Prioritizing the worries of AML patients: Quantifying patient experience using best–worst scaling

Abstract

Context Although patients with acute myeloid leukemia (AML) experience significant toxicities and poor outcomes, few studies have quantified patients' experience. Methods A community‐centered approach was used to develop an AML‐specific best–worst scaling (BWS) instrument involving 13 items in four domains (psychological, physical, decision‐making, and treatment delivery) to quantify patient worry. A survey of patients and caregivers was conducted using the instrument. Data were analyzed using conditional logistic regression. Results The survey was completed by 832 patients and 237 caregivers. Patients were predominantly white (88%), married/partnered (72%), and in remission (95%). The median age was 55 years (range: 19–87). Median time since diagnosis was 8 years (range: 1–40). Patients worried most about “the possibility of dying from AML” (BWS score = 15.5, confidence interval [CI] [14.2–16.7]) and “long‐term side effects of treatments” (14.0, CI [12.9–15.2]). Patients found these items more than twice as worrisome as all items within the domains of care delivery and decision‐making. Patients were least worried about “communicating openly with doctors” (2.50, CI [1.97–3.04]) and “having access to the best medical care” (3.90, CI [3.28–4.61]). Caregiver reports were highly correlated to patients' (Spearman's ρ = 0.89) though noted significantly more worry about the possibility of dying and spending time in the hospital. Conclusion This large convenience sample demonstrates that AML patients have two principal worries: dying from their disease and suffering long‐term side effects from treatment. To better foster patient‐centered care, therapeutic decision‐making and drug development should reflect the importance of both potential outcomes. Further work should explore interventions to address these worries.

Patient, Family Member and Physician Perspectives and Experiences with AML Treatment Decision-Making

“…Despite physicians reporting that they discussed various treatment options with their patients, most patients in this study did not articulate or had limited understanding about the difference between IC, NIC and BSC. Patients noted the overwhelming shock upon diagnosis [39][40][41], which can make it difficult to process complex information about treatment options [5,41] and to feel involved in treatment decisionmaking [42]. Several patients also described inflated concerns regarding the treatment side effects based on their experiences with family/ friends on other chemotherapies.…”

Development of a Patient-Centered Preference Tool for Patients With Hematologic Malignancies: Protocol for a Mixed Methods Study

“…Novel stated preference methods including best-worst scaling (BWS) instruments and discrete choice experiments (DCEs) have recently been developed that can accurately quantify patient preferences [ 17 , 18 ]. BWS instruments have been used to prioritize the values of stakeholders [ 19 ]. Object case BWS instruments (henceforth simply referred to as BWS instruments) are thought to have less cognitive burden on respondents compared with other preference elicitation instruments [ 20 ].…”

“…DCEs require participants to make choices between pairs of hypothetical treatments with different outcomes and have been particularly useful at rigorously quantifying the trade-off preferences of patients for treatments and informing patient-focused drug development [ 7 ]. Through a multistage process involving stakeholder engagement with patients, caregivers, and the FDA, we developed a BWS instrument and a DCE for patients with acute myeloid leukemia (AML) [ 19 , 21 - 23 ]. Using the BWS, we elicited the treatment preferences of 832 patients with AML and demonstrated that patients had the strongest concerns about outcomes in psychosocial and physical domains [ 19 ].…”

“…Through a multistage process involving stakeholder engagement with patients, caregivers, and the FDA, we developed a BWS instrument and a DCE for patients with acute myeloid leukemia (AML) [ 19 , 21 - 23 ]. Using the BWS, we elicited the treatment preferences of 832 patients with AML and demonstrated that patients had the strongest concerns about outcomes in psychosocial and physical domains [ 19 ]. Using the DCE, we elicited specific treatment outcome preferences of 294 patients with AML and demonstrated substantial differences among preferences [ 22 ].…”

“…BWS is a simpler form of a DCE that does not involve changing levels of attributes. A BWS survey was developed by the principal investigator (DRR) of this study to capture the preferences of patients with AML and was used in a previous study, sampling 832 patients, illustrating that patients had the strongest concerns about treatment outcomes in psychosocial and physical domains [ 19 , 21 ].…”