Abstract 240P
Background
An aromatase inhibitor in combination with a cyclin-dependent kinase 4 and 6 inhibitor (CDKi 4/6) is now the standard of care for HR+/HER2-, locally advanced (LA) and metastatic breast cancer (MBC). Palbociclib was the first CDKi 4/6 generally accessible within the National Health Service (NHS) via the Palbociclib Patient Programme (PPP) with 848 patients initiating palbociclib treatment on the PPP. This study examined the use, effectiveness, and toxicity of palbociclib within the PPP.
Methods
Patients with LA/MBC treated at 8 centres within the PPP between April and December 2017 were eligible. Presented here are the initial data of patients treated in the 1st line setting (1L). Clinical records were reviewed with clinico-pathological, treatment, outcomes, and safety data.
Results
Of the 191 patients recruited, 140 were 1L: median age (interquartile range) 57.6 years (48.6 - 68.9); 50% (68/136) pre/peri-menopausal; 29% (n=40) presented with de novo LA/MBC; 33% (46/138) had visceral and 67% (92/138) non-visceral disease, 47% (65/138) bone-only. Observation period (OP) was 24 months from palbociclib initiation. At the end of OP, complete or partial response and stable disease were recorded as best response in 91% (127), progression in 6% (9) and ‘unknown’ response 3% (4); median progression-free survival (mPFS) was 22.5 months, median overall survival (mOS) not reached (NR), mPFS and mOS NR for de novo patients, nor those who relapsed >12 months from end of adjuvant treatment (n=41). At the end of OP, 51% (71) remained on treatment and 49% (n=69) discontinued (80% [55/69] due to disease progression); 43% (n=60) had ≥1 treatment interruptions and dose reductions were: none 57% (80) and >1 reduction 43% (60) (includes: 20% [28] <3 months, 11% [15] 3-6 months and 12% [17] ≥6 months).
Conclusions
These data from the first NHS patients treated with CDK4/6i outside of a clinical trial indicate the real-world effectiveness and tolerability of palbociclib and complements the pivotal studies. Patient access schemes may bridge the gap between regulatory approval and NHS funding for new medicines and can facilitate collection of data to evaluate outcomes in routine practice.
Clinical trial identification
Editorial acknowledgement
Onyinye Diribe, OPEN Health.
Legal entity responsible for the study
Pfizer.
Funding
Pfizer.
Disclosure
C. Palmieri: Financial Interests, Personal, Advisory Board: Pfizer, Roche, Eli Lilly, Novartis, Daiichi Sankyo, Seattle Genetics; Financial Interests, Institutional, Sponsor/Funding: Pfizer and Exact Sciences; Financial Interests, Personal, Invited Speaker: Pfizer. C. Harper-wynne: Financial Interests, Personal, Invited Speaker, Honoraria/advisory boards / educational suppor: AZ, Eisai, Exact Sciences/ Genomic Health, Lilly, Myriad, Novartis, Pfizer, Roche, Veracyte. D. Wheatley: Financial Interests, Personal, Invited Speaker, Ad boards: Roche, AZ, Daiichi Sancho. I. MacPherson: Financial Interests, Personal, Advisory Board: - Roche, Novartis, Pfizer, Eli Lilly, Pierre Fabre, Daiichi Sankyo, AstraZeneca; Financial Interests, Personal, Other, Travel / Conference registration: Roche, Eli Lilly, Daiichi Sankyo. M. Nathan: Other, Personal, Advisory Board, speakers fees and conference attendance money received from: Eisai, Roche, Lilly, Novartis, Pfizer and AstraZeneca. M. Verrill: Financial Interests, Personal, Advisory Board, research fu: Lilly, Novartis, Pfizer and Roche. J. Eva: Financial Interests, Personal, Full or part-time Employment: OPEN Health. C. Doody: Financial Interests, Personal, Full or part-time Employment: Pfizer. R. CHOWDHURY: Financial Interests, Personal, Full or part-time Employment: Pfizer. All other authors have declared no conflicts of interest.