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

Eastern Cooperative Oncology Group (ECOG) and North American Leukemia intergroup

Research output: Contribution to journalArticle

20 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)62-68
Number of pages7
JournalBritish Journal of Haematology
Volume167
Issue number1
DOIs
StatePublished - Oct 1 2014

Fingerprint

Azacitidine
Myelodysplastic Syndromes
Platelet Count
Acute Myeloid Leukemia
Survival
Risk Reduction Behavior
Therapeutics
Bone Marrow
Clinical Trials
Confidence Intervals
Research

Keywords

  • Azacitidine
  • French prognostic scoring system
  • Myelodysplastic syndromes
  • Prognostic models
  • Revised international prognostic scoring system

ASJC Scopus subject areas

  • Hematology
  • Medicine(all)

Cite this

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title = "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",
abstract = "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.",
keywords = "Azacitidine, French prognostic scoring system, Myelodysplastic syndromes, Prognostic models, Revised international prognostic scoring system",
author = "{Eastern Cooperative Oncology Group (ECOG) and North American Leukemia intergroup} and Zeidan, {Amer M.} and Lee, {Ju Whei} and Thomas Prebet and Peter Greenberg and Zhuoxin Sun and Mark Juckett and Smith, {Mitchell R.} and Paietta, {Elisabeth M.} and Janice Gabrilove and Erba, {Harry P.} and Katterling, {Rhett P.} and Tallman, {Martin S.} and Gore, {Steven D.}",
year = "2014",
month = "10",
day = "1",
doi = "10.1111/bjh.13008",
language = "English (US)",
volume = "167",
pages = "62--68",
journal = "British Journal of Haematology",
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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 M.

AU - Gabrilove, Janice

AU - Erba, Harry P.

AU - Katterling, Rhett P.

AU - Tallman, Martin S.

AU - Gore, Steven D.

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

VL - 167

SP - 62

EP - 68

JO - British Journal of Haematology

JF - British Journal of Haematology

SN - 0007-1048

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