CURRENT CLINICAL INTERESTS
Acute myeloid leukemia (AML)
CURRENT RESEARCH INTERESTS
1. Clinical trials in AML, particularly those involving new
2. Management of patients with AML
New designs for clinical trials - patients and physician are
poorly served by the current phase 1-phase 2-phase 3 paradigm. I
am interested in methodology that (a) simultaneously monitors
multiple endpoints (such as response and toxicity, or CR and
survival), and (b) focuses on small randomized trials to select a
therapy to move to a later stage of testing; although such
play-the-winner trials are often viewed as "underpowered"
they can be shown to be preferable to trials that test only 1 new
therapy, because the latter ignore that the worst false negative
results from not studying a drug at all, given the role of empiricism
in drug development.
questions face clinicians caring for patients with AML. For
example, is it reasonable to discharge a patient with a low neutrophil
count, is a day 14 marrow really necessary, how well do I know this
patients' prognosis with standard therapy? Oftentimes answers to
these questions are based more on convention than data. Hence we aim
to collect and analyze data to answer these type questions.
Estey E, Thall PF: New designs for phase II clinical trials. Blood
Giles FJ, Kantarjian HM, Cortes JE, Garcia-Manero G, Verstovsek S, Faderl
S, Thomas DA, Ferrajoli A, O’Brien S, Wathen JK, Xiao L-C, Berry DA,
Estey EH. Adaptive randomized study of Idarubicin and
Cytarabine versus Troxacitabine and Cytarabine versus Troxacitabine
and Idarubicin in untreated patients 50 years or older with adverse
karyotype acute myeloid leukemia. J. Clin. Oncol. 21:1722-1727, 2003.
Estey E, Garcia-Manero G, Ferrajoli A, Faderl S, Verstovsek S, Jones D, Kantarjian
H. Use of all-trans retinoic acid plus arsenic trioxide as an alternative to chemotherapy in untreated acute promyelocytic leukemia.
Blood 107:3469-3473, 2006.
Estey E, Dohner H. Acute myeloid leukemia.
Lancet 368:1894-1907, 2006.
Appelbaum FR, Kopecky KJ, Tallman MS, Slovak ML, Gundacker HM, Kim HT, Dewald GW, Kantarjian HM, Pierce SR,
Estey EH. The clinical spectrum of adult acute myeloid leukemia associated with core binding factor translocations.
Br. J. Haematol.
Estey E. Acute myeloid leukemia and myelodysplastic syndromes in older patients.
J. Clin. Oncol.
Estey E, de Lima M, Tibes R, Pierce S, Kantarjian H, Champlin R, Giralt S. Prospective feasibility analysis of reduced intensity conditioning (RIC) regimens for hematopoetic stem cell transplantation (HSCT) in elderly patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). Blood
Estey E. "Fairest of them
all" for myelodysplastic syndromes. Blood 111:6, 2008.
P, Nguyen H, Estey E.
Patient-specific dose finding based on bivariate outcomes and
covariates. Biometrics 64:1126-36,
Gardner A, Mattiuzzi G,
Faderl S, Borthakur G, Garcia-Manero G, Pierce S, Brandt M, Estey E.
Randomized comparison of “neutropenic” and non-neutropenic
diet in patients undergoing remission
induction therapy for acute myeloid leukemia . J. Clin. Oncol. 26:5684-5688,
Sekeres M, Elson P,
Kalaycio, Advani A, Copelan E, Faderl S, Kantarjian H, Estey
E. Time from diagnosis to treatment initiation
predicts survival in younger, but not older, acute myeloid leukemia
patients. Blood 113:28-36,
Armistead PM, de Lima M, Pierce S, Qiao W, Wang
X, Thall PF, Giralt S, Ravandi F, Kantarjian H, Champlin R, Estey
E. Quantifying the survival benefit for allogeneic hematopoietic stem cell
transplantation in relapsed acute myelogenous leukemia. Biol
Blood Marrow Transplant.
Amadori S, Appelbaum FR, Buchner T, Burnett AK, Dombret H, Fenaux P,
Grimwade D, Larson RA, Lo Coco F, Naoe T, Niederwieser D, Ossenkoppele
GJ, Sanz MA, Sierra J, Tallman MS, Lowenberg B, and Bloomfield
and management of acute myeloid leukemia in adults: recommendations
from an international expert panel, on behalf of the European
Blood 115:453-474, 2010.
Walter R, Kantarjian H, Huang X, Pierce S, Sun
X,Gundaker H, Ravandi F, Faderl S,Tallman M, Appelbaum F, Estey E. The effect
of complete remission(CR) and CR with incomplete platelet recovery on
outcome in acute myeloid leukemia: a combined Eastern Cooperative
Oncology group, Southwest Oncology Group and M.D. Anderson cancer
center study, J. Clin. Oncol.
R, Appelbaum F, Tallman M, Weiss N, Larson R, Estey E.
Shortcomings in the clinical evaluation of new drugs: acute myeloid leukemia as
paradigm. Blood 116:2420-2428, 2010.
Estey E. High
cytogenetic or molecular genetic risk AML. Hematology Am.
Soc. Hematol. Educ. Program 474-481, 2010.
Schanz J, Steidl C,
Fonatsch C, Pfeilstöcker M, Nösslinger T, Tuechler H, Valent P,
Hildebrandt B, Giagounidis A, Aul C, Lübbert M, Stauder R, Krieger O,
Garcia-Manero G, Kantarjian H, Germing U, Haase D, Estey E. Coalesced
multicentric analysis of 2,351 patients with myelodysplastic syndromes
indicates an underestimation of poor-risk cytogenetics of
myelodysplastic syndromes in the international prognostic scoring
system. J. Clin. Oncol.
Newly diagnosed acute promyelocytic leukemia: arsenic moves
front and center. J.
Clin. Oncol. 29:2743-2746, 2011.
Becker PS, Kantarjian HM,
Appelbaum FR, Petersdorf SH, Storer B, Pierce S, Shan J, Hendrie PC,
Pagel JM, Shustov AR, Stirewalt DL, Faderl S, Harrington E, Estey
EH. Clofarabine with
high dose cytarabine and granulocyte colony stimulating factor priming
for relapsed and refractory AMLrabine with high dose cytarabine and
granulocyte colony-stimulating factor (G-CSF) priming for relapsed and
refractory acute myeloid leukaemia. Br.
J. Haematol. 2011.
Walter R, Lee S, Gardner K,
Chai X, Shannon- Dorcy K, Applebaum F, Estey E.
Outpatient management following intensive induction
chemotherapy for myelodysplastic syndromes and acute myeloid leukemia:
a pilot study. Haematologica 96:914-917, 2011.
Walter R, Othus M,
Bortahkur G, Ravandi F, Cortes J, Pierce S, Appelbaum F, Kantarjain H,
Estey E: Prediction of early death following induction
therapy for newly diagnosed AML with pretreatment risk scores: a novel
paradigm for treatment assignment. J. Clin. Oncol. 29:4417-4423, 2011.
Estey E. How to
manage high-risk acute myeloid leukemia. Leukemia 26(5):861-869, 2012.
Estey E. Be Quick But Don't Hurry. Blood 119:5342-5343, 2012.
Estey E. Treatment of AML : Resurrection for Gemtuzumab Ozogamicin. Lancet 379:1468-1469, 2012.
Estey E. BeIntensity of Conditioning for Allogeneic Hematopoetic Cell Transplant in First Complete Remission of Acute Myeloid Leukemia. Lancet Oncology (in press).