TY - JOUR
T1 - Clinical value of event-free survival in acute myeloid leukemia
AU - Maiti, Abhishek
AU - Kantarjian, Hagop M.
AU - Popat, Vinita
AU - Borthakur, Gautam
AU - Garcia-Manero, Guillermo
AU - Konopleva, Marina Y.
AU - DiNardo, Courtney D.
AU - Verstovsek, Srdan
AU - Andreeff, Michael
AU - Kadia, Tapan M.
AU - Ajufo, Helen O.
AU - Goswamy, Rohit V.
AU - Blanco, Carlos
AU - Velasquez, Miguel
AU - Daver, Naval G.
AU - Pemmaraju, Naveen
AU - Pierce, Sherry R.
AU - Wierda, William G.
AU - Kornblau, Steven M.
AU - Ravandi, Farhad
AU - Cortes, Jorge E.
N1 - Funding Information:
This study was supported in part by the Shannon Timmins Endowed Fellowship in Leukemia Research awarded to A.M., and MD Anderson Cancer Center Support Grant CA016672 from the National Institutes of Health, National Cancer Institute.
Publisher Copyright:
© 2020 by The American Society of Hematology
PY - 2020/4/28
Y1 - 2020/4/28
N2 - The value of event-free survival (EFS) as an end point in acute myeloid leukemia (AML) trials has been questioned. We hypothesized that rather than a surrogate for overall survival (OS), improvement in EFS may decrease the use of health care. In this retrospective study, we identified 400 patients with AML who were treated on first-line therapy trials and had OS between 2 and 36 months. We captured health care use from diagnosis until death or until the patient was censored at stem cell transplantation (SCT). We used correlation and regression analysis to determine the relation between health care use and EFS. Among patients with newly diagnosed AML, 35% had adverse-risk AML, 48% received intensive chemotherapy, and 28% received hypomethylating agents. The median EFS censored at SCT was 9.7 months. Longer EFS led to a significant decline in health care use regardless of OS. This held true for all observations, including overall health care use (r 5 20.45), sum of clinic visits, emergency room visits, hospitalizations, consultations (r 5 20.44), sum of invasive procedures, laboratory and imaging studies (r 5 20.51), and blood product transfusions (r 5 20.19). These correlations were stronger for patients who achieved a complete remission and held true across age, treatment, and disease risk subgroups. In patients with newly diagnosed AML, improvement in EFS correlates with a decrease in all health care use irrespective of OS duration.
AB - The value of event-free survival (EFS) as an end point in acute myeloid leukemia (AML) trials has been questioned. We hypothesized that rather than a surrogate for overall survival (OS), improvement in EFS may decrease the use of health care. In this retrospective study, we identified 400 patients with AML who were treated on first-line therapy trials and had OS between 2 and 36 months. We captured health care use from diagnosis until death or until the patient was censored at stem cell transplantation (SCT). We used correlation and regression analysis to determine the relation between health care use and EFS. Among patients with newly diagnosed AML, 35% had adverse-risk AML, 48% received intensive chemotherapy, and 28% received hypomethylating agents. The median EFS censored at SCT was 9.7 months. Longer EFS led to a significant decline in health care use regardless of OS. This held true for all observations, including overall health care use (r 5 20.45), sum of clinic visits, emergency room visits, hospitalizations, consultations (r 5 20.44), sum of invasive procedures, laboratory and imaging studies (r 5 20.51), and blood product transfusions (r 5 20.19). These correlations were stronger for patients who achieved a complete remission and held true across age, treatment, and disease risk subgroups. In patients with newly diagnosed AML, improvement in EFS correlates with a decrease in all health care use irrespective of OS duration.
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U2 - 10.1182/bloodadvances.2019001150
DO - 10.1182/bloodadvances.2019001150
M3 - Article
C2 - 32330243
AN - SCOPUS:85084995559
SN - 2473-9529
VL - 4
SP - 1690
EP - 1699
JO - Blood Advances
JF - Blood Advances
IS - 8
ER -