TY - JOUR
T1 - Platelet count doubling after the first cycle of azacitidine therapy predicts eventual response and survival in patients with myelodysplastic syndromes and oligoblastic acute myeloid leukaemia but does not add to prognostic utility of the revised IPSS
AU - Eastern Cooperative Oncology Group (ECOG) and North American Leukemia intergroup
AU - Zeidan, Amer M.
AU - Lee, Ju Whei
AU - Prebet, Thomas
AU - Greenberg, Peter
AU - Sun, Zhuoxin
AU - Juckett, Mark
AU - Smith, Mitchell R.
AU - Paietta, Elisabeth
AU - Gabrilove, Janice
AU - Erba, Harry P.
AU - Katterling, Rhett P.
AU - Tallman, Martin S.
AU - Gore, Steven D.
N1 - Publisher Copyright:
© 2014 John Wiley & Sons Ltd.
PY - 2014/10/1
Y1 - 2014/10/1
N2 - Summary: Reliable clinical or molecular predictors of benefit from azacitidine therapy in patients with myelodysplastic syndromes (MDS) are not defined. Doubling of platelet count at start of second cycle of azacitidine therapy compared to baseline was associated with achieving response and survival advantage in a Dutch cohort. To validate this observation, we analysed a larger cohort of North American patients, whose data was collected in a prospective clinical trial with a longer median follow-up. We found a significant association between platelet count doubling after first cycle of azacitidine therapy and probability of achieving objective response. Among patients with MDS or oligoblastic acute myeloid leukaemia (<30% bone marrow blasts, n = 102), there was a statistically significant reduction in risk of death for patients who achieved platelet count doubling (n = 23, median OS, 21·0 months) compared to those who did not (n = 79, median OS, 16·7 months, adjusted hazard ratio (no/yes)=1·88, 95% confidence interval, 1·03-3·40, P = 0·04). Nonetheless, the addition of this platelet count doubling variable did not improve the survival prediction provided by the revised International Prognostic Scoring System or the French Prognostic Scoring System. Identification of reliable and consistent predictors for clinical benefit for azacitidine therapy remains an unmet medical need and a top research priority.
AB - Summary: Reliable clinical or molecular predictors of benefit from azacitidine therapy in patients with myelodysplastic syndromes (MDS) are not defined. Doubling of platelet count at start of second cycle of azacitidine therapy compared to baseline was associated with achieving response and survival advantage in a Dutch cohort. To validate this observation, we analysed a larger cohort of North American patients, whose data was collected in a prospective clinical trial with a longer median follow-up. We found a significant association between platelet count doubling after first cycle of azacitidine therapy and probability of achieving objective response. Among patients with MDS or oligoblastic acute myeloid leukaemia (<30% bone marrow blasts, n = 102), there was a statistically significant reduction in risk of death for patients who achieved platelet count doubling (n = 23, median OS, 21·0 months) compared to those who did not (n = 79, median OS, 16·7 months, adjusted hazard ratio (no/yes)=1·88, 95% confidence interval, 1·03-3·40, P = 0·04). Nonetheless, the addition of this platelet count doubling variable did not improve the survival prediction provided by the revised International Prognostic Scoring System or the French Prognostic Scoring System. Identification of reliable and consistent predictors for clinical benefit for azacitidine therapy remains an unmet medical need and a top research priority.
KW - Azacitidine
KW - French prognostic scoring system
KW - Myelodysplastic syndromes
KW - Prognostic models
KW - Revised international prognostic scoring system
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U2 - 10.1111/bjh.13008
DO - 10.1111/bjh.13008
M3 - Article
C2 - 24995683
AN - SCOPUS:84911008768
SN - 0007-1048
VL - 167
SP - 62
EP - 68
JO - British journal of haematology
JF - British journal of haematology
IS - 1
ER -