Patients with AML Worry about More Than Just Dying: Quantifying Patient Experience Using Best-Worst Scaling

Background: Patients with acute myeloid leukemia (AML) face poor outcomes and treatments with significant and debilitating toxicities. New regimens with improved efficacy and an improved side effect profile are needed. Documenting patient experience can assist in identifying unmet needs and in fostering drug development. We sought to understand the experience of patients with AML by prioritizing their AML-related worries as part of a larger patient-focused drug development initiative led by The Leukemia & Lymphoma Society.

Methods: A robust community-centered approach was used to engage diverse expert and community stakeholders in the development of an instrument to prioritize patient worry. A pilot study was completed to refine the instrument which resulted in 13 patient-centered worries encompassing decision-making, treatment delivery, physical side effects, and psychosocial effects. To capture best-worst (BW) scores, patients were presented with repeated choice tasks prompting them to choose the attributes that they were most and least worried about among repeated subsets of attributes chosen using a balanced-incomplete block design. Results were analyzed using a conditional logistic regression with patients' choices of attributes as the dependent variable. A national survey of patients and caregivers was conducted with the instrument. Results were then compared with Likert ratings of the attributes for validation. Sub-group analysis compared patients currently in remission to those not in remission. Additional qualitative worries were also collected.

Results: The survey was delivered to 5,353 potential participants. A total of 892 patients with AML participated; 832 completed all choice tasks. Patients were predominantly white (88%), married or partnered (72%), college-educated (66%), and privately-insured (79%). Many were currently employed (46%) while some were disabled (25%) or retired (22%). Most patients received an allogeneic stem cell transplant (64%) and the large majority (95%) were currently in remission. The mean time from diagnosis was 8 years (range = 1 - 40), and the mean age was 55 years (range = 19 - 87). Patients were most worried about "the possibility of dying from AML" (Regression result = 1.25, Standard Error (SE) = 0.030), followed by "long-term side effects of treatments" (Regression result = 1.05, SE = 0.030), "being a burden to others" (Regression result = 0.32, SE 0.029), and "returning to daily activities" (Regression result = 0.31, SE 0.028). Patients were least worried about "communicating openly with doctors" (Regression result = -1.25, SE 0.030), "having access to the best medical care" (Regression result = -0.75, SE 0.029) and "having enough information about AML" (Regression result = -0.62, SE 0.028). BW scores were highly correlated with Likert ratings (Spearman's rho = 0.93). Patients who were not in remission worried more about "knowing about all the treatment options" than "coping with the emotional demands of AML," "short-term side effects of treatments," "the overall financial cost of AML," and "choosing a treatment in a short amount of time." Overall, patients found it easy to understand the questions (78%), easy to answer all the questions (64%) and answered the questions consistently with their experience (89%).

Conclusions: Identifying and prioritizing the worries of patients with AML are necessary to understand the experience of those affected by the disease. In this large, national sample representing mostly AML survivors, patients had the strongest worries about long-term clinical outcomes including the possibility of dying from their disease and the long-term side effects of therapy. Psychosocial effects were also worrisome including concerns about burdening others, returning to work, emotionally coping with AML, and the cost of treatment. Items related to treatment delivery and decision-making were less prioritized, perhaps illustrating the sufficient expertise and counseling from care providers. Patients who were not in remission were more concerned about understanding treatment options. Representing the largest study to date of the concerns of patients with AML, these data can serve to inform patient care and the development of new treatment options.

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