Lung transplant referrals for individuals with cystic fibrosis: A pediatric perspective on the cystic fibrosis foundation consensus guidelines

Abstract

Lung transplant referral guidelines for individuals with cystic fibrosis (CF) were published recently. Most of the recommendations focus on the specific indications and barriers to transplantation in adults with CF. Although the number of children with CF and end-stage lung disease continues to decrease, the specific issues related to pediatric patients merit further elucidation. We address each recommendation from the recent publication with a pediatric perspective. Furthermore, we note some significant differences between the practice and policy related to lung transplantation between Canada and the United States.

Elexacaftor/tezacaftor/ivacaftor in children aged 6–11 years with cystic fibrosis, at least one F508DEL allele, and advanced lung disease: A 24‐week observational study

“…However, pediatric lung transplantations are often unavailable because of the scarcity of specialized centers, and the patients are at high risk of dying while they are still on the waiting list. 4 It has been shown that ivacaftor, lumacaftor, and tezacaftor (used singly or in combination) are safe and efficacious in children aged 6-11 years. 5 Furthermore, the results of a 24-week, Phase-III open-label trial of ETI have shown that the treatment was as safe and effective in CF patients aged 6-11 years with at least one F508del allele as in adults and adolescents.…”

“…Such cases are extremely difficult to manage and, when conventional treatment fails, the only alternative is lung transplantation. However, pediatric lung transplantations are often unavailable because of the scarcity of specialized centers, and the patients are at high risk of dying while they are still on the waiting list 4 …”

Cystic fibrosis year in review 2020: Section 2 pulmonary disease, infections, and inflammation

“…The conclusion recognizes the ever‐evolving and improving outcomes in CF but reminds us that CF, at its core, remains a progressive disease. Many patients with ALD will be referred for lung transplant and a commentary examines the transplant practices for pediatric CF patients, including remarks on the transplantation guideline recommendations and a timeline and volumes for the eight current independent US pediatric transplant sites 35 …”