PMID- 37386484
OWN - NLM
STAT- MEDLINE
DCOM- 20230703
LR  - 20231121
IS  - 1465-542X (Electronic)
IS  - 1465-5411 (Print)
IS  - 1465-5411 (Linking)
VI  - 25
IP  - 1
DP  - 2023 Jun 29
TI  - Effectiveness of palbociclib with aromatase inhibitors for the treatment of 
      advanced breast cancer in an exposure retrospective cohort study: implications 
      for clinical practice.
PG  - 78
LID - 10.1186/s13058-023-01678-5 [doi]
LID - 78
AB  - BACKGROUND: New drugs for locally advanced or metastatic breast cancer have led 
      to clinical benefits, aside with increasing costs to healthcare systems. The 
      current financing model for health technology assessment (HTA) privileges 
      real-world data. As part of the ongoing HTA, this study aimed to evaluate the 
      effectiveness of palbociclib with aromatase inhibitors (AI) and compare it with 
      the efficacy reported in PALOMA-2. METHODS: A population-based retrospective 
      exposure cohort study was conducted including all patients initiating treatment 
      in Portugal with palbociclib under early access use and registered in the 
      National Oncology Registry. The primary outcome was progression free survival 
      (PFS). Secondary outcomes considered included time to palbociclib failure (TPF), 
      overall survival (OS), time to next treatment (TTNT), and proportion of patients 
      discontinuing treatment due to  adverse events (AEs). The Kaplan-Meier method was 
      used and median, 1- and 2-year survival rates were computed, with two-sided 95% 
      confidence intervals (95%CI). STrengthening the Reporting of OBservational 
      studies in Epidemiology (STROBE) guidelines for reporting observational studies 
      were used. RESULTS: There were 131 patients included. Median follow-up was 
      28.3 months (IQR: 22.7-35.2) and median duration of treatment was 17.5 months 
      (IQR: 7.8-29.1). Median PFS was 19.5 months (95%CI 14.2-24.2), corresponding to a 
      1-year PFS rate of 67.9% (95%CI 59.2-75.2) and a 2-year PFS rate of 42.0% (95%CI 
      33.5-50.3). Sensitivity analysis showed median PFS would increase slightly when 
      excluding those not initiating treatment with the recommended dose, raising to 
      19.8 months (95%CI 14.4-28.9). By considering only patients meeting PALOMA-2 
      criteria, we could observe a major difference in treatment outcomes, with a mean 
      PFS of 28.8 months (95%CI 19.4-36.0). TPF was 19.8 months (95%CI 14.2-24.9). 
      Median OS was not reached. Median TTNT was 22.5 months (95%CI 18.0-29.8). A total 
      of 14 patients discontinued palbociclib because of AEs (10.7%). CONCLUSIONS: Data 
      suggest palbociclib with AI to have an effectiveness of 28.8 months, when used in 
      patients with overlapping characteristics to those used in PALOMA-2. However, 
      when used outside of these eligibility criteria, namely in patients with less 
      favorable prognosis (e.g., presence of visceral disease), the benefits are 
      inferior, even though still favorable.
CI  - (c) 2023. The Author(s).
FAU - Alves da Costa, Filipa
AU  - Alves da Costa F
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal. alvesdacosta.f@gmail.com.
AD  - Research Institute for Medicines (iMED), Faculty of Pharmacy, University of 
      Lisbon, Lisbon, Portugal. alvesdacosta.f@gmail.com.
FAU - Cardoso Borges, Fabio
AU  - Cardoso Borges F
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal.
FAU - Ramos, Adriana
AU  - Ramos A
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal.
FAU - Mayer, Alexandra
AU  - Mayer A
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal.
FAU - Brito, Claudia
AU  - Brito C
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal.
FAU - Ramos, Catarina
AU  - Ramos C
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal.
FAU - Bernardo, Catarina
AU  - Bernardo C
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal.
FAU - Cossito, Mariane
AU  - Cossito M
AD  - Direcao de Avaliacao de Tecnologias de Saude, Autoridade Nacional do Medicamento 
      e Produtos de Saude, I.P. (INFARMED I.P.), Lisbon, Portugal.
FAU - Furtado, Claudia
AU  - Furtado C
AD  - Direcao de Avaliacao de Tecnologias de Saude, Autoridade Nacional do Medicamento 
      e Produtos de Saude, I.P. (INFARMED I.P.), Lisbon, Portugal.
FAU - Ferreira, Arlindo R
AU  - Ferreira AR
AD  - Unidade de Mama, Centro Clinico Champalimaud, Fundacao Champalimaud, Lisbon, 
      Portugal.
AD  - Catolica Medical School, Universidade Catolica Portuguesa, Lisbon, Portugal.
FAU - Martins-Branco, Diogo
AU  - Martins-Branco D
AD  - Servico de Oncologia Medica, Instituto Portugues de Oncologia de Lisboa Francisco 
      Gentil, EPE, Lisbon, Portugal.
AD  - Academic Trials Promoting Team, Institute Jules Bordet, Rue Meylemeersch 90, 
      1070, Brussels, Belgium.
FAU - da Costa Miranda, Ana
AU  - da Costa Miranda A
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal.
FAU - Lourenco, Antonio
AU  - Lourenco A
AD  - Registo Oncologico Nacional (RON), Instituto Portugues de Oncologia de Lisboa 
      Francisco Gentil, EPE, Lisbon, Portugal.
AD  - NOVA Medical School, Universidade Nova de Lisboa, Lisbon, Portugal.
LA  - eng
PT  - Journal Article
DEP - 20230629
PL  - England
TA  - Breast Cancer Res
JT  - Breast cancer research : BCR
JID - 100927353
RN  - 0 (Aromatase Inhibitors)
RN  - G9ZF61LE7G (palbociclib)
SB  - IM
MH  - Humans
MH  - Female
MH  - *Aromatase Inhibitors/adverse effects
MH  - Retrospective Studies
MH  - *Breast Neoplasms/drug therapy
MH  - Cohort Studies
PMC - PMC10308630
OTO - NOTNLM
OT  - Cancer registry
OT  - Effectiveness
OT  - Palbociclib
OT  - Safety
COIS- The authors declare that the research was conducted in the absence of any 
      commercial or financial relationships that could be construed as a potential 
      conflict of interest. DMB declares having received honoraria and advisory board 
      fees from Daiichi Sankyo, Novartis, Merck Sharp & Dohme, Janssen, Pfizer, 
      Angelini, and AstraZeneca; meeting/travel grants from Novartis, Merck Sharp & 
      Dohme, LEO Farmaceuticos, Ipsen, Janssen, Roche, Laboratorios Vitoria, and Gilead 
      Sciences; institutional grants from Novartis and F. Hoffmann-La Roche Ltd, all of 
      which outside the remit of the current project. AF declares having received 
      honoraria and advisory board fees from Daiichi Sankyo, Gilead, Merck Sharp & 
      Dohme, Novartis, Roche; and meeting/travel grants from Roche. FAC, FCB, AR, CB, 
      CR, AL, ACM, MC, CF, AM and ClB declare no competing interests.
EDAT- 2023/06/30 01:06
MHDA- 2023/07/03 06:41
PMCR- 2023/06/29
CRDT- 2023/06/29 23:42
PHST- 2022/10/03 00:00 [received]
PHST- 2023/06/23 00:00 [accepted]
PHST- 2023/07/03 06:41 [medline]
PHST- 2023/06/30 01:06 [pubmed]
PHST- 2023/06/29 23:42 [entrez]
PHST- 2023/06/29 00:00 [pmc-release]
AID - 10.1186/s13058-023-01678-5 [pii]
AID - 1678 [pii]
AID - 10.1186/s13058-023-01678-5 [doi]
PST - epublish
SO  - Breast Cancer Res. 2023 Jun 29;25(1):78. doi: 10.1186/s13058-023-01678-5.