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Handling Incomplete or Late-Onset Toxicities in Early-Phase Dose-Finding Clinical Trials: Current Practice and Future Prospects.
Yin, Zhulin; Mander, Adrian P; de Bono, Johann S; Zheng, Haiyan; Yap, Christina.
Afiliación
  • Yin Z; Clinical Trials and Statistics Unit, Institute of Cancer Research, London, United Kingdom.
  • Mander AP; Centre for Trials Research, Cardiff University, Cardiff, United Kingdom.
  • de Bono JS; Drug Development Unit, The Institute of Cancer Research and the Royal Marsden NHS Foundation Trust, London, United Kingdom.
  • Zheng H; Department of Mathematical Sciences, University of Bath, Bath, United Kingdom.
  • Yap C; Clinical Trials and Statistics Unit, Institute of Cancer Research, London, United Kingdom.
JCO Precis Oncol ; 8: e2300441, 2024 Jan.
Article en En | MEDLINE | ID: mdl-38181316
ABSTRACT

PURPOSE:

The way late-onset toxicities are managed can affect trial outcomes and participant safety. Specifically, participants often might not have completed their entire follow-up period to observe any toxicities before new participants would be recruited. We conducted a methodological review of published early-phase dose-finding clinical trials that used designs accounting for partial and complete toxicity information, aiming to understand (1) how such designs were implemented and reported and (2) if sufficient information was provided to enable the replicability of trial results.

METHODS:

Until March 26, 2023, we identified 141 trials using the rolling 6 design, the time-to-event continuous reassessment method (TITE-CRM), the TITE-CRM with cycle information, the TITE Bayesian optimal interval design, the TITE cumulative cohort design, and the rapid enrollment design. Clinical settings, design parameters, practical considerations, and dose-limiting toxicity (DLT) information were extracted from these published trials.

RESULTS:

The TITE-CRM (61, 43.3%) and the rolling 6 design (76, 53.9%) were most frequently implemented in practice. Trials using the TITE-CRM had longer DLT assessment windows beyond the first cycle compared with the rolling 6 design (52.5% v 6.6%). Most trials implementing the TITE-CRM (91.8%, 56 of 61) failed to describe essential parameters in the protocols or the study result papers. Only five TITE-CRM trials (8.2%, 5 of 61) reported sufficient information to enable replication of the final analysis.

CONCLUSION:

When compared with trials using the rolling 6 design, those implementing the TITE-CRM design exhibited notable deficiencies in reporting essential details necessary for reproducibility. Inadequate reporting quality of advanced model-based trial designs hinders their credibility. We provide recommendations that can improve transparency, reproducibility, and accurate interpretation of the results for such designs.
Asunto(s)

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Teorema de Bayes Tipo de estudio: Diagnostic_studies / Guideline / Prognostic_studies Límite: Humans Idioma: En Revista: JCO Precis Oncol Año: 2024 Tipo del documento: Article País de afiliación: Reino Unido Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Teorema de Bayes Tipo de estudio: Diagnostic_studies / Guideline / Prognostic_studies Límite: Humans Idioma: En Revista: JCO Precis Oncol Año: 2024 Tipo del documento: Article País de afiliación: Reino Unido Pais de publicación: Estados Unidos