Chronic lymphocytic leukaemia (CLLL) and myelodysplastic syndromes (MDS) are paradigmatic chronic haematologic neoplasms, typically involving elderly people and with a prolonged clinical course. They are both incurable except for the few patients eligible for a allogeneic stem cell transplant, which anyway is effective only in a proportion of them and associated with potential life-threatening and fatal complications. Great improvements in the management of patients with CLL or MDS have been accomplished in the last few years, leading to prolongation of disease-free, and overall, survival. In CLL in particular, and in MDS to a more limited extent, targeted oral medicines that are taken continuously until disease progression or intolerable toxicity have been introduced as standard  care. Therefore, quality of life has become key, and in this regard the importance of the patient’s point of view on their health status is fully acknowledged.

Patient-reported outcome (PRO) measures capture any outcome evaluated directly by the patient and are based on patient’s perception of their disease and its treatment(s). In the MyPAL4Adults study we aim at integrating electronic PRO (ePRO) in the standard care to improve the management of patients with CLL or MDS who have been treated or are receiving treatment for their disease.

How may ePROs  help with a clinician’s awareness of a patient’s symptoms? How might ePROs help clinicians become aware of issues which are important to the patient but are sometimes overlooked?

ePROs provide a unique means of capturing the personal and social context of the disease and treatment experience. Doctor’s appointments are frequently very stressful moments for the patients when a lot of information is shared in a limited amount of time. Less recent or milder symptoms are easily overlooked focusing on the most recent or bothersome ones. Some symptoms, that might be considered clinically less relevant for the physician as they are not life-threatening and do not require an immediate intervention rather than observation (e.g. fatigue, somnolence), are those that might have a significantly higher impact on everyday life of the patient and disrupt family and social routine. Early identification and management of such symptoms would be instrumental to improve and restore the quality of life. In this regard, ePRO may help monitor more carefully changes in the patient’s health status and identify earlier unmet needs.

How might they help both the clinician and the patient manage those symptoms? How may ePROs possibly reduce emergency or unnecessary hospitalisations?

In general symptoms might be related to either the underlying disease or the disease-specific treatment. Studies demonstrate that clinicians miss or underestimate a large proportion of the symptomatic adverse events experienced by patients on treatment, while earlier recognition of symptoms might be crucial for the most appropriate management and an increased compliance. Some symptoms can compromise treatment adherence and efficacy, hampering long-term disease control. Others, if left untreated, can rapidly worsen over time and lead to emergency or unnecessary hospitalization. For these reasons, they need to be clearly and swiftly identified in order to implement the appropriate intervention (i.e. switch to a different treatment in case of uncontrolled progressive disease, ancillary medications/dose reduction/treatment interruption or discontinuation in case of treatment-related adverse events). The adoption of ePROs can therefore aid the process of clinical-decision making and improve quality of life in different settings:

  • in patients undergoing continuous treatment with oral targeted agents, where a better management would reasonably lead to increased compliance therefore maximizing the chance of improving treatment outcomes;
  • in patients receiving the best supportive care, where the improved quality of life derived from a better symptom management could lead to a reduction in the number of outpatient visits and hospitalizations.

How may ePROs improve communication with the patient?

ePRO can facilitate the identification and screening of unmet needs which could have been overlooked during regular visits, in particular those regarding the psychological status and social difficulties experienced by the patient and also of their families. Some issues are difficult to  discuss or may seem irrelevant to the patient during the visit, when typically the focus is on the clinical aspects of the disease and time constraints and environmental conditions do not always facilitate a broader dialogue between the patients and their clinicians. ePROs enable reporting and recording the patient’s status between appointments which might be helpful to address the “elephant in the room”, highlight the need of discussing specific patient’s concerns and, ultimately, facilitate and improve the interaction and communication between healthcare professionals, patients and their families. In addition to that, medically relevant psychological distress or physical symptoms that may require an immediate response might be identified more promptly through ePRO reporting, and help can be offered in a timely manner.

Are there any possible downsides to using ePROs in practice? If so, how might they be overcome?

Downsides in the use of ePROs in practice are generally limited. ePRO measurements should be carefully designed to avoid constituting an undue burden to the patient. Limited estimated completion time of ePRO assessments (10-20 minutes) is crucial to avoid patient burden that may reduce patient adherence to ePRO completion. In our study, we considered the possible risk of (serious) distress in completing the questionnaires. As symptom reporting is considered to be part of routine care, we expect the risks to be rather limited because we selected validated questionnaires that address issues discussed in usual care. One should also consider the possible drawback of the continuous recording of (supposedly) symptoms that may inflate the awareness of the disease thus potentially causing deterioration of overall quality of life with a continuous, if not exaggerated, attention to  personal well-being. The unfiltered report of any physical sign and change in mood could weaken the patient’s capacity to react to everyday difficulties that may be unrelated to the disease and could lead to a medicalization of their life, losing the capacity to distinguish between relevant and irrelevant things, somehow delegating responsibilities to the technology and ultimately back to the physician, with an overall decrease of personal empowerment. The study as designed will also address this issue and carefully evaluate the possible negative consequences of ePRO.