Formal Statistical Testing and Inference in Randomized Phase II Trials in Medical Oncology

Imagem de Miniatura
Arquivos
art_RIECHELMANN_Formal_Statistical_Testing_and_Inference_in_Randomized_Phase_2013.PDF (128 KB)
Citações na Scopus
5
Tipo de produção
article
Data de publicação
2013
DOI
Scopus
Web of Science
PubMed
Título da Revista
ISSN da Revista
Título do Volume
Editora
LIPPINCOTT WILLIAMS & WILKINS
Autores
SAAD, Everardo D.
SASSE, Emma C.
BORGHESI, Gustavo
MIRANDA, Vanessa C.
FEDE, Angelo B. S.
SAAD, Lucas S.
OLIVEIRA, Vinicius
BARROS, Eduardo A. C.
PASCOIN, Maira
GIGLIO, Auro del
Citação
AMERICAN JOURNAL OF CLINICAL ONCOLOGY-CANCER CLINICAL TRIALS, v.36, n.2, p.143-145, 2013
Projetos de Pesquisa
Unidades Organizacionais
Fascículo
Resumo
Objectives: With the growing number of new anticancer therapies, randomized phase II trials have been used more often in oncology. Although the primary objective of such trials is not to formally compare results between arms, this practice seems frequent. We sought to quantify the frequency of use of formal statistical testing or inference through the use of P values and confidence intervals (CIs) in randomized phase II trials. Methods: We searched PubMed for randomized phase II trials assessing systemic cancer therapies published in the years 1995/1996 and 2005/2006. For each study, 2 reviewers independently abstracted data, including reporting of P values and CIs for the primary endpoint. Results: We retrieved 288 articles, 107 of which were eligible for analysis. The median number of patients per trial was 94, the primary endpoint was response rate in 71 (66.4%) cases, and a control arm was present in 55 (51.4%) trials. Either P values or CIs for the primary endpoint were reported in 85 (79.4%; 95% CI, 70.8%-86.1%) cases. Year of publication, source of funding, and use of a control group were not associated with this practice. Conclusions: Formal statistical comparisons between arms of randomized phase II trials are frequently undertaken in medical oncology. The extent to which such a practice abrogates phase III testing is unknown.
Palavras-chave
clinical trial, phase II, confidence intervals, neoplasms, research design, statistics
URI
https://observatorio.fm.usp.br/handle/OPI/2246
Referências
  1. Chen EX, 2009, J CLIN ONCOL S, V27
  2. Gray R, 2006, CLIN CANCER RES, V12, P1966, DOI 10.1158/1078-0432.CCR-05-1136
  3. Green S, 2003, CLIN TRIALS ONCOLOGY, P58
  4. Lee JJ, 2005, J CLIN ONCOL, V23, P4450, DOI 10.1200/JCO.2005.03.197
  5. LEE YJ, 1981, SEMIN ONCOL, V8, P403
  6. Liu PY, 1999, CONTROL CLIN TRIALS, V20, P343, DOI 10.1016/S0197-2456(99)00009-4
  7. Perrone F, 2003, LANCET ONCOL, V4, P305, DOI 10.1016/S1470-2045(03)01078-7
  8. Rubinstein L, 2009, CLIN CANCER RES, V15, P1883, DOI 10.1158/1078-0432.CCR-08-2031
  9. Rubinstein LV, 2005, J CLIN ONCOL, V23, P7199, DOI 10.1200/JCO.2005.01.049
  10. SIMON R, 1985, CANCER TREAT REP, V69, P1375
  11. Stenning S, 2003, CLIN TRIALS CANC PRI, P30
  12. Stone A, 2007, CONTEMP CLIN TRIALS, V28, P138, DOI 10.1016/j.cct.2006.05.009
  13. Thall PF, 1996, J CLIN ONCOL, V14, P296
  14. WITTES RE, 1985, CANCER TREAT REP, V69, P1235
  15. Zia MI, 2005, J CLIN ONCOL, V23, P6982, DOI 10.1200/JCO.2005.06.679

Coleções
Artigos e Materiais de Revistas Científicas - HC/ICESP
Artigos e Materiais de Revistas Científicas - ODS/03