Oncology Today with Dr Neil Love

Desmoid Tumors — Microlearning Activity 1 with Dr Ravin Ratan

Mar 16, 2026
Dr Ravin Ratan, clinical investigator and oncologist at MD Anderson specializing in desmoid tumors, discusses clinical trial data and treatment strategies. He reviews long-term niragestat safety and response rates. He covers fertility and pregnancy considerations with GSIs. He debates placebo controls, the evolving role of surgery, and options like cryoablation and radiation.
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INSIGHT

Long Term Outcomes With Niragasostat

  • Niragasostat showed a 41% response rate versus 8% with placebo and produced durable responses in many patients.
  • Some patients stayed on drug >4 years with continued deepening of responses, though post-stop shrinkage could also occur.
INSIGHT

Niragasostat Safety Profile Over Time

  • Prolonged niragasostat exposure showed no new safety signals but expected GI and skin toxicities occurred.
  • Reported side effects include diarrhea, hidradenitis suppurativa–like skin findings, and other skin changes requiring some patients to stop.
ADVICE

Counsel Reproductive Risks Before Gamma Secretase Inhibitors

  • Counsel women of childbearing potential about ovarian toxicity and refer to fertility counseling before starting niragasostat.
  • Amenorrhea with raised FSH/LH and low estradiol appears reversible after stopping, but fertility impact is not yet proven.
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