What's New

Selection Criteria Expand for Cystic Fibrosis Lung Transplant

An image of the lungs of a child with cystic fibrosis (CF). In NewYork-Presbyterian Hospital's newly expanded lung transplant program, there will be no age limit, and children with CF will be considered for transplantation as early as necessary.
Photo courtesy of the Centers for Disease Control and Prevention.

NewYork-Presbyterian Hospital has taken a leadership role in perfecting techniques and patient selection criteria for lung transplantation in patients with cystic fibrosis (CF). As a result, the Hospital is home to the largest CF lung transplant program in New York City. Leveraging this experience, the Hospital has now expanded the program to include patients under the age of 18 years. Although current management strategies reasonably control the disease into adulthood in most pediatric patients, lung transplantation may offer extended survival in pediatric patients with advancing disease who have severe and irreversible airflow obstruction.

"Obviously, the goal is to delay transplant as long as possible, but it is important to have the option of transplantation for any individual with advanced CF," noted Selim Arcasoy, MD, who worked as a co-author of a recent update of the international guidelines for selection of lung transplant candidates (J Heart Lung Transplant 2006;25:745-755). Dr. Arcasoy added that, as part of the expanded program, there will be no age limit, and children will be considered for transplantation as early as necessary.

The indications for transplantation in patients under the age of 18 with CF are the same as they are in older individuals, according to Dr. Arcasoy. Pediatric patients who are considered for transplant typically have irreversible disease that has rendered them dependent on supplemental oxygen. Other signs of advancing disease, such as frequent exacerbations, high blood levels of carbon dioxide, and pulmonary hypertension, are also usually present. CF is the reason for transplant in approximately 20% of the lung transplants performed at NewYork-Presbyterian/Columbia University Medical Center. Of the 39 transplants performed in CF patients since 2001, 2 have been in individuals under the age of 18, and more are anticipated. Outcomes so far have been similar in children and adults.

Selim M. Arcasoy, MD
Joshua R. Sonett, MD

"Our program has generated very good outcomes data in follow-up that now exceeds 5 years in CF patients," Dr. Arcasoy reported. "The 1-year survival for lung transplants overall has been 96%, and the 3-year survival is 90%. In contrast, the national data from UNOS [United Network for Organ Sharing] is currently reporting 1-year survival of 83% and 3-year survival of 67%." A factor likely contributing to the high rates of survival in the NewYork-Presbyterian/Columbia program is that Dr. Arcasoy and his colleague Joshua Sonett, MD, are involved in a variety of research projects that are moving the field forward and inform their clinical practices. Dr. Sonett's skills in lung transplant surgery have won him an international reputation, and his name reached the mainstream media when he operated on former United States President Bill Clinton for a lung complication following his coronary bypass surgery. He has long been involved in a variety of innovative work, not just in lung transplantation, but also in a broad array of pulmonary diseases. In CF, for example, he has been involved in placement of stents in the lung to treat obstructed pulmonary function in pediatric patients.

Additionally, the transplant program has been very active in studies of the pathophysiology of graft rejection, including early detection of genetic signals of an impending rejection episode. Their efforts to provide sufficient immunosuppression to suppress rejection episodes while minimizing the complications of immunosuppressive agents are critical to extending graft and patient survival.

Because of the challenges of transplantation for patients, Dr. Arcasoy emphasized the importance of early referral of patients with advancing CF, whether they are adults or children. He suggested that transplant might be most effective when it is considered and introduced in the context of ongoing care.

"It is best if we can consult with patients long before lung transplantation is necessary," he said. "It not only provides time to perform tests on a timely basis, but also to prepare the patient both physically and psychologically for transplant if it becomes necessary. Patient education is an important component of a successful transplant."

The allocation of donor grafts was modified in 2005. Previously, patients moved up the list in the order that they were inscribed, but now patients are scored according to severity of their lung disease, so that those with the highest scores move to the head of the line. Although recipients and donors are matched by age so that those patients with CF younger than age 12, 12 to 17 years old, and 18 years or older receive organs from matched age groups, the new allocation system has permitted a greater number of very sick CF patients to receive an organ. However, patients younger than age 12 are still prioritized based on waiting time as in the old allocation system for adults, and this has to be considered during listing decisions for transplantation with children.

"Fortunately, there is a limited need for lung transplantation in pediatric CF patients due to the progress in medical management," Dr. Arcasoy noted. "However, there are cases where this is the best option for extending life, and we have been encouraged that outcomes in younger patients appear to be as good as those observed in adults."