High-Dose Chemotherapy and Autologous Stem-Cell Transplantation for Treatment of Ovarian Cancer

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The summary below is from the full report titled "High-Dose Chemotherapy and Autologous Stem-Cell Transplantation for Ovarian Cancer: An Autologous Blood and Marrow Transplant Registry Report." It is in the 3 October 2000 issue of Annals of Internal Medicine (volume 133, pages 504-515). The authors are PJ Stiff, J Veum-Stone, HM Lazarus, L Ayash, JR Edwards, A Keating, JP Klein, DJ Oblon, TC Shea, S Thomé, and MM Horowitz.

What is the problem and what is known about it so far?

Most women with ovarian cancer that has spread outside the ovaries die of the cancer despite treatment; very few survive more than 2 years after the cancer spreads. Researchers are therefore developing new treatment strategies, including autologous stem-cell transplantation (ASCT). In ASCT, patients first receive strong chemotherapy drugs to wipe out the cancer cells. Since the chemotherapy also wipes out normal bone marrow cells that are necessary for life, the bone marrow is then replaced with normal bone marrow–forming cells (stem cells). These normal cells are taken from the patients before chemotherapy.

Why did the researchers do this particular study?

To see how women did after treatment with high-dose chemotherapy followed by ASCT for ovarian cancer.

Who was studied?

From a national registry of persons treated with autologous blood and marrow transplantation, researchers selected 421 women with advanced ovarian cancer who had received high-dose chemotherapy and ASCT.

How was the study done?

The researchers determined how many women lived for at least 2 years without evidence of advancing cancer and how many women survived at least 2 years regardless of whether the cancer advanced. They also examined the relationship between survival and the following patient factors: age, ability to participate in daily activities, stage of cancer at time of diagnosis and at time of ASCT, microscopic characteristics of cancer cells, response to previous therapy for ovarian cancer, transplantation regimen, and year of transplantation.

What did the researchers find?

During the first 100 days after ASCT, 11% of the women died. Two years after ASCT, only 12% of women were alive without evidence of progression of the cancer, and 35% were still living overall. Women fared best if they were younger, were well enough to participate in daily activities, had a certain microscopic type of cancer cells (non–clear cell), were in remission at the time of ACST, and/or had tumors that had previously responded to chemotherapy with certain types of drugs. Even among women with the most favorable characteristics, however, only 55% survived for 2 years.

What were the limitations of the study?

This study included only women who were reported to the registry; those not reported might have had different outcomes. In addition, the study did not compare women who received high-dose chemotherapy and ASCT with similar women who received other treatments. Thus, it does not tell us whether this treatment is better than others.

What are the implications of the study?

Some women with advanced ovarian cancer survive, with or without progression of the cancer, for 2 years after treatment with high-dose chemotherapy and ASCT. Even among women with the most favorable characteristics, however, only about half survived 2 years after treatment. Further studies are needed to compare the effectiveness of this therapy with that of others.