Value of PFS to patients: longitudinal study
Research type
Research Study
Full title
Longitudinal study to Assess the 'VALue' to patients of PROgression Free Survival (AVALPROFS)
IRAS ID
143236
Contact name
Lesley Fallowfield
Contact email
Sponsor organisation
Brighton & Sussex Medical School
Research summary
Considerable controversy exists about progression free survival (PFS) as a clinical trial endpoint. Although PFS is attractive for practical, methodological and financial reasons, it is not necessarily a valid and appropriate surrogate for overall survival (OS). It is debatable whether or not a longer PFS results in discernible clinical benefits (the main treatment goal). Importantly few data demonstrate the presumed benefits of PFS to patients themselves, in particular whether stabilisation of metastatic disease and/or a reduction in the burden of disease symptoms is ‘worth’ any adverse treatment related symptoms (Fallowfield and Fleissig 2011). Few studies address this question directly; trials often omit Patient Reported Outcome (PRO) measures altogether, relying instead on physician reported toxicity grades. Patients with metastatic cancer often have unrealistic perceptions about their prognoses and the therapeutic intent of treatment which impacts decision making. No studies have examined if patients even understand the meaning of PFS.
Patients go through periods of crisis, hope, adaptation and uncertainty when adjusting to cancer treatment and consequences; so is PFS associated with psychological well-being? Whilst intuitively reasonable that knowing PFS status and that tumour growth has been arrested would bring relief (and conversely that lack of response may increase anxiety), no research supports this assumption.
We wish to contribute meaningfully to research in this area through a longitudinal study of patients newly diagnosed with metastatic disease. We have completed the first 2 phases of this project, which were:-1) the development and modification of appropriate patient reported outcome (PRO) measures and 2) a pilot study with patient volunteers to determine the acceptability of measures and suggestions about methods of recruitment (R&D Ref: 12/211/FAL).REC name
London - Surrey Borders Research Ethics Committee
REC reference
14/LO/0045
Date of REC Opinion
27 Jan 2014
REC opinion
Further Information Favourable Opinion