Estimation of vaccine efficacy in a repeated measures study under heterogeneity of exposure or susceptibility to infection

Philosophical Transactions of the Royal Society A: Mathematical, Physical and Engineering Sciences

Volumn 366, Issue 1874, 2008, Pages 2347-2360

  Valim, Clarissa ^a,b,c   Mezzetti, Maura ^d   Maguire, James ^c   Urdaneta, Margarita ^e   Wypij, David ^b,c  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

^c HARVARD SCHOOL OF PUBLIC HEALTH   (United States)

^d UNIVERSITY OF ROME TOR VERGATA   (Italy)

^e MINISTÉRIO DA SAÚDE   (Brazil)

Author keywords

Efficacy; Generalized estimating equation; Generalized linear models; Heterogeneity; Longitudinal analyses; Vaccine

Indexed keywords

HEALTH; MODAL ANALYSIS; PROBABILITY DISTRIBUTIONS; RISK ASSESSMENT; VACCINES;

(PL) PROPERTIES; APPLIED (CO); ENVIRONMENTAL EXPOSURES; MALARIA VACCINE; PROPORTIONAL HAZARDS; RE ANALYSIS; REPEATED MEASURES; SIMULATION STUDIES;

ESTIMATION;

MALARIA VACCINE;

ARTICLE; BIOMETRY; BRAZIL; CLINICAL TRIAL; EPIDEMIOLOGY; HUMAN; INFECTION CONTROL; MALARIA; PROPORTIONAL HAZARDS MODEL; STATISTICAL MODEL; STATISTICS; TREATMENT OUTCOME; VACCINATION;

BIAS (EPIDEMIOLOGY); BIOMETRY; BRAZIL; CLINICAL TRIALS AS TOPIC; HUMANS; INFECTION CONTROL; LINEAR MODELS; MALARIA; MALARIA VACCINES; PROPORTIONAL HAZARDS MODELS; TREATMENT OUTCOME; VACCINATION;

EID: 45749083817     PISSN: 1364503X     EISSN: None     Source Type: Journal    
DOI: 10.1098/rsta.2008.0044     Document Type: Article

References (44)

1. Aalen, O. O. 1988 Heterogeneity in survival analysis. Stat. Med. 7, 1121-1137. (doi:10.1002/sim. 4780071105)
2. Aalen, O. O. 1994 Effects of frailty in survival analysis. Stat. Methods Med. Res. 3, 227-243. (doi:10.1177/096228029400300303)
3. Aide, P., Bassat, Q. & Alonso, P. 2007 Towards an effective malaria vaccine. Arch. Dis. Child. 92, 476-479. (doi:10.1136/adc.2005. 092551)
4. Alonso, P. L. et al. 1994 Randomized trial of efficacy of SPf66 vaccine against Plasmodium falciparum malaria in children in southern Tanzania. Lancet 344, 1175-1181. (doi:10.1016/S0140-6736(94)90505- 3)
5. Alonso, P. L. et al. 2005 Duration of protection with RTS,S/AS02A malaria vaccine in prevention of Plasmodium falciparum disease in Mozambican children: single-blind extended follow-up of a randomized controlled trial. Lancet 366, 2012-2018. (doi:10.1016/S0140-6736(05)67669-6)
6. Anderson, P. K. & Gill, R. D. 1982 Cox's regression model for counting processes: a large sample study. Ann. Statist. 10, 1100-1120. (doi:10.1214/aos/1176345976)
7. Aponte, J. J. et al. 2007 Safety of the RTS,S/AS02D candidate malaria vaccine in infants living in a highly endemic area of Mozambique: a double blind randomized controlled phase I/IIb trial. Lancet 370, 1543-1551. (doi:10.1016/S0140-6736(07)61542-6)
8. Baird, J. K. et al. 1993 Age-specific prevalence of Plasmodium falciparum among six populations with limited histories of exposure to endemic malaria. Am. J. Trop. Med. Hyg. 49, 707-719.
9. Bejon, P. et al. 2006 A phase 2b randomised trial of the candidate malaria vaccines FP9 ME-TRAP and MVA ME-TRAP among children in Kenya. PloS Clin. Trials 1, e29. (doi:10.1371/journal. pctr.0010029)
10. Bejon, P. et al. 2007 Extended follow-up following a phase 2b randomized trial of the candidate malaria vaccines FP9 ME-TRAP and MVA ME-TRAP among children in Kenya. PloS ONE 2, e707. (doi:10.1371/journal. pone.0000707)
11. Bojang, K. A. et al. Efficacy of RTS,S/AS02 malaria vaccine against Plasmodium falciparum infection in semi-immune adult men in The Gambia: a randomized trial. Lancet 358, 1927-1934. (doi:10.1016/S0140-6736(01)06957-4)
12. Chastang, C., Byar, D. & Piantadosi, S. 1988 A quantitative study of the bias in estimating the treatment effect caused by omitting a balanced covariate in survival models. Stat. Med. 7, 1243-1255. (doi:10.1002/sim.4780071205)
13. Collett, D. 1991 Modelling binary data. New York, NY: Chapman and Hall.
14. Diggle, P. J., Liang, K. & Zeger, S. L. 1994 Analysis of longitudinal data. Oxford statistical science series, vol. 13. New York, NY: Oxford University Press.
15. Gail, M. H., Wieand, S. & Piantadosi, S. 1984 Biased estimates of effect in randomized experiments with nonlinear regressions and omitted covariates. Biometrika 71, 431-444. (doi:10.1093/ biomet/71.3.431)
16. Gilbert, P. B., Self, S. G. & Ashby, M. A. 1998 Statistical methods for assessing differential vaccine protection against human immunodeficiency virus types. Biometrics 54, 799-814. (doi:10.2307/ 2533835)
17. Golm, G. T., Halloran, M. E. & Longini, I. M. 1999 Semiparametric methods for multiple exposure mismeasurement and a bivariate outcome in HIV vaccine trials. Biometrics 55, 94-101. (doi:10. 1111/j.0006-341X.1999.00094.x)
18. Graves, P. & Gelband, H. 2001 Vaccines for preventing malaria. Cochrane review. The Cochrane Library, issue no. 3. (Update Software, Oxford, 2001). See http://www.update-software.com/ abstracts/ab000129.htm.
19. Graves, P., Gelband, H. & Garner, P. 1998 The SPf66 malaria vaccine: what is the evidence for efficacy. Parasit. Today 14, 218-220. (doi:10.1016/S0169-4758(98)01242-3)
20. Halloran, M. E., Haber, M. J. & Longini, I. M. 1991 Direct and indirect effects in vaccine efficacy and effectiveness. Am. J. Epidemiol. 133, 323-331.
21. Halloran, M. E., Longini, I. M., Haber, M. J., Struchiner, C. J. & Brunet, R. C. 1994 Exposure efficacy and change in contact rates in evaluating prophylactic HIV vaccines in the field. Stat. Med. 13, 357-377. (doi:10.1002/sim.4780130404)
22. Halloran, M. E., Longini, I. M. & Struchiner, C. J. 1995 Causal inference in infectious diseases. Epidemiology 6, 142-151. (doi:10.1097/00001648-199503000-00010)
23. Halloran, M. E., Longini, I. M. & Struchiner, C. J. 1996 Estimability and interpretation of vaccine efficacy using frailty mixing models. Am. J. Epidemiol. 144, 83-97.
24. Henderson, R. & Oman, P. 1999 Effect of frailty on marginal regression estimates in survival analysis. J. R. Stat. Soc. B 61, 367-379. (doi:10.1111/1467-9868.00182)
25. Hougaard, P. 1991 Modelling heterogeneity in survival data. J. Appl. Prob. 28, 695-701. (doi:10. 2307/3214503)
26. Hougaard, P. 1995 Frailty models for survival data. Lifetime Data Anal. 1, 255-273. (doi:10.1007/ BF00985760)
27. Hougaard, P. 2000 Analysis of multivariate survival data. New York, NY: Springer.
28. Klein, T. A. & Lima, J. B. P. 1990 Seasonal distribution and biting patterns of Anopheles mosquitoes in Costa Marques, Rondônia, Brazil. J. Am. Mosq. Control Assoc. 6, 700-707.
29. Liang, K. & Zeger, S. 1986 Longitudinal data analysis using generalized linear models. Biometrika 73, 13-22. (doi:10.1093/biomet/73.1.13)
30. Lin, D. Y. & Wei, L. J. 1989 The robust inference for the Cox proportional hazards model. J. Am. Stat. Assoc. 84, 1074-1078. (doi:10.2307/2290085)
31. Longini, I. M. & Halloran, M. E. 1996 A frailty mixture model for estimating vaccine efficacy. Appl. Stat. 45, 165-173. (doi:10.2307/2986152)
32. Omori, Y. & Johnson, R. A. 1993 The influence of random effects on the unconditional hazard rate and survival functions. Biometrika 80, 910-914. (doi:10.1093/biomet/80.4.910)
33. Schmoor, C. & Schumacher, M. 1997 Effects of covariate omission and categorization when analysing randomized trials with the Cox model. Stat. Med. 16, 225-237. (doi:10.1002/ (SICI)1097-0258(19970215)16:3 <225::AID-SIM482> 3.0.CO;2-C)
34. Schumacher, M., Olschewski, M. & Schmoor, C. 1987 The impact of heterogeneity on the comparison of survival times. Stat. Med. 6, 773-784. (doi:10.1002/sim.4780060708)
35. Smith, T., Charlwood, J. D., Takken, W., Tanner, M. & Spiegelhalter, D. J. 1995 Mapping the densities of malaria vectors within a single village. Acta Trop. 59, 1-18. (doi:10.1016/0001-706X(94)00082-C)
36. Struchiner, C. J., Halloran, M. E., Brunet, R. C., Ribeiro, J. M. C. & Massad, E. 1994 Malaria vaccines: lessons from field trials. Cad. de Saude Publica, Rio de Janeiro 10, 310-326.
37. Struthers, C. A. & Kalbfleisch, J. D. 1986 Misspecified proportional hazards models. Biometrika 73, 363-369. (doi:10.1093/biomet/73.2. 363)
38. Therneau, T. M. & Grambsch, P. M. 2000 Modeling survival data. Extending the Cox model. New York, NY: Springer.
39. Urdaneta, M., Prata, A., Struchiner, C. J., Tosta, C. E., Tauil, P. & Boulos, M. 1996 SPf66 vaccine trial in Brazil: conceptual framework, study design and analytical approach. Rev. Soc. Bras. Med. Trop. 29, 259-269.
40. Urdaneta, M., Prata, A., Struchiner, C. J., Tosta, C. E., Tauil, P. & Boulos, M. 1998 Evaluation of SPf66 malaria vaccine efficacy in Brazil. Am. J. Trop. Med. Hyg. 58, 378-385.
41. van der Laan, M. J. & Robins, J. M. 2003 Unified methods for censored longitudinal data and causality, p. 72. New York, NY: Springer.
42. Vaupel, J. W., Manton, K. G. & Stallard, E. 1979 The impact of heterogeneity in individual frailty on the dynamics of mortality. Demography 16, 439-454. (doi:10.2307/2061224)
43. WHO 1995 Twelfth program report of the UNDP/World Bank/WHO special program for research and training in tropical diseases (TDR). Bull. WHO 12, 64-76.
44. Zeger, S. L. & Liang, K. 1986 Longitudinal data analysis for discrete and continuous outcomes. Biometrics 42, 121-130. (doi:10.2307/2531248)