Stephanie J. Lee, MD, MPH; Karen M. Kuntz, ScD; Mary M. Horowitz, MD, MS; Philip B. McGlave, MD; John M. Goldman, DM; Kathleen A. Sobocinski, MS; Janet Hegland, BS; Craig Kollman, PhD; Susan K. Parsons, MD, MRP; Milton C. Weinstein, PhD; Jane C. Weeks, MD, MS; Joseph H. Antin, MD
Chronic myelogenous leukemia (CML) is an indolent but ultimately fatal disease. Because the natural history of CML varies and quality of life with CML may be excellent until shortly before death, deciding whether and when to pursue unrelated donor bone marrow transplantation is often difficult.
To compare early transplantation, delayed transplantation, and no transplantation for patients with chronic-phase CML on the basis of discounted, quality-adjusted life expectancy.
A Markov model comparing different strategies was constructed. This model considers patient age, quality of life, risk aversion, and the competing risks for CML progression and transplant toxicity.
Therapeutic decision at the time of diagnosis of CML.
The base case is a 35-year-old patient with intermediate-prognosis CML. Younger and older patients with better and worse prognoses are also evaluated.
Early transplantation, delayed transplantation, and no transplantation.
Quality-adjusted, discounted life expectancy.
For patients with newly diagnosed CML, transplantation within the first year provides the greatest quality-adjusted expected survival, although this benefit decreases with increasing patient age. For a 35-year-old patient with intermediate-prognosis CML, transplantation within the first year results in 5.3 more discounted, quality-adjusted years of life expectancy than does no transplantation. This finding is robust even with varying baseline assumptions.
These results support the use of early unrelated donor bone marrow transplantation for most patients with CML.
Lee SJ, Kuntz KM, Horowitz MM, et al. Unrelated Donor Bone Marrow Transplantation for Chronic Myelogenous Leukemia: A Decision Analysis. Ann Intern Med. 1997;127:1080–1088. doi: https://doi.org/10.7326/0003-4819-127-12-199712150-00005
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Published: Ann Intern Med. 1997;127(12):1080-1088.
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