Desmoid Tumors — Microlearning Activity 2 with Dr Ravin Ratan
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This episode of the Gastrointestinal Cancer Update podcast features Dr. Neil Love interviewing Dr. Ravin Ratan from MD Anderson Cancer Center on the practical management of desmoid tumors, focusing on real-world case discussions. The conversation centers on three complex patient cases: a 40-year-old woman with a large pelvic desmoid who experienced spontaneous regression on active surveillance; a 39-year-old with a symptomatic intra-abdominal desmoid that responded dramatically to niragacostat; and a 41-year-old with recurrent breast desmoid after multiple surgeries and radiation, who achieved sustained remission with systemic therapy. Dr. Ratan emphasizes that not all desmoid tumors require treatment, surgery is no longer the first-line approach due to high recurrence rates, and systemic therapies like niragacostat offer effective alternatives with manageable side effects. He discusses the timing of treatment response—symptoms often improve quickly, while imaging shrinkage lags—and highlights the lack of definitive guidelines on treatment duration, recommending a minimum of six months to a year, with discontinuation possible if the tumor remains stable and symptoms are controlled. The episode also explores the impact of ovarian suppression from targeted therapies, the role of cryoablation in select cases, and the importance of multidisciplinary care. Overall, the discussion underscores a shift toward personalized, non-surgical management strategies based on symptom burden and tumor location rather than size alone. Key takeaways include: 1) Active surveillance is viable for select patients, especially those with large tumors and fertility concerns; 2) Systemic therapies like niragacostat can lead to rapid symptom relief and significant tumor shrinkage; 3) Treatment duration should be time-limited and individualized, with discontinuation considered after 1–2 years if stable; 4) Cryoablation is a promising local option for accessible tumors, though residual findings post-treatment require careful interpretation; 5) Ovarian toxicity from targeted agents is likely reversible but not fully predictable, warranting fertility counseling; 6) Prior surgery or radiation does not preclude response to systemic therapy; 7) Patient-centered decision-making is essential, especially when balancing treatment efficacy with quality of life; 8) Multidisciplinary consultation is strongly advised for complex cases.
Active surveillance is a valid option for large desmoid tumors, especially in patients concerned about fertility and ovarian function.
Systemic therapies like niragacostat can lead to rapid symptom improvement and significant tumor shrinkage, even in large or symptomatic tumors.
Treatment duration should be time-limited (typically 6 months to 2 years), with discontinuation considered if the tumor is stable and symptoms are controlled.
Cryoablation is a viable local therapy for accessible desmoids, offering a one-time intervention with potential for long-term remission.
Ovarian suppression from targeted agents is likely reversible, but fertility impact remains uncertain—fertility counseling is essential before starting therapy.
…and 3 more takeaways available in PodZeus
Introduction and Case 1: Spontaneous Regression in a Large Pelvic Desmoid
“It absolutely happens. I've got a collection of patients with large desmoid tumors that were potentially not in dangerous locations... came to see me with, you know, probably a 10 or 11 centimeter desmoid tumor in his flank. But he just swore up and down, you know, Doc, it just stopped hurting a month ago. And I feel like it's getting better.”
Case 2: Response to Niragacostat in a Symptomatic Intra-Abdominal Desmoid
“In that drug symptoms get better quickly. It's probably true for other GSIs. It's probably true for other therapies that are very active in this disease, but we've seen it demonstrated with naira.”
Case 3: Recurrent Breast Desmoid After Multiple Surgeries and Radiation
“We've done a fair bit of systemic therapy as primary treatment, but now this patient has been refractory to some local therapies. Also important to note in the DEFI trial, many of the patients that had prior local therapy like surgery, and so those patients seem to respond just as well as anyone else to niragasostat.”
Key Principles in Desmoid Tumor Management
Dr. Ratan outlines core principles: not all desmoids need treatment, surgery is no longer preferred, systemic therapies are first-line, and treatment should be time-limited based on response and tolerability.
Local Therapies, Side Effects, and Treatment Discontinuation
The episode concludes with a discussion on cryoablation as a local option, management of side effects like mucositis and fatigue, and the lack of definitive data on treatment duration and retreatment efficacy, emphasizing shared decision-making.
“It absolutely happens. I've got a collection of patients with large desmoid tumors that were potentially not in dangerous locations... came to see me with, you know, probably a 10 or 11 centimeter desmoid tumor in his flank. But he just swore up and down, you know, Doc, it just stopped hurting a month ago. And I feel like it's getting better.”
“We've done a fair bit of systemic therapy as primary treatment, but now this patient has been refractory to some local therapies. Also important to note in the DEFI trial, many of the patients that had prior local therapy like surgery, and so those patients seem to respond just as well as anyone else to niragasostat.”
“In that drug symptoms get better quickly. It's probably true for other GSIs. It's probably true for other therapies that are very active in this disease, but we've seen it demonstrated with naira.”
Host
Guest
Desmoid Tumor
other
Dr. Ravin Ratan
person
Niragacostat
product
MRI
other
Dr. Neil Love
person
Ovarian Suppression
other
MD Anderson Cancer Center
organization
Cryoablation
other
CT Scan
other
Serafinib
product
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