Treatment of progression of Philadelphia-negative myeloproliferative neoplasms to myelodysplastic syndrome or acute myeloid leukemia by azacitidine: a report on 54 cases on the behalf of the Groupe Francophone des Myelodysplasies (GFM).
Transformation of Philadelphia (Ph)-negative myeloproliferative neoplasms (MPNs) to myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) is associated with poor response to chemotherapy and short survival. Fifty-four patients with Ph-negative MPN (including 21 essential thrombocythemia [ET], 21 polycythemia vera [PV], 7 primary myelofibrosis, and 5 unclassified MPN) who had progressed to AML (n = 26) or MDS (n = 28) were treated with azacitidine in a patient-named program. Overall response rate was 52% (24% complete response [CR], 11% partial response [PR], 8% marrow CR or CR with incomplete recovery of cytopenias, 9% hematologic improvement) and median response duration was 9 months. Prognostic factors were for overall response the underlying MPN (71% vs 33% responses in ET and PV, respectively; P = .016); prognostic factors for CR achievement were the underlying MPN (14% CR for PV vs 43% for ET; P = .040) and World Health Organization classification at transformation (36% vs 12% CR in MDS and AML, respectively, P = .038). Recurrence of chronic phase features of the initial MPN was observed in 39% of the responders. Median overall survival was 11 months. Azacitidine gives encouraging results in Ph-negative MPN having progressed to AML or MDS, but response duration is short, and consolidation treatments have to be evaluated.
Thepot
Sylvain
S
Service d'Hematologie Clinique, Hôpital Avicenne, Assistance Publique-Hôpitaux de Paris, Université Paris 13, Bobigny, France.
Itzykson
Raphael
R
Seegers
Valerie
V
Raffoux
Emmanuel
E
Quesnel
Bruno
B
Chait
Yasmine
Y
Sorin
Lucile
L
Dreyfus
Francois
F
Cluzeau
Thomas
T
Delaunay
Jacques
J
Sanhes
Laurence
L
Eclache
Virginie
V
Dartigeas
Caroline
C
Turlure
Pascal
P
Harel
Stephanie
S
Salanoubat
Celia
C
Kiladjian
Jean-Jacques
JJ
Fenaux
Pierre
P
Adès
Lionel
L
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D001374:Azacitidine / Q000627:therapeutic use*; D018450:Disease Progression; D005260:Female; D005602:France / Q000453:epidemiology; D006801:Humans; D053208:Kaplan-Meier Estimate; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000209:etiology* / Q000401:mortality; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000209:etiology* / Q000401:mortality; D009196:Myeloproliferative Disorders / Q000150:complications* / Q000188:drug therapy* / Q000235:genetics; D010677:Philadelphia Chromosome; D011379:Prognosis; D016896:Treatment Outcome
D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine