A Patient-Centred Approach to Understanding the Trade-Offs Associated with Treatment Decision Making in Hematological Cancers with Short Life Expectancy

INTRODUCTION: Patient experience data provides an opportunity to explore patient's perspectives on current and potential treatments. Gaining patient narratives on the expectation, tolerance and attitudes towards treatments can enhance clinical management and inform discussions with payers and regulatory agencies. Patient experience data is particularly beneficial in conditions where there is no clear, prescribed treatment pathway as it can contextualize joint treatment decisions made by patients and their treating physicians. Life expectancy among elderly patients with hematogical cancers such as acute myeloid leukemia (AML) is short. Patients with these conditions may be eligible for intensive chemotherapy (IC) or may face a decision between non-intensive chemotherapy (NIC) or palliative care. Further, eligible patients may not wish to undergo the taxing treatment regimens associated with IC. A targeted literature review was conducted to understand the trade-offs associated with treatment decision making and to gain patient perspectives on the value of extended overall survival (OS) in hematological cancers.

METHODS: Searches of computerized bibliographic databases including PubMed (Medline), EMBASE and PsycINFO were conducted using the OVID platform. Searches were conducted using a combination of keywords and Medical Subject Headings (MeSH) terms. To be selected for review, articles must have contained keywords in the title and/or abstract and include information regarding patient experience or perspectives. Articles focussed on children (<18 years) and those not available in English were not included.

RESULTS: A total of 26 articles were selected for full text review (12 AML-specific, 13 other hematological cancers and one article with findings relevant to both populations). Most articles selected for inclusion were qualitative research studies (n=23), providing rich detail regarding the patient perspective.

AML patients discussed the lack of a clear, defined treatment pathway, unlike other cancers where there is a prescribed course of action. These patients described feeling trapped between two undesirable treatment options (IC versus palliative care) and described this treatment decision as "do or die". Similarly, in other hematological cancers patients reported choosing IC to extend their life, while acknowledging that the treatment itself can be distressing. It appeared that the idea of living longer dominated this trade-off. Some patients expressed regret in choosing IC after experiencing the associated side effects. These findings suggest that patients may not fully understand the treatment options available to them, which highlights the importance of the patient-clinician relationship.

Findings from studies also highlighted quality of life as an important consideration for patients in addition to the quantity or length of life. Specifically, patients emphasized their need to maintain a 'normal life' following their diagnosis and to be able to engage in their hobbies and daily activities. There is a paucity of research into important life milestones for AML patients. However, patients and caregivers with other hematological cancers described their desire to be alive for family occasions, such as births or weddings, as well as a sense of appreciation and gratitude for the time remaining with their loved one. This was particularly evident for those patients who were parents of younger children, who felt unprepared for the untimely passing of their kin.

CONCLUSIONS: Heterogeneity in the presentation, functional status and presence of comorbidities among patients with AML and other hematological cancers presents unique challenges for treatment. Nevertheless, understanding of aetiology and epidemiology of these conditions continues to evolve. In response, medical product development/evaluation, policy and treatment decisions in clinical practice need to reflect patient perspectives of treatment in terms of expectations, goals, experiences and preferences to ensure selection of the most appropriate treatment for a given individual. Existing evidence provides some insight as to factors that are important to patients and may influence treatment decisions. However, further research to understand patient perspectives, particularly among those who may not be eligible or willing to receive IC, is required.