Cystic fibrosis — a progressive, genetic disease that affects the lungs, pancreas, and other organs — will no longer automatically qualify for a wish from the Make-A-Wish Foundation starting in January 2024.
The nonprofit foundation, which grants wishes to children with critical illnesses, made the decision because there have been so many major improvements in treatments for the condition. In the past, children with cystic fibrosis often did not survive past their teens or 20s, but many are now living much longer.
Children with the disease will still be considered for the opportunity to become a princess, virtually travel through space with a real astronaut, or even receive a chicken coop mansion for their beloved birds, but it will be on a case-by-case basis, the foundation announced last week.
A similar change was made for people younger than 18 living with HIV/AIDS.
Kids with cystic fibrosis will be eligible for a wish if their condition is “accompanied by additional complications or factors that make the current situation critical,” which is in line with the foundation’s mission of serving children with any progressive, degenerative, or malignant condition that puts their lives in jeopardy.
“This decision was not made lightly, and we understand it may result in some frustration and disappointment,” the Make-A-Wish Foundation said in an update on its website. “As with all wish referrals, we will carefully consider any CF request that a family member, legal guardian, medical professional, or potential wish child believes meets our guidelines.”
Dr. Bruce Marshall, chief medical officer of the Cystic Fibrosis Foundation, told BuzzFeed News that Make-A-Wish worked with CFF to appropriately update its criteria “so it reflects the nuances of living with cystic fibrosis, and to be sure that meaningful decisions are made based on the individual living with the disease and not the disease itself.”
Overall, Marshall said he’s thrilled about what this change ultimately means for the nearly 40,000 children and adults living with cystic fibrosis in the US.
“Cystic fibrosis is an incredibly individualized disease, and not everyone living with CF benefits from the transformative treatments that currently exist,” Marshall said. “The decision made by the Make-a-Wish Foundation to update its criteria is a reflection of the tremendous story that is cystic fibrosis.”
Because of improved medical treatments and care, people born with cystic fibrosis today are expected to live 15 years longer than those born a decade ago, with more than half expected to live 53 years compared with 38 previously. Those born in the late 1990s were only expected to live into their early 30s. Back in 1955, children with cystic fibrosis rarely reached adulthood.
Now, 60% of people with the disease are adults who are capable of going to college, pursuing careers, and starting families. Advances in care have turned life’s simple pleasures into major wins too, such as cystic fibrosis patients having enough energy to blow up a balloon and keep up with their kids on a soccer field.
The disease is complex and affects everyone in different ways and to varying degrees. It’s caused by mutations in a specific gene that lead to a buildup of thick and sticky mucus in various organs. Mucus in the lungs clogs the airways and traps germs, leading to infections and difficulty breathing; mucus in the pancreas prevents the body from absorbing nutrients from food, resulting in malnutrition and poor growth; and mucus in the liver can cause liver disease.
Common symptoms of cystic fibrosis include persistent coughing, frequent lung infections such as pneumonia and bronchitis, wheezing, greasy or bulky stools, chronic sinus infections, enlarged fingertips and toes, rectal prolapse (when part of the large intestine slips outside the anus), salty-tasting skin, and difficulty with bowel movements.
Most people are diagnosed by age 2, but some are diagnosed as adults. Cystic fibrosis is the most common, life-threatening genetic condition in the US.
Fortunately, new treatments and expansions in eligibility for therapies have resulted in immense improvements in patients’ quality of life.
In the last two years, there’s been a substantial decrease in worsening lung symptoms in people with cystic fibrosis. In 2019, exacerbations were reported in 41% of adults and 22% of children. In 2021, those percentages dropped to 14% and 10%, respectively.
Experts say the pandemic increased awareness of infection prevention and the use of Trikafta, the brand name for a combination of three medications known as “cystic fibrosis transmembrane conductance regulator modulator therapies.” They work by correcting malfunctions in a specific protein caused by gene mutations.
There are four different CFTR modulators: Trikafta (elexacaftor/tezacaftor/ivacaftor), Symdeko (tezacaftor/ivacaftor), Orkambi (lumacaftor/ivacaftor), and Kalydeco (ivacaftor).
Lung transplants have also decreased dramatically since 2016, when 271 people with cystic fibrosis were reported to receive one. In 2021, only 52 people had the procedure done.
Other therapies available for those with cystic fibrosis include clearing airways to loosen and remove thick mucus from the lungs, inhaling medicines to open the airways and fight infections, and taking pancreatic enzyme supplements to improve nutrient absorption.
Many of these treatments require out-of-pocket costs, however. Data show that 41% of all people with cystic fibrosis and 52% of children under age 10 are on Medicaid, the government-funded health insurance for people with limited income and resources.