Non-Fusion Versus Fusion Surgery in Pediatric Idiopathic Scoliosis: What Trade-Offs in Outcomes Are Acceptable for the Patient and Family?

BACKGROUND: Vertebral body tethering and other non-fusion techniques for the treatment of pediatric idiopathic scoliosis are increasing in popularity. There is limited physician consensus on this topic as the result of a paucity of published data regarding which patients most benefit from non-fusion strategies. Thus, much of the decision-making is left to patients and parents, who must select a treatment based on their goals and values and the information available from health-care providers, the internet, and social media. We sought to understand patient and family preferences regarding the attributes of fusion versus non-fusion surgery that drive these choices. METHODS: Patients and families were recruited from 7 pediatric spine centers and were asked to complete a survey-based choice experiment that had been jointly developed with the U.S. Food and Drug Administration (FDA) to evaluate patient preferences. Choices between experimentally designed alternatives were analyzed to estimate the relative importance of outcomes and requirements associated with the choice options (attributes). The attributes included appearance, confidence in the planned correction, spinal motion, device failure, reoperation, and recovery period. The inclusion criteria were (1) an age of 10 to 21 years and (2) a diagnosis of adolescent idiopathic scoliosis in patients who were considering, or who had already undergone, treatment with fusion or non-fusion surgery. Preference weights were estimated from the expected changes in choice given changes in the attributes. RESULTS: A total of 344 respondents (124 patients, 92 parents, and 128 parent/patient dyads) completed the survey. One hundred and seventy-three patients were enrolled prior to surgery, and 171 were enrolled after surgery. Appearance and motion were found to be the most important drivers of choice. For the entire cohort, fusion was preferred over non-fusion. For patients who were considering surgery, the most important attributes were preservation of spinal motion and appearance. CONCLUSIONS: Patients and families seeking treatment for idiopathic scoliosis value appearance and preservation of spinal motion and, to a lesser extent, reoperation rates when considering fusion versus non-fusion surgery.

Preferences for potential benefits and risks for gene therapy in the treatment of sickle cell disease.

Objective of this study is to quantify benefit-risk tradeoffs pertaining to potential gene therapies among adults and parents/caregivers of children with sickle cell disease (SCD). A discrete-choice experiment survey was developed in which respondents selected their preferred treatment alternatives in a series of experimentally controlled pairs of hypothetical gene therapies and a "no gene therapy" option. Gene therapy alternatives were defined based on the chance of eliminating SCD symptoms, expected increases in life expectancy they could offer, treatment-related risk of death, and potential increases in lifetime cancer risk. Respondents made selections based on their current disease severity and in the context of expectations of worsened disease. Three clinical sites and 1 patient organization recruited 174 adult patients and 109 parents of children with SCD to complete the survey. Adult and parent respondents were generally willing to choose gene therapies, but the adults required higher expected levels of efficacy (ie, higher chance of eliminating symptoms) than parents to choose gene therapies that conferred mortality risks of ≥10%. When adults and parents of children with less severe symptoms were asked to consider scenarios of higher levels of disease severity, the increased risk tolerance, and the lowest acceptable level of efficacy for gene therapies with mortality risks dropped by >50%. Baseline SCD symptoms are a major driver of gene therapy acceptability. Adults and parents of patients with milder symptoms may prefer other treatment options; however, an expectation of symptoms deterioration triggers strong reassessment of the acceptable benefit-risk balance of this novel technology.

Patient perspectives on considerations, tradeoffs, and experiences with multiple myeloma treatment selection: a qualitative descriptive study.

BACKGROUND: Advances in multiple myeloma treatment and a proliferation of treatment options have resulted in improved survival rates and periods of symptom-free remission for many multiple myeloma patients. As a result, health-related quality of life (HRQoL) concerns related to myeloma treatments have become increasingly salient for this patient population and represent an important consideration guiding patients' treatment choices. To gain an understanding of patients' experiences with choosing myeloma therapies and explore the HRQoL concerns that are most important to them, we interviewed a diverse sample of US-based multiple myeloma patients about their treatment considerations. METHODS: We conducted a qualitative descriptive study using in-depth interviews. Participants reflected on (1) the factors that were most important to them when thinking about multiple myeloma treatment and how these have changed over time, (2) how they might weigh the importance of treatment efficacy vs. side effects, (3) trade-offs they would be willing to make regarding efficacy vs. HRQoL, and (4) treatment changes they had experienced. Interviews were audio-recorded and transcribed, and narratives were analyzed using applied thematic analysis. RESULTS: We interviewed 21 patients, heterogeneous in their disease trajectory and treatment experience. Participants were 36 to 78 years, 52% female, and 38% Black. Efficacy was named as the most important treatment consideration by almost two-thirds of participants, and over half also valued HRQoL aspects such as the ability to maintain daily functioning and enjoyment of life. Participants expressed concern about potential treatment side effects and preferred more convenient treatment options. Although participants stated largely trusting their clinicians' treatment recommendations, many said they would stop a clinician-recommended treatment if it negatively impacted their HRQoL. Participants also said that while they prioritized treatment efficacy, they would be willing to change to a less efficacious treatment if side effects became intolerable. CONCLUSIONS: Our findings link to other reports reflecting considerations that are important to multiple myeloma patients, including the importance placed on increasing life expectancy and progression-free survival, but also the tension between treatment efficacy and quality of life. Our results extend these findings to a racially diverse US-based patient population at different stages in the disease trajectory.

Patient-Preference Diagnostics: Adapting Stated-Preference Methods to Inform Effective Shared Decision Making.

BACKGROUND: While clinical practice guidelines underscore the need to incorporate patient preferences in clinical decision making, incorporating meaningful assessment of patient preferences in clinical encounters is challenging. Structured approaches that combine quantitative patient preferences and clinical evidence could facilitate effective patient-provider communication and more patient-centric health care decisions. Adaptive conjoint or stated-preference approaches can identify individual preference parameters, but they can require a relatively large number of choice questions or simplifying assumptions about the error with which preferences are elicited. METHOD: We propose an approach to efficiently diagnose preferences of patients for outcomes of treatment alternatives by leveraging prior information on patient preferences to generate adaptive choice questions to identify a patient's proximity to known preference phenotypes. This information can be used for measuring sensitivity and specificity, much like any other diagnostic procedure. We simulated responses with varying levels of choice errors for hypothetical patients with specific preference profiles to measure sensitivity and specificity of a 2-question preference diagnostic. RESULTS: We identified 4 classes representing distinct preference profiles for patients who participated in a previous first-time anterior shoulder dislocation (FTASD) survey. Posterior probabilities of class membership at the end of a 2-question sequence ranged from 87% to 89%. We found that specificity and sensitivity of the 2-question sequences were robust to respondent errors. The questions appeared to have better specificity than sensitivity. CONCLUSIONS: Our results suggest that this approach could help diagnose patient preferences for treatments for a condition such as FTASD with acceptable precision using as few as 2 choice questions. Such preference-diagnostic tools could be used to improve and document alignment of treatment choices and patient preferences. HIGHLIGHTS: Approaches that combine patient preferences and clinical evidence can facilitate effective patient-provider communication and more patient-centric healthcare decisions. However, diagnosing individual-level preferences is challenging, and no formal diagnostic tools exist.We propose a structured approach to efficiently diagnose patient preferences based on prior information on the distribution of patient preferences in a population.We generated a 2-question test of preferences for the outcomes associated with the treatment of first-time anterior shoulder dislocation.The diagnosis of preferences can help physicians discuss relevant aspects of the treatment options and proactively address patient concerns during the clinical encounter.

A Guide to Observable Differences in Stated Preference Evidence.

BACKGROUND AND OBJECTIVE: In health preference research, studies commonly hypothesize differences in parameters (i.e., differential or joint effects on attribute importance) and/or in choice predictions (marginal effects) by observable factors. Discrete choice experiments may be designed and conducted to test and estimate these observable differences. This guide covers how to explore and corroborate various observable differences in health preference evidence. METHODS: The analytical process has three steps: analyze the exploratory data, analyze the confirmatory data, and interpret and disseminate the evidence. In this guide, we demonstrate the process using dual samples (where exploratory and confirmatory samples were collected from different sources) on 2020 US COVID-19 vaccination preferences; however, investigators may apply the same approach using split samples (i.e., single source). RESULTS: The confirmatory analysis failed to reject ten of the 17 null hypotheses generated by the exploratory analysis (p < 0.05). Apart from demographic, socioeconomic, and geographic differences, political independents and persons who have never been vaccinated against influenza are among those least likely to be vaccinated (0.838 and 0.872, respectively). CONCLUSIONS: For all researchers in health preference research, it is essential to know how to identify and corroborate observable differences. Once mastered, this skill may lead to more complex analyses of latent differences (e.g., latent classes, random parameters). This guide concludes with six questions that researchers may ask themselves when conducting such analyses or reviewing published findings of observable differences.