Major clinical events and healthcare resource use among patients with long-chain fatty acid oxidation disorders in the United States: Results from LC-FAOD Odyssey program.

Major clinical events (MCEs) related to long-chain fatty acid oxidation disorders (LC-FAOD) in triheptanoin clinical trials include inpatient or emergency room (ER) visits for three major clinical manifestations: rhabdomyolysis, hypoglycemia, and cardiomyopathy. However, outcomes data outside of LC-FAOD clinical trials are limited. The non-interventional cohort LC-FAOD Odyssey study examines data derived from US medical records and patient reported outcomes to quantify LC-FAOD burden according to management strategy including MCE frequency and healthcare resource utilization (HRU). Thirty-four patients were analyzed of which 21 and 29 patients had received triheptanoin and/or medium chain triglycerides (MCT), respectively. 36% experienced MCEs while receiving triheptanoin versus 54% on MCT. Total mean annualized MCE rates on triheptanoin and MCT were 0.1 and 0.7, respectively. Annualized disease-related inpatient and ER events were lower on triheptanoin (0.2, 0.3, respectively) than MCT (1.2, 1.0, respectively). Patients were managed more in an outpatient setting on triheptanoin (8.9 annualized outpatient visits) vs MCT (7.9). Overall, this shows that those with LC-FAOD in the Odyssey program experienced fewer MCEs and less HRU in inpatient and ER settings during triheptanoin-treated periods compared with the MCT-treated periods. The MCE rate was lower after initiation of triheptanoin, consistent with clinical trials.

The Clinician's Tardive Inventory (CTI): A New Clinical Tool for Documenting and Rating Tardive Dyskinesia.

Objective: Current clinician-rated tardive dyskinesia (TD) symptom scales have not addressed the expanding clinical signs and functional impact of TD. The study objective was to develop and test the reliability of a new integrated instrument.Methods: A movement disorder neurologist devised the outline of the rating scale. A Steering Committee (5 neurologists and 2 psychiatrists) provided revisions until consensus was reached. The Clinician's Tardive Inventory (CTI) assesses abnormal movements of the eye/eyelid/face, tongue/mouth, jaw, and limb/trunk; complex movements defined as complicated movements different from simple patterned movements or postures; and vocalizations. The CTI rates frequency of symptoms from 0 to 3 (ranging from absent to constant). Functional impairments, including activities of daily living (ADL), social impairment, symptom distress, and physical harm, are rated 0-3 (ranging from unawareness to severe impact). The CTI underwent interrater and test-retest reliability testing between February and June 2022 based on videos and accompanying vignettes, which were reviewed by 2 movement disorder specialists to determine adequacy. Four clinicians rated each video/vignette. Interrater agreement was analyzed via 2-way random-effects intraclass correlation (ICC), and test-retest agreement was assessed utilizing the Kendall tau-b.Results: Forty-five video/vignettes were assessed for interrater reliability and 16 for test-retest reliability. The most prevalent movements were those of the tongue and mouth (77.8%) and jaw (55.6%). ICCs for movement frequency for anatomic symptoms were as follows: anatomic symptom summary score 0.92, abnormal eye movement 0.89, abnormal tongue/mouth movement 0.91, abnormal jaw movement 0.89, abnormal limb movement 0.76, complex movement 0.87, and abnormal vocalization 0.77; ICCs for functional impairments were as follows: total impairment score 0.92, physical harm 0.82, social embarrassment 0.88, ADLs 0.83, and symptom bother 0.92; Retests were conducted a mean (SD) of 15 (3) days later with correlation coefficients ranging from 0.66 to 0.87.Conclusions: The CTI is a new integrated instrument with proven reliability in assessing TD signs and functional impacts. Future validation study is warranted.

Racial and Ethnic Disparities in Patients With Inflammatory Bowel Disease: An Online Survey.

BACKGROUND: Data regarding care access and outcomes in Black/Indigenous/People of Color/Hispanic (BIPOC/H) individuals is limited. This study evaluated care barriers, disease status, and outcomes among a diverse population of White/non-Hispanic (W/NH) and BIPOC/H inflammatory bowel disease (IBD) patients at a large U.S. health system. METHODS: An anonymous online survey was administered to adult IBD patients at Ochsner Health treated between Aug 2019 and Dec 2021. Collected data included symptoms, the Consumer Assessment of Healthcare Providers and Systems and Barriers to Care surveys, health-related quality of life (HRQOL) via the Short Inflammatory Bowel Disease Questionnaire, the Medication Adherence Rating Scale-4, and the Beliefs about Medicines Questionnaire. Medical record data examined healthcare resource utilization. Analyses compared W/NH and BIPOC/H via chi-square and t tests. RESULTS: Compared with their W/NH counterparts, BIPOC/H patients reported more difficulties accessing IBD specialists (26% vs 11%; P = .03), poor symptom control (35% vs 18%; P = .02), lower mean HRQOL (41 ± 14 vs 49 ± 13; P < .001), more negative impact on employment (50% vs 33%; P = .029), worse financial stability (53% vs 32%; P = .006), and more problems finding social/emotional support for IBD (64% vs 37%; P < .001). BIPOC/H patients utilized emergency department services more often (42% vs 22%; P = .004), reported higher concern scores related to IBD medication (17.1 vs 14.9; P = .001), and worried more about medication harm (19.5% vs 17.7%; P = .002). The survey response rate was 14%. CONCLUSIONS: BIPOC/H patients with IBD had worse clinical disease, lower HRQOL scores, had more medication concerns, had less access to specialists, had less social and emotional support, and used emergency department services more often than W/NH patients.

This study examined care access and outcomes in a diverse population of inflammatory bowel disease patients, comparing White/non-Hispanic and Black/Indigenous/People of Color/Hispanic individuals. The analysis revealed that Black/Indigenous/People of Color/Hispanic patients reported greater difficulties accessing inflammatory bowel disease specialists, poorer symptom control, and lower quality of life, and faced challenges in employment, financial stability, and finding social/emotional support. Additionally, they utilized emergency department services more frequently, expressed higher medication concerns, and had increased worries about medication harm.

Clinical and Patient-Reported Outcomes of Advanced Urothelial Carcinoma Following Discontinuation of PD-1/L1 Inhibitor Therapy.

INTRODUCTION: The patterns of care and attrition of locally advanced or metastatic urothelial carcinoma (la/mUC) patients eligible for systemic therapy following PD-1/L1 inhibitors are unclear. The objective of this study was to evaluate the clinical characteristics and treatment patterns among patients with la/mUC following discontinuation of first-line (1L) or second-line (2L) PD-1/L1 inhibitor therapy. METHODS: An ambispective, multisite, chart review study was conducted in the United States, including patients with la/mUC. Eligible patients had initiated and subsequently discontinued PD-1/L1 therapy in the 1L or 2L setting for la/mUC between May 2016 and July 2018; with follow-up through October 2019. Patient characteristics, treatments, and overall survival (OS) were described. Patients had the option to complete a 1-time patient reported outcomes (PRO) survey. RESULTS: Among 300 patients included in the chart review, 198 (66%) received 1L PD-1/L1 inhibitor and 102 (34%) received 2L PD-1/L1 inhibitor. Following discontinuation of PD-1/L1 inhibitor therapy, 34% (n = 68) received subsequent therapy in 2L and 29% (n = 30) in third-line (3L). The median OS post-1L PD-1/L1 inhibitor was 9.4 (95% CI 8.6-NA) and 2.5 months (95% CI 2.24-3.50) for those who received and did not receive subsequent therapy, respectively. Following 2L PD-1/L1 inhibitor discontinuation, the median OS was 5.7 (95% CI 5.1-7.8) and 3.98 (95% CI 3.29-4.87) months for those who received and did not receive subsequent therapy, respectively. Among those with PRO data, 64% reported experiencing cancer-related pain and 29.6% received an opioid. Only 12.7% reported having a caregiver, requiring approximately 13 h/d of service. CONCLUSION: The symptom and caregiver burden are high among real-world patients with la/mUC who discontinued 1L or 2L PD-1/L1 inhibitors and outcomes are dismal, with a minority receiving subsequent therapy. Patterns of care in the setting of 1L maintenance avelumab and novel agents require further investigation.

Development of a patient decision aid for treatment resistant depression.

BACKGROUND: Shared decision-making (SDM) involves patients and clinicians choosing treatment jointly. SDM in mental health is hampered by lack of well-developed supporting tools. We describe an evidence-based patient decision aid (PDA) to facilitate SDM for treatment-resistant depression (TRD) following US National Quality Forum standards which are based upon the International Patient Decision Aid Standards (IPDAS). METHODS: A web-based PDA was developed by a multidisciplinary steering committee of clinicians, patient advocates, patients and a decision scientist. Development included creating content consistent with decision-making domains that are impacted by patient preference in TRD. Development was guided by literature review, group conference calls/discussions, patient and clinician interviews (N = 8), high and lower literacy focus groups (N = 11) and pilot study (N = 5). The PDA presents risk-benefit information on domains (e.g., effectiveness, mode of administration, side effects, cost) and includes values clarification exercises. Pilot study patients were administered the Decisional Conflict Scale (DCS) and Decision Self-Efficacy Scale (DSES) prior to and following PDA interaction and clinician SDM. RESULTS: During the pilot, prior to PDA interaction, mean (standard deviation) DCS score was 42.2 (14.4) and DSES score was 86.0 (14.6) out of 100. Following PDA interaction and SDM, DCS decreased (improved) to 28.1 (SD 4.1) and DSES increased to 95.5 (6.7). All patients endorsed that the PDA helped them to: recognize pros and cons of options; understand how treatments were administered, possible side-effects, and likelihood of benefit; recognize what was important relative to the decision; organize thoughts and prepare for a discussion with their clinician. CONCLUSIONS: This PDA may support SDM in TRD. A future trial to determine impact of the present SMD on decision-making quality is warranted. It also highlights gaps in comparative effectiveness trials that could guide equitable shared decision-making.

Health-related quality of life (QoL) in patients with advanced melanoma receiving immunotherapies in real-world clinical practice settings.

BACKGROUND: Pembrolizumab (PEMBRO) and ipilimumab + nivolumab (IPI + NIVO) are approved advanced melanoma (AM) immunotherapies. To address limited health-related quality of life (QoL) real-world evidence with immunotherapies in AM, we compared QoL in AM patients receiving either treatment in clinical practice. METHODS: A prospective US observational study enrolled adult AM patients initiating first-line PEMBRO or IPI + NIVO between June 2017 and March 2018. Endpoints included the QLQ-C30 global health score (GHS) and EuroQol visual analog scale (EQ-VAS) scores. Mean changes were compared using repeated measures mixed-effects models and are presented covariate adjusted. RESULTS: 225 PEMBRO and 187 IPI + NIVO patients were enrolled. From baseline through week 24, PEMBRO was associated with 3.2 mean GHS score increase (95% CI 0.5, 5.9; p = .02), while no change was observed with IPI + NIVO; 0.2 (95% CI - 2.6, 3.0; p = 0.87). Among objective treatment-responders, GHS scores associated with PEMBRO increased 6.0 (95% CI 3.1, 8.8; p < .0001); IPI + NIVO patients increased 3.8 (95% CI 0.8, 6.9; p = .01). In treatment non-responders, IPI + NIVO was associated with GHS/QoL deterioration of - 3.7 (95% CI - 6.8, - 0.6; p = .02), PEMBRO non-responders demonstrated no change; 0.7 (95% CI - 2.3, 3.7; p = 0.6). Between treatments, PEMBRO patients increased 2.6 greater in EQ-VAS (95% CI 0.6, 4.5; p = .01) vs IPI + NIVO at 24 weeks. CONCLUSIONS: PEMBRO was associated with better 24-week QoL compared to IPI + NIVO in actual clinical practice settings. Real-world data has known limitations, but with further confirmation these results may have implications for treatment selection.

Hospitalization and emergency department utilization in patients with advanced melanoma receiving pembrolizumab versus ipilimumab plus nivolumab in US academic centers.

Background: Both pembrolizumab (PEMBRO) and ipilimumab + nivolumab (IPI + NIVO) are FDA-approved immunotherapy regimens for advanced melanoma (AM). Each regimen has different toxicity profiles potentially impacting healthcare resource utilization (HCRU). This study compared real-world hospitalization and emergency department (ED) utilization within 12 months of therapy initiation of each regimen.Methods: A retrospective cohort study was conducted in AM patients ≥18 years old initiating PEMBRO or IPI + NIVO between January 1, 2016-December 30, 2017. Patients were identified from 12 US-based academic and satellite centers. All-cause hospitalization ED visits were identified. These events were used to calculate rates per 1,000 patient months. Utilization between groups was compared using multivariate logistic regression.Results: In total, 400 patients were included (200 PEMBRO, 200 IPI + NIVO). PEMBRO vs IPI + NIVO patients had poorer Eastern Cooperative Group (ECOG) performance status, 29% 2-4, vs 12% (p < .001); more diabetes, 21% vs 13% (p = .045); were more often PD-L1 expression positive, 77% vs 63% (p = .011); and less likely BRAF mutant, 35% vs 50% (p = .003). The proportion with more than one hospitalization over 12 months was 17% PEMBRO vs 24% IPI + NIVO. Less than 2% had more than one admission and none had more than two. Unadjusted mean (SD) hospitalizations per 1,000 patient-months were 16 (37) and 20 (38), PEMBRO and IPI + NIVO, respectively. Adjusted odds ratio for hospitalization was 0.6 (95% CI = 0.3-0.9; p = .027) for PEMBRO vs IPI + NIVO. ED visits occurred in 18% vs 21%, PEMBRO and IPI + NIVO, respectively, 0.7 (p = .186).Conclusions: PEMBRO patients had a significantly lower probability of hospitalization through 12 months vs IPI + NIVO. The probability of ED visits did not differ.

Factors associated with immunotherapy selection in patients with advanced melanoma.

AIM: To explore factors associated with pembrolizumab (PEMBRO) versus ipilimumab + nivolumab (IPI+NIVO) selection in advanced melanoma. MATERIALS & METHODS: Total of 12 academic and satellite clinics contributed to this study. Descriptive and logistic regression analyses were conducted to explore associations between clinical characteristics and treatment choice.  Results: Total of 400 patients were included: 200 PEMBRO and 200 IPI+NIVO. Patients were significantly more likely to receive PEMBRO versus IPI+NIVO if they had poorer Eastern Cooperative Oncology Group score, 2-4 versus 0-1 (odds ratio [OR]: 6.6; 95% CI: 3.0-14.7), if they were PD-L1 positive (OR: 4.5; 95% CI: 1.9-10.4) or had BRAF wild-type tumor (OR: 2.2; 95% CI: 1.4-3.6). CONCLUSION: Patient factors are significantly associated with treatment selection in advanced melanoma. Outcomes comparisons should take this into consideration.

Changing Patients' Treatment Preferences and Values with a Decision Aid for Type 2 Diabetes Mellitus: Results from the Treatment Arm of a Randomized Controlled Trial.

INTRODUCTION: Failure to intensify treatment for type 2 diabetes mellitus (T2DM) when indicated, or clinical inertia, is a major obstacle to achieving optimal glucose control. This study investigates the impact of a values-focused patient decision aid (PDA) for T2DM antihyperglycemic agent intensification on patient values related to domains important in decision-making and preferred treatments. METHODS: Patients with poorly controlled T2DM who were taking a metformin-containing regimen were recruited through physicians to access a PDA presenting evidence-based information on T2DM and antihyperglycemic agent class options. Participants' preferences for treatment, decision-making, and the relative importance they placed on various values related to treatment options (e.g., dosing, weight gain, side effects) were assessed before and after interacting with the PDA. Changes from baseline were calculated (post-PDA minus pre-PDA difference) and assessed in univariate generalized linear models exploring associations with patients' personal values. RESULTS: Analyses included 114 diverse patients from 27 clinics across the US. The importance of avoiding injections, concern about hypoglycemia, and taking medications only once a day significantly decreased after interacting with the PDA [- 1.1 (p = 0.002), - 1.3 (p < 0.001), - 1.1 (p = 0.004), respectively], while the importance of taking medications that avoided weight gain increased [0.8 (p = 0.004)]. Prior to viewing the PDA, most patients (58.8%) had not begun thinking about the decision of adding a medication, and few (12.3%) indicated that they had already made a decision. Post-PDA, 46.5% could state a medication preference. CONCLUSION: The values-focused PDA for T2DM medication intensification prepared patients to make a shared decision with their clinician and changed patients' values regarding what was important in making that decision. Helping patients understand their options and underlying values can promote shared decision-making and may reduce clinical inertia delaying treatment intensification. FUNDING: Janssen Scientific Affairs, LLC.

Impact of breakthrough pain on community-dwelling cancer patients: results from the National Breakthrough Pain Study.

OBJECTIVE: To characterize cancer-related breakthrough pain (BTcP) among community-dwelling patients with cancer. METHODS: Data from the National Breakthrough Pain Study, a cross-sectional observational survey describing breakthrough pain (BTP) in the community, were analyzed for a subset of patients with BTcP. Eligible patients were community-dwelling adults with commercial insurance and insurance claims consistent with cancer and chronic pain who consented to a structured telephone interview. Assessments included the Brief Pain Inventory-Short Form (BPI), the Short Form 12 Health Survey, the Sheehan Disability Scale, the Work Performance Questionnaire, Generalized Anxiety Disorder-7 Screener, and Patient Health Questionnaire-2. RESULTS: In total, 112 patients with cancer pain also experienced BTcP; 83.3% were in remission. Most patients reported experiencing ≥2 BTcP episodes per day, a median time to BTcP peak of 15 minutes, and a median duration of BTcP of 30 minutes. Mild pain at onset that gradually worsened was reported by 54.5% of patients, and incidental pain triggered by physical activity was reported by 58.0%. Most patients who reported using a medication to manage BTcP received an oral immediate-release opioid, such as oxycodone or hydrocodone, and only 1 received a rapid-onset opioid; few (24.1%) reported that pharmacologic treatment for BTcP worked every time. Patients reported mean (standard deviation) BPI scores of 4.2 (1.75) and 5.7 (1.98) for average and worst pain intensity during the preceding 24 hours, respectively, and high interference with activity, mood, ability to walk and work, social relations, sleep, and enjoyment of life. CONCLUSION: Results indicate that BTcP among community-dwelling patients with cancer continues to be a health burden and reveals opportunities to improve its management.

The impact of patient support programs on adherence, clinical, humanistic, and economic patient outcomes: a targeted systematic review.

BACKGROUND: Patient support programs (PSPs), including medication management and counseling, have the potential to improve care in chronic disease states with complex therapies. Little is known about the program's effects on improving clinical, adherence, humanistic, and cost outcomes. PURPOSE: To conduct a targeted review describing medical conditions in which PSPs have been implemented; support delivery components (eg, face-to-face, phone, mail, and internet); and outcomes associated with implementation. DATA SOURCES: MEDLINE - 10 years through March 2015 with supplemental handsearching of reference lists. STUDY SELECTION: English-language trials and observational studies of PSPs providing at minimum, counseling for medication management, measurement of ≥1 clinical outcome, and a 3-month follow-up period during which outcomes were measured. DATA EXTRACTION: Program characteristics and related clinical, adherence, humanistic, and cost outcomes were abstracted. Study quality and the overall strength of evidence were reviewed using standard criteria. DATA SYNTHESIS: Of 2,239 citations, 64 studies met inclusion criteria. All targeted chronic disease processes and the majority (48 [75%]) of programs offered in-clinic, face-to-face support. All but 9 (14.1%) were overseen by allied health care professionals (eg, nurses, pharmacists, paraprofessionals). Forty-one (64.1%) reported at least one significantly positive clinical outcome. The most frequent clinical outcome impacted was adherence, where 27 of 41 (66%) reported a positive outcome. Of 42 studies measuring humanistic outcomes (eg, quality of life, functional status), 27 (64%) reported significantly positive outcomes. Only 15 (23.4%) programs reported cost or utilization-related outcomes, and, of these, 12 reported positive impacts. CONCLUSION: The preponderance of evidence suggests a positive impact of PSPs on adherence, clinical and humanistic outcomes. Although less often measured, health care utilization and costs are also reduced following PSP implementation. Further research is needed to better quantify which support programs, delivery methods, and components offer the greatest value for any particular medical condition.

Knowledge of binge eating disorder: a cross-sectional survey of physicians in the United States.

OBJECTIVES: Binge eating disorder (BED)--now a designated disorder in the DSM-5--is the most prevalent eating disorder (ED), affecting 2-3% of the US population. This survey of US physicians assesses how BED is diagnosed, treated and referred. METHODS: Internists, family practitioners, obstetrics/gynecologist (OB/GYNs) and psychiatrists were randomly selected from a nationally-representative panel. Participants completed an online survey and reviewed case vignettes consistent with DSM-5-defined BED, then answered questions to elicit whether they would assess for psychiatric conditions including EDs. Those reporting they would screen and who correctly identified BED in vignettes received additional questions about BED diagnosis, treatment, and referral patterns. RESULTS: Of 278 physicians surveyed, 96% were board-certified and 87% had practiced >10 years. 23% were psychiatrists, 27% family practitioners, 31% internists and 19% OB/GYNs. 92% were 'somewhat likely' to screen for ED after reviewing DSM-5-consistent vignettes. 206 (74%) correctly identified BED. Of these, 33% and 68% reported they proactively screen eating habits for all patients and obese patients, respectively. 10% reported not screening eating habits even in the presence of ED symptoms. Fewer than half reported using DSM criteria in Diagnosing BED, and 56 (27%) did not recognize BED to be a discreet ED. CONCLUSION: Although ED awareness is improving, understanding BED as a distinct ED is lacking, which may result in low rates of screening and diagnosis. This study illustrates how taking a complete patient history (including probing BED characteristics) may be an effective first-line strategy for clinicians to facilitate optimal care for these patients.

Effect of a patient decision aid (PDA) for type 2 diabetes on knowledge, decisional self-efficacy, and decisional conflict.

BACKGROUND: Patients with type 2 diabetes (T2DM) often have poor glycemic control on first-line pharmacologic therapy and require treatment intensification. Intensification decisions can be difficult because of many available options and their many benefits and risks. The American Diabetes Association recommends patient-centered, evidence-based tools supporting shared decision-making between patients and clinicians. We developed a patient decision aid (PDA) targeting decisions about treatment intensification for T2DM. Our objective was to determine the effectiveness of this PDA for patients with T2DM on metformin who require treatment intensification. METHODS: This study was a pragmatic randomized controlled trial conducted in 27 US primary care and endocrinology clinics. Subjects were English-speaking adults with T2DM receiving metformin with persistent hyperglycemia who were recommended to consider medication intensification. Subjects were randomized to receive either the PDA or usual care (UC). Main outcome measures were change in knowledge, decisional self-efficacy, and decisional conflict. RESULTS: Of 225 subjects enrolled, 114 were randomized to the PDA and 111 to UC. Mean [SD] age was 52 [1] years, time since T2DM diagnosis was 6 [+/-6] years, 45.3% were male, and most (55.5%) were non-Caucasian. Compared to UC, PDA users had significantly larger knowledge gains (35.0% [22.3] vs 9.9% [22.2]; P < 0.0001) and larger improvements in self-efficacy (3.7 [16.7] vs-3.9 [19.2]; P < 0.0001) and decisional conflict (-22.2 [20.6] vs-7.5 [16.6]; P < 0.0001). CONCLUSIONS: The PDA resulted in substantial and significant improvements in knowledge, decisional conflict and decisional self-efficacy. Decisional conflict scores after PDA use were within the range that correlates with effective decision-making. This PDA has the potential to facilitate shared-decision-making for patients with T2DM. TRIAL REGISTRATION: NCT02110979.

A Retrospective, Single-Center Comparative Cost Analysis of OnabotulinumtoxinA and AbobotulinumtoxinA for Cervical Dystonia Treatment.

BACKGROUND: Chemodenervation with botulinum neurotoxin (BoNT) is recommended as first-line treatment for the management of cervical dystonia. The choice of BoNT for treatment is subject to the consideration of several factors, including cost. OBJECTIVE: To compare the costs incurred by patients and payers for onabotulinumtoxinA (ONA) or abobotulinumtoxinA (ABO) for the treatment of cervical dystonia. METHODS: We conducted a retrospective, noninterventional closed cohort study of cervical dystonia patients within a single U.S. private neurological practice. Patient and payer incurred costs from medical billing records for patients satisfying inclusion and exclusion criteria treated from November 1, 2009, through January 1, 2013, were de-identified and included in the analysis. Forty-seven patients initially treated with at least 3 consecutive cycles of ONA, followed by at least 3 consecutive cycles of ABO were included, representing 282 injection cycles available for analysis. Patients were required to have had a positive response to treatment with both agents and no concomitant treatment with BoNT for any other condition during the analysis period. The analysis compared the primary endpoint of median overall payer and patient incurred costs reimbursed to the clinic under each treatment regimen. For the purposes of this cost analysis, comparable clinical outcomes on both therapies was assumed.   RESULTS: Switching from ONA to ABO resulted in an overall incurred reimbursement cost savings for payers and patients. Median costs per injection cycle for ONA were $1,925 ($0-$2,814) compared with $1,214 ($229-$2,899; P less than 0.0001) for ABO, representing an approximate 37% reduction in incurred reimbursement costs inclusive of toxin and procedure. Overall toxin reimbursement costs, patient out-of-pocket toxin costs, and the cost of unavoidable waste were also lower when patients were treated with ABO.  CONCLUSIONS: For patients treated for cervical dystonia, switching from ONA to ABO resulted in payer and patient reimbursement cost reductions in a single U.S. private practice with outcomes assumed to be similar.

Development of a patient decision aid for type 2 diabetes mellitus for patients not achieving glycemic control on metformin alone.

PURPOSE: To describe the process used to develop an evidence-based patient decision aid (PDA) that facilitates shared decision-making for treatment intensification in inadequately controlled type 2 diabetes mellitus (T2DM) consistent with International Patient Decision Aids Standards. METHODS: A PDA was developed by a multidisciplinary steering committee of clinicians, patient advocate, nurse, certified diabetes educators, and decision scientist, using a systematic development process. The process included defining the PDA scope and purpose, outlining the framework, content creation, and designing for integration into clinical practice. This was accomplished through a review of the literature and publically available educational materials and input from practicing clinicians and patients during development and iteratively refining content based on input. Patients with poorly controlled T2DM on metformin considering additional medication assessed the PDA during a pilot. RESULTS: Testing identified six preference-sensitive domains important for choosing T2DM treatment: degree of glycemic response, avoiding weight gain, hypoglycemia risk and other adverse events, avoiding injections, convenience of dose administration, blood glucose monitoring, and cost of therapy. Patient feedback guided content revision. Treatment options were offered after presenting medication class risk-benefit information and eliciting patient values, goals, and preferences. The PDA received the highest International Patient Decision Aids Standards global score to date, 88/100, with 100% of criteria fully met for the following dimensions: development process, disclosures, evaluation process, evidence quality, guidance for users, information quality, language/readability, testing, and eliciting patient values. CONCLUSION: A PDA was developed to help T2DM patients make decisions regarding medication choice. This approach may be applicable to other chronic conditions.

Improving access to shared decision-making for Hispanics/Latinos with inadequately controlled type 2 diabetes mellitus.

PURPOSE: To describe the cultural and linguistic adaptation and Spanish translation of an English-language patient decision aid (PDA) for use in supporting shared decision-making in Hispanics/Latinos with type 2 diabetes mellitus (T2DM), a group at a high risk for complications. PATIENTS AND METHODS: A steering committee of endocrinologists, a primary care physician, a certified diabetes educator, and a dietician, each with extensive experience in providing care to Hispanics/Latinos was convened to assess a PDA developed for English-speaking patients with T2DM. English content was reviewed for cultural sensitivity and appropriateness for a Hispanic/Latino population. A consensus-building process and iterative version edits incorporated clinician perspectives. The content was adapted to be consistent with traditional Hispanic/Latino cultural communication precepts (eg, avoidance of hostile confrontation; value for warm interaction; respect for authority; value of family support for decisions). The PDA was translated by native-speaking individuals with diabetes expertise. RESULTS: The PDA underwent testing during cognitive interviews with ten Spanish-speaking Hispanics/Latinos with T2DM to ensure that the content is reflective of the experience, understanding, and language Hispanic/Latino patients use to describe diabetes and treatment. Content edits were made to assure a literacy level appropriate to the audience, and the PDA was produced for online video dissemination. CONCLUSION: High-quality, well-developed tools to facilitate shared decision-making in populations with limited access to culturally sensitive information can narrow gaps and align care with individual patient preferences. A newly developed PDA is available for shared decision-making that provides culturally appropriate treatment information for inadequately controlled Hispanics/Latinos with T2DM. The impact on the overall health of patients and care management of T2DM requires further study.

National Breakthrough Pain Study: prevalence, characteristics, and associations with health outcomes.

The National Breakthrough Pain Study is a large observational study that assessed breakthrough pain (BTP) in a population of commercially insured community-dwelling patients with opioid-treated chronic pain. Eligible patients were identified from an administrative claims database, and consenting patients were asked to complete a structured telephone interview and several validated questionnaires. Questionnaires assessed pain interference with function (Brief Pain Inventory-Short Form), health status (Short Form 12 [SF-12] Health Survey), disability (Sheehan Disability Scale), work performance (World Health Organization Health and Work Performance Questionnaire), and mood (Generalized Anxiety Disorder-7 Screener [GAD-7] and Patient Health Questionnaire-2 [PHQ-2]). Of 2198 patients interviewed, 1278 patients had persistent pain controlled with opioid therapy; 1023 (80%) of these patients reported BTP. Patients had a median of 2.0 episodes of BTP per day (range, 1-50) and a median duration of BTP of 45 minutes (range, 1-720). Compared with patients without BTP, patients with BTP had more pain-related interference in function (Brief Pain Inventory, mean ± SD: 34.2 ± 15.6 vs 25.0 ± 15.7 [P < 0.001]), worse physical health (SF-12 physical component score: 29.9 ± 9.6 vs 35.1 ± 10.4 [P < 0.001]) and mental health (SF-12 mental component score: 47.4 ± 11.3 vs 49.3 ± 10.4 [P < 0.001]), more disability (Sheehan Disability Scale global impairment score: 15.1 ± 9.1 vs 10.6 ± 8.5; World Health Organization Health and Work Performance Questionnaire absolute absenteeism: 12.4 ± 59.9 vs 7.7 ± 44.9 hours [both P < 0.001]), and worse mood (GAD-7 score: 7.4 ± 5.9 vs 5.9 ± 5.4; PHQ-2 anhedonia score: 1.2 ± 1.1 vs 0.9 ± 1.0 [both P < 0.001]). In this population of community-dwelling patients with opioid-treated chronic pain, BTP was highly prevalent and associated with negative outcomes. This burden of illness suggests the need for specific treatment plans.

Middle-of-the-night hypnotic use in a large national health plan.

STUDY OBJECTIVES: Although difficulty maintaining sleep (DMS) is the most common nighttime insomnia symptom among US adults, many FDA-approved hypnotics have indications only for sleep onset, stipulating bedtime administration to offset residual sedation. Given the well-known self-medication tendencies of insomniacs, concern arises that maintenance insomniacs might be prone to self-administer their prescribed hypnotics middle-of-the-night (MOTN) after nocturnal awakenings, despite little efficacy-safety data supporting such use. However, no US data characterize the actual population prevalence or correlates of MOTN hypnotic use. METHODS: Telephone interviews assessed patterns of prescription hypnotic use in a national sample of 1,927 commercial health plan members (ages 18-64) receiving prescription hypnotics within 12 months of study. The Brief Insomnia Questionnaire assessed insomnia symptoms. RESULTS: 20.2% of respondents reported MOTN hypnotic use, including 9.0% who sometimes used twice-per-night (once at bedtime plus once MOTN) and another 11.2% who sometimes used MOTN, but never twice-per-night. The remaining 79.8% used exclusively at bedtime. Among exclusive MOTN users, only 14.0% used MOTN on the advice of their physician (52.6% of those seen by sleep medicine specialists and 42.6% by psychiatrists vs. 5.2% to 13.6% seen by other physicians). MOTN use predictors included DMS being the most bothersome sleep problem, long duration of hypnotic use, and low frequency of DMS. CONCLUSIONS: One-fifth of patients with prescription hypnotics used MOTN, only a minority on advice from their physicians. Since significant next-day cognitive and psychomotor impairment is documented with off-label MOTN hypnotic use, prescribing physicians should question patients about unsupervised MOTN dosing.

Prevalence of axial spondyloarthritis in United States rheumatology practices: Assessment of SpondyloArthritis International Society criteria versus rheumatology expert clinical diagnosis.

OBJECTIVE: New classification criteria for axial spondyloarthritis (SpA) have been validated by the Assessment of SpondyloArthritis international Society (ASAS) working group. We applied these criteria to estimate prevalence of SpA in randomly selected, retrospectively reviewed medical records from representative US rheumatology practices. METHODS: Rheumatologists from 101 US practices identified at-risk patients, ages 18-44 years, with chronic back pain. Medical records were reviewed against ASAS criteria. The proportion of patients meeting ASAS criteria was compared to an estimate of the total number of at-risk patients treated at participating sites and, following weighting, was extrapolated to 5,520 US rheumatology practices. US Census data were used to estimate national prevalence. RESULTS: In a sample of 816 randomly selected records, 514 (63%) at-risk patients (95% confidence interval [95% CI] 59.6-66.3%) met ASAS criteria. By applying this proportion to 1,217,097 Americans estimated at risk, 766,652 were projected to meet ASAS criteria. This projection corresponds to a national prevalence of 0.70% (95% CI 0.38-1.1%) or 701 per 100,000 individuals. The prevalence estimates of ankylosing spondylitis and nonradiographic axial SpA are 0.35% (95% CI 0.18-0.554%) and 0.35% (95% CI 0.18-0.554%), respectively. Rheumatologists diagnosed axial SpA in 491 (60%) of those at risk, corresponding to 0.67% (95% CI 0.36-1.01%) prevalence overall. However, of 514 patients meeting ASAS criteria, 124 (24%) were undiagnosed by rheumatologists. CONCLUSION: This is the first systematic epidemiology study of axial SpA using ASAS criteria. Better recognition of axial symptoms is needed, as rheumatologists' expert clinical diagnoses are not always in agreement with ASAS criteria.

The associations of insomnia with costly workplace accidents and errors: results from the America Insomnia Survey.

CONTEXT: Insomnia is a common and seriously impairing condition that often goes unrecognized. OBJECTIVES: To examine associations of broadly defined insomnia (ie, meeting inclusion criteria for a diagnosis from International Statistical Classification of Diseases, 10th Revision, DSM-IV, or Research Diagnostic Criteria/International Classification of Sleep Disorders, Second Edition) with costly workplace accidents and errors after excluding other chronic conditions among workers in the America Insomnia Survey (AIS). DESIGN/SETTING: A national cross-sectional telephone survey (65.0% cooperation rate) of commercially insured health plan members selected from the more than 34 million in the HealthCore Integrated Research Database. PARTICIPANTS: Four thousand nine hundred ninety-one employed AIS respondents. MAIN OUTCOME MEASURES: Costly workplace accidents or errors in the 12 months before the AIS interview were assessed with one question about workplace accidents "that either caused damage or work disruption with a value of $500 or more" and another about other mistakes "that cost your company $500 or more." RESULTS: Current insomnia with duration of at least 12 months was assessed with the Brief Insomnia Questionnaire, a validated (area under the receiver operating characteristic curve, 0.86 compared with diagnoses based on blinded clinical reappraisal interviews), fully structured diagnostic interview. Eighteen other chronic conditions were assessed with medical/pharmacy claims records and validated self-report scales. Insomnia had a significant odds ratio with workplace accidents and/or errors controlled for other chronic conditions (1.4). The odds ratio did not vary significantly with respondent age, sex, educational level, or comorbidity. The average costs of insomnia-related accidents and errors ($32 062) were significantly higher than those of other accidents and errors ($21 914). Simulations estimated that insomnia was associated with 7.2% of all costly workplace accidents and errors and 23.7% of all the costs of these incidents. These proportions are higher than for any other chronic condition, with annualized US population projections of 274 000 costly insomnia-related workplace accidents and errors having a combined value of US $31.1 billion. CONCLUSION: Effectiveness trials are needed to determine whether expanded screening, outreach, and treatment of workers with insomnia would yield a positive return on investment for employers.