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Theodore G. Liou, MD; Frederick R. Adler, PhD; Barbara C. Cahill, MD; Stacey C. FitzSimmons, PhD; David Huang, DPH; Jonathan R. Hibbs, MD; Bruce C. Marshall, MD

JAMA. 2001;286:2683-2689.

Context  Patients with cystic fibrosis (CF) are the second largest group of lung transplant recipients in the United States. The survival effect of transplantation on a general CF population has not previously been measured.

Objective  To determine the impact of bilateral lung transplantation on survival in patients with CF.

Design, Setting, and Patients  Retrospective observational cohort study of 11 630 CF patients who did not undergo lung transplantation (controls) and 468 transplant recipients with CF from 115 CF centers in the United States, 1992-1998. Patients were stratified into 5 groups based on a 5-year survival prediction model (survival group 1: <30%; survival group 2: 30 to <50%; survival groups 3-5: 50 to <100%.)

Main Outcome Measure  Five-year survival from date of transplantation in 1992-1997 in the transplant group and from January 1, 1993, in the control group.

Results  Lung transplantation increased 5-year survival of CF patients in survival group 1. Survival group 2 had equivocal survival effects, and groups 3-5 had negative survival effects from transplantation. From 1994-1997, there was a mean annual prevalence of 238 patients in survival group 1 and mean annual incidence of 154 patients entering the group, approximately 1.5 times the number of lung transplantations performed each year in CF patients (mean, 104). Use of the criterion of forced expiratory volume in 1 second of less than 30% resulted in an equivocal survival benefit and identified 1458 potential candidates for transplantation in 1993.

Conclusions  Cystic fibrosis patients in group 1 have improved 5-year survival after lung transplantation. The majority of patients with CF have equivocal or negative survival effects from the procedure. Selection of patients with CF for transplantation based on group 1 survival predictions maximizes survival benefits to individuals and may reduce the demand for scarce donor organs.

Author Affiliations: Division of Respiratory, Critical Care and Occupational Pulmonary Medicine (Drs Liou, Cahill, and Marshall), Departments of Mathematics and Biology (Dr Adler), Lung Transplantation Program (Dr Cahill), and Intermountain Cystic Fibrosis Center (Drs Liou and Marshall), University of Utah, Salt Lake City; Medical Department, Cystic Fibrosis Foundation, Bethesda, Md (Dr FitzSimmons); Salt Lake Veterans Affairs Medical Center, Salt Lake City, Utah (Dr Marshall); School of Public Health, University of California, Los Angeles (Dr Huang); and the New York State Department of Health, Albany (Dr Hibbs). Dr FitzSimmons is now with FitzSimmons and Associates.

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