Is allogeneic stem cell transplantation for myelofibrosis still indicated at the time of molecular markers and JAK inhibitors era?

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 sec...

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Published in:European journal of haematology 2017-07, Vol.99 (1), p.60-69
Main Authors: Lestang, Elsa, Peterlin, Pierre, Le Bris, Yannick, Dubruille, Viviane, Delaunay, Jacques, Godon, Catherine, Theisen, Olivier, Blin, Nicolas, Mahe, Beatrice, Gastinne, Thomas, Garnier, Alice, Touzeau, Cyrille, Voldoire, Maud, Bene, Marie C., Le Gouill, Steven, Milpied, Noel, Mohty, Mohamad, Moreau, Philippe, Guillaume, Thierry, Chevallier, Patrice
Format: Article
Language:English
Subjects:
Acute myeloid leukemia
Adult
Aged
allogeneic transplantation
Biomarkers
calreticuline
Female
Hematopoietic Stem Cell Transplantation - adverse effects
Hematopoietic Stem Cell Transplantation - methods
Humans
intent‐to‐treat analysis
JAK inhibitor
JAK2
Janus kinase 2
Janus Kinase 2 - genetics
Janus Kinase Inhibitors - administration & dosage
Janus Kinase Inhibitors - adverse effects
Janus Kinase Inhibitors - therapeutic use
Male
Medical prognosis
Middle Aged
Molecular Targeted Therapy
Mutation
Myelodysplastic syndrome
Myelofibrosis
Myeloid leukemia
myeloproliferative leukemia virus
primary myelofibrosis
Primary Myelofibrosis - diagnosis
Primary Myelofibrosis - genetics
Primary Myelofibrosis - mortality
Primary Myelofibrosis - therapy
Prognosis
Retrospective Studies
secondary myelofibrosis
Stem cell transplantation
Stem cells
Survival
Transplantation
Transplantation, Homologous
Treatment Outcome
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Summary: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 JAK2V617F burden (>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.
ISSN:0902-4441
1600-0609
DOI:10.1111/ejh.12891