Treatment Satisfaction Questionnaire For Medication Research Articles

Abstract 4136334: Treatment Satisfaction With an “Inclisiran First” Implementation Strategy Versus Usual Care in Patients With Atherosclerotic Cardiovascular Disease

Background: Despite the availability of effective lipid-lowering therapies (LLTs), most patients (pts) with ASCVD do not meet guideline-recommended LDL-C goals. In VICTORION-INITIATE (NCT04929249), an “inclisiran first” (IF) strategy of adding inclisiran immediately on failure to achieve LDL-C <70 mg/dL, despite maximally tolerated statin therapy, in pts with ASCVD led to significantly higher LDL-C goal attainment vs usual care (UC) at Day 330. Objective: To evaluate change from baseline to Day 330 in pt-reported treatment satisfaction with an IF implementation strategy vs UC in pts with ASCVD enrolled in VICTORION-INITIATE. Methods: VICTORION-INITIATE, a Phase 3b, open-label, prospective trial, was conducted at 45 representative clinical sites across 20 US states. Pts randomized 1:1 (stratified by insurance status) received inclisiran 284 mg (300 mg inclisiran sodium) at Days 0, 90, and 270 plus UC or UC alone (lipid management directed by treating physicians). We evaluated change from baseline to Day 330 in pt-reported satisfaction with IF vs UC using the validated 11-question Treatment Satisfaction Questionnaire for Medication (TSQM)-II comprising 4 domains (effectiveness, side effects, convenience, and global satisfaction) scored from 0–100. As a post-hoc analysis of a pre-specified exploratory endpoint, we analyzed the difference in least-squares (LS) mean change in TSQM-II domain scores for IF vs UC using ANCOVA with baseline scores as a covariate. Results: We randomized 450 patients: median age 67 years, 30.9% female, 25.8% with a history of statin intolerance. From baseline to Day 330, difference in LS mean change (95% confidence interval) in TSQM-II scores for IF (n=139) vs UC (n=105) was significant (p<0.001) for effectiveness (10.4 [5.4, 15.5]), convenience (10.5 [5.8, 15.2]), and global satisfaction (12.1 [7.2, 16.9]). Statistical comparison was not applicable for the side effects domain at Day 330 due to the small sample size (IF: n=6, UC: n=7). On Day 330, mean scores for IF exceeded UC for each TSQM-II question (Figure). Conclusions: An IF implementation strategy resulted in significantly greater change from baseline to Day 330 across multiple domains of pt-reported treatment satisfaction vs UC.

TEC-ADHERE: Real-World Persistence and Adherence on Dimethyl Fumarate in Patients with Relapsing-Remitting Multiple Sclerosis in the French OroSEP Patient-Support Program.

Treatment persistence and adherence are essential for achieving therapeutic goals in patients with multiple sclerosis (MS). OroSEP is an independent patient-support program (PSP) in France for patients with relapsing-remitting MS (RRMS) receiving oral disease-modifying therapies. TEC-ADHERE (NCT04221191; 08/19/2019-09/15/2022) was a prospective, non-interventional, phase4 study to assess the effect of OroSEP on persistence and adherence to dimethyl fumarate (DMF; Tecfidera™) in patients with RRMS. Outcomes were compared for patients in OroSEP versus non-OroSEP patients who received their neurologists' standard of care (SoC). Patients initiated DMF at month0 (M0); follow-up visits occurred at M3 and M6. Primary outcome was persistence at M6. Secondary outcomes included persistence at M1 and M3, adherence at M6 (Girerd questionnaire), anxiety (Generalized Anxiety Disorder Assessment), patient satisfaction at M6 (Treatment Satisfaction Questionnaire for Medication), patient and neurologist satisfaction with OroSEP participation, and adverse events (AEs). Per-protocol population included 341 patients (OroSEP, n = 135; SoC, n = 206). Persistence was similar for OroSEP vs SoC (M6, 75.9% vs 76.6%; M1, 96.0% vs 92.4%; M3, 85.5% vs 89.0%). At M6, mean adherence was higher for OroSEP (5.4) vs SoC (4.7; p < 0.0001), and good adherence (Girerd score = 6) was achieved by more OroSEP patients (55.7%) than SoC patients (29.6%; p < 0.01). Mean anxiety scores were lower in the OroSEP group than in the SoC group at baseline (7.1 vs 8.8; p = 0.02) and M6 (3.4 vs 6.1; p < 0.001). Mean satisfaction scores at M6 were higher for OroSEP (77.4) vs SoC (64.2; p < 0.01). Most neurologists (n = 11/14) agreed that OroSEP helped improve adherence. Treatment-related AEs occurred in 62 (36.3%) OroSEP patients and 76 (42.9%) SoC patients; most common were flushing, diarrhea, hot flush, and abdominal pain. These outcomes support the value of PSPs in encouraging adherence, alleviating anxiety, improving patient satisfaction, and supporting patients to be more independent in managing MS. ClinicalTrials.gov identifier, NCT04221191.

Treatment satisfaction and effectiveness of Lurasidone on quality of life and functioning in adult patients with schizophrenia in the real-world Italian clinical practice: a prospective 3-month observational study.

Although second-generation antipsychotics (SGAs) have proven to be effective therapeutic options for patients with schizophrenia, there is a notable lack of evidence on patients' subjective perspectives regarding their well-being, quality of life, and satisfaction with these medications. This study aimed to evaluate the treatment satisfaction and effectiveness of lurasidone on quality of life and functioning in adult patients with schizophrenia in real-world Italian clinical practice. This was a multicentre, national, non-interventional, single-arm, 3-month prospective study. Patients who were naive to lurasidone treatment and whose treating physician had decided to start them on this medication were enrolled and evaluated over a 3-month period. Eligible patients were adults (≥ 18 years of age) with a primary diagnosis of schizophrenia who were being treated with lurasidone (for the first time [i.e., they were lurasidone naive]) as part of routine clinical practice. Efficacy endpoints were changes in patient/caregiver treatment satisfaction (seven-point Likert scale from the Treatment Satisfaction Questionnaire for Medication), patient quality of life and functioning (QLS), investigator-rated global assessment of functioning (CGI-S, IAQ) after 6 weeks and 3 months of lurasidone, and number of relapses and hospitalizations. Sixty-one patients were enrolled and 59 completed the study. The median dosage of lurasidone at baseline was 37.00mg/day. The median duration of titration was 86.0 days (Min 28; Max 115 days); the median number of dosage changes was 1.0. At the end of 3-month observation period, the median dose of lurasidone was 74.00mg/day. QoL and Functioning Score showed a trend of improvement over time, reaching a mean change from baseline of 9.8 at the end of the study. According to the CGI-S, the percentage of patients who were "markedly or severely ill" showed a continuous decrease from baseline to 3 months, from 62.29% to 8.20%. Patient satisfaction increased over time, with 80.32% of patients reporting that they were somewhat, fairly, or very satisfied (including 63.93% who were completely or very satisfied) at the end of the study. No relapses/hospitalizations for psychiatric reasons were reported. Lurasidone was well tolerated with no safety concerns or discontinuations due to AEs. Lurasidone represents a valid option for the treatment of schizophrenia and positively affects subjective well-being, quality of life and satisfaction. NCT06527885 retrospectively registered (01/08/2024).

Therapeutic Effectiveness SNOT 22-based interdose interval adjustment of dupilumab for CRSwNP.

This study aims to evaluate whether Sino-nasal Outcome Test (SNOT-22)-based interdose interval adjustment of dupilumab demonstrates enduring efficacy and patient satisfaction in chronic rhinosinusitis with nasal polyps (CRSwNP). A retrospective analysis was conducted using planned data collection from 44 patients who received dupilumab and had been followed up for more than 6 months, targeting individuals with CRSwNP based on the EPOS 2020 Criteria. Patients were treated with add-on dupilumab by adjusting the interdose interval based on SNOT-22, which was tapered when subjects with >40 SNOT-22 at pretreatment evaluation were controlled at the level of ≤20 SNOT-22; when subjects with ≤40 SNOT-22 at pretreatment evaluation were controlled at the level of ≥50% improvement. SNOT-22, nasal polyp scores (NPS), and a subjective satisfaction questionnaire modified from the Treatment Satisfaction Questionnaire for Medication (TSQM v.1.4) were assessed at each visit. The SNOT-22-based interdose interval adjustment of dupilumab showed sustained improvement in patients' subjective symptoms, satisfaction, and NPS. Mean scores (S.D) of SNOT-22 improved from 46.04 (22.30) to 14.72 (13.66) at 6 months (p=0.000); NPS improved from 3.20 (2.24) to 1.72 (1.46) at 6 months (p=0.000). Satisfaction scores (0-5) were well maintained above 3.5 points for up to 6 months(p=0.166). There was also a significant correlation between the improvement in the nasal symptom domain of SNOT-22 scores and higher satisfaction scores. Adjusting dupilumab intervals based on SNOT-22 from the start leads to sustained efficacy and patient satisfaction in Korean CRSwNP. This will be of great clinical help in determining the optimal dupilumab interval.

Quality of life measures with etripamil self-administration for acute episodes of paroxysmal supraventricular tachycardia in a medically unsupervised setting: patient-reported outcomes from NODE-303

Abstract Background Etripamil nasal spray (NS) is a fast-acting, self-administered calcium-channel blocker in development for the conversion of AV-nodal dependent paroxysmal supraventricular tachycardia (PSVT) in a medically unsupervised setting. Prior studies show favorable safety and efficacy in converting single episodes of PSVT to sinus rhythm (SR). Purpose In NODE-303, patient-reported outcome (PRO) instruments assessed quality of life (QoL) over multiple etripamil-treated episodes of PSVT. Methods NODE-303 was an event-driven, multi-center, open-label Phase 3 study in North and South American patients with documented PSVT that evaluated the safety and efficacy of etripamil in PSVT termination, over multiple episodes and without the need for prior test dosing. Enrolled patients who perceived PSVT symptoms applied an ambulatory ECG cardiac monitoring system (CMS), performed a pre-trained vagal maneuver and, if symptoms persisted, self-administered etripamil NS 70 mg. A study amendment allowed a repeat dose (etripamil NS, 70 mg) if symptoms persisted 10 min after the first dose. Each patient could self-treat up to 4 episodes. Safety was assessed through adverse events, clinical data, and ECG CMS recordings. The Treatment Satisfaction Questionnaire for Medication (TSQM; validated for acute episodic assessments) and other PROs were administered, and healthcare utilization data were acquired. Patients completed PROs at baseline (BL), monthly, and per PSVT episode, which collected information on symptoms, episode characteristics, rescue medications, and emergency room visits. Additional PROs were the Brief Illness Perception Questionnaire (BIPQ), Cardiac Anxiety Questionnaire (CAQ), and 36-Item Short Form Health Survey (SF-36). Results There were higher proportions of patients reporting mild, minimal or no disease severity, every 6 months, than at BL; extremely severe, severe, and moderate disease severity was reported by smaller proportions of patients than at BL, though incomplete surveys at later visits may limit this analysis. Anxiety and stress about future PSVT episodes were consistently reduced from BL. Measures of treatment satisfaction, effectiveness, and convenience were consistent across multiple episodes, and were at levels considered favorable for the TSQM instrument (Table 1). Rates of emergency-room visits and hospital admissions across multiple, treated PSVT episodes were similar to those observed in trials assessing single episodes (Table 2). The BIPQ, CAQ or SF-36 had no notable changes from BL. Conclusions Etripamil self-administration was associated with patient-reported improvements in degree of disease severity, decreased anxiety and stress over future PSVT episodes, and levels of treatment satisfaction, effectiveness, and convenience considered favorable for respective PRO instrument. Using a symptom-prompted, self-administered treatment for PSVT could potentially improve QoL measures and reduce healthcare resource utilization.

Residual disease is the main, but not the only factor impacting satisfaction in psoriatic patients undergoing biological therapies.

Despite advancements in psoriasis treatment, a gap remains in aligning patient satisfaction with clinical outcomes. Our study aimed to evaluate which clinical and psychological factors may impact treatment satisfaction in psoriatic patients undergoing long-term biological therapies. We performed an observational, cross-sectional, single-center study involving adult patients with moderate-to-severe psoriasis treated with biologics for at least 12 months. We collected sociodemographic characteristics and data on the course of the psoriasis. We also assessed the absolute (residual) Psoriasis Area and Severity Index (PASI), the site of the residual disease, and the severity of pruritus through the Visual Analogue Scale (VAS). Satisfaction was evaluated using the Treatment Satisfaction Questionnaire for Medication (TSQMv.II). The Type D Personality Scale (DS14 questionnaire and Patient Health Questionnaire-9 assessed the psychological profile. Overall, 146 patients were included, and 82.1% were globally satisfied (global satisfaction TSQM score >75). Linear regression analysis showed a negative correlation between global satisfaction scoring and residual PASI. The multivariable analysis found a higher VAS-pruritus score (OR = 1.20, 95% CI = 1.01-1.44; P = 0.043) and not reaching a residual PASI < 2 (OR = 0.30, 95% CI = 0.09-0.94, P = 0.039) as the strongest predictors of global unsatisfied patients (TSQM < 75%). Other factors unrelated to residual disease, such as gender, class of biologic agent, and type D personality, have also been found to impact patient satisfaction. Our study's findings underscore the complexity of patient satisfaction in psoriasis management and highlight the multifactorial nature of treatment success beyond traditional clinical measures.

7377 CAM2029, Octreotide Subcutaneous Depot, Improves Patient-Reported Outcomes From Standard-of-Care Baseline in Patients With Acromegaly: Interim Results From a Phase 3 Study (ACROINNOVA 2)

Abstract Disclosure: D. Ferone: Consulting Fee; Self; Camurus AB, Ipsen, Novartis-AAA, Recordati. Grant Recipient; Self; Camurus AB, Ipsen, Novartis-AAA, Recordati. Research Investigator; Self; Camurus AB. P. Maffei: Research Investigator; Self; Camurus AB. J. Silverstein: Consulting Fee; Self; Xeris Pharmaceuticals. Research Investigator; Self; Camurus AB. J.L. Spencer-Segal: Advisory Board Member; Self; Camurus AB. Research Investigator; Self; Camurus AB. A. Gilis-Januszewska: None. M. Doknic: Speaker; Self; Pfizer, Inc., Novo Nordisk, Merck, Sandoz. P. Kadioglu: None. P. Freda: Research Investigator; Self; Camurus AB. L. Katznelson: Advisory Board Member; Self; Camurus AB, Recordati. G. Fidan Yaylali: None. M. Harrie: Employee; Self; Camurus AB. Stock Owner; Self; Camurus AB. A. Svedberg: Employee; Self; Camurus AB. Stock Owner; Self; Camurus AB. A.M. Pedroncelli: Employee; Self; Camurus AB. Stock Owner; Self; Camurus AB. F. Tiberg: Employee; Self; Camurus AB. Stock Owner; Self; Camurus AB. Introduction: Standard-of-care (SoC) therapies for acromegaly typically require healthcare provider administration and can pose a significant treatment burden. CAM2029 is a novel subcutaneous octreotide depot designed for convenient once-monthly self-administration as a ready-to-use syringe or injection pen. In a 24-week Phase 3 trial (ACROINNOVA 1), CAM2029 achieved superior insulin-like growth factor 1 (IGF-1) response vs placebo in acromegaly patients (pts) previously controlled with SoC (octreotide LAR/lanreotide Autogel). We report patient-reported outcome (PRO) data from an interim analysis of a Phase 3, open-label trial of CAM2029 (ACROINNOVA 2; NCT04125836). Methods: ACROINNOVA 2 enrolled pts with acromegaly who completed ACROINNOVA 1 (prior CAM2029 or placebo groups) and new pts with IGF-1 ≤2x upper limit of normal during stable SoC. Pts received once-monthly CAM2029 20 mg for up to 52 weeks (28 weeks for prior placebo pts [week 24−52]). Primary endpoint was adverse events. PROs were assessed using Acromegaly Index of Severity (AIS, 0−18; sum of 6 scores [0−3; none−severe] for headache, sweating, fatigue, joint pain, paresthesia, soft tissue swelling) for symptoms; Acromegaly Quality of Life Questionnaire (AcroQoL, 0−100) for QoL; Treatment Satisfaction Questionnaire for Medication (TSQM, 0−100) and patient satisfaction scale (0−5) for treatment satisfaction; and Self-Injection Assessment Questionnaire (SIAQ v2.0, 0−10) for satisfaction with self-administration. Higher scores signify improvements, except for AIS. Data are shown for the overall population. Results: At data cutoff (May 23, 2023), 54 roll-over pts (prior CAM2029 n=36; prior placebo n=18) and 81 new pts were enrolled; mean CAM2029 exposure was 56.3 weeks. CAM2029 was well tolerated with no unexpected side effects. Mean AIS score improved from baseline SoC to week 52 (−1.2; 95% CI: −1.8, −0.7); estimated proportion of pts with any acromegaly symptoms reduced by 15.1% (95% CI: −23.9, −6.3). At week 52 vs baseline: mean total AcroQoL increased by 3.5 (95% CI: 1.3, 5.7); mean TSQM increased for ‘convenience’ by 15.6 (95% CI: 11.8, 19.3), ‘effectiveness’ by 6.3 (95% CI: 2.8, 9.8), ‘satisfaction’ by 4.7 (95% CI: 1.1, 8.4), and for ‘side effects’ by 2.4 (95% CI: −2.0, 6.8). Mean patient satisfaction score at week 52 was 4.1 (95% CI: 3.9, 4.3); 32.6% and 19.3% of pts reported a ‘much better’ or ‘slightly better’ experience, respectively, with CAM2029 vs prior SoC. Mean SIAQ increased at week 48 vs pre-treatment, ‘satisfaction with way of taking medication’ by 1.6 (95% CI: 0.9, 2.2) and ‘feelings about injections’ by 0.8 (95% CI: 0.3, 1.3). Conclusion: CAM2029 improved symptom burden, QoL, treatment convenience, and patient satisfaction vs baseline SoC, including in pts with stable or controlled disease at baseline, supporting the clinical benefit of CAM2029 and its potential to address unmet needs for pts with acromegaly. Presentation: 6/2/2024

The patient-reported outcomes for the new brand-generic teriflunomide in relapsing-remitting multiple sclerosis

BackgroundPatient-reported outcomes (PROs) provide valuable insights into the impact of disease-modifying therapies (DMTs) on patients' daily lives and disease progression. This study evaluates treatment satisfaction and tolerability among patients using a brand-generic Teriflunomide (Tebazio®, 14 mg tablet) manufactured by Zistdaru Danesh Biopharmaceuticals. Materials and MethodsA Phase IV observational study was conducted on patients with Relapsing-Remitting Multiple Sclerosis (RRMS) who were either initiated on or switched to Teriflunomide 14 mg. The primary focus was on the medication's safety. Patient satisfaction was measured using the Treatment Satisfaction Questionnaire for Medication [Version 1.4] (TSQM-14). Additionally, medication adherence and discontinuation rates were monitored. ResultsOf the 235 RRMS patients enrolled, participated in this study, all received the Teriflunomide treatment orally on a daily basis. Over the 18-month follow-up period, 25.96 % of patients discontinued the treatment. Discontinuation was mainly due to adverse events (11 %), lack of patient willingness to continue (12.7 %), and disease progression (4.2 %). The most commonly reported adverse events included dermatologic disorders, elevated liver enzymes, and gastrointestinal issues. TSQM-14 scores demonstrated significant improvements over the 18-month period. A high medication adherence rate of 98.1 % was also recorded. ConclusionPatients reported notable satisfaction with Teriflunomide, as reflected in their TSQM scores, which suggests a likelihood of improved patient adherence. The 14 mg brand-generic Teriflunomide was well-accepted by Iranian RRMS patients, with no significant concerns arising during the study. These findings also highlight the significance of patient-reported outcomes in DMTs, with potential benefits for adherence and clinical practice.

If patients had a choice - Treatment satisfaction and patients' preference in therapy of actinic keratoses.

Actinic keratoses (AK) are increasing in incidence and represent the most common (pre-)cancerous lesion in the fair-skinned population, with a high unmet medical need. In order to increase treatment adherence, it is very important to assess patients' therapy-related evaluations of different treatment options. 100 patients with AK who were treated with at least two different treatment options were included. They rated their therapies using the Treatment Satisfaction Questionnaire for Medication (TSQM, maximum 100 points per category) and a Likert scale (LS, 1 = very satisfied; 6 = not satisfied). Patients were also asked about their needs in terms of treatment goal, cost, type, duration, and location of treatment. 81% of the study participants were male and on average 74 years old. 95% had field cancerization. Eight frequently used therapy procedures were evaluated by the patients (surgery, cryotherapy, various topical agents, photodynamic therapy). The TSQM satisfaction scores ranged from 78.47 ±16.07 (surgical procedures) to 53.03 ±22.13 (diclofenac-HA). Statistically significant differences between the procedures were only found in the area of efficacy. Side effects were classified as low. Low recurrence rate and safe removal were the most important treatment goals (LS: 1.18 ±0.44 and 1.27 ±0.53, respectively). Understanding patient preferences is essential for adherence and is therefore of great importance for the success of AK therapy. Personalized approaches should be considered in the choice of therapy.

Evaluation of therapy satisfaction with cladribine tablets in patients with RMS: Final results of the non-interventional study CLEVER

BackgroundCladribine tablets for the treatment of relapsing multiple sclerosis (RMS) are administered in two pulsed treatment courses in two consecutive years, totalling a maximum of 20 treatment days. Here we present data collected shortly after the launch of cladribine tablets, focusing on the patient's perspective. The objective was to investigate patients’ perceived effectiveness, tolerability, and convenience, as well as global satisfaction of and with cladribine tablets. MethodsCLEVER was a non-interventional multicentre study conducted in Germany from 12/2017 to 7/2020. Adult patients with RMS initiating therapy with cladribine tablets were included. Observation time per patient was 24 weeks, comprising 3 visits (baseline, week 4 and 24). The primary endpoint was overall treatment satisfaction at week 24, assessed by the Treatment Satisfaction Questionnaire for Medication 14 items (TSQM 1.4). Subgroup analyses included stratification by prior treatment. ResultsIn total, 491 patients (69.2 % female; mean (±SD) age 40.3 (±11.5) years, 85.1 % pre-treated, median EDSS 2.5) initiated therapy with cladribine tablets and were included in the analysis. At week 24, the mean (±SD) global TSQM satisfaction score was 75.6 (±19.0). For patients switching from either injectables or oral medication, the change in therapy satisfaction from baseline to week 24 was positive in all TSQM domains with clinically meaningful effect sizes in the global satisfaction and side effects domains. Most patients (85.5 %) remained relapse-free over 24 weeks. Out of 491 patients, 187 (38.1 %) experienced at least one adverse event and 8 patients (1.6 %) one serious adverse event. ConclusionTreatment satisfaction with cladribine tablets was high. The switch from prior injectables or oral medication translated into increased treatment satisfaction at week 24 with clinically meaningful effects in the global satisfaction and side effects domains. Effectiveness and safety were consistent with results from clinical studies.

Medication Adherence and Treatment Satisfaction With Lipid-Lowering Drugs Among Patients With Diabetes and Dyslipidemia.

Poor adherence to lipid-lowering drugs in diabetic patients with dyslipidemia increases has been linked with an increased cardiovascular risk. A better understanding of the determinants of adherence to lipid-lowering drugs and treatment satisfaction among people with diabetes and dyslipidemia is crucial. We aimed to assess the prevalence of adherence to lipid-lowering drugs, identify its determinant factors, and evaluate treatment satisfaction among users of lipid-lowering drugs who have diabetes and dyslipidemia. We surveyed 398 adult patients with diabetes and dyslipidemia, using a validated medication adherence survey (Adherence to Refills and Medications Scale) and a validated treatment satisfaction survey (Treatment Satisfaction Questionnaire for Medication, TSQM). Sociodemographic and medical history data were collected through questionnaires. The prevalence of poor medication adherence was 36%. Factors associated with poor adherence included adverse reactions to medications, lack of medication availability, and lack of family support. Adherent patients reported lower low-density lipoprotein-cholesterol (LDL-C) and total cholesterol levels, higher treatment satisfaction, and a higher prevalence of cardiovascular disease and comorbidities. Having a family history of dyslipidemia was negatively associated with adherence, while the number of comorbidities positively influenced it. The scores of TSQM components such as effectiveness, global satisfaction, and convenience were significantly higher in people who were adherent or achieved the LDL-C target. Our findings highlight the need for interventions targeting several factors impacting adherence to lipid-lowering drugs in patients with diabetes and dyslipidemia. Managing adverse effects, leveraging family support, and ensuring medication access represent crucial aspects of improving adherence and potentially mitigating cardiovascular risks in this high-risk population.

Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS-A multicenter observational study over 18 months.

In amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations (SOD1-ALS), tofersen received accelerated approval in the United States and is available via expanded access programs (EAP) outside the United States. This multicenter study investigates clinical and patient-reported outcomes (PRO) and serum neurofilament light chain (sNfL) during tofersen treatment in an EAP in Germany. Sixteen SOD1-ALS patients receiving tofersen for at least 6 months were analyzed. The ALS progression rate (ALS-PR), as measured by the monthly change of the ALS functional rating scale-revised (ALSFRS-R), slow vital capacity (SVC), and sNfL were investigated. PRO included the Measure Yourself Medical Outcome Profile (MYMOP2), Treatment Satisfaction Questionnaire for Medication (TSQM-9), and Net Promoter Score (NPS). Mean tofersen treatment was 11 months (6-18 months). ALS-PR showed a mean change of -0.2 (range 0 to -1.1) and relative reduction by 25%. Seven patients demonstrated increased ALSFRS-R. SVC was stable (mean 88%, range -15% to +28%). sNfL decreased in all patients except one heterozygous D91A-SOD1 mutation carrier (mean change of sNfL -58%, range -91 to +27%, p < .01). MYMOP2 indicated improved symptom severity (n = 10) or yet perception of partial response (n = 6). TSQM-9 showed high global treatment satisfaction (mean 83, SD 16) although the convenience of drug administration was modest (mean 50, SD 27). NPS revealed a very high recommendation rate for tofersen (NPS +80). Data from this EAP supported the clinical and sNfL response to tofersen in SOD1-ALS. PRO suggested a favorable patient perception of tofersen treatment in clinical practice.

Satisfaction With and Adherence to Off-Label Corticosteroids in Adolescents and Adults With Eosinophilic Esophagitis: Results of a Web-Based Survey in the United States.

We assessed satisfaction with and adherence to off-label corticosteroids in patients with eosinophilic esophagitis (EoE) in the United States. EoE is a chronic inflammatory disease for which there are currently no US Food and Drug Administration-approved swallowed topical corticosteroids. This noninterventional, cross-sectional, web-based survey included caregivers of adolescents (aged 11 to 17 y) and adults (aged 18 years or older) with a self-reported [or caregiver-reported (adolescents)] physician diagnosis of EoE who were receiving corticosteroids. Participants were recruited through 2 nonprofit, patient advocacy groups. The 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) was used to assess satisfaction across effectiveness, convenience, and global satisfaction domains (scale: 1 to 100 per domain); higher scores indicated greater satisfaction. The 4-item Morisky Green Levine Medication Adherence Scale (MGL-4) was used to assess adherence; an MGL-4 score of <3 indicated adherence. Participants also reported reasons for nonadherence. Overall, 201 participants (caregivers of adolescents, n=98; adults, n=103) were included in this study. Mean TSQM-9 scores indicated low satisfaction with off-label corticosteroids across all 3 satisfaction domains in adolescents (≤61.1) and adults (≤55.7). Slightly fewer adolescents (37.1%) than adults (40.8%) were considered adherent. Forgetfulness was the most frequently reported reason for nonadherence; some patients chose not to take their medications, owing to poor palatability (adolescents), difficulty taking medications at specific times (adults), or feeling depressed/overwhelmed (adolescents and adults). Satisfaction with and adherence to off-label corticosteroids were low in this web-based survey of adolescents and adults with EoE in the United States.

Patient-Reported Satisfaction with Thyroid Hormone Replacement Therapy for Subclinical Hypothyroidism in Older Adults: A Pooled Analysis of Individual Participant Data from Two Randomized Controlled Trials.

Background: The benefit of levothyroxine treatment of subclinical hypothyroidism (SCH) is subject to debate. This study compared treatment satisfaction between older adults with SCH using levothyroxine or placebo. Methods: We analyzed pooled individual participant data from two randomized, double-blind, placebo-controlled trials investigating the effects of levothyroxine treatment in older adults with SCH. Community-dwelling participants aged ≥65 years, with SCH (persistent thyrotropin levels 4.60-19.99 mIU/L for >3 months and normal free T4 level), were included. Intervention dose titration until thyrotropin levels normalized, with a mock dose adjustment of placebo. Treatment satisfaction was determined during the final study visit using the Treatment Satisfaction Questionnaire for Medication (TSQM), encompassing perceived effectiveness, side effects, convenience, and global satisfaction, along with the participants' desire to continue study medication after the trial. Results: We included 536 participants. At baseline, the median (interquartile range [IQR]) age was 74.9 (69.7-81.4) years, and 292 (55%) were women. The median (IQR) thyrotropin levels were 5.80 (5.10-7.00) mIU/L at baseline in both groups; at final visit, 4.97 (3.90-6.35) mIU/L in the placebo and 3.24 (2.49-4.41) mIU/L in the levothyroxine group. After treatment, the groups did not differ significantly in global satisfaction (mean difference [CI] -1.1 [-4.5 to 2.1], p = 0.48), nor in any other domain of treatment satisfaction. These results held true regardless of baseline thyrotropin levels or symptom burden. No major differences were found in the numbers of participants who wished to continue medication after the trial (levothyroxine 35% vs. placebo 27%), did not wish to continue (levothyroxine 27% vs. placebo 30%), or did not know (levothyroxine 37% vs. placebo 42%) (p = 0.14). In a subpopulation with high symptom burden from hypothyroid symptoms at baseline, those using levothyroxine more often desired to continue the medication after the trial than those using placebo (mean difference [CI]: -21.1% [-35.6% to -6.5%]). Conclusion: These pooled data from two RCTs showed no major differences in treatment satisfaction between older adults receiving levothyroxine or placebo. This finding has important implications for decision-making regarding initiating levothyroxine treatment for SCH. Our findings generally support refraining from routinely prescribing levothyroxine in older adults with SCH.

Patient- and Clinician-Reported Outcomes for Tirbanibulin in Actinic Keratosis in Clinical Practice Across the United States (PROAK).

The Patient-Reported Outcomes in Actinic Keratosis (PROAK) study evaluated patient- and clinician-reported outcomes (PRO; ClinRO) during 24 weeks of follow-up among adult patients with actinic keratosis (AK) on the face or scalp who were administered tirbanibulin 1% ointment in real-world community practices in the United States.&nbsp; Methods: Quality of life (QoL) was assessed by Skindex-16 at week (W) 8. Additionally, effectiveness (Investigator Global Assessment [IGA]), PRO and ClinRO (Treatment Satisfaction Questionnaire for Medication and Expert Panel Questionnaire), safety, and tolerability were assessed at W8 and W24. The safety population included 300 patients; the full analysis set included 290 patients (278 patients at W24). At W8, a statistically significant difference (P&lt;0.03) was observed for Skindex-16 domains in all assessed subgroups. Clinicians and patients reported high global satisfaction (mean [SD] scores of 74.9 [23.9] and 72.0 [24.6], respectively) at W24. Overall skin appearance improved from baseline to W24 (83.6% clinicians; 78.5% patients). IGA success (IGA score of 0-1) was achieved by 71.9% of patients at W24 with a similar % at W8 (73.8%) suggesting a stable effectiveness over time. About 5% of patients reported at least one adverse event, 4% reported at least one serious adverse event and no patients reported serious adverse drug reactions. At W8, the most frequently reported local skin reactions were mild/moderate erythema (47.6%) and flaking/scaling (49.6%). Treatment with tirbanibulin demonstrated effectiveness in the management of AK lesions and a favorable safety and tolerability profile. Furthermore, QoL was improved as early as W8, and both patients and clinicians reported high levels of treatment satisfaction, independently of patients' characteristics. J Drugs Dermatol. 2024;23(5):338-346. doi:10.36849/JDD.8264.

Patient satisfaction with 6-month paliperidone palmitate versus other long-acting injectable antipsychotics

IntroductionLong-acting injectable antipsychotics (LAIs) offer advantages for schizophrenic patients compared to oral antipsychotics: less frequent dosing, lower relapse rates, better adherence, and lower healthcare costs. LAIs include paliperidone, aripiprazole, olanzapine, risperidone, and zuclopenthixol. Paliperidone palmitate is the only antipsychotic with two formulations with an administration interval longer than one month (3-monthly and 6-monthly), which could be better for the patient and help ensure treatment continuity, especially in cases of limited access to the health care system.Objectives To assess the satisfaction of patients under treatment with 6-month paliperidone palmitate compared to other long-acting injectable antipsychotics with a higher frequency of administration.Methods We analyzed the satisfaction level of a sample of patients receiving treatment with LAIs at the Mental Health Center of El Escorial. All patients had a diagnosis of schizophrenia or other psychotic disorders (according to DSM-5). Patients who met the inclusion criteria completed the Treatment Satisfaction Questionnaire for Medication (TSQM), a generic questionnaire of treatment satisfaction that measures four dimensions: side effects, treatment efficacy, comfort of use, and overall satisfaction. Other clinical and socio-demographic variables were collected, as well as the type of injectable, dose, and frequency of administration.ResultsData from approximately 30 patients will be analyzed and discussed later.ConclusionsLess frequent administration of LAIs may result in greater patient satisfaction and be just as beneficial clinically. Treatment satisfaction is positively associated with an improvement in psychotic symptoms and seems to be related to better adherence.Disclosure of InterestNone Declared

Risdiplam therapy in adults with 5q-SMA: observational study on motor function and treatment satisfaction

BackgroundWe aimed to describe the experience of a single neuromuscular center in Germany in treating adult spinal muscular atrophy (SMA) patients with risdiplam and to analyze motor function and treatment satisfaction during a follow-up period up to 20 months.MethodsFourteen patients with type 2 or 3 SMA (seven with SMA type 2, six with SMA type 3; age range: 18–51) were included. The Revised Upper Limb Module (RULM) and the Hammersmith Functional Motor Scale Expanded (HFMSE) were recorded at baseline and at follow-up (month 4, 8, 12, 16, 20). Treatment adverse events were collected at every follow-up visit. Patients’ treatment satisfaction was assessed by the Treatment Satisfaction Questionnaire for Medication (TSQM).ResultsHalf of the patients reached the 20-month follow-up. Based on the HFMSE score, no patients had clinically meaningful improvement. Twelve remained stable (92.3%), two showed transient clinically meaningful deterioration (15.4%) and one experienced lasting clinically meaningful deterioration (7.7%). Based on the RULM scores, seven patients were either stable or demonstrated clinically meaningful improvement (53.8%) and six showed clinically meaningful deterioration (46.2%). There was no treatment withdrawal during the follow-up. The most common adverse events were skin rash/increased skin sensitivity to sunlight (n = 3), diarrhea (n = 3), aphthous ulcer (n = 3) and abdominal pain (n = 2). Most patients stated to be at least “satisfied” with the medication.ConclusionsRisdiplam was well tolerated. Half of the patients remained stable or improved after risdiplam initiation. Larger and multicentric studies are needed to better understand the long-term effects of risdiplam in adult SMA.

553 - Tralokinumab real-world patient-reported outcomes in moderate-to-severe atopic dermatitis adult patients in the United States: 6-month interim analysis

Abstract Introduction/Background Tralokinumab, an IL-13 targeted biologic approved in the United States (US) for adult patients with moderate-to-severe atopic dermatitis (AD), improved patient-reported outcomes (PROs) in clinical trials and after 4 weeks of use in the real-world setting. Objectives This 6-month interim analysis evaluated the real-world impact of tralokinumab on PROs in adult patients. Methods This is an interim analysis of an ongoing 52-week patient survey study enrolling US AD patients from the AdbryTM AdvocateTM Program. Patients completed the baseline survey close to tralokinumab initiation. Data on demographics and PROs including weekly itch numeric rating scale (NRS), eczema-related weekly sleep NRS, Dermatology Life Quality Index (DLQI), and Treatment Satisfaction Questionnaire for Medication (TSQM-9) were collected. Percent and absolute changes from baseline to 6 months of treatment were calculated. Outcomes are stratified by previous dupilumab use. The percentage of patients experiencing minimal clinically important difference (MCID) was also calculated for itch NRS (3-point reduction) and DLQI (4-point reduction). Results As of May 2023, 102 patients completed baseline, 1-month, and 6-month surveys. Of these, 59.8% were female, mean age was 44.2 years (SD=15.7), 82.4% were white, and 85.3% had private insurance. 84.3% were previously treated with topical corticosteroids (TCS) and 52.9% were dupilumab-experienced patients. Over 6 months, there was an improvement in the mean sleep interference NRS (40%), average weekly itch NRS (39%), worst weekly itch NRS (33%), PO-SCORAD (37%), and DLQI (52%). There were improvements in mean TSQM-9 global satisfaction (11.06 points), TSQM-9 convenience (3.92 points), and TSQM-9 effectiveness (14.60 points) scores. Median MCIDs were met by 57.1% on the sleep interference NRS, 51.7% of patients on the average weekly itch NRS, 50.0% on the worst weekly itch NRS, and 68.7% on the DLQI. Relative to the dupilumab-experienced cohort, the dupilumab-naïve patients showed markedly improved outcomes [median MCIDs of 65.6% v 48.4% on sleep NRS; 59.5% v 44.4% on average weekly itch NRS; 61.4% v 39.6% on worst weekly itch NRS; 61.4% v 35.3% on PO-SCORAD; and 75.6% v 61.9% on DLQI]. Conclusions This 6-month interim analysis shows that tralokinumab improved quality of life outcomes related to itch, sleep, and treatment satisfaction when used either in dupilumab-naïve or in dupilumab-experienced patients. These patients will continue to be followed for up to 52 weeks of treatment with tralokinumab.

(118) Clinical Predictors of Long-Term Satisfaction with Treatment of Collagenase Clostridium Histolyticum for Patients with Peyronie’s Disease

Abstract Introduction Collagenase clostridium histolyticum (CCH) is the only FDA-approved medication for Peyronie’s Disease (PD) in the United States. Roughly half of patients who received CCH experience improvements in curvature by more than 20% in the IMPRESS I/II trials. Current literature reports varying satisfaction rates of 67-81% within one year of treatment completion. However, little is known of long-term patient satisfaction following CCH treatment and its clinical predictors. Objective The aim of this study was to identify clinical predictors of long-term satisfaction with CCH for PD. Methods An Institutional Review Board approved retrospective chart review and standardized questionnaire was conducted for all patients with PD who received CCH from 2009-2022. Patients who received CCH before presenting to our institution were excluded. The questionnaire utilized the Treatment Satisfaction Questionnaire for Medication (TSQM), which is validated across the subdomains of satisfaction with effectiveness, side effects (SE), convenience, and global satisfaction (GS), and summed to generate the TSQM total score. Statistical analyses were performed in R using Wilcoxon rank sum test and independent T-test to compare continuous variables with TSQM scores, and Spearman’s rank correlation to compare categorical variables with TSQM scores. Results Of the 243 eligible patients, 80 (32.9%) responded (Table 1). Of these, 34 (42.5%) reported satisfaction with CCH treatment. On average, surveys were completed 4.7 (SD 2.3) years after last CCH injection. Age at presentation, at last CCH injection, and at survey completion were significantly correlated with satisfaction with side effects (Spearman's ρ= 0.32, 0.32, 0.33, respectively; p&amp;lt;0.004 for all) (Table 2). Curvature degree, plaque diameter, and plaque volume measured before CCH treatment and the amount of change in these parameters were not significantly associated with TSQM scores. However, having any improvement in curvature significantly increased the GS subdomain score without affecting the TSQM total score (GS means 37.6 change v 20.7 no change; p=0.03). Greater final curvature degree and plaque volume after CCH completion were significantly correlated with lower TSQM total scores (Spearman's ρ= -0.36, -0.32, respectively; p&amp;lt;0.04 for both). 10 (12.5%) patients reported depression, which significantly decreased scores of the effectiveness subdomain, GS subdomain, and TSQM total (p&amp;lt;0.04 for all). 10 (12.5%) patients proceeded to surgical intervention after CCH treatment and reported significantly decreased scores in the effectiveness subdomain and TSQM total (p= 0.008, 0.001, respectively). Conclusions Our data suggests that older patients exhibit greater long-term satisfaction and tolerability of side effects from CCH. Additionally, final curvature and plaque volume after CCH completion were the most important objective predictors of long-term satisfaction rather than the degree of change in these parameters. Patients with depression also reported lower long-term satisfaction rates with CCH treatment. Disclosure Any of the authors act as a consultant, employee or shareholder of an industry for: M.K. acts as a consultant for AbbVie, Marius, Tolmar, Endo, Petros, Boston Scientific, Coloplast. M.K. is an investor of Sprout. L.L. acts as a consultant for AbbVie, Aytu BioScience, Contraline, Lipocine. L.L. acts as an advisor for Inherent Bioscences. L.L. acts as consultant/speaker for Endo Pharmaceuticals. L.L. acts as a speaker for American Medical Systems/Boston Scientific.

(398) The Correlates of Satisfaction with Long-term Outcomes Following Collagenase Clostridium Histolyticum Treatment for Peyronie’s Disease

Abstract Introduction Collagenase Clostridium histolyticum (CCH) is the only FDA-approved medication for Peyronie’s Disease (PD) in the United States. Roughly half of patients who received CCH experienced improvements in curvature by more than 20% in the IMPRESS I/II trials. Despite objective improvements in PD severity, some patients still report dissatisfaction with CCH treatment. The effects of subjective reasons of greatest satisfaction and dissatisfaction on long-term overall treatment satisfaction is even less clear. Objective The aim of this study was to assess how subjective patient responses regarding reasons for satisfaction and dissatisfaction correlate to long-term satisfaction with CCH treatment. Methods An Institutional Review Board approved retrospective chart review and standardized questionnaire was conducted for all patients with PD who received CCH from 2009-2022. Patients who received CCH before presenting to our institution were excluded. The questionnaire utilized the Treatment Satisfaction Questionnaire for Medication (TSQM), which is validated across the subdomains of satisfaction with effectiveness, side effects (SE), convenience, and global satisfaction (GS), and summed to generate the TSQM total score. The questionnaire also included multiple-choice questions on CCH insurance coverage, reasons for greatest satisfaction and dissatisfaction with a free-response “other” answer choice, and likelihood of recommending CCH to other patients with PD on a scale from 0-6. Statistical analyses were performed in R using Wilcoxon rank sum test and independent T-test to compare continuous variables with TSQM scores, Spearman’s rank correlation to compare categorical variables with TSQM scores, and Fisher's exact test to compare categorical variables with one other. Results Of the 243 eligible patients, 80 (32.9%) responded (Table 1). Of these, 34 (42.5%) reported satisfaction with CCH treatment. On average, surveys were completed 4.7 (SD 2.3) years after last CCH injection. When queried on what factors patients were the most satisfied with, improvements in curvature and sexual function were significantly correlated with increased scores in the effectiveness subdomain, GS subdomain, and TSQM total (p&amp;lt;0.01 for all) (Table 2). 30 (37.5%) patients self-reported no satisfaction because of no perceived improvement in the same question, which significantly correlated with decreased scores in the effectiveness subdomain, GS subdomain, and TSQM total (p&amp;lt;0.0001 for all). Of this cohort, 26 (86.7%) patients would not recommend CCH to other patients with PD (OR 10.27, 95% CI 2.94-46.85, p=0.00002). When queried on what factors patients were the most dissatisfied with, pain with injection, duration of CCH series, and no improvements in curvature were all significantly associated with lower scores in effectiveness subdomain, GS subdomain, and TSQM total (p&amp;lt;0.003 for all). Patients with insurance coverage had lower odds of reporting cost of CCH as their greatest dissatisfaction (OR 0.05, 95% CI 0.004-0.62, p=0.03). Conclusions Our data suggests that the most important factors for long-term patient satisfaction with CCH treatment are improvements in curvature and sexual function while the most important factors for patient dissatisfaction are pain with injection, duration of CCH series, and no improvements. While pain with injection is temporally brief, it continues to negatively affect patient satisfaction even years after completion of CCH. Disclosure Any of the authors act as a consultant, employee or shareholder of an industry for: M.K. acts as a consultant for AbbVie, Marius, Tolmar, Endo, Petros, Boston Scientific, Coloplast. M.K. is an investor of Sprout. L.L. acts as a consultant for AbbVie, Aytu BioScience, Contraline, Lipocine. L.L. acts as an advisor for Inherent Bioscences. L.L. acts as consultant/speaker for Endo Pharmaceuticals. L.L. acts as a speaker for American Medical Systems/Boston Scientific.