More than 30,000 Americans have been diagnosed with cystic fibrosis (CF), a disease that in the 1960s and 1970s ended its victims' lives in their teens. Today, required newborn screening and advances in personalized medicine are changing the CF story.
Although doctors diagnose about 1,000 cases each year, CF is now considered a treatable disease, with patients living into their 50s. In fact, the cystic fibrosis community is quite confident that with the new therapies available and those still to come, the median survival age will continue to rise rapidly.
A common uncommon disease
Cystic fibrosis is an inherited disease. Each biological parent must contribute a recessive, mutated gene to their child. Unfortunately, most parents of children with CF – about 1 in every 31 Americans – are "silent carriers" of the mutated gene. These carriers are unaware of the mutated gene because they have never had any CF symptoms.
Today, screening of carriers is available to help inform family-planning decisions. Plus, as of 2010, all 50 states are required to conduct newborn screening for cystic fibrosis. More than 75% of those with CF are diagnosed by age 2.
Cystic fibrosis impairs the transfer of electrolytes – chloride, salt and bicarbonate (a pH regulator) – in various organs, such as the pancreas and liver. Pulmonary manifestations of the disease are almost universal. Without a lung transplant, patients with CF typically die from lung disease.
An accredited pediatric, adult CF center
The University of Kansas Health System and Children's Mercy Kansas City are the only accredited pediatric cystic fibrosis centers in the Kansas City metropolitan area. Our CF teams meet face to face to initiate and streamline the transition of care from childhood to adulthood.
In addition, The University of Kansas Health System is the only accredited adult CF center in the Kansas City metropolitan area. To become accredited, we underwent the Cystic Fibrosis Foundation's rigorous accreditation program. Every 5 years, a select group of physicians from top-performing centers reviews our processes, vets our unique diagnostic testing and evaluates the program.
In 2017, we were 1 of only 15 hospitals nationwide to receive the Cystic Fibrosis Foundation's Quality Care Award. Recipients chosen by the CF Foundation excel at meeting quality improvement and performance standards. The foundation believes "this elevated level of specialized care has led to longer, more productive lives for people living with CF."
An active clinical research center
The University of Kansas Health System also holds distinction as a Therapeutic Development Center, as designated by the CF Foundation. Participating centers conduct and advance CF research to help improve treatment options.
The process to make this elite list is highly competitive. Each center must be a Cystic Fibrosis Foundation-accredited care center with a solid research support system, qualified personnel and demonstrated expertise in conducting CF clinical research.
Multispecialty care team
Anytime a physician suspects CF, the patient should receive an evaluation by a specialist. Cystic fibrosis is a disease best managed by a multidisciplinary team that understands the disease's complexities and has experience in treating it.
Adult patients referred to our clinic meet with a multidisciplinary team that includes a CF physician, a nurse coordinator, a social worker, a respiratory therapist, a dietitian and a pharmacist. Our cystic fibrosis team also works closely with an endocrinologist who specializes in CF endocrine problems, such as pancreatic disease and diabetes. (Diabetes develops in up to 40% of adult patients because of the impact the disease has on the pancreas.)
Each patient's appointment, approximately 1-2 hours, culminates with the team discussing the patient's current situation and areas of concern and recommending a 3-month treatment plan.
Our range of services and expertise makes a significant difference for our CF patients. They have a better experience, effective treatment options and are in the best position to receive optimal care at an accredited CF center.
Breakthroughs in treatment
CF is one of the first rare diseases to have FDA-approved personalized medicines, meaning the medicines are specific for each patient's particular gene mutation. The customized medicines restore electrolyte function.
The CF community believes broad distribution of these life-changing drugs to all CF patients, regardless of gene type, will be possible within the next 10 years. Even better, each generation of medication is becoming more potent and effective.
Our overall goal is to grow old with our adult patients. By providing outstanding, collaborative care, educating others about cystic fibrosis management and looking for the treatments of tomorrow by participating in scientific research today, we will reach that goal.
To consult with a physician or refer a patient, call 913-588-5862 or 877-588-5862.