SANTA MONICA, CALIF. — Patients with gynecologic cancer who chose to participate in phase I clinical trials survived for twice as long as similar patients who did not participate in such trials, according to a poster presentation by Dr. Francesco Legge at the biennial meeting of the International Gynecologic Cancer Society.
These results are surprising because phase I clinical trials are intended only to assess safety and to establish dosage ranges. Any clinical benefit would be a secondary consideration.
The retrospective analysis involved patients with gynecologic cancer who were referred to phase I clinical trials at the Royal Marsden Hospital in Sutton, England, over a 2-year period. Dr. Legge, who is affiliated with the Catholic University of the Sacred Heart in Rome and in Campobasso, Italy, and his colleagues compared 32 patients who chose to participate in those clinical trials with 36 patients who were referred but chose not to participate.
Median survival time of the patients who enrolled was 8 months, compared with 4 months for the patients who did not enroll, a statistically significant difference. After a multivariate analysis that controlled for various demographic measures and disease characteristics, three factors emerged as being significantly associated with a patient's decision on whether to enroll in a clinical trial.
The longer the travel time from the patient's home to the hospital, the less likely she was to enroll. Of patients who lived within 1 hour of the hospital, 61% chose to participate, compared with 48% of the patients who lived 1–2 hours from the hospital, and 8% of patients who lived more than 2 hours from the hospital.
The better a patient's health was, as measured by her performance status, the more likely she was to participate. Of the patients with an initial Eastern Cooperative Oncology Group (ECOG) performance status of grade 0 (indicating a patient who is fully active), 59% chose to participate, compared with 51% of the patients with an initial performance status of grade 1 (indicating a patient who is able to carry out light or sedentary work). None of the patients with an initial performance status greater than grade 1 chose to participate in a trial.
Abnormal liver function tests also predicted nonenrollment in clinical trials.
Among the factors for which there was no significant relationship between enrollment or nonenrollment were age; marital status; occupation; ethnicity; interval from diagnosis; number of previous surgical procedures or previous courses of radiotherapy; and the junior or senior physician status of the individual discussing the phase I trial with the patient.
Ovarian cancer was the most common type of cancer among the patients in the study, accounting for 21 of 32 (66%) patients who chose to participate in the clinical trials and 21 of 36 (58%) patients who chose not to participate. The majority of the remaining patients had cervical cancer.
The patients participated in a wide variety of clinical trials, including those testing angiogenesis inhibitors, epidermal growth factor-receptor inhibitors, methyltransferase inhibitors, DNA-repair inhibitors, and new cytotoxics.
“The practical limitations imposed by long-distance travel, together with the potential clinical benefit due to the participation [in] these trials, should encourage more investigators to develop phase I units in major cancer centers,” the investigators concluded.