EFFECT: a randomized phase II study of efficacy and impact on function of two doses of nab-paclitaxel as first-line treatment in older women with advanced breast cancer: Breast Cancer Research

L. Biganzoli, S. Cinieri, R. Berardi, R. Pedersini, A. McCartney, A.M. Minisini, E.R. Caremoli, S. Spazzapan, E. Magnolfi, A. Brunello, E. Risi, R. Palumbo, S. Leo, M. Colleoni, S. Donati, S. De Placido, L. Orlando, M. Pistelli, V. Parolin, A. MislangD. Becheri, F. Puglisi, G. Sanna, E. Zafarana, L. Boni, G. Mottino

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Background: Limited data are available regarding the use of nab-paclitaxel in older patients with breast cancer. A weekly schedule is recommended, but there is a paucity of evidence regarding the optimal dose. We evaluated the efficacy of two different doses of weekly nab-paclitaxel, with a specific focus on their corresponding impact on patient function, in order to address the lack of data specifically relating to the older population. Methods: EFFECT is an open-label, phase II trial wherein 160 women with advanced breast cancer aged ≥ 65 years were enrolled from 15 institutions within Italy. Patients were randomly assigned 1:1 to receive nab-paclitaxel 100 mg/m2 (arm A) or 125 mg/m2 (arm B) on days 1, 8, and 15 on a 28-day cycle, as first-line treatment for advanced disease. The primary endpoint was event-free survival (EFS), wherein an event was defined as disease progression (PD), functional decline (FD), or death. In each arm, the null hypothesis that the median EFS would be ≤ 7 months was tested against a one-sided alternative according to the Brookmeyer Crowley test. Secondary endpoints included objective response rate (ORR), clinical benefit rate (CBR), progression-free survival (PFS), overall survival (OS), and safety. Results: After a median follow-up of 32.6 months, 140 events were observed in 158 evaluable patients. Median EFS was 8.2 months (90% CI, 5.9-8.9; p = 0.188) in arm A vs 8.3 months (90% CI, 6.2-9.7, p = 0.078) in arm B. Progression-free survival, overall survival, and response rates were similar in both groups. A higher percentage of dose reductions and discontinuations due to adverse events (AEs) was noted in arm B. The most frequently reported non-haematological AEs were fatigue (grade [G] 2-3 toxicity occurrence in arm A vs B, 43% and 51%, respectively) and peripheral neuropathy (G2-3 arm A vs B, 19% and 38%, respectively). Conclusion: Pre-specified outcomes were similar in both treatment arms. However, 100 mg/m2 was significantly better tolerated with fewer neurotoxicity-related events, representing a more feasible dose to be recommended for older patients with advanced disease. Trial registration: EudraCT, 2012-002707-18. Registered on June 4, 2012. NIH ClinicalTrials.gov, NCT02783222. Retrospectively registered on May 26, 2016. © 2020 The Author(s).
Original languageEnglish
JournalBreast Cancer Res.
Issue number1
Publication statusPublished - 2020


  • Breast cancer
  • Functional decline
  • Metastatic
  • Nab-paclitaxel
  • Older adults
  • Toxicity
  • paclitaxel
  • 130-nm albumin-bound paclitaxel
  • albuminoid
  • antineoplastic agent
  • aged
  • alopecia
  • anemia
  • arthralgia
  • Article
  • bone marrow toxicity
  • breast cancer
  • Brookmeyer Crowley test
  • cancer mortality
  • clinical assessment tool
  • controlled study
  • daily life activity
  • diarrhea
  • disease exacerbation
  • dose response
  • drug efficacy
  • drug safety
  • drug tolerability
  • dyspnea
  • event free survival
  • fatigue
  • febrile neutropenia
  • female
  • follow up
  • functional decline
  • geriatrics
  • human
  • infection
  • instrumental activity of daily living
  • Italy
  • leukopenia
  • liver toxicity
  • major clinical study
  • multiple cycle treatment
  • myalgia
  • nausea and vomiting
  • nephrotoxicity
  • neurotoxicity
  • neutropenia
  • null hypothesis
  • objective response rate
  • open study
  • overall survival
  • peripheral neuropathy
  • phase 2 clinical trial
  • primary health care
  • progression free survival
  • randomized controlled trial
  • survival time
  • treatment response
  • age
  • breast tumor
  • cancer staging
  • clinical trial
  • multicenter study
  • pathology
  • prognosis
  • survival rate
  • tumor invasion
  • very elderly
  • Age Factors
  • Aged
  • Aged, 80 and over
  • Albumins
  • Antineoplastic Agents, Phytogenic
  • Breast Neoplasms
  • Dose-Response Relationship, Drug
  • Female
  • Humans
  • Neoplasm Invasiveness
  • Neoplasm Staging
  • Paclitaxel
  • Prognosis
  • Survival Rate


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