Impact of Tumor Assessment Frequency on Statistical Power in Randomized Cancer Clinical Trials Evaluating Progression-Free Survival

Nakagawa, Yuki; Yoshimoto, Takuya; Nakagawa, Shintaro; Sugitani, Yasuo; Yamamoto, Hideharu; Asakawa, T.

doi:10.1007/s43441-021-00328-2

Cited by 1 publication

(1 citation statement)

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“…Similarly, the use of PFS, associated with RECIST-oriented computer tomography and/or magnetic resonance imaging, as a primary endpoint in a real-world effectiveness study is subjected to uncertainty. The schedules of PFS assessment may add further statistical uncertainty [ 49 ]. On the one hand, PFS allows for assessing outcomes of a given treatment independently from post-treatment therapies, which we could not comprehensively monitor, due to data privacy rules.…”

Section: Discussionmentioning

confidence: 99%

COMBI-r: A Prospective, Non-Interventional Study of Dabrafenib Plus Trametinib in Unselected Patients with Unresectable or Metastatic BRAF V600-Mutant Melanoma

Berking,

Livingstone,

Debus

et al. 2023

Cancers

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Combined BRAF/MEK-inhibition constitutes a relevant treatment option for BRAF-mutated advanced melanoma. The prospective, non-interventional COMBI-r study assessed the effectiveness and tolerability of the BRAF-inhibitor dabrafenib combined with the MEK-inhibitor trametinib in patients with advanced melanoma under routine clinical conditions. Progression-free survival (PFS) was the primary objective, and secondary objectives included overall survival (OS), disease control rate, duration of therapy, and the frequency and severity of adverse events. This study enrolled 472 patients at 55 German sites. The median PFS was 8.3 months (95%CI 7.1–9.3) and the median OS was 18.3 months (14.9–21.3), both tending to be longer in pre-treated patients. In the 147 patients with CNS metastases, PFS was similar in those requiring corticosteroids (probably representing symptomatic patients, 5.6 months (3.9–7.2)) compared with those not requiring corticosteroids (5.9 months (4.8–6.9)); however, OS was shorter in patients with brain metastases who received corticosteroids (7.8 (6.3–11.6)) compared to those who did not (11.9 months (9.6–19.5)). The integrated subjective assessment of tumor growth dynamics proved helpful to predict outcome: investigators’ upfront categorization correlated well with time-to-event outcomes. Taken together, COMBI-r mirrored PFS outcomes from other prospective, observational studies and confirmed efficacy and safety findings from the pivotal phase III COMBI-d/-v and COMBI-mb trials.

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Section: Discussionmentioning

confidence: 99%