Skip to main content
PMC home page
Thorax logoLink to Thorax
. 1997 Apr;52(4):313–317. doi: 10.1136/thx.52.4.313

A prognostic model for the prediction of survival in cystic fibrosis

K M Hayllar, S G Williams, A E Wise, S Pouria, M Lombard, M E Hodson, D Westaby
PMCID: PMC1758530  PMID: 9196511

Abstract

BACKGROUND: The treatment for endstage cystic fibrosis is, where appropriate, double-lung, heart-lung or, occasionally, heart-lung-liver transplantation. Optimising the timing of transplantation depends upon an accurate prediction of survival, but while current criteria give some guidance to this, they are not based upon statistically derived prognostic models. METHODS: Data collected prospectively on 403 patients with cystic fibrosis, recruited between 1969 and 1987 (cohort A), were analysed by log rank and univariate Cox regression analysis to determine variables that accurately predict survival. The significant variables were then subject to time dependent multivariate Cox regression analysis to generate a prognostic model. The model was validated, within the study population, using split sample testing, and was subsequently validated in a further cohort of patients recruited since October 1988 (cohort B). RESULTS: One hundred and eighty eight (50.4%) of the study cohort died within the study period. Percentage predicted forced expiratory volume in one second (FEV1), percentage predicted forced vital capacity (FVC), short stature, high white cell count (WBC), and chronic liver disease (as evidenced by the presence of hepatomegaly) were negatively correlated with survival. These variables, when combined into a prognostic index, accurately predicted one year survival in the study population and in the cohort recruited since 1988. CONCLUSION: This prognostic index may prove valuable in predicting prognosis in other cohorts with cystic fibrosis and thereby improve the timing of transplantation. 




Full Text

The Full Text of this article is available as a PDF (132.5 KB).

Selected References

These references are in PubMed. This may not be the complete list of references from this article.

  1. Britton J. R. Effects of social class, sex, and region of residence on age at death from cystic fibrosis. BMJ. 1989 Feb 25;298(6672):483–487. doi: 10.1136/bmj.298.6672.483. [DOI] [PMC free article] [PubMed] [Google Scholar]
  2. Christensen E., Schlichting P., Andersen P. K., Fauerholdt L., Schou G., Pedersen B. V., Juhl E., Poulsen H., Tygstrup N. Updating prognosis and therapeutic effect evaluation in cirrhosis with Cox's multiple regression model for time-dependent variables. Scand J Gastroenterol. 1986 Mar;21(2):163–174. doi: 10.3109/00365528609034642. [DOI] [PubMed] [Google Scholar]
  3. Corey M., McLaughlin F. J., Williams M., Levison H. A comparison of survival, growth, and pulmonary function in patients with cystic fibrosis in Boston and Toronto. J Clin Epidemiol. 1988;41(6):583–591. doi: 10.1016/0895-4356(88)90063-7. [DOI] [PubMed] [Google Scholar]
  4. Elborn J. S., Cordon S. M., Western P. J., Macdonald I. A., Shale D. J. Tumour necrosis factor-alpha, resting energy expenditure and cachexia in cystic fibrosis. Clin Sci (Lond) 1993 Nov;85(5):563–568. doi: 10.1042/cs0850563. [DOI] [PubMed] [Google Scholar]
  5. Elborn J. S., Shale D. J., Britton J. R. Cystic fibrosis: current survival and population estimates to the year 2000. Thorax. 1991 Dec;46(12):881–885. doi: 10.1136/thx.46.12.881. [DOI] [PMC free article] [PubMed] [Google Scholar]
  6. Farrant J. M., Hayllar K. M., Wilkinson M. L., Karani J., Portmann B. C., Westaby D., Williams R. Natural history and prognostic variables in primary sclerosing cholangitis. Gastroenterology. 1991 Jun;100(6):1710–1717. doi: 10.1016/0016-5085(91)90673-9. [DOI] [PubMed] [Google Scholar]
  7. Gaskin K. J., Waters D. L., Howman-Giles R., de Silva M., Earl J. W., Martin H. C., Kan A. E., Brown J. M., Dorney S. F. Liver disease and common-bile-duct stenosis in cystic fibrosis. N Engl J Med. 1988 Feb 11;318(6):340–346. doi: 10.1056/NEJM198802113180602. [DOI] [PubMed] [Google Scholar]
  8. Henry R. L., Mellis C. M., Petrovic L. Mucoid Pseudomonas aeruginosa is a marker of poor survival in cystic fibrosis. Pediatr Pulmonol. 1992 Mar;12(3):158–161. doi: 10.1002/ppul.1950120306. [DOI] [PubMed] [Google Scholar]
  9. Huang N. N., Schidlow D. V., Szatrowski T. H., Palmer J., Laraya-Cuasay L. R., Yeung W., Hardy K., Quitell L., Fiel S. Clinical features, survival rate, and prognostic factors in young adults with cystic fibrosis. Am J Med. 1987 May;82(5):871–879. doi: 10.1016/0002-9343(87)90147-1. [DOI] [PubMed] [Google Scholar]
  10. Hudson I., Phelan P. D. Are sex, age at diagnosis, or mode of presentation prognostic factors for cystic fibrosis? Pediatr Pulmonol. 1987 Sep-Oct;3(5):288–297. doi: 10.1002/ppul.1950030503. [DOI] [PubMed] [Google Scholar]
  11. Kerem E., Reisman J., Corey M., Canny G. J., Levison H. Prediction of mortality in patients with cystic fibrosis. N Engl J Med. 1992 Apr 30;326(18):1187–1191. doi: 10.1056/NEJM199204303261804. [DOI] [PubMed] [Google Scholar]
  12. Levy L. D., Durie P. R., Pencharz P. B., Corey M. L. Effects of long-term nutritional rehabilitation on body composition and clinical status in malnourished children and adolescents with cystic fibrosis. J Pediatr. 1985 Aug;107(2):225–230. doi: 10.1016/s0022-3476(85)80130-x. [DOI] [PubMed] [Google Scholar]
  13. Madden B. P., Hodson M. E., Tsang V., Radley-Smith R., Khaghani A., Yacoub M. Y. Intermediate-term results of heart-lung transplantation for cystic fibrosis. Lancet. 1992 Jun 27;339(8809):1583–1587. doi: 10.1016/0140-6736(92)91842-v. [DOI] [PubMed] [Google Scholar]
  14. Nixon P. A., Orenstein D. M., Kelsey S. F., Doershuk C. F. The prognostic value of exercise testing in patients with cystic fibrosis. N Engl J Med. 1992 Dec 17;327(25):1785–1788. doi: 10.1056/NEJM199212173272504. [DOI] [PubMed] [Google Scholar]
  15. SPROUL A., HUANG N. GROWTH PATTERNS IN CHILDREN WITH CYSTIC FIBROSIS. J Pediatr. 1964 Nov;65:664–676. doi: 10.1016/s0022-3476(64)80151-7. [DOI] [PubMed] [Google Scholar]
  16. Scott-Jupp R., Lama M., Tanner M. S. Prevalence of liver disease in cystic fibrosis. Arch Dis Child. 1991 Jun;66(6):698–701. doi: 10.1136/adc.66.6.698. [DOI] [PMC free article] [PubMed] [Google Scholar]
  17. Scott J., Higenbottam T., Hutter J., Hodson M., Stewart S., Penketh A., Wallwork J. Heart-lung transplantation for cystic fibrosis. Lancet. 1988 Jul 23;2(8604):192–194. doi: 10.1016/s0140-6736(88)92290-8. [DOI] [PubMed] [Google Scholar]
  18. Sharples L., Hathaway T., Dennis C., Caine N., Higenbottam T., Wallwork J. Prognosis of patients with cystic fibrosis awaiting heart and lung transplantation. J Heart Lung Transplant. 1993 Jul-Aug;12(4):669–674. [PubMed] [Google Scholar]
  19. Shepherd S. L., Hovell M. F., Slymen D. J., Harwood I. R., Hofstetter C. R., Granger L. E., Kaplan R. M. Functional status as an overall measure of health in adults with cystic fibrosis: further validation of a generic health measure. J Clin Epidemiol. 1992 Feb;45(2):117–125. doi: 10.1016/0895-4356(92)90004-7. [DOI] [PubMed] [Google Scholar]
  20. Sokol R. J., Stall C. Anthropometric evaluation of children with chronic liver disease. Am J Clin Nutr. 1990 Aug;52(2):203–208. doi: 10.1093/ajcn/52.2.203. [DOI] [PubMed] [Google Scholar]
  21. Webb L. J., Ross M., Markham R. L., Webster A. D., Thomas H. C., Sherlock S. Immune function in patients with extrahepatic portal venous obstruction and the effect of splenectomy. Gastroenterology. 1980 Jul;79(1):99–103. [PubMed] [Google Scholar]
  22. Whitehead B., Helms P., Goodwin M., Martin I., Lask B., Serrano E., Scott J. P., Smyth R. L., Higenbottam T. W., Wallwork J. Heart-lung transplantation for cystic fibrosis. 1: Assessment. Arch Dis Child. 1991 Sep;66(9):1018–1026. doi: 10.1136/adc.66.9.1018. [DOI] [PMC free article] [PubMed] [Google Scholar]
  23. Whitehead B., Helms P., Goodwin M., Martin I., Lask B., Serrano E., Scott J. P., Smyth R. L., Higenbottam T. W., Wallwork J. Heart-lung transplantation for cystic fibrosis. 1: Assessment. Arch Dis Child. 1991 Sep;66(9):1018–1026. doi: 10.1136/adc.66.9.1018. [DOI] [PMC free article] [PubMed] [Google Scholar]
  24. Whitehead B., Helms P., Goodwin M., Martin I., Scott J. P., Smyth R. L., Higenbottam T. W., Wallwork J., Elliott M., de Leval M. Heart-lung transplantation for cystic fibrosis. 2: Outcome. Arch Dis Child. 1991 Sep;66(9):1022–1026. doi: 10.1136/adc.66.9.1022. [DOI] [PMC free article] [PubMed] [Google Scholar]

Articles from Thorax are provided here courtesy of BMJ Publishing Group

RESOURCES