Mental Health Challenges Faced by Children with Cystic Fibrosis Are the Focus of a Major, Multi-Site Study Led by UB

BUFFALO, N.Y. — A University at Buffalo psychiatrist who has played a critical role in getting mental health screening and treatment integrated into routine care for adults and adolescents with cystic fibrosis (CF) has been awarded $3 million from the Cystic Fibrosis Foundation to focus on the mental health of children with the disease.

Led by UB and launched this summer, the new study is an outgrowth of The International Depression Epidemiological Study (TIDES), which began in 2014 and was the largest study of mental health in adolescents and adults with CF. As a result of TIDES, annual screening for depression and anxiety is now part of routine CF care for nearly 90% of adults and adolescents with CF in the U.S.

“That’s the goal of this new study, which we are calling TIDES 2.0,” says Beth A. Smith, MD, principal investigator, interim chair of psychiatry in the Jacobs School of Medicine and Biomedical Sciences at UB, medical director of the Children’s Psychiatry Clinic in Oishei Children’s Hospital and founding chair of the Cystic Fibrosis Foundation’s Mental Health Advisory Committee. “It will allow us to take what we have done nationally and internationally for adolescents and adults with CF and do it for children from 18 months up to 11 years old.”

The study will evaluate the national prevalence of mental health concerns in children with CF under 12 years of age. It will identify the best ways to screen for mental health issues in these children, and it will characterize those issues most often seen in children being treated with the new therapies that have essentially revolutionized CF. It will identify potential risk factors and likely lead to the adoption of new international guidelines on mental health screening for children with CF, just as the original TIDES did.

Often diagnosed in infants, cystic fibrosis is a rare, chronic disease without a cure. A progressive, genetic disease, it affects the whole body, including the ability to breathe and digest food. Managing the disease is complex, and people with CF can spend several hours a day doing treatments to clear airways of mucus and treat other complications; some individuals eventually require a lung transplant.

Dramatic improvements

Fortunately, the prognosis for people with CF has improved dramatically in the past 20 years. That’s due to improvements in multidisciplinary, specialized care, as well as the development of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, which precisely correct the malfunctioning protein that is made by the CFTR gene. UB and Oishei researchers have been at the forefront of this research; in 2009 they enrolled the first U.S. patient in a multisite clinical trial of these drugs.

Whereas in the 1950s, when children born with CF rarely lived long enough to attend elementary school, today, many people with CF grow up, marry, have families and live into retirement.

The impact these pharmaceutical advances have had cannot be overstated, but the story of the disease has grown more complicated.

Previous studies at UB and elsewhere have shown that depression in people with CF is linked with worse health outcomes, including decreased lung function, lower body mass index, increased exacerbations and hospitalizations, and increased mortality.

In a small study Smith and her colleagues published in 2010, they found that children with CF as young as 7 years old start to have depressive symptoms. “Kids become sad and irritable, they start to have a lot of negative thoughts about their lives, they notice that they’re different from other kids,” Smith says.

Googling one’s disease for example, only intensifies those feelings. “Even though life expectancy has improved so much, there are a lot of comorbidities now that people are living longer,” says Smith. “They have much higher rates of colorectal cancer, they get CF-related diabetes because their pancreas gets bogged down, and boys find out they might be sterile. There’s just a lot that gets unfolded over time for these young kids.”

Parents, of course, are also deeply affected and their concerns can, in turn, affect the child. So, Smith says, it is critical to identify and address mental health issues early on.

Changing the trajectory

“Looking back on when I first began working with patients with CF in the early 2000s, I think if mental health screening had been more prominent and we had been addressing it in a different way, maybe we would have been able to change some of the patients’ trajectories,” she says.

In a previous study, Smith and colleagues found that people with CF with chronic depressive symptoms who had not received treatment were more likely to die than those who had had their depression addressed. “It’s not that the other patients didn’t have depression,” she says. “They did, but it was addressed.”

Changing those trajectories is part of Smith’s mission with the new grant focused on children. “Can we change these trajectories if we treat the depression and treat it well and help with developing coping skills and disease self-management? Can we change the outcome so a positive depressive screening in someone with CF isn’t associated with a doubling of the risk of mortality?”

Part of the care team

One key to doing that requires that the entire CF care team be committed to including mental health screening as part of standard patient care. Danielle M. Goetz, MD, director of the Cystic Fibrosis Center of Western New York at Oishei Children’s Hospital, clinical associate professor of pediatrics and the leading CF pulmonologist as well as a co-PI on the new grant, plays a critical role.

“The pulmonologists are so important,” says Smith. “They run the clinics; they’re the boots on the ground. As a mental health clinician, I can say that mental health screening is important, but it won’t happen unless the pulmonologists understand that this is important to total CF care. At our clinic, Dr. Goetz has been foundational to making sure this study works.”

TIDES 2.0 will enroll patients at the Cystic Fibrosis Center of Western New York at Oishei Hospital in Buffalo and at 15 other sites nationwide including Massachusetts General Hospital/Harvard Medical School and the Johns Hopkins University School of Medicine.

People interested in enrolling in the study should contact Smith at balucas@buffalo.edu or research coordinator Julianne Hergenroder at jhergenroder@upa.chob.