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Quality-Adjusted Survival After Treatment for Acute Myeloid Leukemia in Childhood: A Q-TWiST Analysis of the Pediatric Oncology Group Study 8821

From the Children's Hospital and Dana-Farber Cancer Institute, Massachusetts General Hospital, Harvard Medical School, Boston, MA; Dartmouth Medical School, Lebanon, NH; Children's Hospital of Michigan, Barbara Ann Karmanos Cancer Institute and Wayne State University, Detroit, MI; Texas Children Cancer Center and Baylor College of Medicine, Houston, TX; Emory University School of Medicine, Atlanta, GA; and Pediatric Oncology Group Statistical Office, Department of Statistics, Gainsville, FL.

Address reprint requests to Susan K. Parsons, MD, Department of Pediatric Oncology, Dana-Farber Cancer Institute, 44 Binney St, Boston, MA 02115; email parsons{at}a1.tch.harvard.edu

PURPOSE: To describe quality-of-life considerations in postremission therapies for children with acute myelogenous leukemia.

PATIENTS AND 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).

CONCLUSION: 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.

A preliminary report on this study was presented at the July 1997 Joint Meeting of the Society for Clinical Trials and International Society for Clinical Biostatistics, Boston, MA.

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