and the median duration of therapy being 5 months. The over-all response rate was 40% for azacitidine and 30% for decitabine. Fifty-three (24%) patients underwent an allogeneic hematopoietic stem cell transplant (AHSCT), with a response rate of 56% and a non-relapse mortality of 19%. Survival in young adults with CMML, although higher than in older patients, is poor and even worse in the presence of ASXL1 and SRSF2 mutations. Treatment outcome was more impressive with AHSCT than with HMA and neither was influenced by ASXL1/SRSF2 mutations or karyotype.
authors.affiliation: Department of Medicine, Division of Hematology, Mayo Clinic, Rochester, MN, USA.
authors.forename: M M
authors.initials: MM
authors.lastname: Patnaik
authors.affiliation: Department of Medicine, Division of Hematology, Mayo Clinic, Rochester, MN, USA.
authors.forename: E A
authors.initials: EA
authors.lastname: Wassie
authors.affiliation: Malignant Hematology and Immunology Program, H. Lee Moffitt Cancer Center, Tampa, FL, USA.
authors.forename: E
authors.initials: E
authors.lastname: Padron
authors.affiliation: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Onida
authors.affiliation: Hematology département, Hospital Saint-Louis, Paris, France.
authors.forename: R
authors.initials: R
authors.lastname: Itzykson
authors.affiliation: Department of Medicine, Division of Hematology, Mayo Clinic, Rochester, MN, USA.
authors.forename: T L
authors.initials: TL
authors.lastname: Lasho
authors.affiliation: 1] Université Paris Descartes, Paris, France [2] Institut Cochin, Paris, France.
authors.forename: O
authors.initials: O
authors.lastname: Kosmider
authors.affiliation: Department of Medicine, Division of Hematology, Mayo Clinic, Rochester, MN, USA.
authors.forename: C M
authors.initials: CM
authors.lastname: Finke
authors.affiliation: Division of Hematopathology, Mayo Clinic, Rochester, MN, USA.
authors.forename: C A
authors.initials: CA
authors.lastname: Hanson
authors.affiliation: Division of Cytogenetics, Mayo Clinic, Rochester, MN, USA.
authors.forename: R P
authors.initials: RP
authors.lastname: Ketterling
authors.affiliation: Malignant Hematology and Immunology Program, H. Lee Moffitt Cancer Center, Tampa, FL, USA.
authors.forename: R
authors.initials: R
authors.lastname: Komrokji
authors.affiliation: Department of Medicine, Division of Hematology, Mayo Clinic, Rochester, MN, USA.
authors.forename: A
authors.initials: A
authors.lastname: Tefferi
authors.affiliation: 1] Institut Gustave Roussy, Paris, France [2] INSERM U1009, Institut Gustave Roussy, Paris, France.
authors.forename: E
authors.initials: E
authors.lastname: Solary
mesh_terms: D000328:Adult; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D015477:Leukemia, Myelomonocytic, Chronic / Q000188:drug therapy* / Q000453:epidemiology / Q000235:genetics* / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009154:Mutation; D009687:Nuclear Proteins / Q000235:genetics*; D011379:Prognosis; D012261:Ribonucleoproteins / Q000235:genetics*; D000068103:Serine-Arginine Splicing Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D009687:Nuclear Proteins; D012261:Ribonucleoproteins; C073288:SRSF2 protein, human; D000068103:Serine-Arginine Splicing Factors