Abstract OT3-03-01: MADELINE: A prospective observational study of mobile app-based patient reported outcomes in advanced breast cancer
Authors: McRoy LL , Mitra D , Hollis K , Kaye JA , Zelnak A , Cheyl J 
Published: March 2017

BACKGROUND: There have been few studies evaluating the day-to-day effects of advanced breast cancer (ABC) and its treatment on patients in a real-world setting.Palbociclib is a novel CDK4/6 inhibitor approved in the US for hormone-receptor positive, human epidermal growth factor receptor negative (HR+, HER2-) ABC/metastatic breast cancer (MBC) in combination with letrozole as initial endocrine based therapy in postmenopausal women or with fulvestrant in women with disease progression following endocrine therapy. With the introduction of this first-in-class drug it is important to understand the experiences of patients initiating ABC therapies including palbociclib in real-world settings and to document the management of these therapies. A smartphone-based mobile application has been developed to collect patient-reported outcome (PRO) data to assess the impact of ABC and associated treatment on symptoms, functioning and quality of life (QOL) as reported by patients. The application is further designed to provide patients initiating palbociclib with a virtual community to connect to others enrolled in the study for peer support. Additionally, clinical data on therapy management (e.g. dose modifications, interruptions, discontinuations, adverse event management and monitoring) will be obtained from patients' medical records to explore the association between patient reported functioning and neutropenia.

Study Design: A prospective, observational, non-interventional study. PROs collected via a mobile application and clinical data via case report forms completed by investigator.

Eligibility Criteria: Women with HR+/HER2– ABC receiving palbociclib in combination with letrozole or fulvestrant as per US label (Group 1) orapproved therapies for ABC other than palbociclib (Group 2). No comparison is intended between the 2 groups.

Specific Aims: The primary goals are to describe changes in patients' general health status as measured by monthly administration of the 12-Item Short Form Health Survey, describe changes in patients' psychological distress as measured by monthly administration of the Center for Epidemiological Studies Depression Scale, and describe the extent to which ABC and its treatment are associated with changes in patients' lives in terms of symptoms, functioning and QOL as measured by daily and weekly administration of targeted patient-reported questions. Additionally, for patients who are employed at baseline, time lost from work in relation to breast cancer and its treatment will be quantified.Data from patients' medical records will be used to document changes in therapy as well as the incidence, severity, and duration of neutropenia. The association between patient reported functioning and neutropenia will also be assessed. Finally, real-world monitoring patterns will be assessed.

Statistical Methods: Descriptive statistics will be used to summarize all endpoints. Meta-data regarding use of virtual community resources will be explored for relationships to PRO data.

Present Accrual and Target Accrual: Approximately 450 patients from up to 30 US sites will be enrolled. Study duration will be approximately 12 months assuming 6 months of recruitment. It is expected the study will start enrollment Q3 2016.

Sponsor: Pfizer.

Citation Format: McRoy LL, Mitra D, Hollis K, Kaye JA, Zelnak A, Cheyl J. MADELINE: A prospective observational study of mobile app-based patient reported outcomes in advanced breast cancer [abstract]. In: Proceedings of the 2016 San Antonio Breast Cancer Symposium; 2016 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2017;77(4 Suppl):Abstract nr OT3-03-01.