Quality-adjusted survival after treatment for acute myeloid leukemia in childhood: A Q-TWiST analysis of the Pediatric Oncology Group Study 8821.
OBJECTIVE
To describe quality-of-life considerations in post-remission therapies for children with acute myelogenous leukemia.


METHODS
A quality-adjusted survival analysis, using the quality-adjusted time without symptoms or toxicity (Q-TWiST) method, was applied to Pediatric Oncology Group Trial 8821, which compared randomized assignment with intensive consolidation chemotherapy (CC) or autologous bone marrow transplantation (ABMT). Nonrandomized assignment to allogeneic bone marrow transplantation (allo BMT) on the basis of availability of a matched related donor was also evaluated. A 25-patient cohort provided data for modeling chronic graft-versus-host disease. The Q-TWiST analysis was performed based on the intent-to-treat principle.


RESULTS
As previously reported, the 3-year event-free survival was not significantly different between the randomized arms (CC v ABMT). At a median follow-up of 5 years (of the censoring distribution), the CC group had less time in toxicity (TOX) and more time without symptoms or toxicity (TWiST), relapse-free time, and alive time than patients assigned to ABMT (none of these were statistically significant). Compared with the CC group, allo BMT patients spent more time in TOX (P <.001), more time in TWiST (P =.06), and had more relapse-free time (P =.03) and time alive (P =.07). Allo BMT was superior to ABMT with greater time in TWiST (P =.02), relapse-free time (P =.01), and time alive P =.002).


CONCLUSIONS
The Q-TWiST analysis is a powerful decision aid in choosing among alternative therapies. Prospective information on patient preferences will facilitate future trials evaluating treatment outcomes. Refinements in the Q-TWiST method could be included to further enhance the power of this patient care decision-making tool.
Parsons
S K
SK
Children's Hospital and Dana-Farber Cancer Institute, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02115, USA. parsons@al.tch.harvard.edu
Gelber
S
S
Cole
B F
BF
Ravindranath
Y
Y
Ogden
A
A
Yeager
A M
AM
Chang
M
M
Shuster
J
J
Weinstein
H J
HJ
Gelber
R D
RD
D000208:Acute Disease; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002648:Child; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data; D003661:Decision Support Techniques; D006801:Humans; D007951:Leukemia, Myeloid / Q000401:mortality / Q000628:therapy*; D011788:Quality of Life; D016019:Survival Analysis; D014481:United States / Q000453:epidemiology
D000970:Antineoplastic Agents