DOI: 10.1097/01.SA.0000307934.03986.8e
,
Issn Print: 0039-6206
Publication Date: 2008/08/01
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Excerpt
Theodore G. Liou,*§∥ Frederick R. Adler,†‡ David R. Cox,# and Barbara C. Cahill*¶
(N Engl J Med, 357:2143-2152, 2007)
Departments of *Internal Medicine, †Mathematics, ‡Biology, and §Pediatrics, and the ∥Intermountain Cystic Fibrosis Center; and the ¶Lung Transplant Program, University of Utah, Salt Lake City, UT; and #Nuffield College, Oxford, United Kingdom
End-stage lung disease causes about 80% of all deaths among patients with cystic fibrosis (CF) at a median age of about 25 years. In children, CF is the most frequent indication for lung transplantation, an aggressive and costly therapy, with an uncertain effect on the child's quality of life.
Data from the Cystic Fibrosis Foundation Patient Registry and the Organ Procurement and Transplantation Network were used to identify children with CF who were on a waiting list for lung transplantation from 1992 through 2002. Proportional-hazards methods with transplantation as a covariate that changed at the time of transplantation were used to estimate how the procedure altered the risk of death.
A total of 53,073 children with CF from the 2 registries were reviewed and from these, after exclusion of patients for various reasons, 514 patients (85%) of all children with CF remained listed for transplantation during the study period. The median survival for those who died before transplantation was 223 days; median time to transplantation for the 248 who underwent the procedure was 427days; and the median survival after transplantation was 1037 days. A total of 141 patients on the waiting list died, with 91% of the deaths caused by respiratory failure. Of the 120 patients who died after undergoing transplantation, 60% of deaths were the result of complications associated with transplantation, mostly allograft rejection; 29% were owing to respiratory failure and 11% were ascribed to other causes. Using proportional-hazards modeling, 4 variables other than transplantation were associated with changes in survival. Older age at study entry was associated with improved survival before transplantation but decreased survival postoperatively. Diabetes before study entry reduced survival before transplantation but had no effect on survival postoperatively. Infection with Staphylococcus aureus was associated with improved survival before transplantation but with greatly reduced survival afterward. Burkholderia cepacia infection decreased survival whether or not the patient underwent transplantation. Examination of the effects of lung function indicated that poorer functional status was associated with decreased survival among those for whom data were available, but it did not have an interaction with transplantation. Using hazard factors according to age, diabetes status, and S. aureus infection at the time of placement on the waiting list for each of the 514 children showed 4 categories: significant estimated benefit (5 patients), significant risk of harm (315 patients), insignificant benefit (76 patients), and insignificant risk of harm (118 patients). This distribution was similar for the 248 patients who received lung transplants. Quality-of-life issues were uncertain because, after transplantation, the number of days of hospitalization decreased, but complications increased.
Improved survival was estimated for only 5 of the 514 patients on the waiting list for lung transplantation, thus indicating that prolonging life by lung transplantation should not be anticipated for children with CF. An improvement in quality of life can no longer be assumed and further study through a prospective trial is needed to show the effect of lung transplantation on survival.
