Extremity Sarcoma Trial Utilizes Patient Willingness Outcomes to Gauge Accrual

R. Lor Randall, MD

Most patients with extremity sarcoma are willing to participate in a randomized clinical trial exploring various postoperative cancer surveillance regimens, according to a results of a cross-sectional patient survey published in BMJ Open.

In the prospective, cross-sectional patient survey, investigators sought to assess their patients’ perspectives on their willingness to participate in the SAFETY (NCT03944798) trial, which would randomize patients to 1 of 4 different surveillance regimens following surgery for their extremity sarcoma. Patients, who were from outpatient sarcoma clinics in Canada, the United States, and Spain between May 2017 and April 2020, were also surveyed on the burden of cancer care, factors that impact their comfort with randomization, and the significance of cancer research.

Of the 130 surveys that were completed and obtained, 85.5% of patients responded that they would agree to participate in this study. Of those who chose not to participate, their reason was that it was too much of a burden for them at a time when they are feeling overwhelmed. Overall, most patients agreed that oncology research will assist investigators in improving their understanding of and treating patients with cancer.

In an interview with OncLive, R. Lor Randall, MD, the David Linn Endowed Chair for Orthopedic Surgery, as well as professor and chair of the Department of Orthopedic Surgery at University of California Davis Comprehensive Cancer Center, and an author on the published survey, discussed the results of this survey focused on patient willingness for clinical trial enrollment in extremity sarcoma.

OncLive: What was the purpose of this survey in extremity sarcoma?

Randall: This is a study that we just published in BMJ Open. It is interesting; we're doing a surveillance study called SAFETY where patients who get extremity sarcoma get randomized after their treatment to chest radiograph versus chest CT, [and then at an interval of every] 3 months versus every 6 months; it's a 2 x 2 design.

We say, "chest imaging," and some people think that should be a chest CT, and some people think that should be a chest radiograph. Frankly, we don't know if it really makes a difference in terms of outcomes or just detection up front.

Therefore, Michelle Ghert, MD, and the team of McMaster University put this [survey] together for this surveillance study. The question became: would patients be willing to enroll [in this study]? The intuition tells us that patients would say, "Well, if I get a chest CT, I'm more likely to find that disease earlier, and therefore maybe I'll be cured sooner. So, why would I want to be randomized to just getting a chest radiograph?"

When [thought leaders from around the world] started putting this trial together, people said, "Well, maybe patients won't enroll." So, we said, "All right, let's send that first step—let's do a survey and see." All of us at the primary opening sites put together a survey. Between May 2017 and April 2020, we surveyed a bunch of patients who are getting extremity sarcoma treatment, who were going to go into surveillance. We asked, "Would you mind answering some questions about whether you would be willing to enroll in a study?"

We found that 85.5% of patients said they would agree to do this kind of trial. That was it; we needed to convince the funding sources that the patients would be willing to do it. We just published this willingness study in the literature and now the actual study is open, but the actual study wouldn't have been open if we hadn't been able to prove that patients were willing to enroll.

Could this be an approach that could cascade into multiple cancer types before opening up a clinical trial?

That's exactly why I think it's exciting. In many cancer trials, there is backbone therapy, right? Patients are going to get the standard therapy, or they're going to get standard therapy plus a new agent. Less often, there are placebo trials, right? This is a good way for trials with either a placebo or non-placebo to see if you will get the accrual. In academic centers and sarcoma, this is particularly relevant because all of us want to have all the latest trials available to us.

However, they are expensive to open. Then you get a few patients, and then some patients don't want to enroll in the trial. It becomes a real problem. Therefore, if you do things like these willingness surveys, I think it could really be a Litmus test [to determine] the success of the trial.

How was the survey distributed and organized?

It was a combination of either electronic [or paper if] some people didn't have access to [technology]. They were also given the opportunity [to take the survey] in clinic.

What are some of the goals of the SAFETY trial, now that it is accruing? What do you really hope to answer and address with this trial?

We want to be able to find out if chest radiographs will actually be sufficient to pick up burden of disease; it's not an outcomes study, but we'll be able to see if there is more detection with chest radiographs, as well as frequency of the imaging. The idea is also to do a cost-effective analysis in that, if chest radiographs do what we want them to do, then we don't need to be putting patients through chest CT. Then, perhaps we can do [the imaging] less frequently, as well.

Do you think the high percentage rate would still be reported if this survey was administered 5 or 10 years ago?

What was really great to have documented was that we also asked them, "Why would you [enroll in the trial]?" The answer, almost uniformly, was, "to help patients of tomorrow."

That is really inspiring, right?

Hopefully none of these patients will get chest metastasis; however, for the few that do, their concern is, "Would it have helped if it had been picked up earlier?" The answer is, "I'm willing to take that risk because we'll get an answer and help the patients of tomorrow." We need more of that—all of us being a little bit more focused on the big picture.

I do think it is an exciting topic, because this was not hard to do. It may become that people, especially foundations that are going to fund trials, may ask for people to do a willingness survey first. On behalf of the collaboration, which is mostly being driven by Michelle Ghert, MD, of McMaster University, we really hope that more sarcoma centers will be opening the trial and enrolling patients. It's sort of in the uptick right now.

Reference

1. Schneider P, Giglio V, Ghanem D, et al. Willingness of patients with sarcoma to participate in cancer surveillance research: a cross-sectional patient survey. BMJ Open. 2021;11(2):e042742.doi:10.1136/bmjopen-2020-042742