TY - JOUR
T1 - Prognostic factors for response and overall survival in 282 patients with higher-risk myelodysplastic syndromes treated with azacitidine
AU - Itzykson, Raphael
AU - Thépot, Sylvain
AU - Quesnel, Bruno
AU - Dreyfus, Francois
AU - Beyne-Rauzy, Odile
AU - Turlure, Pascal
AU - Vey, Norbert
AU - Recher, Christian
AU - Dartigeas, Caroline
AU - Legros, Laurence
AU - Delaunay, Jacques
AU - Salanoubat, Célia
AU - Visanica, Sorin
AU - Stamatoullas, Aspasia
AU - Isnard, Francoise
AU - Marfaing-Koka, Anne
AU - De Botton, Stephane
AU - Chelghoum, Youcef
AU - Taksin, Anne Laure
AU - Plantier, Isabelle
AU - Ame, Shanti
AU - Boehrer, Simone
AU - Gardin, Claude
AU - Beach, C. L.
AU - Adès, Lionel
AU - Fenaux, Pierre
PY - 2011/1/13
Y1 - 2011/1/13
N2 - Prognostic factors for response and survival in higher-risk myelodysplastic syndrome patients treated with azacitidine (AZA) remain largely unknown. Two hundred eighty-two consecutive high or intermediate-2 risk myelodysplastic syndrome patients received AZA in a compassionate, patient-named program. Diagnosis was RA/RARS/RCMD in 4%, RAEB-1 in 20%, RAEB-2 in 54%, and RAEB-t (AML with 21%-30% marrow blasts) in 22%. Cytogenetic risk was good in 31%, intermediate in 17%, and poor in 47%. Patients received AZA for a median of 6 cycles (1-52). Previous low-dose cytosine arabinoside treatment (P = .009), bone marrow blasts > 15% (P = .004), and abnormal karyotype (P = .03) independently predicted lower response rates. Complex karyotype predicted shorter responses (P = .0003). Performance status ≥ 2, intermediate- and poor-risk cytogenetics, presence of circulating blasts, and red blood cell transfusion dependency ≥ 4 units/8 weeks (all P < 10-4) independently predicted poorer overall survival (OS). A prognostic score based on those factors discriminated 3 risk groups with median OS not reached, 15.0 and 6.1 months, respectively (P < 10-4). This prognostic score was validated in an independent set of patients receiving AZA in the AZA-001 trial (P = .003). Achievement of hematological improvement in patients who did not obtain complete or partial remission was associated with improved OS (P < 10-4). In conclusion, routine tests can identify subgroups of patients with distinct prognosis with AZA treatment.
AB - Prognostic factors for response and survival in higher-risk myelodysplastic syndrome patients treated with azacitidine (AZA) remain largely unknown. Two hundred eighty-two consecutive high or intermediate-2 risk myelodysplastic syndrome patients received AZA in a compassionate, patient-named program. Diagnosis was RA/RARS/RCMD in 4%, RAEB-1 in 20%, RAEB-2 in 54%, and RAEB-t (AML with 21%-30% marrow blasts) in 22%. Cytogenetic risk was good in 31%, intermediate in 17%, and poor in 47%. Patients received AZA for a median of 6 cycles (1-52). Previous low-dose cytosine arabinoside treatment (P = .009), bone marrow blasts > 15% (P = .004), and abnormal karyotype (P = .03) independently predicted lower response rates. Complex karyotype predicted shorter responses (P = .0003). Performance status ≥ 2, intermediate- and poor-risk cytogenetics, presence of circulating blasts, and red blood cell transfusion dependency ≥ 4 units/8 weeks (all P < 10-4) independently predicted poorer overall survival (OS). A prognostic score based on those factors discriminated 3 risk groups with median OS not reached, 15.0 and 6.1 months, respectively (P < 10-4). This prognostic score was validated in an independent set of patients receiving AZA in the AZA-001 trial (P = .003). Achievement of hematological improvement in patients who did not obtain complete or partial remission was associated with improved OS (P < 10-4). In conclusion, routine tests can identify subgroups of patients with distinct prognosis with AZA treatment.
UR - http://www.scopus.com/inward/record.url?scp=78650172030&partnerID=8YFLogxK
U2 - 10.1182/blood-2010-06-289280
DO - 10.1182/blood-2010-06-289280
M3 - Article
C2 - 20940414
AN - SCOPUS:78650172030
SN - 0006-4971
VL - 117
SP - 403
EP - 411
JO - Blood
JF - Blood
IS - 2
ER -