title: Is allogeneic stem cell transplantation for myelofibrosis still indicated at the time of molecular markers and JAK inhibitors era?
abstract: OBJECTIVE
  The role of allogenic stem cell transplantation (ASCT) is still debated in myelofibrosis (MF).
  
  
  METHODS
  A retrospective analyzed was performed to compare the outcome of 71 patients with intermediate-2 or high-risk Dynamic International Prognosis Scoring System+ (DIPSS+) primary (PMF) or secondary (SMF) myelofibrosis with an indication of ASCT as they ultimately underwent the procedure (n=34) or not (n=37).
  
  
  RESULTS
  Five-year overall survival (OS) was not statistically different between both groups (allograft: 52% vs no allograft: 34%, P=.12). However, progression to myelodysplastic syndrome or acute myeloid leukemia at 5 years was significantly lower in transplanted patients (14% vs 50%, P=.01). In univariate analysis, 5-year OS was significantly higher for transplanted vs non-transplanted patients with unfavorable karyotype (75% vs 0%, P=.001), SMF (71% vs 20%, P=.001) or high DIPSS+ score (46% vs 15%, P=.03). There was also a trend for better 5-year OS in allografted patients with high JAK2V617Fburden (>65%) (75% vs 8%, P=.07). Interestingly, the survival of patients who did not proceed to ASCT was dramatically increased by the use of ruxolitinib.
  
  
  CONCLUSIONS
  Not all intermediate-2/high-risk DIPSS+ MF patients benefit from ASCT, especially since the introduction of JAK2 inhibitors.
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Elsa
authors.initials: E
authors.lastname: Lestang
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Pierre
authors.initials: P
authors.lastname: Peterlin
authors.affiliation: Hematology/Biology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Yannick
authors.initials: Y
authors.lastname: Le Bris
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Viviane
authors.initials: V
authors.lastname: Dubruille
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Jacques
authors.initials: J
authors.lastname: Delaunay
authors.affiliation: Hematology/Biology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Catherine
authors.initials: C
authors.lastname: Godon
authors.affiliation: Hematology/Biology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Olivier
authors.initials: O
authors.lastname: Theisen
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Nicolas
authors.initials: N
authors.lastname: Blin
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Beatrice
authors.initials: B
authors.lastname: Mahe
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Thomas
authors.initials: T
authors.lastname: Gastinne
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Alice
authors.initials: A
authors.lastname: Garnier
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Cyrille
authors.initials: C
authors.lastname: Touzeau
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Maud
authors.initials: M
authors.lastname: Voldoire
authors.affiliation: Hematology/Biology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Marie C
authors.initials: MC
authors.lastname: Bene
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Steven
authors.initials: S
authors.lastname: Le Gouill
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Noel
authors.initials: N
authors.lastname: Milpied
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Mohamad
authors.initials: M
authors.lastname: Mohty
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Philippe
authors.identifier: http://orcid.org/0000-0003-1780-8746
authors.initials: P
authors.lastname: Moreau
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Thierry
authors.initials: T
authors.lastname: Guillaume
authors.affiliation: Hematology Department, CHU Hotel-Dieu, Nantes, France.
authors.forename: Patrice
authors.identifier: http://orcid.org/0000-0003-3142-5581
authors.initials: P
authors.lastname: Chevallier
mesh_terms: D000328:Adult; D000368:Aged; D015415:Biomarkers; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods; D006801:Humans; D053614:Janus Kinase 2 / Q000235:genetics; D000075242:Janus Kinase Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D058990:Molecular Targeted Therapy; D009154:Mutation; D055728:Primary Myelofibrosis / Q000175:diagnosis / Q000235:genetics / Q000401:mortality* / Q000628:therapy*; D011379:Prognosis; D012189:Retrospective Studies; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D015415:Biomarkers; D000075242:Janus Kinase Inhibitors; D053614:Janus Kinase 2
keywords: JAK inhibitor; JAK2; allogeneic transplantation; calreticuline; intent-to-treat analysis; myeloproliferative leukemia virus; primary myelofibrosis; secondary myelofibrosis