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38 Phase II study of enzalutamide in androgen receptor positive (AR+) recurrent ovarian cancer: final results
  1. R Grisham1,
  2. D Giri2,
  3. M Henson3,
  4. A Iasonos4,
  5. Q Zhou4,
  6. A McDonnell3,
  7. J Girshman5,
  8. R O’Cearbhaill3,
  9. D Zamarin3 and
  10. C Aghajanian3
  1. 1Memorial Sloan Kettering Cancer Center, Department of Medicine- Gynecologic Medical Oncology, New York, USA
  2. 2Memorial Sloan Kettering Cancer Center, Department of Pathology, New York, USA
  3. 3Memorial Sloan Kettering Cancer Center, Department of Medicine- Gynecologic Medical Oncology Service, New York, USA
  4. 4Memorial Sloan Kettering Cancer Center, Department of Statistics, New York, USA
  5. 5Memorial Sloan Kettering Cancer Center, Department of Radiology, New York, USA


Objectives This was a single institution, phase II, Simon 2-stage with safety lead-in study of the oral androgen-receptor antagonist, enzalutamide, in patients with recurrent AR+ ovarian cancer with measurable disease and 1–3 prior lines of chemotherapy. The primary objective was to determine the proportion of patients surviving progression free for 6 months (PFS6) and overall response rate by RECIST 1.1 Criteria; with 7/58 responses or PFS6 of 13 being considered a positive study.

Methods Following consent, archival tissue was screened for AR+ by IHC with ≥5% considered positive. Enrolled patients were treated with enzalutamide 160mg po daily until progression of disease or treatment discontinuation. A cycle was 28 days. Adverse events were graded by CTCAE V 4.0.

Results Between 11/2013–7/2018 160 patients were screened and 59 patients [45 high grade serous(HGS), 14 low grade serous(LGS)] consented to treatment on the study (1 patient was replaced; efficacy cohort=58, safety cohort=59). There was 1 confirmed and 1 unconfirmed partial response (PR), PFS6 was 22% (90% CI: 15.1–100%) with PFS6 for those with HGS 19.8% (90% CI: 12.7–100%) and for LGS 38.5% (21.7%-100%). Median PFS was 3.5 months. There were no toxicities >grade 3 related to study drug. Related grade 3 toxicities occurred in 6 patients [fatigue (1), rash (2), hypertension (1), anemia (1), and transaminase elevation (1)].

Conclusions The study met its primary endpoint, with 13 patients (22%) remaining progression free at 6 months, however the response rate was low. Enzalutamide was well tolerated and may offer a good treatment option in select patients.

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