Monitoring rare serious adverse events from a new treatment and testing for a difference from historical controls

Clinical Trials

Volumn 4, Issue 6, 2007, Pages 598-610

  Fay, Michael P ^a,c   Huang, Chiung Yu ^a   Twum Danso, Nana A Y ^b  

^a NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^b Task Force for Child Survival and Development   (United States)

^c NONE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALBENDAZOLE; IVERMECTIN;

ADD ON THERAPY; ARTICLE; CALCULATION; DRUG EFFICACY; DRUG MONITORING; DRUG SAFETY; DRUG SURVEILLANCE PROGRAM; DRUG WITHDRAWAL; FILARIASIS; LYMPHOID TISSUE; METHODOLOGY; NULL HYPOTHESIS; ONCHOCERCIASIS; PRIORITY JOURNAL; PROBABILITY; SAMPLE SIZE; STATISTICAL SIGNIFICANCE; STATISTICS; DRUG COMBINATION; ELEPHANTIASIS; EXPERIMENTAL THERAPY; HUMAN; STATISTICAL MODEL; TREATMENT OUTCOME;

ALBENDAZOLE; DRUG MONITORING; DRUG THERAPY, COMBINATION; ELEPHANTIASIS, FILARIAL; HUMANS; MODELS, STATISTICAL; THERAPIES, INVESTIGATIONAL; TREATMENT OUTCOME;

EID: 40949115994     PISSN: 17407745     EISSN: 17407753     Source Type: Journal    
DOI: 10.1177/1740774507084978     Document Type: Article

References (29)

1. Gyapong JO, Kumaraswami V, Biswas G, et al. Treatment strategies underpinning the global programme to eliminate lymphatic filariasis. Expert Opinion on Pharmacotherapy 2005; 6(2): 179-200.
2. World Health Organization. Lympatic filariasis. Weekly Epidemiological Record 2001; 76: 149-54.
3. World Health Organization. Onchocerciasis and its Control: Report of a WHO Expert Committee on Onchocerciasis Control. WHO: Geneva. 1995.
4. Pinder M. Loa loa a neglected filarial. Parasitology Today 1988; 4: 279-84.
5. Boussinesq M, Gardon J. Prevalences of Loa loa microfilaremia throughout the area endemic for the infection. Annals of Tropical Medicine & Parasitology 1997; 91: 573-89.
6. Thylefors B, Alleman M. Towards the elimination of onchocerciasis. Annals of Tropical Medicine and Parasitology 2006; 100: 733-46.
7. Horton J, Witt C, Ottesen EA, et al. An analysis of the safety of the single doses, two drug regimens used in programmes to eliminate lymphatic filariasis. Parasitology 2000; 121: S147-60.
8. Awadzi K, Edwards G, Duke BOL, et al. The co-administration of ivermectin and albendazole safety, pharmacokinetics and efficacy against Onchocerca volvulus. Annals of Tropical Medicine & Parasitology 2003; 97: 165-78.
9. World Health Organization. Report on active surveillance for adverse events following the use of drug co-administrations in the global programme to eliminate lymphatic filariasis. Weekly Epidemiological Record 2003; 78: 315-7.
10. Boussinesq M, Gardon J, Gardon-Wendel N, et al. Three probable cases of Loa loa encephalopathy following ivermectin treatment for onchocerciasis. American Journal of Tropical Medicine and Hygiene 1998; 58: 461-9.
11. Ducorps M, Gardon-Wendel N, Ranque S, et al. Effets secondaires du traitement de la loase hypermicrofilaremique par 1 ivermectine. Bulletin de la Societe de Pathologie Exotique et de ses Filiales 1995; 88: 105-12.
12. Gardon J, Gradon-Wendel N, Demanga-Ngangue, et al. Serious reactions after mass treatment of onchocerciasis with ivermectin in an area endemic for Loa loa infection. Lancet 1997; 350: 18-22.
13. Lehmann EL, Romano JP. Testing statistical hypotheses, 3rd edition. Springer, New York, 2005.
14. Kepner J, Wackerly D. Some observations on the Makuch/Simon approach to sample size determination in clinical trials with historical controls. Communications in Statistics: Simulations 2001; 30: 611-21.
15. Lee JJ, Tseng C-H. Uniform power method for sample size calculation in historical control studies with binary response. Controlled Clinical Trials 2001; 22: 390-400.
16. Korn EL, Freidlin B. Conditional power calculations for clinical trials with historical controls. Statistics in Medicine 2006; 25: 2922-31.
17. Chan ISF. Exact tests of equivalence and efficacy with a non-zero lower bound for comparative studies. Statistics in Medicine 1998; 17(12): 1403-13.
18. Berger RL, Boos DD. p-Values maximized over a confidence set of nuisance parameter. Journal of the American Statistical Association 1994; 89: 1012-16.
19. Cytel Statistical Software. StatXact Procs for SAS Users, User Manual, Version 6. Cytel Software Corporation, Cambridge, MA, 2004.
20. Chan ISF, Bohidar NR. Exact power and sample size for vaccine efficacy studies. Communications in Statistics - Theory and Methods 1998; 27: 1305-22.
21. Jennison C, Turnbull BW. Group sequential methods with applications to clinical trials. Chapman and Hall/CRC, Washington, DC, 2000.
22. O'Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics 1979; 35: 549-56.
23. Fay MP, Kim H-J, Hachey M. On using truncated sequential probability ratio test boundaries for Monte Carlo implementation of hypothesis tests. (to appear in Journal of Computational and Graphical Statistics, 2007; available at http:/www3.niaid.nih.gov/about/organization/dcr/BRB/staff/ michael.htm) Accessed October 17, 2007.
24. Dragalin V, Fedorov V, Cheuvart B. Statistical approaches to establishing vaccine safety. Statistics in Medicine 2002; 21: 877-93.
25. Chan ISF. Proving non-inferiority or equivalence of two treatments with dichotomous endpoints using exact methods. Statistical Methods in Medical Research 2003; 12: 37-58.
26. Makuch RW, Simon RM. Sample size considerations for non-randomized comparative studies. Journal of Chronic Diseases 1980; 3: 175-81.
27. Xiong X, Tan M, Boyett J. A sequential procedure for monitoring clinical trials against historical controls. Statistics in Medicine 2007; 26: 1497-511.
28. Pocock SJ. Group sequential methods in the design and analaysis of clinical trials. Biometrika 1977; 64: 191-9.
29. Lehmann EL. Elements of Large-sample Theory. Springer, New York, 1999.