Prognostication in advanced cancer: update and directions for future research

The objective of this review is to provide an update on prognostication in patients with advanced cancer and to discuss future directions for research in this field. Accurate prognostication of survival for patients with advanced cancer is vital, as patient life expectancy informs many important personal and clinical decisions. The most common prognostic approach is clinician prediction of survival (CPS) using temporal, surprise, or probabilistic questions. The surprise and probabilistic questions may be more accurate than the temporal approach, partly by limiting the time frame of prediction. Prognostic models such as the Glasgow Prognostic Score (GPS), Palliative Performance Scale (PPS), Palliative Prognostic Score (PaP), Palliative Prognostic Index (PPI), or Prognosis in Palliative Care Study (PiPS) predictor model may augment CPS. However, care must be taken to select the appropriate tool since prognostic accuracy varies by patient population, setting, and time frame of prediction. In addition to life expectancy, patients and caregivers often desire that expected treatment outcomes and bodily changes be communicated to them in a sensible manner at an appropriate time. We propose the following 10 major themes for future prognostication research: (1) enhancing prognostic accuracy, (2) improving reliability and reproducibility of prognosis, (3) identifying the appropriate prognostic tool for a given setting, (4) predicting the risks and benefits of cancer therapies, (5) predicting survival for pediatric populations, (6) translating prognostic knowledge into practice, (7) understanding the impact of prognostic uncertainty, (8) communicating prognosis, (9) clarifying outcomes associated with delivery of prognostic information, and (10) standardizing prognostic terminology

Gender as a Modifying Factor Influencing Myotonic Dystrophy Type 1 Phenotype Severity and Mortality: A Nationwide Multiple Databases Cross-Sectional Observational Study

International audienceBACKGROUND: Myotonic Dystrophy type 1 (DM1) is one of the most heterogeneous hereditary disease in terms of age of onset, clinical manifestations, and severity, challenging both medical management and clinical trials. The CTG expansion size is the main factor determining the age of onset although no factor can finely predict phenotype and prognosis. Differences between males and females have not been specifically reported. Our aim is to study gender impact on DM1 phenotype and severity.METHODS: We first performed cross-sectional analysis of main multiorgan clinical parameters in 1409 adult DM1 patients (\textgreater18y) from the DM-Scope nationwide registry and observed different patterns in males and females. Then, we assessed gender impact on social and economic domains using the AFM-Téléthon DM1 survey (n = 970), and morbidity and mortality using the French National Health Service Database (n = 3301). RESULTS: Men more frequently had (1) severe muscular disability with marked myotonia, muscle weakness, cardiac, and respiratory involvement; (2) developmental abnormalities with facial dysmorphism and cognitive impairment inferred from low educational levels and work in specialized environments; and (3) lonely life. Alternatively, women more frequently had cataracts, dysphagia, digestive tract dysfunction, incontinence, thyroid disorder and obesity. Most differences were out of proportion to those observed in the general population. Compared to women, males were more affected in their social and economic life. In addition, they were more frequently hospitalized for cardiac problems, and had a higher mortality rate.CONCLUSION: Gender is a previously unrecognized factor influencing DM1 clinical profile and severity of the disease, with worse socio-economic consequences of the disease and higher morbidity and mortality in males. Gender should be considered in the design of both stratified medical management and clinical trial

Patient selection of DM-Scope registry.

<p>Patient selection of DM-Scope registry.</p