Treatment Satisfaction Questionnaire For Medication Research Articles

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.

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.

(115) Side Effect Profile of Collagenase Clostridium Histolyticum for Peyronie’s Disease Significantly Affects Long-term Patient Satisfaction

Abstract Introduction Collagenase Clostridium histolyticum (CCH) is the only FDA-approved medication for Peyronie’s Disease (PD) in the United States. While treatment-related side effects are commonly considered mild or moderate in severity, including bruising, pain, and swelling, there is currently limited literature on how these side effects affect patient satisfaction, especially long-term satisfaction, with CCH treatment. Objective The aim of this study was to better understand the side effect profile of CCH treatment for PD and its effects on 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. 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. Patients experienced an average of 2.2 (SD 1.5) notable side effects, and increased number of side effects was significantly correlated with decreased scores of SE subdomain, convenience subdomain, and TSQM total (Spearman's ρ= –0.46, –0.29, –0.28; p&amp;lt;0.009 for all). Most common side effects noted were bruising, swelling, and pain with injection (Table 2). Other than swelling, all SE noted were associated with significantly lower SE subdomain scores (p&amp;lt;0.03 for all). New erectile dysfunction (ED) was correlated with the largest reduction in the TSQM total score (mean 105.4 ED vs. 192.1 no ED, p=0.006). Hematoma and pain with injection were the only other side effects associated with significant reductions in the TSQM total score (p=0.03 for both), and experiencing no SE was correlated with significantly increased TSQM total scores (mean 256.1 no SE vs. 166.7 SE, p=0.003). When rating pain with injection on a scale 1 to 5, mean rating was 3.1 (SD 1.2), and rating was significantly associated with scores of SE subdomain, convenience subdomain, and TSQM total (Spearman's ρ= -0.33, –0.52, –0.33; p&amp;lt;0.003 for all). Conclusions Our data suggests that impairment of sexual function through new ED has the most significant negative impact on long-term overall satisfaction with CCH treatment, followed closely by pain with injection and hematoma. Though the latter two side effects are typically considered mild to moderate in severity, they continue to profoundly impact overall satisfaction nearly five years after completion of 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.

Rationale and design of the NODE-303 study: evaluating the safety of symptom-prompted, self-administered etripamil for paroxysmal supraventricular tachycardia episodes in real-world settings

Paroxysmal supraventricular tachycardia (PSVT) is a common episodic arrhythmia characterized by unpredictable onset and burdensome symptoms including palpitations, dizziness, chest pain, distress, and shortness of breath. Treatment of acute episodes of PSVT in the clinical setting consists of intravenous adenosine, beta-blockers, and calcium channel blockers (CCBs). Etripamil is an intranasally self-administered L-type CCB in development for acute treatment of AV-nodal dependent PSVT in a non-medical supervised setting. This paper summarizes the rationale and study design of NODE-303 that will assess the efficacy and safety of etripamil. In the randomized, double-blinded, placebo-controlled, Phase 3 RAPID trial, etripamil was superior to placebo in the conversion of single PSVT episodes by 30 minutes post initial dose when administered in the non-healthcare setting; this study required a mandatory and observed test dosing prior to randomization. The primary objective of NODE-303 is to evaluate the safety of symptom-prompted, self-administered etripamil for multiple PSVT episodes in real-world settings, without the need for test dosing prior to first use during PSVT. Secondary endpoints include efficacy and disease burden. Upon perceiving a PSVT episode, the patient applies an electrocardiographic monitor, performs a vagal maneuver, and, if the vagal maneuver is unsuccessful, self-administers etripamil 70 mg, with an optional repeat dose if symptoms do not resolve within 10 minutes after the first dose. A patient may treat up to four PSVT episodes during the study. Adverse events are recorded as treatment-emergent if they occur within 24 hours after the administration of etripamil. Efficacy endpoints include time to conversion to sinus rhythm within 30 and 60 mins after etripamil administration, and the proportion of patients who convert at 3, 5, 10, 20, 30, and 60 min. Patient-reported outcomes are captured by the Brief Illness Perception Questionnaire, the Cardiac Anxiety Questionnaire, the Short Form Health Survey 36, the Treatment Satisfaction Questionnaire for Medication and a PSVT survey. Overall, these data will support the development of a potentially paradigm-changing long-term management strategy for recurrent PSVT.

Tralokinumab Real-World Patient-Reported Outcomes in Moderate-to-Severe Atopic Dermatitis Adult Patients in the United States: 6-Month Interim Analysis

Introduction: 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. This 6-month interim analysis evaluated the real-world impact of tralokinumab on PROs  in adult patients.&#x0D; 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).&#x0D; 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].&#x0D; Conclusion: 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.

Patient Satisfaction with Oral Testosterone Undecanoate in Men Who Received Prior Testosterone Therapy: An Open-Label, Single-Center Clinical Trial.

To evaluate patient satisfaction and symptom control in hypogonadal men transitioning from other testosterone therapies to oral testosterone undecanoate (TU). In this open-label clinical trial, men aged 18 to 75 years with hypogonadism were switched to oral TU after a sufficient washout of previous testosterone therapies. Treatment satisfaction and symptom control were primarily measured using the 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) and quantitative androgen deficiency in aging males (qADAM) questionnaires, respectively. Secondary outcomes included changes in serum testosterone (T), estradiol (E2), hematocrit (HCT), and prostate-specific antigen (PSA) levels. Forty-one men participated, with significant improvements in all TSQM-9 scores observed over 6 months. Symptom control as measured by qADAM remained consistent. There was a significant increase in serum T and E2 levels, but HCT and PSA levels remained stable. Switching to oral TU from other testosterone therapies is associated with increased patient satisfaction and stable hypogonadal symptom control.

Treatment access and satisfaction on disease-modifying therapies of neuromyelitis optica spectrum disorder patients in China: a cross-sectional survey.

Neuromyelitis optica spectrum disorder (NMOSD) is a rare and debilitating disease that has become more widely recognized in China. Legislative measures have been implemented by the government to improve treatment access for rare diseases. To investigate the diagnostic journey, treatment status, treatment accessibility, and treatment satisfaction of the NMOSD patients on disease-modifying therapies (DMTs) in China. A patient online survey. This cross-sectional online survey was conducted between November 2022 and January 2023. Patients over 18 years old and diagnosed with NMOSD were included. The questionnaire consisted of five sections covering demographics, diagnostic and treatment experiences, DMTs availability, cost and affordability, and treatment satisfaction using the Treatment Satisfaction Questionnaire for Medication (version II). Patient opinions and demands were also collected at the end of the survey. A total of 375 patients diagnosed with NMOSD were recruited, of which 321 patients used DMTs. It required 1.22 ± 3.22 years and 3.58 ± 4.24 hospital visits for a definitive diagnosis. One-third of the patients still needed to travel for over 2 h to access DMTs. The total treatment expenditure was estimated to be CNY 59,827.00 (USD 8315.95) a year. Drug expenses alone accounted for 52.22% of the average annual household income. The most common challenges perceived were the inability to afford treatment and a lack of effective options. No significant difference was found in treatment satisfaction among DMTs, except that rituximab scored lowest in convenience compared to other DMTs. Patients' age and travel time required to obtain medications were negatively associated with global treatment satisfaction. In China, patients with NMOSD face challenges in obtaining proper treatment due to diagnostic difficulties, distant medication access, and high costs. Policies should prioritize improving disease education and alleviating financial burdens for the patients.

Disease burden and treatment satisfaction in patients with prurigo nodularis in Japan.

Prurigo nodularis (PN) is a chronic inflammatory skin disorder with a high disease burden. In this cross-sectional, web-based survey, Global Questions (GQ), the Numerical Rating Scales (NRS) for pruritus, burning sensation and sleep disturbance, the Short-Form-8 (SF-8) Health Survey, Dermatology Life Quality Index (DLQI), Patient Health Questionnaire 9 (PHQ-9), Work Productivity and Activity Impairment (WPAI), and Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) scores were used to assess the current disease burden and treatment satisfaction among patients with PN in Japan. In total, 97 patients were included (55.7% male, median age 51 years, median duration of PN 36 months). Based on GQ scores, 35.1% of patients had mild disease, 50.5% moderate, and 14.4% severe disease. Disease burden increased as the severity of PN increased, as indicated by worsening of pruritus NRS scores and quality of life (DLQI, PHQ-9, WPAI presenteeism, work productivity loss, and activity impairment scores). Patients with comorbid atopic dermatitis (AD) also had more intense pruritus than those without AD. Mean ± standard deviation TSQM-9 scores for effectiveness, convenience, and global satisfaction were 54.7 ± 18.1%, 62.4 ± 15.2%, and 57.4 ± 15.9%, respectively. TSQM-9 scores were lowest in patients receiving the most intensive guideline-directed treatment (i.e., topical corticosteroids + systemic oral corticosteroids or cyclosporine), highlighting an unmet need for more effective treatment options for patients with PN. In summary, Japanese patients with PN reported increased disease burden and reduced treatment satisfaction with increased disease severity, despite the use of guideline-recommended therapies.

Preliminary Evidence of Efficacy, Safety, and Treatment Satisfaction with Tirbanibulin 1% Ointment: A Clinical Perspective on Actinic Keratoses.

Actinic keratosis is a common precancerous skin lesion that can progress into invasive squamous cell carcinomas. Many topical treatments for actinic keratoses often have poor tolerability and prolonged duration. Tirbanibulin is a novel synthetic drug with potent antitumor and antiproliferative activities. We conducted a single-center, prospective and observational study using tirbanibulin ointment on a 25 cm2 area for 5 consecutive days on 30 participants with AKs on the face or scalp. They were followed for at least 57 days to assess the safety profile and efficacy of the drug as well as treatment satisfaction. We evaluated six signs of local skin reaction (LSR): erythema, scaling, crusting, swelling, blisters/pustules, and erosions/ulcerations, grading the severity as mild, moderate, or severe. The effectiveness was evaluated both clinically and dermoscopically. The treatment satisfaction was assessed using the Treatment Satisfaction Questionnaire for Medication (TSQM 1.4). On day 57, 70% of the patients showed a complete clinical and dermoscopic response. The highest scores obtained from the TSQM 1.4 were more evident in the convenience and side effects domains. Most LSRs, including erythema (83.3%), scaling (30%), and swelling (3.3%), occurred on day 8 but resolved spontaneously. Tirbanibulin is a viable therapeutic option with a short regimen treatment and good tolerability, which favors therapy adherence.

Paradoxical Findings in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors: Suboptimal Disease Control Perceived As Satisfactory

Introduction: The introduction of C5 inhibitors (C5i) for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) has resulted in lower morbidity and mortality rates. However up to 86% of patients still experience anemia and fatigue in 63% of them which may impact their quality of life (QoL). The SATURNO study aimed to assess PNH patient and physician satisfaction with C5i treatment, along with the impact of the disease and clinical outcomes. Methods: A multicenter, observational, cross-sectional study conducted in PNH patients receiving C5i treatment for at least 3 months in Spain. Key clinical and laboratory data were collected, including red blood cells (RBC) transfusions, hemolytic crisis, hemoglobin (Hb), haptoglobin (Hp), lactate dehydrogenase (LDH), absolute reticulocyte count (ARC) and bilirubin. Patient satisfaction was assessed using the Treatment Satisfaction Questionnaire for Medication (TSQM) and Visual Analog Scale (VAS). Physician satisfaction was measured using the TSQM general item and VAS. Fatigue and QoL were evaluated using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), respectively. The Work Productivity and Activity Impairment questionnaire (WPAI) was also used. Results: A total of 56 patients and 13 physicians participated in the study. Median (IQR) age of patients was 54.0 (44.5; 63.5) years, 35.7% were female and median (IQR) duration of treatment with C5i was 6.6 (3.3; 11.6) years. Overall, 94.6% of patients and 90.6% of physicians reported to be satisfied, very satisfied, or extremely satisfied with the treatment. Mean (SD) VAS score for patient satisfaction was 9.0 (1.2) and for physicians 7.9 (1.7) out of 10. Mean (SD) EORTC QLQ-C30 score was 71.7 (21.7) out of 100. Mean (SD) FACIT-F score was 39.7 (10.6) out of 52 and fatigue was reported by 55.2% of patients. Clinical and laboratory parameters showed a significant proportion of patients exhibiting abnormal values. Hb and Hp were below the normal ranges in 67.9% and 65.1% of patients, respectively, whereas elevated LDH, ARC and indirect bilirubin were observed in 50.0%, 83.0% and 75.9% of them, respectively. In the last 12 months, 30.9% of patients had received at least one RBC transfusion, with a mean (SD) number of 8.6 (8.4) RBC transfusions, and 33.9% experienced at least one hemolytic crisis with a mean (SD) of 4.9 (9.4). Work productivity assessment revealed that 60.7% of patients were currently unemployed, a mean (SD) percentage of absenteeism of 5.7% (12.4) and presenteeism of 23.8% (26.0). Overall mean (SD) percentage of work productivity impairment was 25.6% (27.3). Activity impairment was reported by 36.4% (28.3) of patients. Conclusions: The SATURNO study showed that although patients and physicians reported satisfaction with C5i treatment, this might be challenged. A substantial number of PNH patients treated with C5i still experience anemia, fatigue and abnormal laboratory values, indicative of ongoing hemolysis and suboptimal control. Moreover, patients with PNH had affected work productivity and a proportion of them were experiencing activity impairment. These findings highlight the need for comprehensive management strategies to address the impact of the disease on patients with PNH receiving C5i treatment.

Hereditary angioedema outcomes in US patients switched from injectable long-term prophylactic medication to oral berotralstat

BackgroundBerotralstat, a first-line, once-daily, oral plasma kallikrein inhibitor for long-term prophylaxis of hereditary angioedema (HAE), is an effective and well-tolerated treatment option. ObjectiveTo summarize the safety, effectiveness, and impact on treatment satisfaction in patients who switched from injectable long-term prophylactics to oral berotralstat monotherapy (150 mg daily) at US sites in the international open-label APeX-S study. MethodsAPeX-S was an open-label, Phase II study of berotralstat conducted in 22 countries. Here, we focus on APeX-S patients enrolled at US sites who switched from injectable long-term prophylactics to berotralstat 150 mg once-daily monotherapy. ResultsA total of 34 patients discontinued lanadelumab (n = 21), subcutaneous C1 esterase inhibitor (n = 11), or intravenous C1 esterase inhibitor (n = 2) and switched to berotralstat 150 mg monotherapy. Vomiting, diarrhea, and upper respiratory tract infection were the most common adverse events (each 11.8%). Mean monthly attack rates were consistently low after the switch to berotralstat. The mean (SEM) monthly attack rate was 0.29 (0.11) at Month 1, 0.48 (0.15) at Month 6, and 0.58 (0.23) at Month 12. The median attack rate was 0 attack/mo throughout 12 months of treatment. Improvements were observed in the Treatment Satisfaction Questionnaire for Medication from baseline to Month 12 after the switch to berotralstat monotherapy, with the greatest improvements in convenience. ConclusionThe transition from injectable prophylactic medication to berotralstat was generally well tolerated. Patients switching to berotralstat monotherapy maintained good control of their HAE symptoms and reported improved treatment satisfaction. Trial RegistrationClinicalTrials.gov Identifier: NCT03472040.

Treatment satisfaction of adult patients with moderate-to-severe atopic dermatitis treated with baricitinib in France, Germany, and the United Kingdom: results from a cross-sectional international patient survey

IntroductionWe explored patient satisfaction with baricitinib, an oral Janus kinase inhibitor, in patients with atopic dermatitis (AD) treated in routine clinical practice.MethodsAdults with moderate-to-severe AD treated with baricitinib in clinical practice for ≥4 weeks in France, Germany, and the UK completed a one-time online survey under market research methodologies. Treatment satisfaction was assessed using a Likert scale and abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9). Patients reported demographic, disease, and treatment information. Data were analyzed descriptively.ResultsThe survey was completed by 170 patients with a mean age of 39.3 years (SD = 13.5), 59% (n = 101) were female. At baricitinib initiation, 79% rated their AD as “Severe”, yet 28% reported body surface area (BSA) involvement ≥10%. Most were “Satisfied” or “Very satisfied” (76%/18%) with baricitinib, with high rates reported for controlling itch (36%/56%). Itch improvements were noted by 97% of patients. Some tapered/stopped (50%/32%) topical corticosteroid use, aligned with reported improvements on the patient global assessment and BSA. Mean TSQM-9 convenience score was 78.0 (SD = 14.0).ConclusionsSatisfaction with itch control was particularly high, reflected in rates of improvement in itch since starting baricitinib. On the TSQM-9, the convenience score was the highest. Many patients tapered/stopped concomitant topicals, indicating baricitinib’s effect in controlling AD symptoms.

Patient- and Clinician-Reported Outcomes of Tirbanibulin 1% in the Treatment of Actinic Keratosis on the Face and Scalp (PROAK Study)

Introduction: Actinic Keratosis (AK) has been shown to negatively affect emotional and social functioning and skin-related quality of life [1]. The objective was to evaluate clinician- and patient-reported outcomes (ClinRO; PRO) for tirbanibulin treatment satisfaction and effectiveness among patients administered tirbanibulin in routine clinical practice across the U.S.&#x0D; Methods: A single-arm, prospective cohort study (PROAK: NCT05260073) was conducted in adults with AKs on the face or scalp as part of usual care. Patients and clinicians completed surveys and clinical assessments at baseline, Week-8, and Week-24. ClinRO and PRO comprised Treatment Satisfaction Questionnaire for Medication (TSQM-9) (with 3 domains: treatment effectiveness, convenience of use, and global satisfaction with treatment) and Expert Panel Questionnaire (EPQ) (assessing overall skin appearance, satisfaction with improvement in “skin texture” and “how skin looks”, and likelihood to consider tirbanibulin again). Clinicians assessed AK responses using Investigator's Global Assessment (IGA) and patient’s skin photodamage severity scale. IGA success was defined as achieving IGA score of 0-1 (≥75% AK lesions clearance). Data at Week-8 was already published [1]. Here we present the results obtained at Week-24.&#x0D; Results: A total of 278 patients completed study assessments at Week-24 (mean age: 66.3 years; 68.6% males; Fitzpatrick skin type II: 71.4%). Mean (standard deviations [SD]) TSQM-9 score in effectiveness, convenience and global satisfaction was 74.3 (21.2), 84.5 (15.6) and 74.9 (23.9) for clinicians and 73.4 (21.4), 85.0 (14.7) and 71.9 (24.7) for patients. 77.3% of clinicians and 78.4% of patients reported a high likelihood for considering tirbanibulin treatment in the future. At Week-24, 83.6% of clinicians and 78.5% of patients rated overall skin appearance after tirbanibulin treatment to be mostly somewhat/much improved. At Week-24, the proportion of patients with completely/partially cleared AK (IGA 0/1) was 71.9%, similar to that obtained at Week-8 (73.8%) [1].&#x0D; Conclusions: Effectiveness ≥75% clearance was achieved by 71.9% of patients, consistent with the results of the Phase III trials [2]. ClinRO and PRO demonstrated high satisfaction with once-daily tirbanibulin treatment for 5 consecutive days at Week-24, and both clinicians and patients reported a desire to consider tirbanibulin treatment in the future.&#x0D; [1] Schlesinger T et al. Skin. 2023;7(3):771-787.&#x0D; [2] Blauvelt A et al. N Engl J Med. 2021;384(6):512–20.

Efficacy, safety, and patient satisfaction with oral semaglutide: first single-centre clinical experience.

To explore the effects of oral semaglutide on glycaemic parameters, body weight, and satisfaction in the first recipient patients with type 2 diabetes mellitus in Slovenia, in a real-world clinical practice setting. The first consecutive adult patients with type 2 diabetes who were eligible for oral semaglutide treatment were included in this prospective, open-label interventional study. Patients received increasing doses of oral semaglutide and were evaluated at inclusion, at 1 month, then 3-5 months after starting treatment. Fasting blood glucose, glycosylated haemoglobin (HbA1c), body weight, patient satisfaction with oral semaglutide treatment (using the validated Treatment Satisfaction Questionnaire for Medication), and adverse effects, were analysed. Statistical analyses were performed using one-way analysis of variance, and, when significant interactions were found, Bonferroni post-hoc test. A P-value <0.05 was considered statistically significant. Twenty patients (11 male: 9 female; mean age, 59.9 ± 1.5 years; mean diabetes duration, 8.5 ± 1.4 years) were included. Oral semaglutide (7 and 14 mg) significantly decreased HbA1c (from 9.4 ± 0.3% to 8.2 ± 0.2% and 7.8 ± 0.2%, respectively) and fasting plasma glucose (from 11.2 ± 0.5 mmol/L to 9.2 ± 0.7 mmol/L and 8.9 ± 0.4 mmol/L, respectively). Oral semaglutide (14 mg) significantly decreased body weight (from 100.9 ± 2.7 kg to 92.7 ± 2.4 kg). Patients reported that treatment was easy to use and expressed high global satisfaction. Mild and transient, mostly gastrointestinal, adverse effects were reported in 10 patients. Oral semaglutide, the first oral glucagon-like peptide 1 receptor agonist, was effective and safe, and associated with high patient satisfaction, in its first recipients in Slovenia. The results are important for daily clinical practice involving patients with type 2 diabetes, however, due to the small study population, lack of placebo control, and short exposure to oral semaglutide, the effectiveness of oral semaglutide in clinical practice requires further investigation.

THU070 Randomized, Placebo-controlled Phase 3 Trial Protocol Assessing The Efficacy And Safety Of Octreotide Subcutaneous Depot (CAM2029) In Patients With Acromegaly

Abstract Disclosure: D. Ferone: Advisory Board Member; Self; Recordati, Novartis-AAA, Sandoz, Camurus. Grant Recipient; Self; Bristol-Myers Squibb, Novartis-AAA, Ipsen, Camurus. Research Investigator; Self; Camurus. P. Freda: Research Investigator; Self; Camurus. L. Katznelson: Advisory Board Member; Self; Recordati, Camurus, Amryt. Research Investigator; Self; Camurus. F. Gatto: Research Investigator; Self; Camurus. A. Gilis-Januszewska: Research Investigator; Self; Camurus. M.B. Gordon: Advisory Board Member; Self; Camurus, Crinetics, HRA Pharma, Novo Nordisk, Recordati. Grant Recipient; Self; Amryt, Ascendis, Camurus, Corcept, Crinetics, Ipsen, Novartis Pharmaceuticals, Novo Nordisk, Opko, Pfizer, Inc., Strongbridge. Research Investigator; Self; Camurus. P. Kadioglu: Research Investigator; Self; Camurus. P. Maffei: Research Investigator; Self; Camurus. J.R. Seufert: Research Investigator; Self; Camurus. J. Silverstein: Advisory Board Member; Self; Amryt, Recordati. Research Investigator; Self; Camurus. J.L. Spencer-Segal: Grant Recipient; Self; Camurus. Research Investigator; Self; Camurus. M. Harrie: Employee; Self; Camurus. A. Svedberg: Employee; Self; Camurus. F. Tiberg: Employee; Self; Camurus. Extended-release formulations of the somatostatin analogues (SSAs), octreotide and lanreotide, are first-line medical therapies for patients with acromegaly who have contraindications for, or are not cured by, surgical intervention and/or radiotherapy. Treatment goals, regardless of modality, include biochemical normalization of insulin-like growth factor 1 (IGF-1) and growth hormone (GH), symptom relief, tumor control, and maintenance of pituitary function. From the patient perspective, treatment satisfaction, autonomy, and quality of life may be as important as biochemical control. Constraints of current SSA formulations include incomplete biochemical and symptom control, and a lack of convenient self-administration options. Octreotide subcutaneous (SC) depot (CAM2029) is a novel FluidCrystal® formulation of octreotide designed for easy administration by patients, in a ready-to-use prefilled syringe or injection pen. In previous clinical trials, CAM2029 provided more than five-fold higher bioavailability compared to the currently marketed long-acting octreotide formulation.The efficacy and safety of CAM2029 in acromegaly is being evaluated in a 6-month, randomized, double-blind, placebo-controlled phase 3 trial (NCT04076462). 72 patients were randomized 2:1 to monthly SC doses of CAM2029 20 mg or placebo, with the option for self-administration. Key inclusion criteria included a biochemical and clinical diagnosis of acromegaly due to a pituitary adenoma with persistent or recurrent disease, treatment with a stable dose of octreotide long-acting release or lanreotide Autogel for at least 3 months as monotherapy, and controlled disease with IGF-1 ≤1 x upper limit of normal (ULN). Key exclusion criteria included GH ≥2.5 μg/L at screening, pituitary surgery within 6 months of screening, prior pituitary irradiation, and recent treatment with pasireotide, pegvisomant, or dopamine agonists. The primary endpoint is the proportion of patients with normalized IGF-1 (≤1 x ULN) after 6 months of treatment. Other endpoints include change from baseline of IGF-1, GH and patient-reported outcomes (Treatment Satisfaction Questionnaire for Medication, Self-Injection Assessment Questionnaire, Acromegaly Quality of Life Questionnaire, EQ-5D-5L), safety and tolerability, clinical signs and symptoms of acromegaly, use of rescue medication and octreotide pharmacokinetics. The estimated primary completion date for the trial is May 2023. After completing study treatment, patients may switch to open-label treatment in a phase 3 long-term safety and extension trial of CAM2029 (NCT04125836). Presentation: Thursday, June 15, 2023