The Long Wait: Life, Fertility, and Uncertainty in Desmoid Tumor Care
In mid-December, Smart Patients invited Dr. Dale Shepard, a sarcoma specialist at the Cleveland Clinic and one of the nation’s leading investigators in desmoid tumor trials, for a three-day Ask the Expert conversation. The wide-ranging conversation covered hormones and surveillance, surgery and uncertainty—and how people make life decisions in the shadow of a rare, unpredictable tumor.
We started off with a young member’s question that many others would echo over the next several days. “I’ve been hard-pressed to find an OB/GYN or reproductive endocrinologist who can answer questions in the context of desmoids,” she wrote.
Would trying to preserve her fertility before starting treatment for a desmoid tumor make her disease worse? It was a question that captured a broader tension running through the entire discussion: how to plan a future when science offers probabilities, not certainties. How—and when—these questions are addressed often determines whether patients feel able to move forward with treatment, surveillance, or clinical trials.
For young women living with desmoid tumors, hormones and reproductive planning are not abstract concerns. Dr. Shepard was clear about what is known and what remains uncertain:
Desmoid tumors are 2–3 times more common in women.
Estrogen exposure—through pregnancy or estrogen-containing contraceptives—has been associated with tumor growth in some patients.
Progesterone’s role is far less clear.
But the questions went beyond biology. Our members wanted guidance that could help them make irreversible life decisions.
Would short-term, low-dose estrogen for IVF matter?
Could pregnancy trigger new tumors elsewhere?
Could surgical trauma—even an IVF needle—cause another tumor to form?
Dr. Shepard explained that short-term, low-dose estrogen exposure during IVF is unlikely to significantly affect tumor growth—guidance that helped shift the discussion from “Is this allowed?” to “How can this be managed safely?” He noted, "If there is growth, it can usually be managed afterward.” Sporadic desmoid tumors rarely appear in multiple locations. Surgical trauma can increase risk, but the range is broad and poorly defined - estimated to be anywhere from 25% to 75%, depending on context.
“A needle used for IVF would be unlikely to cause enough damage to stimulate growth of a desmoid tumor. ”
As members discussed therapy and its place within the arc of their lives, it was clear from this conversation that fertility preservation, family planning, career timing, and mental health are not “adjacent concerns.” They are central to treatment decisions, adherence, quality of life, and perceived treatment value.
The Unpredictability of Desmoid Tumors - and the Art of Waiting
Desmoid tumors are rare connective tissue tumors that do not metastasize but can behave aggressively, infiltrating nearby organs and causing pain, dysfunction, or obstruction. They are driven almost exclusively by two genetic pathways: CTNNB1 mutations (sporadic disease) and APC mutations associated with familial adenomatous polyposis (FAP). 1
Yet despite decades of study, one of the most important clinical shifts in recent years has been learning when not to treat. This shift—toward watching carefully rather than intervening immediately—has emerged gradually, shaped by both clinical evidence and patient experience.2
Dr. Shepard explained, “In many cases, active surveillance really is the best therapy.”
He cited observational data following roughly 100 patients with asymptomatic, extra-abdominal desmoid tumors over three years:
About 30% experienced spontaneous tumor shrinkage
About 30% remained stable
About 40% showed growth
For patients accustomed to the urgency that follows a cancer diagnosis, this approach can feel unsettling. But it reflects a growing consensus in the field: because desmoid tumors do not spread, stability without symptoms is often a meaningful success.
This theme surfaced repeatedly across the Smart Patients discussion, revealing how patients recalibrate expectations as they live with surveillance over months or years. Patients described tumors discovered incidentally, partially resected, then never seen again. Others spoke of years spent under MRI surveillance—living full lives while learning to tolerate uncertainty.
What’s Next for Desmoid Tumors?
Questions about what comes next—new drugs, clinical trials, and changing standards of care—emerged after patients felt grounded in the discussion. Today, nirogacestat is the only FDA-approved drug for desmoid tumors. Others are on the horizon. During the session, Dr. Shepard outlined how the treatment landscape is beginning to expand:
A second gamma secretase inhibitor with promising data
FOG-001, an investigational infusion therapy now in multicenter trials
Earlier-stage approaches that target TGF-beta and transducin beta-like protein 1
There are currently about 70 desmoid tumor clinical trials with roughly 20 actively recruiting worldwide. Yet the discussion made clear that access is uneven. Patients described barriers ranging from geography and strict eligibility criteria to the day-to-day burden of participation—despite understanding that trials can offer access to therapies years before approval.
What We Learned
Our Ask the Expert session on desmoid tumors answered questions and revealed patterns in how patients make decisions, where uncertainty slows action, and what support enables people to move forward with confidence. Several themes emerged that will continue to shape how Smart Patients and others can support the desmoid tumor community and engage more effectively.
1. Fertility and hormones are not side questions—they are decision drivers
For younger patients, uncertainty around fertility and hormone exposure directly affects when (or whether) they pursue treatment, enroll in trials, or delay care. Addressing these questions early helps reduce hesitation and supports more informed, timely decisions.
2. “Working” is defined by life impact, not just scans
Across the discussion, Smart Patients consistently weighed symptom relief, daily functioning, and mental health more heavily than radiographic tumor shrinkage. This aligns with growing recognition that patient-defined outcomes—pain, fatigue, ability to work or to plan a family—are central to perceived treatment value.
3. Active surveillance succeeds only when expectations are shared
While surveillance is often the preferred clinical approach, patients described needing clearer explanations, regular touchpoints, and reassurance to feel confident “waiting.” Structured communication—not just imaging schedules—emerged as essential to sustaining trust and adherence.
4. Trial participation depends on trust, timing, and fit
Patients understood the importance of clinical trials and expressed interest in new therapies, but participation hinged on personal readiness and practical barriers—geography, eligibility, fertility concerns, and day-to-day burden. Conversations that address these factors early help patients see trials as viable, not intimidating.
What makes conversations like this especially powerful is not that they happen once, but that they build over time. On Smart Patients, individual questions—about hormones, surveillance, fertility, or trials—accumulate into shared understanding. Patterns emerge. Misconceptions surface and get corrected. Confidence grows. This ongoing exchange allows important viewpoints to evolve alongside science, reflecting how real people adapt as evidence, options, and life circumstances change.
References
1Kasper B, et al. An update on the management of sporadic desmoid-type fibromatosis: a European Consensus Initiative between Sarcoma Patients EuroNet and European Organization for Research and Treatment of Cancer /Soft Tissue and Bone Sarcoma Group. Ann Oncol. 2017 Oct 1;28(10). https://pubmed.ncbi.nlm.nih.gov/28961825/
2Bonvalot S, et al. Spontaneous regression of primary abdominal wall desmoid tumors: more common than previously thought. Ann Surg Oncol. 2013 Dec;20(13). https://pubmed.ncbi.nlm.nih.gov/24052312/
This Ask the Expert conversation will remain available on Smart Patients as a living resource so members can return to it as new questions arise, treatments evolve, and personal priorities shift. As part of an ongoing dialogue within the desmoid tumor community, it contributes to a growing body of real-world patient-perspectives that helps patients feel more prepared, clinicians better informed, and future research more closely aligned with what truly matters to patients in daily life.