CF Patients Satisfied With Care, but Not Life Quality, US Survey Finds

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by Marta Figueiredo PhD |

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US survey | Cystic Fibrosis News Today | survey illustration

Most people with cystic fibrosis (CF) responding to a U.S.-based online survey were generally satisfied with their treatment plan and were not considering changing their medication. The survey was conducted by Cystic Fibrosis News Today.

Shortness of breath was the most commonly reported symptom among CF patients, as well as the one that had the greatest impact on their daily life, the survey showed.

Despite overall treatment satisfaction, nearly a third of survey respondents were dissatisfied with their quality of life, suggesting that more should be done in terms of symptom management and patient support/care.

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The online survey was conducted in collaboration with the Business Intelligence arm of BioNews, which publishes this website.

“BioNews is dedicated to improving the lives of patients living with disease,” Jacob Harney, PhD, operations manager for Business Intelligence, said in a statement to Cystic Fibrosis News Today.

“We continue to research the impacts of disease(s) on quality of life in the hopes that better understanding results in better treatments and therapies being available in the future,” he said.

Conducted from Feb. 16 through July 6, 2021, the survey sought to gain a greater understanding of the CF community, focusing on disease management, treatment plans and side effects, insurance, and patient quality of life.

Who answered the survey?

A total of 232 U.S. residents completed the survey, and more than a quarter of them — 80, or 27.3% — were people living with CF. The remaining respondents included family members or friends of CF patients (63.5%) — most (77.3%) of whom provided care to a person with CF — healthcare professionals (3.8%), and researchers studying CF (5.5%). 

Subsequent analyses focused on patients only. This group had a median age of 48.5 years, based on 59 responses, and had lived with a CF diagnosis for a median of 29 years, according to 71 responses.

A total of 77 patients answered survey questions about their gender, race and ethnicity, educational background, and annual income.

More than two-thirds (68.8%) identified as female, 29.9% as male, and one patient as non-binary or another gender. Most respondents (94.8%) were White, while one patient each identified as Latino/Hispanic, Black/African American, Native American/Indigenous, and mixed race.

Most held either a bachelor’s degree (27.3%) or a graduate degree (24.7%; master’s, doctorate, law, or medical degree). Another 20.8% had completed an associate’s or trade school degree, while 11.7% had a high school diploma or GED. The remaining 15.6% reported another education level.

More than a third of respondents (35.1%) earned up to $30,000 per year, 23.4% had an income of $30,001–$60,000, 16.9% of $60,001–$100,000, and 6.5% made more than $100,000.

Most (89%) of the 73 patients responding reported knowing one or both of their disease-causing mutations in the CFTR gene, with the most common being F508del (78.1%), according to 64 responses. This is consistent with information from the general CF patient population.

Notably, the second-most commonly reported mutation was R117H (6.3%), with mutations other than the 25 listed in the survey being reported by nearly one-third of respondents (32.8%). Also, 29.7% said they carried two copies of the same mutation.

The most common CF symptom — based on 69 patients who answered this question — was shortness of breath (55.1%), followed by frequent lung/sinus infections (50.7%), damaged airways (bronchiectasis; 49.3%), very salty-tasting skin (46.4%), and persistent coughing (40.6%).

Shortness of breath also was most commonly reported (26.6%) as having the greatest impact on daily activities, followed by diabetes (15.6%), persistent coughing (14.1%), frequent lung/sinus infections (10.9%), and damaged airways (9.4%).

“Shortness of breath and persistent coughing are commonly appreciated symptoms but diabetes, resulting in the persistent elevation in blood sugar, is a side effect associated with mucus buildup and scarring of pancreatic tissue,” Harney said.

When asked about health insurance, most of the 77 patients who answered reported having private insurance (57.1%) or Medicare (51.9%). More than a third were covered by Medicaid, while 11.7% used other types of health insurance. Two patients preferred not to disclose their health insurance.

How were patients being cared for?

Nearly all of the CF patients surveyed (66 of 71, or 93%) reported seeing a pulmonologist, and more than three in five (64.8%) were seeing a general or primary care doctor. Others reported being seen by a respiratory therapist (52.1%), an endocrinologist (43.7%), an ear/nose and throat doctor (38%), and a cardiologist (18.3%).

All patients were taking medications to treat CF, based on 67 responses, while 60 of 70 patients (85.7%) were on medications not related to the disease.

The most commonly used disease-targeting therapy — that is, medications that target CF’s underlying cause — was Trikafta (elexacaftor/tezacaftor/ivacaftor), taken by 53% of 66 respondents. This was followed by Kalydeco (ivacaftor; 6%) and Symdeko (tezacaftor/ivacaftor; 3%).

Many patients also used treatments to manage CF symptoms, with the most common being Pulmozyme (dornase alfa; 69.7%), hypertonic saline (47%) — both being mucus-thinning agents — Creon (pancrelipase; 45.4%), a type of pancreatic/digestive enzymes, and Zithromax (azithromycin; 42.4%), an antibiotic.

Notably, this information was consistent with that provided by relatives or friends of CF patients, showing Trikafta as the most common disease-targeting therapy (50.4%), and Pulmozyme as the most common non-disease-targeting medication (69%), according to 113 responses.

Patients were on the same treatment plan for a median of 25.5 years, based on the answers of 59 of them.

The most common side effects, as reported by 60 patients, were anxiety (53.3%), joint and muscle pain (43.3%), sinus congestion (41.6%), nasal congestion (38.3%), depression (35%), and bloating (35%). Joint and muscle pain, along with extreme tiredness, were the most bothersome, based on 56 responses.

Most patients — 55 of 65, or 84.6% — had stopped a medication previously. The most common were two antibiotics, Tobi (tobramycin; 64.6%) and Levoquin (levofloxacin; 41.7%), followed by Symdeko (27.1%), based on 48 respondents.

Nearly a fifth (18.8%) reported having previously stopped Orkambi (lumacaftor/ivacaftor), another disease-targeting therapy.

According to 47 patients, common reasons for stopping these treatments included side effects (29.8%) and lack of effectiveness (23.4%).

The most frequent side effects that 35 patients experienced while on prior treatments included chest discomfort (25.7%), bronchospasm — a sudden tightening in the walls of the airways (22.8%) — nausea/vomiting (20%), and bloody mucus (20%). The most bothersome were chest discomfort, bronchospasm, and hives.

Based on 74 responses, nine (12.2%) patients reported having undergone an organ transplant due to CF, all comprising both lungs.

How patients viewed their life quality and care?

Among the 77 patients who answered a question about their quality of life, more than half (55.9%) reported being satisfied. That included 29.9% of respondents who said they were “somewhat satisfied” with their quality of life and 26% who were “very satisfied.”

“The encouraging findings from the present survey include a greater percentage of CF patients (55.9%) being satisfied with their quality of life compared to those being unsatisfied (32.4%) and 81.8% report being satisfied with their treatment plan,” Harney said.

“Since 100% of patients report being on medications for their disease, the high satisfaction rate is encouraging for the CF community,” Harney added.

However, nearly a third of patients reported being “somewhat” (20.8%) to “very unsatisfied” (11.7%), and 11.7% were “neutral.”

Similarly, based on 76 responses, more than half (57.9%) expressed optimism about their future: 30.3% were “somewhat optimistic” and 27.6% were “very optimistic.” More than a quarter (26.3%) were “neutral,” and only 15.8% of patients reported being “somewhat pessimistic,” with none saying they were “very pessimistic.”

Also, more than 80% of patients were “somewhat” (33.3%) to “extremely satisfied” (48.5%) with their current treatment plan, according to 66 responses. The remaining were “somewhat dissatisfied” (4.5%) or “neither satisfied nor dissatisfied” (13.6%); none were “extremely dissatisfied.”

In line with these treatment satisfaction rates, only 10 (15.2%) of 66 respondents were considering switching their current medications. The most common reason for considering such a change were concerns about effectiveness (63.6%), according to 11 respondents.

More than three-quarters of patients also reported being satisfied with their health insurance provider: 40.3% of 72 patients were “extremely satisfied” and 37.5% were “somewhat satisfied.”  By contrast, fewer than two in 10 patients were unhappy with their insurance, with 8.3% responding “somewhat dissatisfied” and 5.6% “extremely dissatisfied.” The remaining 8.3% reported being neutral on their healthcare provider.

Notably, nearly 70% of the 61 patients who answered a question about clinical trials expressed interest in participating. Among them, 37.7% said they were “extremely likely” and 31.2% were “somewhat likely” to take part. Only 11.5% reported being unlikely to participate in a clinical trial, and 19.7% said they were “neither likely nor unlikely.”

Overall, the survey results highlight generally high rates of satisfaction with care and health insurance among CF patients, but also indicate that a relevant proportion of patients are dissatisfied with their quality of life.

These findings highlight areas of unmet needs and call for better care and support to improve the lives of these patients.