Doctors can be clearer in explaining treatment decisions: Study

Patients, caregivers noted concerns, expectations for CFTR modulators

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Doctors should take care not to oversimplify when talking to patients about treatment decisions for chronic illnesses, according to patients, caregivers, and physicians interviewed for a study focusing on CFTR modulator therapy for cystic fibrosis (CF).

It’s also important to have “compassionate conversations” with patients who aren’t eligible for potentially life-changing treatments, researchers said.

The study, “Exploring perceptions of and decision-making about CFTR modulators,” was published in the journal Pediatric Pulmonology.

Over the last decade, some 90% of CF patients have become eligible for CFTR modulator therapies — the first CF treatments that directly target the disease’s underlying cause. Four of the medications are approved in the U.S.: Kalydeco, Orkambi, Symdeko, and Trikafta, all of which are sold by Vertex Pharmaceuticals.

Accumulating evidence demonstrates the potentially profound benefits these medications have for CF patients in terms of lung function, life quality, and survival. Particularly substantial benefits are expected from the most recently approved therapy, Trikafta, a combination of three different modulators.

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‘Few specific guidelines’ on how to approach treatment decisions

The growing availability of these medications points to a need for “thoughtful and comprehensive” discussions and “shared decision-making,” the researchers wrote.

“CF treatment has focused on CFTR modulators, but few specific guidelines for CF care team members exist on how to provide education and support to patients regarding initiation of these new therapies,” the team noted.

The researchers conducted semi-structured interviews with nine adult CF patients and 11 caregivers for pediatric patients in CF programs at the University of North Carolina. Eight clinicians involved in the programs also were interviewed.

All but one of the adult patients were taking CFTR modulators; the one patient wasn’t eligible for them.  Among the pediatric patients, six used CFTR modulators, four were ineligible, and one was eligible but not taking the therapy.

Caregivers and clinicians were generally enthusiastic about the availability of CFTR modulators and their possible benefits. Adult patients “were more measured,” with some skepticism mixed with hope, the researchers said.

Patients and caregivers said they used multiple sources of information when making decisions about starting CFTR modulators. But many said the decision to use them wasn’t difficult, given the substantial clinical benefits.

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Avoiding overly optimistic expectations

Clinicians said their discussions with patients about whether to use Trikafta, a highly effective modulator, were simple. One clinician said it was “kind of a no-brainer to try it and see what happens.”

Respondents said they understood the side effects of CFTR modulators and generally found them acceptable, though “both people living with CF and CF clinicians express[ed] concerns about potential immediate and long-term side effects,” such as weight gain, the researchers said.

The physicians also reported concerns that patients would have heightened expectations about the medications that could not be met. “I’m very worried about people being built up that this is a cure… then not tolerate it,” one said.

On the other hand, the researchers wrote, “it is important that clinicians not let their own enthusiasm simplify conversations surrounding potential … side effects of modulators, [or] the possibility that patients may have a less robust response to the medication than they hope to have.”

When clinicians were asked about non-clinical effects of CFTR modulators, such as finances, education, and family planning, “several respondents seemed surprised by the question and struggled with responses,” the researchers wrote.

Patients and caregivers, in contrast, described the positive impacts of CFTR modulators on their lives, and expressed optimism about the future. “It’s given my child a life,” one caregiver said.

We hope that lessons from this study … might be generalized to help inform shared decision-making.

Some patients who had benefited from the medications said they wished they had planned better for everyday aspects of life and taken steps such as planning for retirement.

Survey respondents across all three groups said they appreciated having a multidisciplinary team involved in conversations about goals and treatment decision-making.

Those who were found not eligible for modulator therapies expressed a range of emotions, including devastation about their own lack of access and gratitude that the medications could help others.

When treatment-eligible patients and caregivers were asked to imagine a scenario where they or their child were not eligible, most expressed support and empathy. While some clinicians reflected similar distress, others “seemed resigned or neutral,” according to the authors.

“We hope that lessons from this study and others like it might be generalized to help inform shared decision‐making for future ‘life‐changing’ therapies for CF and other serious chronic illnesses,” the researchers wrote.