Treatment Adherence Research Articles

Drug utilization and medication adherence for the treatment of psoriatic arthritis: an Italian study.

This study aims to evaluate the persistence, treatment adherence and drug cost associated with biologic and targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs) in the management of psoriatic arthritis (PsA) in Italy, with a focus on biosimilar drugs. This was a retrospective observational study involving eight hospital pharmacies, between January 2017 and December 2020, on naïve patients with at least one b/tsDMARD dispensation indicated for PsA. Patients were followed up for 12 months and persistence and adherence were evaluated by proportion of days covered (PDC). The originator and biosimilar for adalimumab and etanercept were compared. Furthermore, the real annual cost per patient based on adherence to therapy was calculated. Patients initiating b/tsDMARDs for PsA had a mean persistence of 263 days and 48.6% remained persistent for 1 year. Adherent patients (PDC ≥ 0.8) were 47.6% for the overall population. Similar persistence and adherence were observed between patients treated with the adalimumab originator and its biosimilar, while patients treated with the etanercept originator showed lower persistence and adherence compared to those treated with its biosimilar (mean persistence: 222 vs. 267 days, patient persistent at 1 year: 29.4% vs. 51.5%, mean PDC: 0.53 vs. 0.70, adherent patients: 23.5% vs. 51.5%). The average annual drug cost ranged from €8,724 (etanercept) to €14,783 (ustekinumab), with an annual saving of more than €2,500 by using biosimilars. Poor adherence to medications contributes to suboptimal clinical outcomes. The comparison between biosimilar and originator offers further evidence in support of the biosimilar to optimizing resources in healthcare.

Acupuncture Treatment for Chronic Post-Traumatic Headache in Individuals with Mild Traumatic Brain Injury: A Pilot Study.

Chronic post-traumatic headache (CPTH) after a mild traumatic brain injury (mTBI) has been reported in up to 60% of patients and can be extremely debilitating. While pharmacological treatments are typically used for CPTH, they frequently cause side effects and have limited effectiveness, leading individuals with CPTH to be unsatisfied with current treatment options and to seek nonpharmacological options. Acupuncture has been identified as a potential treatment option; however, the evidence in this population remains limited. The overall goal of this study was to examine the effect of a once weekly (e.g., low dose) versus twice weekly (e.g., high dose) of acupuncture treatment on CPTH in individuals with mTBI. Thirty-eight individuals were randomized to receive either 5 or 10 acupuncture treatments using a standard protocol over 5 weeks. The protocol consisted of 14 points using traditional acupuncture and 4 points using electroacupuncture. Headache outcomes, safety, treatment adherence, sleep quality, and quality of life (QOL) were assessed. The results showed that while there were no differences between dose groups for any of the outcomes assessed, acupuncture significantly reduced the number of headache days and headache pain intensity in individuals with CPTH. There were no significant changes in acute medication use or sleep quality. While there were some QOL improvements identified, these results should be interpreted with caution. Overall, acupuncture was shown to be safe and well-tolerated in people with CPTH after mTBI, and five acupuncture treatments using a standardized protocol shows promise in providing headache relief for this population.

Drug-Induced Acne in Inflammatory Bowel Disease: A Practical Guide for the Gastroenterologist.

Drug-induced acne is a common side effect to a wide array of pharmacological therapies and is characterized by a monomorphic, papulopustular eruption typically affecting the face, scalp, and the upper thorax. Corticosteroids and Janus kinase inhibitors (JAKi) are commonly used for the treatment of inflammatory bowel disease (IBD) and are known to aggravate a prior tendency to acne or trigger the development of new acneiform eruptions. Recent attention on managing drug-induced acne has been driven by the increasing use of JAKi, an expanding therapeutic class in IBD and several other immune-mediated inflammatory diseases. Both randomized controlled trials and real-world studies have identified acne as one of the most common treatment-emergent adverse events in JAKi. Left untreated, this common skin reaction can significantly impact patient self-esteem and quality of life leading to poor treatment adherence and suboptimal IBD control. This review examines the characteristics of drug-induced acne in IBD treatments, provides a practical guide for gastroenterologists to manage mild to moderate occurrences, and highlights when to seek specialist dermatology advice. Such approaches enable early treatment of a common and often distressing adverse event and optimizes the management of IBD by preventing the premature discontinuation or dose reduction of efficacious IBD drugs.

Impact of pharmacist-led intervention in medication adherence and inhaler usage on asthma and chronic obstructive pulmonary disease control: a quasi-experimental study.

Despite recent advances in the management of asthma and chronic obstructive pulmonary disease (COPD), patients still experience suboptimal disease control largely due to medication non-adherence and inappropriate use of inhaler. This study evaluates the impact of pharmacist-led intervention in medication adherence and inhaler usage on asthma and COPD control among out-patients attending the premier tertiary hospital in Nigeria. A quasi-experimental study carried-out among eligible out-patients attending pulmonology clinic of University College Hospital, Ibadan. Baseline questionnaire explored medication adherence using a comprehensive-medication-adherence-assessment-scale (CMAAS-12) developed by the study co-investigators, use of pressurized-metered-dose (pMDI) and Diskus inhalers, as well as asthma/COPD control using validated asthma control test (ACT) and COPD assessment test (CAT). Subsequently, patients were allocated into control (n = 65) or intervention group (n = 65) using odd or even number. Intervention group received 2-month follow-up educational and/or cognitive-behavioural interventions to resolve identified adherence barriers, while control group continued with traditional care. Descriptive statistics, Chi-square and Wilcoxon-signed-ranked tests were used for analysis at p < 0.05. Overall, patients with optimal adherence were 11(18.6%) and 16(27.1%), p = 0.132 (control), but 20(33.3%) and 38(63.3%), p < 0.001 (intervention) at baseline and post-baseline, respectively. Specifically, in the intervention group, the identified adherence barriers at baseline were summarized into knowledge (120;40.4%), practical (115;38.7%) and attitudinal (62;20.9%). Patients with correct use of pMDI were 11(21.6%) baseline and 19(36.5%) post-baseline, p = 0.011 (control), but 13(22.8%) and 46(80.7%) respectively, p < 0.001 (intervention). Correct use of Diskus inhaler were 5(50.0%) and 4(40.0%), p = 0.157 (control), but 7(35.0%) and 14(70.0%), p = 0.025 (intervention) at baseline and post-baseline, respectively. Patients with 'well-controlled asthma' were 25(44.6%) and 26 (47.3%), p = 0.025 (control), but 18(35.3%) and 32(60.4%), p < 0.001 (intervention) at baseline and post-baseline, respectively. The COPD-specific health status indicated that 0(0.0%) and 1(14.3%), p = 0.059 (control), but 0(0.0%) and 7(50.0%), p < 0.001 (intervention) at baseline and post-baseline, respectively, belonged to 'low COPD impact'. Pharmacist-led intervention significantly enhanced medication adherence and appropriate use of inhaler among the intervention cohort, with subsequent significant improvement in asthma control and reduced COPD impact compared with the control group. This underscores the need for active involvement of pharmacists in collaborative management of patients with chronic respiratory diseases in clinical practice. ClinicalTrials.gov identifier: NCT06417931. Retrospectively-registered.

Physical Activity in Mild Vascular Cognitive Impairment: Results of the AFIVASC Randomized Controlled Trial at 6 Months.

Vascular cognitive impairment is frequent, in mild (mVCI) or severe forms (vascular dementia). To do a randomized controlled-trial to evaluate the impact of physical activity on cognition (primary outcome), neurocognitive measures, quality of life, functional status, and physical function (secondary outcomes), in patients with mVCI. A hundred and four patients with mVCI (mean age 71.2 years; 53 women) were randomized for a six-month intervention of moderate physical activity (60-minute sessions, 3 times/week) (n = 53) or best-practice "usual care" (n = 51). Comprehensive evaluations of primary and secondary outcomes included an objective measure of physical activity through accelerometry at baseline and after intervention. Mean session attendance was 58%. Adverse events were negligible. After 6 months, no significant primary outcome change was observed, either in the intervention or 'usual care' group. The intervention group improved significantly in some secondary outcomes in physical function - aerobic capacity (U = 403; p = 0.000) and agility (U = 453; p = 0.005) after 6 months. Regardless of randomization arm, a post-hoc analysis based on fulfilling at least 21.5 minutes/day of moderate or 10.7 minutes/day of vigorous physical activity (World Health Organization-WHO standards) revealed improvements. These were not only in motor capacity but also on the global measure of cognition, executive functions and memory. Physical activity was safe and beneficial regarding domains of physical function. No significant cognitive decline was registered over 6-months, regardless of intervention allocation. Larger samples, longer follow-ups and focus on intervention adherence are needed to fully analyze the impact of WHO recommendations for physical activity in mVCI populations.

Effectiveness of Antileishmanial Treatments in Three Endemic Areas of Colombia: A Community-Based Cohort Study.

The effectiveness of treatment of cutaneous leishmaniasis (CL) in rural settings remains underexplored. This study assessed the effectiveness of standard antileishmanial treatments using a community-based approach supported by mobile health (mHealth) in three rural areas of Colombia. From January 2018 to September 2021, we assessed treatment outcomes, adherence, and adverse drug reactions in CL patients, with the support of the Guaral+ST app. Treatment decisions were made by providers at health facilities at each site in accordance with national guidelines, whereas treatment follow-up and presumptive case identification were made by trained community leaders and health agents at the community level. In total, 231 participants received antileishmanial treatment (63 received miltefosine, 110 meglumine antimoniate, and 58 pentamidine). Disease presentation was mild (median number of lesions = 1, interquartile range [IQR]: 1-2) and of short duration (1.5 months, IQR: 1-3). The strategy yielded information on the therapeutic outcomes in 81% of study participants. Effectiveness, measured as the proportion of cure at 90 to 180 days, was 86.3% (95% CI: 73.3-93.48) for miltefosine; 77.6% (67.5-85.3) for meglumine antimoniate, and 73.1% (59.0-83.6) for pentamidine. The effectiveness of pentamidine in children ≤10 years old was 79.4% (61.6-90.3). This is one of the few reports of effectiveness of pentamidine in children with prospective data collection in the Americas. Adverse drug reactions occurred in 32% of patients, most frequently with meglumine antimoniate. Our findings demonstrate that standard antileishmanial treatments are effective in rural areas where the disease is endemic and that mHealth has a pivotal role in improving patient follow-up and data collection on therapeutic outcomes.

Adherence to antiretroviral therapy among HIV patients at a hospital in Tacna, Peru

ObjectiveThis study aims to provide an overview of antiretroviral therapy adherence and its related factors within a hospital in southern Peru. Materials and methodsWe conducted an analytical cross-sectional study spanning from April to October 2022. This study involved a review of medical records and the administration of a survey to individuals living with Human Immunodeficiency Virus. Adherence to antiretroviral treatment was assessed using the CEAT-HIV (Questionnaire for the Evaluation of Antiretroviral Treatment Adherence), and was categorized as high, adequate, insufficient, or low. The statistical analysis included the calculation of crude and adjusted prevalence ratios (PRs). ResultsOur study comprised 300 participants, with a median age of 30 years, predominantly male (78.3 %). Among these individuals, 83 % exhibited low or insufficient adherence to antiretroviral treatment according to the CEAT-VIH scale. Notably, patients with a viral load >50,000 copies/ml scored higher on the CEAT-VIH scale than those with a viral load <400 (adjusted PR: 2.78, 95 % CI: 1.21 to 6.40). Furthermore, a higher level of education and a viral load below 400 copies/ml were associated to higher scores in the treatment compliance dimension of the CEAT-VIH scale. ConclusionEight out of ten evaluated patients exhibited low or insufficient adherence. While lower adherence was correlated with lower education, the connection between viral load and adherence remains inconclusive.

The Effectiveness and Safety Analysis of Duloxetine in Treating Comorbid Depression in Parkinson's Disease: A Retrospective Study.

Parkinson's disease (PD) is a neurodegenerative disorder characterized by both motor and non-motor symptoms, including depression, which significantly impacts the quality of life of affected individuals. This study aims to investigate the real-world effectiveness and safety of duloxetine in treating comorbid depression in patients with Parkinson's disease and to compare its outcomes with traditional treatment approaches. This study included adult patients diagnosed with Parkinson's disease combined with depression from December 2020 to December 2023. Based on the use of duloxetine, the cohort was divided into a traditional treatment group and a duloxetine group (traditional treatment combined with duloxetine). Patients with incomplete medical records, concurrent antidepressant therapy, or major psychiatric or neurological disorders were excluded. Retrospective data, including demographic information, treatment adherence, and various assessment scores, were collected from medical records by trained research staff. In total, 106 patients were analyzed, with 50 patients receiving traditional treatment and 56 patients receiving duloxetine. The duloxetine group exhibited significantly lower scores than the traditional treatment group in the Unified PD Rating Scale (p = 0.015), Hamilton Depression Rating Scale (p = 0.013), Beck Depression Inventory (p = 0.031), Parkinson's disease Questionnaire-39 (p = 0.006), and Clinical Global Impression-Improvement (p < 0.001) scores. In motor function assessment, the duloxetine group demonstrated improvements in kinetic tremor scores (p = 0.017), gait speed (p < 0.001), Timed Up and Go Test performance (p < 0.001), dyskinesia severity (p = 0.017), and rigidity (p = 0.019) compared to the traditional treatment group. Additionally, the duloxetine group exhibited better cognitive function across various assessments, including the Symbol Digit Modalities Test (p = 0.024), Stroop Color-Word Test (p = 0.048), and Montreal Cognitive Assessment (p = 0.024). Duloxetine is associated with superior efficacy in improving motor and non-motor symptoms, overall clinical status, and cognitive function. These findings support the potential utility of duloxetine as a comprehensive treatment option for comorbid depression in Parkinson's disease.

7933 Baseline Demographics and Design of the SkybriGHt Registry Study. A US Multi-center, Prospective, Non-interventional, Long-term, Effectiveness and Safety Study of Children and Adolescents with Growth Hormone Deficiency Treated with Lonapegsomatropin

Abstract Disclosure: P.F. Backeljauw: Advisory Board Member; Self; Ascendis Pharma, Inc., Novo Nordisk, BioMarin. Consulting Fee; Self; Ascendis Pharma, Inc., Novo Nordisk, BioMarin, Cavalry, Upsher-Smith. Speaker; Self; Ascendis Pharma, Inc., BioMarin. A.K. Maniatis: Advisory Board Member; Self; Alexion Pharmaceuticals, Inc., Ascendis Pharma, Inc., BioMarin, Novo Nordisk, Pfizer, Inc.. Research Investigator; Self; Alexion Pharmaceuticals, Inc., Ascendis Pharma, Inc., OPKO, Novo Nordisk, Pfizer, Inc.. Speaker; Self; Alexion Pharmaceuticals, Inc., Ascendis Pharma, Inc., BioMarin, Novo Nordisk, Pfizer, Inc.. Other; Self; Alexion Pharmaceuticals, Inc., Ascendis Pharma, Inc., BioMarin, Novo Nordisk, Pfizer, Inc. M. Abuzzahab: Advisory Board Member; Self; Ascendis Pharma, Inc., Pfizer, Inc.. Consulting Fee; Self; Rhythm. Research Investigator; Self; Ascendis Pharma, Inc., Novo Nordisk, Lumos, Rhythm, Soleno, Medtronic, Inhale. Other; Self; DSMB for LG. S.D. Chernausek: Advisory Board Member; Self; Ascendis Pharma, Inc. T. Lipman: Speaker; Self; Novo Nordisk. A.K. Dewan: Advisory Board Member; Self; Pfizer, Novo Nordisk. Consulting Fee; Self; Novo Nordisk. Research Investigator; Self; Medtronic. Speaker; Self; Ascendis Pharma, Inc., Novo Nordisk. Other; Self; Twistle. G. Prakasam: Advisory Board Member; Self; Ascendis Pharma, Inc., Novo Nordisk, AstraZeneca, Johnson &amp;Johnson, Lilly USA, LLC, Medtronic, Mannkind Corporation, Pfizer, Inc., Ultragenyx. Consulting Fee; Self; Ascendis Pharma, Inc., Novo Nordisk, AstraZeneca, Johnson &amp;Johnson, Lilly USA, LLC, Medtronic, Mannkind Corporation, Pfizer, Inc., Ultragenyx. Grant Recipient; Self; Mannkind Corporation. Research Investigator; Self; Ascendis Pharma, Inc., Novo Nordisk, AstraZeneca, Johnson &amp;Johnson, Lilly USA, LLC, Medtronic, Mannkind Corporation, Pfizer, Inc., Ultragenyx. Speaker; Self; Ascendis Pharma, Inc., Novo Nordisk, AstraZeneca, Johnson &amp;Johnson, Lilly USA, LLC, Medtronic, Mannkind Corporation, Pfizer, Inc., Ultragenyx. Other; Self; Ultragenyx, Pfizer, Inc., Mannkind Corporation, Medtronic, Lilly USA, LLC, Johnson &amp;Johnson, AstraZeneca, Novo Nordisk, Ascendis Pharma, Inc. M.B. Kabbani: Employee; Self; The Pediatric Endocrine &amp; Diabetes Clinic, PC. E. Huang: Employee; Self; Ascendis Pharma, Inc. S. Raveendran: Employee; Self; Ascendis Pharma, Inc. C. Zhao: Employee; Self; Ascendis Pharma, Inc. J.N. Caminis: Employee; Self; Ascendis Pharma, Inc. A.D. Rogol: Advisory Board Member; Self; Ascendis Pharma, Inc., Tolmar. Consulting Fee; Self; Ascendis Pharma, Inc., Tolmar. Other; Self; United States Anti-doping Agency, World Anti-doping agency, Tomlar, Ascendis Pharma, Inc., Up To Date. Objectives: Lonapegsomatropin is a prodrug that delivers unmodified somatropin and is approved by the FDA and EC as a once-weekly treatment for pediatric growth hormone deficiency (pGHD). The overall objectives of the observational SkybriGHt study are to assess safety and effectiveness of lonapegsomatropin in the post-marketing setting. The primary objectives are to describe demographics, clinical characteristics, and treatment patterns. Secondary objectives are to describe clinical outcomes assessments (COAs), long-term outcomes of treatment, treatment adherence and persistence, and outcomes in patients transitioning from childhood to adulthood. Exploratory objectives will examine the impact of adherence on clinical outcomes, and assess all cause and GHD-related resource utilization and costs. A preliminary description of the baseline characteristics and demographics of the first 50 patients enrolled is described here. Methods: In the ongoing SkybriGHt study approximately 900 participants from the US, diagnosed with pGHD and prescribed lonapegsomatropin consistent with routine clinical practice, will be invited to enroll by their HCP. The decision to treat with lonapegsomatropin is made prior to and independently of any potential participation in the study. Participants are enrolled over a 5 year period with the study intended to conclude after 10 years. COAs are collected using questionnaires assessing health-related quality of life in functioning and well-being, treatment satisfaction, and patient preference. Safety is assessed via reporting of adverse events and laboratory test results. Results: Of the first 50 patients enrolled, 11 (22%) had participated in previous trials of lonapegsomatropin, 42 (84%) were male, the median age was 9.9 years (range 3.1, 15.0), and median age at diagnosis was 7.2 years. No participants were previously treated with long-acting GH prior to lonapegsomatropin, and 36 (72.0%) were treated previously with daily GH. Conclusions: The SkybriGHt registry will evaluate the long-term safety, effectiveness, and treatment use patterns (including demographics), with lonapegsomatropin in clinical practice. This preliminary baseline data show that patients were predominantly male, consistent with prior treatment patterns of GHD in the United States. Description of baseline characteristics of additional enrolled patients will be presented. Presentation: 6/3/2024

6811 RESTORE: a Real-World Study of Setmelanotide in Patients With MC4R Pathway Diseases

Abstract Disclosure: C. Huber: Employee; Self; Rhythm Pharmaceuticals, Inc.. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. B. Sweeney: Consulting Fee; Self; Rhythm Pharmaceuticals, Inc., Novo Nordisk. Research Investigator; Self; Rhythm Pharmaceuticals, Inc.. Speaker; Self; Rhythm Pharmaceuticals, Inc. J. Pomeroy: Research Investigator; Self; Rhythm Pharmaceuticals, Inc. U. Mallya: Employee; Self; Rhythm Pharmaceuticals, Inc.. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. S. Zhang: Other; Self; Employer received funding from Rhythm Pharmaceuticals, Inc. to support this research. M. Yang: Other; Self; Employer received funding from Rhythm Pharmaceuticals, Inc. to support this research. S. Malhotra: Employee; Self; Rhythm Pharmaceuticals, Inc.. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. A.M. Haqq: Advisory Board Member; Self; Rhythm Pharmaceuticals, Inc., Novo Nordisk, Foundation for Prader-Willi Research USA. Grant Recipient; Self; Weston Family Microbiome Initiative and Canadian Institutes of Health Research. Background: Setmelanotide has demonstrated efficacy in clinical trials of patients with obesity due to rare melanocortin-4 receptor pathway diseases. Real-world evidence can provide a greater understanding of patient outcomes, such as hyperphagia and quality of life, in a clinical practice setting. Real-world Evidence of SeTmelanotide fOr Hyperphagia and ChRonic Weight ManagEment (RESTORE) is a prospective, observational, longitudinal study aimed at assessing patient- and caregiver-reported real-world effectiveness of setmelanotide among patients with Bardet-Biedl syndrome and proopiomelanocortin, proprotein convertase subtilisin/kexin type 1, or leptin receptor deficiency and their caregivers. Methods: Patients aged ≥6 years and caregivers aged ≥18 years who are able to respond to a survey in English and consented to the Rhythm Intune Program are eligible for the study. Patients must have been prescribed, but not yet initiated, setmelanotide, not have been previously treated with setmelanotide, and not have previously participated in or be currently enrolled in any clinical trial, including setmelanotide trials. Caregivers must be confirmed legal guardians of patients without prior exposure to setmelanotide, have cared for their patient for ≥6 months, and not be paid professional caregivers. Participants will complete secure online surveys at baseline (ie, before treatment) and at 6 posttreatment time points over a 1-year study period. Patient surveys collect information on hyperphagia-related signs and symptoms, medical history, treatment adherence, weight management programs and treatment, comorbidities/complications, comedications, disease impact on psychological well-being and health-related quality of life, treatment satisfaction, work/school/activity status, and patient global assessment. Caregiver surveys include assessments on psychological well-being, treatment satisfaction, and impact on activities of daily living, work status, and family dynamics. A subset of study patients will be invited to receive a wearable device (CentrePoint® Insight Watch) to assess activity and sleep patterns. Treatments and outcomes among patients will be described and compared with baseline for all post-treatment initiation assessment time points. Current status: RESTORE is the first study to assess the real-world effectiveness of setmelanotide, has been IRB approved, and is actively enrolling. Presentation: 6/3/2024

12481 Evaluating The Device Handling And Preference Assessment Questionnaire For Growth Hormone Deficiency: Results From Content Validation Interviews

Abstract Disclosure: J. Neergaard: Employee; Self; Novo Nordisk. S. Akhtar: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. B. Berg: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. S. Hamilton: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. E. Hildebrand: Consulting Fee; Self; Novo Nordisk. J. Medina: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. G. Ter-Borch: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. N. Rasmussen: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. Background: Patient experiences with injection devices may affect treatment adherence, and hence it is important to ensure patient-reported outcomes (PRO) instruments used to assess experience are supported by evidence of validity. As part of a study that compared patient preference and ease of use between the devices used to administer Somapacitan (Sogroya®, Novo Nordisk A/S) and Somatrogon (Ngenla®, Pfizer), we assessed relevance, comprehensiveness and comprehension of a PRO instrument, the Device Handling and Preference Assessment Questionnaire (DHPAQ), in adolescent patients with growth hormone deficiency (GHD) and their caregivers. Methods: We recruited 33 adolescent patients with GHD and 37 caregivers across five sites in the US for an open label, randomized, crossover study in which participants simulated an injection with each device using an injection pad or mannequin. The DHPAQ was used to assess patients’ device preference. Two-sided statistical tests with 5% significance levels were performed using DHPAQ responses. After completing the DHPAQ, a random subgroup of 10 adolescents and 10 caregivers were offered additional compensation to participate in a cognitive interview (CI). The CIs were conducted in accordance with the US Food and Drug Administration 2022 Principles for PRO Instruments and 2009 PRO Guidance for Industry. Trained interviewers used a structured guide to evaluate questions, statements, tasks and response options in the DHPAQ. Participants’ interview responses for each question were coded and added to summary tables. These summary tables were evaluated by the study team to determine the relevance, comprehensiveness and participant comprehension of instructions, item stems and response options. Results: When asked if any questions in the DHPAQ did not seem relevant to their device experience, all 20 CI participants answered “no”. Questions on “learning how to use the device”, “selecting the correct dose” and “injecting the dose” were ranked as most relevant. Participants responses from the CIs supported perceived comprehensiveness of all DHPAQ items. All CI participants reported comprehension of all test items in the DHPAQ, and only three items did not receive a “no difficulty” response from all 20 participants. In the main study, the number of participants who preferred the somapacitan device was significantly greater than those who preferred the somatrogon device (84.3% vs 12.9%, p&amp;lt;0.0001). More participants found the somapacitan device easy or very easy to use compared with the somatrogon device (98.6% vs 74.3%). Conclusions: The results from the CIs indicated that the instructions, item stems and response options of the DHPAQ were relevant, comprehensive and fully comprehended by all participants. In the main study, patients with GHD and their caregivers were found to prefer the somapacitan device to the somatrogon device. Presentation: 6/2/2024

Knowledge and perception of asthma among parents of children with asthma.

Parents' knowledge is an integral part of healthcare quality, impacting treatment adherence, patient loyalty and healthcare utilisation in pediatric asthma. Parental knowledge is particularly crucial as parents influence decision-making for their child's healthcare. To assess parents' knowledge and perceptions of their children's asthma and to identify areas for weakness in therapeutic education. This is transversal survey study based on validated and translated self-administered questionnaire (Arabic asthma knowledge questionnaire AAKQ). The study was conducted in 2 departments: pediatric primary care department at Elomrane and pediatric pneumology outpatient clinics at Bechir Hamza children's hospital of Tunis during 6 months (March2022- October2022). One hundred forty-four parents were involved in the study. Most of the participants were mothers. The mean age was 38 years. Only 27,0% had higher education and smoking in parents was observed in 58% of cases. Our results showed moderate knowledge in 67,9% of participants. The mean of the total knowledge score was 57,7/85. Forty-six per cent of parents had poor knowledge for the first category of the questionnaire. The second category was the most successful part where only 14% had poor knowledge. The mean score of the third category was 13,5/20 with 35,4% of parents having poor knowledge. The mean of total knowledge was found to be higher among participants with higher education (P=0,000), when the gender of the child is masculine (P=0,038), when the parents were asked in Hospital of children of Tunis (P=0,003) and when there is a history of asthma in the siblings (P=0,05). No difference in caregiver's knowledge was based on their age or gender or history of asthma, on the age of the child, his hospitalization for asthma, his asthma control level or his compliance with medication. Knowledge of parents and their children suffering from asthma is acceptable but insufficient. An approach focusing on improving patient education skills is needed to help families understand and accept the disease and achieve optimal control.

In-depth analysis of antipsychotic-induced akathisia: An integrative literature review

Background: Antipsychotic-induced akathisia poses a significant challenge in clinical practice due to its frequent oversight in diagnosis and associated severe consequences such as suicidal behavior and compromised treatment adherence. Understanding the underlying mechanisms and implementing effective management strategies are imperative in addressing this clinical concern. Objective: provide an updated understanding of the correlation between antipsychotics and akathisia, discussing its pathophysiology, diagnosis, assessment, and current as well as prospective treatment options. Methods: An integrative literature review was conducted utilizing Pubmed and Virtual Health Library databases, supplemented by additional resources to broaden the study's scope. Results and Discussion: Among the 204 studies analyzed, 17 met the criteria for inclusion in the review, complemented by 7 articles focusing on assessment scales. The study explores the impact of neuroleptic medications on akathisia's pathophysiology, diagnostic approaches, symptomatology, assessment tools, and management strategies. Conclusion: Robust research endeavors are crucial to consolidate evidence, identify gaps, and offer a holistic perspective on akathisia. The profound comprehension of the condition reveals paths to enhance patients' quality of life.

Treatment Adherence in Pediatric Atopic Dermatitis: A Systematic Review.

Adherence to pediatric atopic dermatitis (AD) treatment regimens can be complex and a major challenge to optimizing treatment outcome. We aimed to review factors associated with nonadherence in pediatric AD and propose interventions to improve adherence. PubMed and EMBASE databases were systematically searched for articles from 2000 to February 2023 related to AD and adherence, with an additional update in December 2023. Non-human studies, reviews, commentaries, and meta-analyses were excluded. Articles were sorted into pediatric versus adult study population based on volume. Herein, we examine the results of papers discussing adherence factors related to pediatric patients. A total of 62 studies met inclusion criteria. Thirty-six studies surveyed patients and caregivers (N = 10,268) to identify barriers to treatment adherence. None of the included studies were specific to systemic medications. Barriers includedpoor caregiver quality of life, inadequate AD-related education, topical corticosteroid (TCS) phobia, unclear therapy-related instructions, and dissatisfaction with physician interaction. Five studies solely measured adherence using medication electronic monitoring systems, Morisky medication adherence scale, or self-reported adherence to measure adherencetotopical medications. Twenty-one studies described interventions involving nurse-led or web-based education programs, text message or email reminders, and TCS education. Adherence was improved with caregiver education programs, daily text-message reminders, eczema action plans, TCS potency "traffic light" color system, and frequent follow-up visits. Adherence to pediatric AD treatment poses a multifactorial challenge for caregivers and patients. This study provides an index of strategies to optimize adherence, as it is essential for prevention of long-term sequela associated with AD in children. As the AD treatment landscape rapidly expands, further studies are vital to assess pediatric adherence to new topical, oral, and injectable medications. PROSPERO: CRD42023488557.

The Influence of Medication Non-Adherence on the Occurrence of Drug-Resistant Tuberculosis

Drug-resistant tuberculosis (DR-TB) presents a significant challenge to global TB control efforts, with medication non-adherence identified as a major contributing factor. This study aimed to investigate the influence of medication non-adherence on the occurrence of DR-TB in Serang City, Indonesia. Utilizing an observational analytic study with a case-control design, the research included 76 TB patients, comprising 38 DR-TB cases and 38 drug-sensitive TB controls. Data were collected from secondary sources and validated questionnaires and analyzed using univariate and bivariate methods, including Chi-square tests and Odds Ratios (OR). The findings revealed a significant relationship between medication non-adherence and DR-TB, with an OR of 11.56, indicating that non-adherent patients are 11.5 times more likely to develop DR-TB compared to adherent patients. However, demographic factors such as age, occupation, and education did not significantly influence the incidence of DR-TB. The study concludes that medication non-adherence is a critical risk factor for the development of DR-TB, underscoring the need for targeted interventions to improve treatment adherence, particularly through educational campaigns and community-based support. Health authorities are encouraged to implement intensive educational programs focused on the risks of non-adherence and ensure that TB treatment is accessible and supported by community health workers. Policymakers should allocate sufficient resources to these initiatives to effectively reduce the incidence of DR-TB.

Integrating the Memory Support Intervention into the Transdiagnostic Intervention for Sleep and Circadian Dysfunction (TranS-C): can improving memory for treatment in midlife and older adults improve patient outcomes? Study protocol for a randomized controlled trial

BackgroundPoor memory for treatment is associated with poorer treatment adherence and poorer patient outcomes. The memory support intervention (MSI) was developed to improve patient memory for treatment with the goal of improving patient outcomes. The aim of this study protocol is to conduct a confirmatory efficacy trial to test whether a new, streamlined, and potent version of the MSI improves outcomes for midlife and older adults. This streamlined MSI is comprised of constructive memory supports that will be applied to a broader range of treatment content. The platform for this study is the Transdiagnostic Intervention for Sleep and Circadian Dysfunction (TranS-C). We will focus on midlife and older adults who are low income and experiencing mobility impairments.MethodsParticipants (N = 178) will be randomly allocated to TranS-C + MSI or TranS-C alone. Both intervention arms include eight 50-min weekly sessions. Assessments will be conducted at pre-treatment, post-treatment, 6-, and 12-month follow-up (6FU and 12FU). Aim 1 will compare the effects of TranS-C + MSI versus TranS-C alone on sleep and circadian functioning, daytime functioning, well-being, and patient memory. Aim 2 will test whether patient memory for treatment mediates the relationship between treatment condition and patient outcomes. Aim 3 will evaluate if previously reported poor treatment response subgroups will moderate the relationship between treatment condition and (a) patient memory for treatment and (b) treatment outcome. Exploratory analyses will compare treatment condition on (a) patient adherence, patient-rated treatment credibility, and patient utilization of treatment contents, and (b) provider-rated acceptability, appropriateness, and feasibility.DiscussionThis study has the potential to provide evidence for (a) the efficacy of a new simplified version of the MSI for maintaining health, well-being, and functioning, (b) the wider application of the MSI for midlife and older adults and to the treatment of sleep and circadian problems, and (c) the efficacy of the MSI for sub-groups who are likely to benefit from the intervention.Trial registrationClinicalTrials.gov NCT05986604. Registered on 2 August 2023.

HECO-01 CONSTRAINTS AND CHALLENGES OF TREATMENT ABANDONMENT IN PEDIATRIC CANCER PATIENTS DIAGNOSED WITH BRAIN TUMORS, LOW MIDDLE INCOME SETTING

Abstract ABSTRACT Treatment abandonment is a critical issue in pediatric oncology that significantly impacts patient outcomes and survival rates. This study investigates the prevalence and factors contributing to treatment abandonment among pediatric solid cancer patients. A comprehensive literature review was conducted to explore the cultural, socioeconomic, and healthcare system-related factors influencing treatment abandonment in this population. Data was collected from 148 pediatric pediatric cancer patients diagnosed with Brain tumors in National Cancer institute, Cairo University over a 2 year period. The study also examines the implications of treatment abandonment on disease progression, patient prognosis, and overall healthcare burden. RESULTS The study found that 14 % of pediatric Brain tumors ‘patients at our institute experienced treatment abandonment during their course of care. Factors contributing to treatment abandonment included financial constraints (34 %), lack of awareness about the importance of treatment adherence (26 %), distance to healthcare facilities (20%), and cultural beliefs (20). The impact of treatment abandonment on disease progression and patient outcomes was substantial, with [58 % p value &amp;lt;0.01}. By understanding the complex factors contributing to treatment abandonment in Egyptian pediatric cancer patients, healthcare providers and policymakers can implement targeted interventions to improve treatment adherence, enhance patient outcomes, and reduce the burden of pediatric cancer in Egypt.

Patient and Caregiver Perspectives on Gender Disparity in CKD: An Interview Study.

Chronic kidney disease (CKD) affects more women than men worldwide, however, men comprise the majority of patients who receive kidney replacement therapy. We aimed to describe the perspectives of patients and their caregivers regarding gender disparities in CKD. Semi-structured interviews were conducted with 45 patients with CKD (20 women) and 14 caregivers (12 women) from seven clinics in Austria. The interviews were analyzed thematically. Five themes were identified in this study. Participants perceived that women were "disadvantaged and vulnerable" (silent and intimidated, single mother predicament, impeded access to care and support due to socioeconomic disadvantage, had to fend for themselves); "fulfilling gender roles and norms" (primarily responsible for childcare, pressure to perform well as homemakers, put others' needs before their own, encouraging husband's treatment adherence), and "protecting their own health" (self-disciplined, vigilant, confronted health challenges, advocated for their needs). Men were seen to "place the onus of care on others" (expected help from family, relied on others for decisions). Both men and women experienced a "disease-related identity crisis and distress" (women: impaired body image, mental distress; men: denial and self-destruction, emasculated by sickness). Women with CKD felt vulnerable and were inclined to fulfill gender norms and responsibilities as caregivers but were also vigilant about protecting their own health. Men tended to be reluctant to accept CKD and appeared to depend on others for disease management. Better awareness and addressing these concerns can inform strategies to minimize gender disparities in access to care and outcomes in CKD.

Knowledge, attitude and perceived barriers related to directly observed treatment, short-course among patients and caregivers attending tuberculosis clinics: a cross-sectional survey.

Tuberculosis (TB) is a major worldwide health problem, particularly in India, where it accounts for a quarter of infections. Adherence to the directly observed treatment, short-course (DOTS), which is necessary for TB treatment, is difficult in resource-limited circumstances. This study addressed the knowledge, attitude, and perceived barriers related to TB and DOTS adherence among patients and caregivers. A cross-sectional survey was undertaken at the selected TB clinics between November 2023 and March 2024. Purposive sample yielded 180 patients and 217 caregivers. Data were obtained using validated, self-structured questionnaires that assessed knowledge, attitude, and perceived barriers to TB and DOTS adherence. Statistical analysis was carried out with IBM SPSS version 23.0. The study found that caregivers had considerably greater TB knowledge than patients (p<0.05). Many patients misunderstood the cause of TB (50.6%) and embraced various misconceptions about the disease, such as TB spreading by shaking hands or sharing food, swimming in a holy river to cure diseases, and TB being the result of sin or karmic retribution. Distance to treatment facilities (95.6%), the necessity to take time off from work (91.7%), and social stigma (65.0%) were identified as the top three barriers to DOTS adherence. The study highlighted considerable gaps in knowledge, distant healthcare facilities, busy work schedules, and social stigma as barriers to TB treatment adherence in Deoghar, Jharkhand. Addressing these challenges via focused education and support activities is essential for enhancing DOTS adherence and TB treatment outcomes.

Evaluation of Body Composition and Biochemical Parameters in Adult Phenylketonuria

Background/Objectives: Phenylketonuria is a hereditary metabolic disorder characterized by a deficiency of phenylalanine hydroxylase. The main treatment for PKU is a phenylalanine-restricted diet. The exclusion of protein rich natural foods and inclusion of low-Phe substitutes may give rise to an imbalanced diet, and the increased risk of overweight and obesity in PKU is a cause for concern. We aimed to evaluate the body composition and nutritional biochemical biomarkers in adult PKU patients who are on Phe-restricted and essential amino acid-supplemented nutrition therapy and to investigate the relationships between these parameters and patient gender, adherence to dietary therapy, and disease type, defined as mild or classic PKU. Methods: The study group comprised 37 PKU patients and 26 healthy siblings as controls. The participants were assessed based on an analysis of anthropometric parameters, body composition, and biochemical test results. Results: PKU patients do not have a higher incidence of overweight and obesity than healthy controls, the proportion of energy derived from carbohydrates in their diets was below the recommended level, and their total energy intake was below the recommended daily allowance. It was remarkable that patients with a treatment adherence ratio of &lt;50% displayed a higher prevalence of overweight and abdominal obesity in comparison to those with a more favorable treatment adherence ratio. Conclusions: In view of the growing prevalence of overweight in the general population, PKU patients should be kept under close long-term follow-up. Particularly in the group with low treatment compliance, more caution should be taken in terms of adverse outcomes.