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High-Dose Chemotherapy and Autologous Stem-Cell Transplantation for Ovarian Cancer: An Autologous Blood and Marrow Transplant Registry Report

Patrick J. Stiff, MD; Judith Veum-Stone, MS; Hillard M. Lazarus, MD; Lois Ayash, MD; John R. Edwards, MD; Armand Keating, MD; John P. Klein, PhD; David J. Oblon, MD; Thomas C. Shea, MD; Stephan Thomé, MD; and Mary M. Horowitz, MD, MS
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Copyright ©2004 by the American College of Physicians

Ann Intern Med. 2000;133(7):504-515. doi:10.7326/0003-4819-133-7-200010030-00009
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Background: Autologous transplantation is increasingly used to treat epithelial ovarian cancer. However, it is not clear which patients may benefit.

Objective: To determine overall and progression-free survival and factors associated with favorable outcome after autotransplantation for ovarian cancer.

Design: Observational cohort study.

Setting: 57 centers reporting to the Autologous Blood and Marrow Transplant Registry (ABMTR).

Patients: 421 women who received transplants between 1989 and 1996.

Interventions: High-dose chemotherapy using diverse regimens with hematopoietic stem-cell rescue.

Measurements: Primary outcomes were progression-free survival and overall survival. Multivariate analyses using Cox proportional-hazards regression considered the following factors: age, Karnofsky performance score, initial stage, histologic characteristics, previous therapy, remission status, extent of disease, graft source, transplant regimen, and year of transplantation.

Results: Most patients had extensive previous chemotherapy. Forty-one percent had platinum-resistant tumors, and 38% had tumors at least 1 cm in diameter. Only 34 patients (8%) received transplants as part of initial therapy. The probability of death within 100 days was 11% (95% CI, 8% to 14%). Two-year progression-free survival was 12% (CI, 9% to 16%), and 2-year overall survival was 35% (CI, 30% to 41%). Younger age, Karnofsky performance score of at least 90%, non–clear-cell disease, remission at transplantation, and platinum sensitivity were associated with better outcomes. Progression-free and overall survival were 22% (CI, 12% to 33%) and 55% (CI, 42% to 66%), respectively, for women with a high Karnofsky performance score and non–clear-cell, platinum-sensitive tumors.

Conclusions: Some subgroups of patients with ovarian cancer seem to have good outcomes after autotransplantation, although several biases may have affected these observations. Phase III trials are needed to compare such outcomes with outcomes of conventional chemotherapy.


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Figure 1.
Probabilities of overall survival (solid line) and progression-free survival (dotted line) after autotransplantation in 421 women with advanced ovarian cancer.
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Figure 2.
Probabilities of survival (top) and progression-free survival (bottom) after autotransplantation according to number of adverse prognostic factors.

Adverse prognostic factors were age at least 48 years, Karnofsky performance score less than 90%, platinum-resistant tumors, and receipt of transplant while not in first complete remission. The solid line represents patients with one or fewer adverse prognostic factors, the dotted line represents patients with two adverse prognostic factors, and the dashed line represents patients with more than two adverse prognostic factors.

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