PROsEXPLOR : exploratory evaluation of patients with mesothelioma and rare tumors
SUMMARY OF THE RESEARCH PROJECT
FUNDED BY THE BUZZI UNICEM FOUNDATION
Proposer: Federica Grosso
PATIENT REPORTED OUTCOMES (PROs): EXPLORATORY EVALUATION OF PATIENTS WITH MESOTHELIOMA AND RARE TUMORS (PROsEXPLOR)
As part of our routine clinical practice, we propose implementing a proactive monitoring system of symptoms associated with mesothelioma and rare tumors and their treatments in a sample number of patients with these diseases based on the patient-reported outcomes of the CTCAE (Common Terminology Criteria for Adverse Events) (NCI-PRO-CTCAETM ITEMS ITALIAN).
The term patient-centeredness – or "patient-centered care” – is currently used to describe a method of care that places the patient at the center of the clinical decision, as opposed to disease-centered or therapy-centered care, which has been the most commonly used method thus far. The patient is his/her own best doctor, and actively involving the patient in his/her care whenever possible and using the most appropriate methods for each individual is not only an exciting prospect but also has significant prognostic impact.
A study was presented at ASCO this year (Basch E, et al, ASCO 2017 NCT00578006), which underlined the importance of proactively monitoring the toxicity of systemic treatments in accordance with a data collection technique known as patient reported outcomes (PROs) or "outcomes / results reported by the patient". The study compared a group of patients whose symptoms were proactively monitored between scheduled visits vs. patients who were monitored only during scheduled visits, which showed a statistically significant 5-month survival advantage, which maintained significance during multivariate analysis of patients whose symptoms were proactively monitored. These patients also had less need of emergency room services. Possible explanations for the increased survival lie in the fact that earlier intervention avoids the development of complications that are more difficult to control, more intensive monitoring of the symptoms improves the control of adverse events resulting from chemotherapy thus allowing for better control of them and consequently better compliance with the treatment leads to longer treatment, and lastly symptom control permits improved functional status which has been proven to be associated with increased survival.
In the PROs method, the patient is prompted by requests from the attending team to report/communicate by a previously agreed method (email, SMS, phone call) any changes in his/her health status after starting treatment or while being monitored even if not receiving any specific treatment. It is therefore the patients themselves who provide information about the state of their health and the impact of any treatment on their physical or mental condition. The PROs do not call for intermediation or interpretation by professionals or subjects other than the patient, who may ask the care giver to provide a better description of events if he/she is not completely self-sufficient.
The utility of PROs lies in the observation that external technical observers often overestimate or underestimate certain events. For example, the psychological impact of treatments and pain on everyday life is often underestimated. The patient’s perspective is a key element of diagnosis and treatment because:
The information provided by the patient serve as complementary and unique indicators of the development of the underlying disease and the efficacy of the treatment
Numerous scientific societies recognize the key role played by the information provided by the patient in the diagnosis and treatment of the disease, as demonstrated by its increasing inclusion in clinical guidelines
Patient-reported outcomes in clinical studies provide important information for assessing the efficacy of new treatments
Information regarding PROs is often essential for good clinical practice and often anticipates intervention aimed at addressing symptoms that are more readily resolved
The objective (medical) and subjective (patient) information should be integrated and should be complementary both during treatment and if participating in a clinical study. During treatment, PROs are useful for evaluating opportunities to personalize individual therapy programs; in clinical research, a survey of PROs can be used to recommend and guide the studies towards more acceptable therapy for patients. More importantly, the impact of treatment on the quality of life is fundamental in diseases with a poor prognosis.
IMPLEMENTATION METHODS FOR THE IDEA:
In order to use the information contained in the PROs, the data must be processed to make it scientifically measurable and classifiable. The NCI (National Cancer Institute) in the U.S. has initiated a project to validate a collection tool known as the PRO-CTCAE. The goal of the project is to use rigorous scientific methods to create a system that can be used by patients to report adverse events in cancer studies. The system generates useful data for researchers, regulatory bodies, physicians and patients. The Italian version will be available in a few days. It would be a good idea to implement this tool not only in clinical trials but also in current clinical practice for a more patient-centered approach.
The tool consists of a questionnaire of simple questions and a well-defined set of possible answers, in which the patient simply checks the appropriate boxes to indicate his/her answers. The questionnaires include questions aimed at evaluating various aspects such as symptoms, well-being, quality of life, perception of treatment, risk, satisfaction with a treatment and satisfaction with the communication with the medical staff. The direct opinion of the patient allows the staff to evaluate events that cannot be observed, and in any case the patient is always in the best position to judge those that are usually easier to observe. Early diagnosis of adverse events (AE) is of utmost importance for the initiation of appropriate and timely therapy.
The objective of this project is to implement a proactive monitoring system of symptoms through the Italian version of the CTCAE-PROs in the subgroup of patients with mesothelioma and rare tumors who are receiving standard therapy outside the framework of a clinical study and who are willing to fill out the questionnaires. The project will be purely exploratory with the aim of verifying the feasibility of this approach outside the scope of a clinical study. At the beginning of each treatment, the patient and the caregiver will be asked to complete the PROCs CTCAE questionnaire on a weekly basis or during the period between two scheduled visits. Information will be sent to the general practitioners of the patients asking them to collaborate in the management of those symptoms that the attending oncologist deems do not require evaluation by a specialist.
The main functions of the dedicated staff will be as follows:
1. Administering the PROs CTCAE questionnaires to the patient and caregiver within the specified timeframes
2. Entering the data into a database together with the relative analysis
3. Notifying the oncologists, general practioners and specialists of symptoms by telephone or email
4. Supporting clinical activities, including making appointments for the examinations and specialist visits requested by the oncologist or general practitioner
The ultimate purpose is to improve the quality of the clinical service and care provided to patients receiving standard therapy.
SUMMARY OF THE FINANCIAL PLAN:
Support staff for the medical personnel
for 12 months
25,000 (includes the overall costs
for managing the grant)