TY - JOUR
T1 - Design and analysis of trials of salvage therapy in acute myelogenous leukemia
AU - Estey, Elihu
AU - Thall, Peter
AU - David, Cynthia
N1 - Copyright:
Copyright 2020 Elsevier B.V., All rights reserved.
PY - 1997/6
Y1 - 1997/6
N2 - Results obtained with a given regimen in relapsed or refractory acute myelogenous leukemia (AML) are variable. This often reflects variability in patient selection. We have developed a system to account for such variability that stratifies patients with refractory or relapsed AML into four groups: group I, first complete response (CR) duration ≤2 years and receiving first salvage treatment (S1); group 2, first CR duration 1-2 years and receiving S1; group 3, first CR duration 0-1 years and receiving SI; and group 4, first CR duration 0-1 years and receiving S2, S3, or S4 after failing S1. CR rates achieved in the four groups are 73%, 47%, 14%, and 0, respectively. This system is useful for comparing results obtained with different therapies and for assigning patients to treatment. At our institution, patients in group 4 are enrolled in phase I studies, and phase I 1/4 -II studies are carried out separately in patients in groups 2, 3, and 4. A phase I 1/4 study refers to one in which the intent is to select for phase II therapies emerging from phase I trial results. The design is Bayesian, and although false-negative rates are relatively high, they are lower than those obtained if a drug for phase II testing is arbitrarily selected.
AB - Results obtained with a given regimen in relapsed or refractory acute myelogenous leukemia (AML) are variable. This often reflects variability in patient selection. We have developed a system to account for such variability that stratifies patients with refractory or relapsed AML into four groups: group I, first complete response (CR) duration ≤2 years and receiving first salvage treatment (S1); group 2, first CR duration 1-2 years and receiving S1; group 3, first CR duration 0-1 years and receiving SI; and group 4, first CR duration 0-1 years and receiving S2, S3, or S4 after failing S1. CR rates achieved in the four groups are 73%, 47%, 14%, and 0, respectively. This system is useful for comparing results obtained with different therapies and for assigning patients to treatment. At our institution, patients in group 4 are enrolled in phase I studies, and phase I 1/4 -II studies are carried out separately in patients in groups 2, 3, and 4. A phase I 1/4 study refers to one in which the intent is to select for phase II therapies emerging from phase I trial results. The design is Bayesian, and although false-negative rates are relatively high, they are lower than those obtained if a drug for phase II testing is arbitrarily selected.
KW - Bayesian design
KW - Leukemia
KW - Phase I trials
KW - Phase II trials
KW - Relapsed/refractory acute myelogenous
KW - Stratification
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U2 - 10.1007/s002800051054
DO - 10.1007/s002800051054
M3 - Article
C2 - 9272127
AN - SCOPUS:0030788301
SN - 0943-9404
VL - 40
SP - S9-S12
JO - Cancer Chemotherapy and Pharmacology, Supplement
JF - Cancer Chemotherapy and Pharmacology, Supplement
ER -