Main Goal Of Treatment Research Articles

Review of Guideline Recommendations for Optimal Anti-VEGF Therapy in Age-Related Macular Degeneration.

Neovascular age-related macular degeneration is a progressive, blinding macular disease that has become a burden both in healthcare systems and the global economy. The vascular endothelial growth factor (VEGF) is the main agent involved in the pathogenic process of the disease. The main goal of the age-related macular degeneration treatment is to maintain and improve visual acuity by injecting intravitreal anti-VEGF agents in either a reactive or proactive manner. Subretinal and intraretinal fluids are the main biomarkers that should be considered when managing the frequency of the therapy. This review discusses both functional and morphological treatment criteria according to current recommendations as opposed to real-life situations encountered during day-to-day clinical practice and highlights situations in which the benefits of continuing therapy are arguable in terms of improving patients' quality of life. Optimizing the treatment regimen represents an important aim of current clinical ophthalmological practice, as age-related macular degeneration patients usually have a long follow-up period.

Osteoarthritis in athletes – surgical and non-surgical aspects of treatment

Introduction: Osteoarthritis (OA) is a degenerative chronic process that primarily affects the elderly, but there are also reports of OA in the young and sports-active population. Symptoms of OA usually include pain, joint stiffness, and crepitation. Physical, radiologic, and laboratory tests are required for diagnosis. The main goal of OA treatment is to restore normal function to the joint affected by OA. Methods: A literature search on PubMed was used to collect facts about the relationship between sports and OA and possible treatment methods. All forms of articles were considered in the literature search. Articles that were not written in English were not included. Literature from the library of the Faculty of Medicine in Mostar was an additional source of information. Results: OA occurs more frequently in people who play or have played professional sports. On the other hand, daily exercise has a positive effect on cartilage metabolism. Most articles reported the occurrence of OA in former professional athletes. The joint most commonly affected by OA was the knee. Injuries were a predictive factor for the occurrence of OA, and most studies talk about anterior cruciate ligament injuries. Running and football were the most commonly studied sports. Treatment options include conservative treatment, surgical methods, and biological therapy. Conclusion: Frequent strain and injury in athletes can cause cartilage damage and potentially lead to the development of OA. The benefits of exercise far outweigh the risks of developing OA. In addition, some studies report a significant stimulating effect of exercise on cartilage cell metabolism. Treatment of OA consists of the use of analgesics, intra-articular therapy, and physiotherapy. Surgical methods are considered when conservative approaches are unsuccessful. Biological therapy and similar treatments are the new potential treatments.

Congenital Adrenal Hyperplasia: A Review of Current Knowledge and Future Directions

Introduction: Congenital adrenal hyperplasia (CAH) is a group of diseases in which genetic defects occur that disturb the synthesis of cortisol. The most common variant of CAH (95%-99%) is caused by 21-hydroxylase deficiency as a result of mutations in the CYP21A2 gene and is one of the most common monogenic diseases. CAH is characterized by androgen overproduction along with variable degrees of cortisol and aldosterone deficiency. Age at diagnosis can provide some information about the underlying mutations, with those diagnosed at birth/early infancy being at greater risk of developing serious enzyme defects. Classic and non-classical forms of this disorder have been described in the literature. CAH diagnosis is based on the clinical presentation, hormonal panel, Adrenocorticotropic Hormone (ACTH) stimulation test, and genetic testing. The main goals of treatment for congenital adrenal hyperplasia are glucocorticoid/mineralcorticoid supplementation, control of high adrenal androgen levels, fertility control, genetic counseling, and optimization of patients’ quality of life. Purpose of the work: This study aims to review and characterize the clinical and pathophysiological aspects of congenital adrenal hyperplasia. Materials and methods: A comprehensive analysis of research papers available on PubMed, Google Scholar, Web of Science, Embase and Scopus was undertaken using the searchterms encompassing the following keywords: congenital adrenal hyperplasia, 21-hydroxylase deficiency, CYP21A2 Results: Congenital adrenal hyperplasia is a heterogeneous group of genetic disorders that can lead to serious and even fatal complications in the form of adrenal crisis. Therefore, screening tests and early diagnosis are crucial and can save the lives of newborns. Treatment should be individualized and allow patients to achieve normal growth, sexual development, fertility and a better quality of life.

Current Advances in Diagnosis, Therapeutics, and Surgical Interventions for the Management of Refractory Gastroesophageal Reflux Disease (GERD): An Update.

Gastroesophageal reflux disease (GERD) is a chronic illness characterized by complications arising from the reflux of stomach contents, which significantly lower the quality of life, increase morbidity, and increase medical expenses associated with treating the condition. The main goal of treatment in GERD is symptomatic relief, relapse prevention, and healing of erosive esophagitis. The treatment mainly involves lifestyle changes to control acid production and proton pump inhibitors (PPIs) as the first line of treatment. Endoscopic interventions or anti-reflux surgery may be beneficial in relieving symptoms in people whose symptoms are triggered by reflux. In this review, we discuss the pathophysiology and newer diagnostic and treatment modalities including available surgical management options to manage refractory GERD.

Looking for the ideal medication for heart failure with reduced ejection fraction: a narrative review.

The main goals of the pharmacological treatment of Heart failure with reduced ejection fraction (HFrEF) are the reduction of mortality and the prevention of hospitalizations. However, other outcomes such as improvements in cardiac remodeling and clinical status, functional capacity and quality of life, should be taken into account. Also, given the significant inter-individual and intra-individual variability of HF, and the fact that patients usually present with comorbidities, an appropriate treatment for HFrEF should exert a clinical benefit in most patient profiles irrespective of their characteristics or the presence of comorbidities, while providing organ protection beyond the cardiovascular system. The aim of this narrative review is to determine which are the proven effects of the guideline-directed treatments for HFrEF on five key clinical outcomes: cardiovascular mortality and hospitalization due to HF, sudden death, reverse cardiac remodeling, renal protection and evidence in hospitalized patients. Publications that fulfilled the pre-established selection criteria were selected and reviewed. Renin-angiotensin system (RAS) inhibitors, namely angiotensin-converting enzyme inhibitors (ACE-I) and angiotensin II receptor blockers (ARBs) or angiotensin receptor-neprilysin inhibitors (ARNI), beta-blockers (BB), mineralocorticoid receptor antagonists (MRA), sodium-glucose co-transporter 2 inhibitors (SGLT2i) show a benefit in terms of mortality and hospitalization rates. ARNI, BB, and MRA have demonstrated a significant positive effect on the incidence of sudden death. ARB, ARNI, BB and SGLT2i have been associated with clear benefits in reverse cardiac remodeling. Additionally, there is consistent evidence of renal protection from ARB, ARNI, and SGLT2i in renal protection and of benefits for hospitalized patients from ARNI and SGLT2i. In conclusion, the combination of drugs that gather most beneficial effects in HFrEF, beyond cardiovascular mortality and hospitalization, would be ideally pursued.

Current concept of osteoporosis diagnosis and treatment in children

Osteoporosis (OP) is a disease characterized by a decrease in bone strength, leading to an increased risk of fractures. Bone fragility in children may be due to genetic disorders, chronic underlying conditions or taking medications that negatively affect bone metabolism. The article considers the modern classification of OP in children, approaches to its diagnosis, the role of dual-energy X-ray absorptiometry for diagnosis.The main goal of OP prevention and treatment is to achieve a higher peak of bone mass, improve bone microarchitectonics, and, as in adults, reduce the risk of fractures, prevent skeletal deformities, improve mobility, independence and quality of life. Recommendations for lifestyle changes are presented, including adequate calcium intake and vitamin D, taking antiresorptive drugs, as well as prospective management of children with OP.

Drugs for COPD.

The main goals of treatment of chronic obstructive pulmonary disease (COPD) are to relieve symptoms, reduce the frequency and severity of exacerbations, prevent disease progression, and reduce mortality. GOLD (Global Initiative for Chronic Obstructive Lung Disease) guidelines for treatment of COPD were updated recently.1 Treatment of acute exacerbations is not discussed here. Drugs available for treatment of COPD are listed in Tables 1 and 3.

ОСОБЛИВОСТІ ФІЗИЧНОЇ ТЕРАПІЇ У ПРОЦЕСІ РЕАБІЛІТАЦІЇ ОСІБ ПОХИЛОГО ВІКУ З ПОРУШЕННЯМ ФУНЦІЙ ХРЕБТА

According to literary sources, osteochondrosis of the cervical and thoracic spine is one of the most common diseases. This disease affects 22% of the world's population and ranks first in the group of diseases of the musculoskeletal system. The main goal of restorative treatment of elderly patients with cervical and thoracic osteochondrosis is to reduce muscle spasticity, decrease the level of heart rate, arterial systolic and diastolic pressure, reduce pain, facilitate the performance of household tasks, etc. In the program of physical therapy, we included therapeutic physical culture, therapeutic massage, and physiotherapeutic means. Therefore, the purpose of the study was to study of the peculiarities of physical therapy in the rehabilitation process of elderly people with osteochondrosis. Research methods. 28 people (12 men and 16 women) with osteochondrosis of the cervical-thoracic spine took part in the study. The average age of the patients was 64.5 years. The pedagogical experiment was aimed at determining the effectiveness of the program of physical therapy developed by us for elderly people with osteochondrosis on indicators of mobility in the cervical spine, intensity of pain sensations. Results and conclusions. The results of the conducted research indicate the positive impact of classes in the complex program of physical therapy for the elderly with cervicothoracic osteochondrosis. Thus, after the completion of the formative study, we established an improvement in the functional state of the cervical spine in elderly persons with cervical-thoracic osteochondrosis. The average group value of the pain intensity index in patients at the end of the study corresponded to the assessment of «very mild pain», while at the beginning of the study it corresponded to the assessment of «pain above average».

Diffuse large B-cell lymphoma and follicular lymphoma: problem state in Russia

Background. Non-Hodgkin's lymphomas (NHL) are a heterogeneous group of hematological malignancies, the vast majority of which are B-cell tumors. The most common variant of NHL is diffuse large B-cell lymphoma (DLBCL), characterized by an aggressive course, which accounts for 30–40% of all NHL. The second most common is follicular lymphoma (FL), traditionally classified as an indolent variant accounting for up to 25% of all NHL. Current therapies for these lymphoproliferative disorders which includes innovative drugs in the 1st line of therapy (LT) have demonstrated high efficacy. However, some patients develop a relapse or a refractory disease. Despite recent significant progress in the development and implementation of innovative targeted drugs, in most cases, it is not possible to achieve persistent long-term remissions after disease relapse, which leaves patients with an unmet need for effective and well-tolerated treatment options. Aim. To obtain objective data on the incidence, clinical course, and effectiveness of therapy for the most common variants of NHL in real-world practice in Russia. Materials and methods. From February to March 2023, 130 hematologists and oncologists from 30 regions of Russia were surveyed to update the data on DLBCL and FL. Results. Over the past 12 months, 5,689 patients with NHL were observed, of which 56% had DLBCL; 62% of them received the 1st LT, 22% received the 2nd LT, 10% received the 3rd LT, and only a few reached later lines. Analysis of the administered treatment options in the 2nd and 3rd LTs shows that there is no standard of care for this population, and the effectiveness of the regimens used in real-world Russian practice is extremely low. FL accounted for 23% of all 5,689 patients with NHL, of which 56% were newly diagnosed and 44% received treatment for relapse. The majority of patients with FL who received ≥3 LTs had an inferior prognosis and rapid disease progression: the median time from diagnosis to the beginning of the 3rd LT was only 26.4 months. The analysis of treatment options for patients with relapsed FL indicates a lack of standard and effective therapies. Conclusion. Relapsed and refractory DLBCL and FL represent a complex clinical situation where the main goal of treatment is disease control due to the impossibility of achieving stable remissions with existing treatment options. Clinicians with great hope are looking to the emergence of new classes of drugs that will be able to improve the prognosis for this complex population.

Algorithm of complex therapy and prevention of emotional burnout in doctors-psychiatrists during the war and its effectiveness

Abstract. The purpose - to develop and determine the effectiveness of the algorithm of complex therapy and prevention of emotional burnout (EB) among psychiatrists during the war, to resolve the issue of its recommendation for wide implementation in the system of mental health care institutions. The research sample, which was formed in compliance with the principles of biomedical ethics and based on informed consent, included 120 psychiatrists who worked in the city of Kyiv in the period from February 24, 2022. During 2022, the persons under examination were divided into three groups: the first group included 37 persons (30.8%) – doctors with no signs of professional maladjustment (PMA), and a low level of EB; the second group consisted of 57 persons (47.5% ) with signs of professional PMA, and a moderate level of EB; the third group consisted of 26 persons ( 21.7%) and they were respondents with clinically established and pronounced PMA, and a high level of EB. As a result of the study, the personal, psychopathological, psychosocial, professional and emotional-interpersonal factors of the formation and progression of EB among psychiatrists during the war were established, and their interrelationships in its genesis, on the basis of which the basic principles of a comprehensive approach to the therapy and prevention of EB were formulated for psychiatrists (complexity, personalization, phasing and availability), the main goals of treatment and prevention measures are defined and 4 therapeutic strategies of diagnosis, therapy and prevention are proposed within the framework of providing specialized medical care to psychiatrists during the war. These are recommended to be implemented taking into account the degree PMA as a key descriptor of EB, and in accordance with the key vectors of pathological changes in the respondents. The implementation and further evaluation of the effectiveness of the mentioned measures proved their effectiveness in eliminating manifestations of EB and PMA, normalization of mental state with levelling of psychopathological symptoms, improvement of social and psychological adaptation and quality of life of psychiatrists. The results obtained make it possible to recommend this algorithm for wide implementation in the practice of mental health care institutions.

Treatment strategies for vertical maxillary excess: Cases with and without anterior open bite

This comprehensive review investigates the treatment of vertical maxillary excess (VME) with and without anterior open bite (AOB) in seven unique case studies. These cases illustrate the implementation of advanced orthodontic techniques, such as miniscrew-assisted intrusion of the maxillary and mandibular arches. The multifaceted nature of AOB must be considered, highlighting the need for clear diagnostic criteria and an understanding of the long-term impacts of treatment strategies. The main goal of treatment is to reduce the vertical dimension of the maxillomandibular complex. A single miniscrew in the midpalatal region for the maxillary arch and two miniscrews in the buccal areas of the mandibular arch can be applied for the effective intrusion of the posterior teeth. The review then delves into the specific challenges and treatment modalities for managing VME without AOB, stressing the critical role of individualized intervention plans. Each case study demonstrates a different aspect of the treatment approach: from differential intrusion of molars to address esthetic concerns and functional issues to comprehensive strategies involving the intrusion of entire dental arches to correct severe malocclusions. The outcomes emphasize the importance of controlled orthodontic mechanics in achieving and maintaining optimal posttreatment stability. This review underscores the effectiveness of these treatment strategies in significantly improving facial profiles and dental function while highlighting the adjunctive use of myofunctional therapy and other retention techniques to prevent relapse. Collectively, these cases provide valuable insights into the tailored treatment of complex orthodontic conditions, offering guidance on achieving long-term success in clinical practice.

Current treatment approaches in the management of the heart failure

Heart failure is a clinical syndrome in which the function of the heart muscle and neurohormonal regulation are impaired, resulting in an inability to meet the metabolic needs of tissues. Many different mechanisms are involved in the pathophysiology of heart failure. Therefore, this disease is defined as a chronic disease in which treatment management is difficult for both the patient and the physician. Therefore, although the main goal is to take the necessary precautions before the disease develops, this may not always be possible. The management of the disease and the treatment options are being provided by finding drugs that are effective against existing mechanisms or against new mechanisms that have been discovered. The main goal of treatment is to stop the chain of events that cause and worsen heart failure based on the physiopathology. For this reason, there are various treatment modalities accepted in current guidelines to manage the current process. The aim of these treatments is to reduce symptoms, improve quality of life, and reduce mortality and morbidity rates. Recently, many important developments in the field of heart failure have started to come one after another. In this article, the mechanisms of action of the current treatment options and their effects on mortality have been mentioned, but rather than the current pharmacologic treatments, promising new treatment options, especially phase 3 and phase 4 trials, have been evaluated.

Understanding the enigma of neurobehavior in children with epilepsy and the need for mandatory screening: a prospective case control study

Background: In epilepsy management, control of seizures is the prime objective and reduction in seizure frequency is the main goal for successful treatment. There is a high prevalence of neurobehavioral problem. Hence, there is a need to screen, and intervene. Aim was to identify the neurobehavioral profile and the impact on children with epilepsy Methods: After IRB clearance, 100 consecutive school-aged children 4-17 years with normal IQ attending epilepsy clinic were enrolled as cases, and 102 age, gender and socio-economic status-matched children without epilepsy were chosen as controls. The strength and difficulties questionnaire are administered to both groups. It addresses five domains: emotional, conduct, hyperactivity, and peer problems which contribute to the total difficulties score, and fifth dimension prosocial behaviour. Results: Among the children with epilepsy, emotional issues were observed in 41%, conduct issues in 67%, hyperactivity behaviour in 54%, and peer problems in 64% while pro-social behaviour was only 27%, and total difficulty score was 60%, which was statistically significant with p<0.001 in all domains when compared to control. The impact of the disease was 73%, home environment was affected in 67%, classroom learning 45%, 28% in leisure activities, and 10% in friendship, which was statistically significant with p<0.001 in all domains when compared to control. Conclusions: We have identified emotional, conduct, hyperactivity and peer problems having significant impact on children with epilepsy. Hence a screening in various behavioural domains helps in early identification and prompt intervention of neurobehavior.

Можно ли вылечить ревматоидный артрит, изучив факторы, препятствующие выздоровлению?

Remission is the current goal of treatment for rheumatoid arthritis (RA). However, the ultimate goal should be complete cure of the disease and not just lifelong control of inflammation. Moreover, on the one hand, there is a certain saturation effect of anti-inflammatory drugs in RA, and on the other hand, there is increasing evidence that the pathological processes underlying the development of RA are independent of modern therapeutic agents. Therefore, in the future, the main goal of RA treatment should be considered to be the cure of the disease, i.e. recovery, not just suppression of symptoms

The Diagnostic Utility of Biochemical Markers and Intestinal Ultrasound Compared with Endoscopy in Patients with Crohn's Disease and Ulcerative Colitis: A Systemic Review and Meta-Analysis.

Background: Inflammatory bowel disease (IBD) consists of Crohn's disease (CD) and Ulcerative colitis (UC). The main goal of treatment is to obtain mucosal healing via endoscopy. More recently, intestinal ultrasounds, along with biochemical markers, have been increasingly popular as point-of-care testing to monitor treatment response. This systemic review and meta-analysis aimed to assess the diagnostic test performance of ultrasonography and biochemical markers (C-reactive protein and fecal calprotectin) compared with endoscopy for detecting inflammation in IBD. Methods: A comprehensive literature search was conducted using PubMed Medline, EMBASE, ScienceDirect, and CINAHL from 1 January 2018 to 1 January 2024. The included studies were prospective and retrospective observational studies, clinical trials, and cross-sectional studies investigating the diagnostic sensitivity and specificity of ultrasonography, biochemical markers, and endoscopy. Studies were selected based on the Preferred Reporting Items for Systematic Review and Meta-analysis Statement (PRISMA). Results: Of the 1035 studies retrieved, 16 met the inclusion criteria, and most of the included studies were prospective observational studies. Diagnostic test accuracy was conducted, and the pooled sensitivity and specificity of all the studies revealed that ultrasonography has the highest pooled sensitivity, at 85% (95% CI, 78 to 91%), and specificity, at 92% (95% CI, 86 to 96%), as compared with biochemical markers and endoscopy. More specifically, biochemical markers had a pooled sensitivity and specificity of 85% (95% CI, 81 to 87%) and 61% (95% CI, 58 to 64%), respectively, and endoscopy had 60% (95% CI, 52 to 68%) and 82% (95% CI, 76 to 87%), respectively. However, the results also show substantial heterogeneity in the studies because of various populations, protocols, and outcomes in the studies included. This was especially noted in the assessment of biochemical markers, in which a metaregression was performed showing a nonsignificant p-value of 0.8856 for the coefficient. Conclusions: IUS was found to have the highest pooled sensitivity and specificity of all the included studies for diagnosing inflammation in patients with CD and UC, and this, coupled with biochemical markers, can improve diagnostic utility.

Patient Burden of Nonsegmental Vitiligo: A US Real-World Survey of Dermatologists and Their Patients.

Vitiligo is a chronic autoimmune disease characterized by destruction of melanocytes, leading to skin depigmentation. Vitiligo can have a high quality-of-life burden and profound impact on psychosocial well-being. The objectives of this study were to describe the self-reported patient burden among patients with nonsegmental vitiligo with≤10% affected body surface area, summarize the physician-reported psychosocial and psychological impact of vitiligo on patient lives, and describe disease characteristics and treatment history, goals, and satisfaction. Data were drawn from the Adelphi Vitiligo Disease Specific Programme™, a real-world, cross-sectional survey with retrospective data collection of physicians and patients with vitiligo, collected in the United States between October 2021 and April 2022. Separate surveys for dermatologists and patients contained questions on clinical and demographic characteristics of patients with vitiligo and burden of vitiligo. Treatment history, goals, and satisfaction were assessed together with the impact of vitiligo on quality of life. Sixty-one dermatologists provided data for 326 patients with≤10% affected body surface area (adults, n=221; adolescents, n=105); 90 of those patientsalso responded to the survey. The most common treatments were topical corticosteroids, topical calcineurin inhibitors, and narrow-band ultraviolet-B phototherapy, with the main treatment goal being repigmentation. Physician-reported treatment satisfaction was 56%; 25% of patients reported frustration with treatment options. Physicians reported impact of vitiligo on everyday life in 46% of patients. Patients reported 12.7% overall work impairment; mean scores for Hospital Anxiety and Depression Scale anxiety and depression domains were 3.5 and 2.2, respectively, and mean Vitiligo-specific Quality of Life index score was 26.9. Patients with facial involvement experienced higher burden than those without. A high patient burden was reported by dermatologists and their patients with vitiligo who had≤10% affected body surface area, including psychosocial and psychological consequences. Thesefindings highlight an unmet need in the treatment of vitiligo.

Evaluating the cost-effectiveness of voclosporin for the treatment of lupus nephritis in the United States.

Lupus nephritis (LN) is a severe manifestation of systemic lupus erythematosus; up to 30% of patients with LN will develop end-stage kidney disease (ESKD). One of the main treatment goals for LN is preservation of kidney function, with early decreases in proteinuria associated with improved long-term outcomes. Voclosporin, a second-generation calcineurin inhibitor, was approved in the United States in 2021 for the treatment of active LN combined with background immunosuppression. The AURORA 1 study found that the use of voclosporin with low doses of mycophenolate mofetil and glucocorticoids yielded significant reductions in proteinuria. The AURORA 2 study showed long-term efficacy and safety of voclosporin over a 3-year period with kidney function preservation. The Institute for Clinical and Economic Review (ICER) is a nonprofit organization that evaluates medical evidence to help improve patient outcomes and control costs. In 2021, ICER published an economic model to estimate the impact and cost-effectiveness of LN therapies. From a US health care perspective, voclosporin was cost-effective at $149,260 per quality-adjusted life-year (QALY) and $131,528 per equal value of life-years gained (evLYG). At the time of the LN cost-effectiveness model (CEM) development, voclosporin was not yet approved in the United States and the cost of treating patients with LN with ESKD was not captured in the literature. To evaluate the cost-effectiveness of voclosporin given the emergence of new data. The LN CEM uses a short-term trial-based Markov model and long-term extrapolation using partitioned survival modeling data assuming adults with LN start with active disease, transitioning to complete or partial renal response, kidney failure, or death. In the current analysis, clinical data for voclosporin, duration of voclosporin treatment for nonresponders, and drug costs reflecting the 2023 price of voclosporin were updated. Additionally, health care payer costs of disease management were incorporated based on real-world claims data on the costs of treating patients with LN. Using the LN CEM with inputs reflecting the latest and most relevant evidence, the incremental cost of voclosporin per QALY was $88,076 and per evLYG was $77,643. For a subpopulation of Black, Hispanic, and Latino patients, the incremental cost of voclosporin per QALY was $77,435 and per evLYG was $67,828. Following the inclusion of updated data in the cost-effectiveness analysis, voclosporin remains a cost-effective therapy for the treatment of active LN including in a Black, Hispanic, and Latino subpopulation, substantially below the ICER willingness-to-pay threshold of $150,000/QALY.

Abstract PO1-18-01: Patient Preferences for HR+/HER2- Metastatic Breast Cancer Treatments in Italy: A Qualitative Assessment

Abstract Objectives: Factors that influence treatment preferences of patients with hormone receptor-positive/HER2-negative (HR+/HER2-) metastatic breast cancer (mBC) are being investigated using discrete choice experiment (DCE) methodology. Appropriate attributes for the ongoing quantitative online survey phase were identified by this initial qualitative pilot study. Methods: To generate hypotheses and inform variables of the quantitative survey, the pilot phase consisted in online telephone interviews with patients with a diagnosis of Stage IV HR+/HER2- breast cancer at various stages of treatment. Interviews focused on exploring patients' perspectives on their interaction with physicians, preferences, and treatment experiences. Transcripts were coded using NVivo software to identify key themes. The ongoing DCE online survey aims to recruit patients meeting the same selection criteria as in the qualitative phase, also at various stages of treatment. Results: Fifteen mBC patients (median age, 43 years [range, 29-64]), including nine chemotherapy-treated and six without chemotherapy exposure, were enrolled in the pilot qualitative study. Eight and four patients reported receiving little information about their treatment plan and their risk status beyond the risk of relapse, respectively. Most participants did not know what to expect from their therapy but expressed trust in their physician’s decision in terms of treatment goals. Few patients (n=3) looked for a second opinion before starting treatment. Four out of fifteen patients declared that they were actively involved in their treatment plan decision, although all relied on their physician’s knowledge and decision. Reduced symptoms and improved quality of life (QoL) were patients’ main treatment goals, and side-effects and efficacy of treatment were their main concerns. When asked about the degree of acceptability of certain side effects, some participants considered hair loss and vomiting as unacceptable side effects, regardless of severity, while others considered them to be the most acceptable. This discrepancy is to be interpreted in the light of participants’ own treatment journey. Severity and duration of side-effects were mentioned by three patients as acceptability factors. In a choice task, patients were asked to express preferences between two hypothetical treatments. Efficacy (n=9) and administration mode (n=6) were spontaneously deemed important for treatment selection. While assessing the difference in risk between the two hypothetical treatments, ten patients explained that the main choice feature was extended progression-free survival (PFS) rate. Risk of infection and neutropenia (n=12) were concerns expressed by most patients in the post-COVID-19 period. Considering their substantial impact on QoL, alopecia (n=9) and vomiting (n=9) were presented by patients as particularly important, regardless of their degree of severity; whereas almost all patients reported that only severe fatigue (n=12), diarrhea (n=13) or nausea (n=13) would be an issue. These results allowed selection of the following attributes for further quantification of their preference weights in a DCE: PFS, risk of neutropenia, risk of alopecia, risk of vomiting, risk of diarrhea, risk of grade 3/4 side effects and mode of administration. Conclusions: This qualitative pilot study allowed for identification of what matters most to patients when selecting a treatment for mBC, which will be further assessed in the DCE. It also highlighted the need of patients with HR+/HER2- mBC for information about their treatment, potential side-effects, and health management. This research suggests that there is a need for patients to be more involved in treatment plans, and that taking patient preferences into account may help improve the treatment selection experiences. Citation Format: Grazia Arpino, Carmine De Angelis, Lorenzo Gerratana, Matteo Lambertini, Sarah Igidbashian, Martina Bellini, Serena Giuntoli, Xavier Guillaume, Julie Behillil, Claire Graziani-Taugeron. Patient Preferences for HR+/HER2- Metastatic Breast Cancer Treatments in Italy: A Qualitative Assessment [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO1-18-01.

A feasibility study to assess a co-designed behaviour change intervention to support people living with achalasia

Abstract Introduction Achalasia is a rare oesophageal condition that affects the motility of the oesophageal body and the relaxation of the lower oesophageal sphincter. Even the most effective treatments are unlikely to be curative. The main goal of medical treatments and interventions are mitigation of symptoms. The medical interventions are pharmacologic, endoscopic and surgical treatments to achieve symptom relief. As all medical treatments only help to alleviate symptoms, it is important for people living with achalasia to use non-pharmacological interventions to manage their condition. Aim This study aimed to evaluate the feasibility of recruitment and assess the acceptability of a co-designed behaviour change intervention, targeting eating in a social setting for people living with achalasia. Methods The mixed-method study used pre- and post-intervention questionnaires and semi-structured post-intervention interviews. Achalasia Action (a UK-based support group) emailed members the study information and participants contacted the researcher to take part. The intervention was a workbook designed collaboratively by the researchers and people living with achalasia, with strategies built on the COM-B model (Capability, Opportunity, Motivation).[1] Participants were given up to two months to complete the workbook. The interview was optional and it was an opportunity for participants to give further feedback on the intervention. Quantitative measures were subjected to descriptive analysis reporting recruitment and retention rates, and self-reported changes in eating behaviours. Qualitative data were analysed descriptively using the APEASE criteria (Affordability, Practicality, Effectiveness and cost-effectiveness, Acceptability, Side-effects/safety, and Equity).[2] Results The study aimed to recruit 20 participants, and this target was achieved, resulting in a 100% recruitment rate. However, the post-intervention questionnaires were completed by only 10 participants, indicating a 50% retention rate from baseline. Quantitative measures demonstrated an improvement in confidence and enjoyment levels while eating in a social setting post-intervention and a decrease in symptoms such as pain, regurgitation, stress and anxiety. Qualitative feedback (n=5) on the intervention described enhanced social support and improved symptom management of achalasia in a social setting. Furthermore, the intervention met the APEASE criteria, indicating its usability and acceptability. Conclusion The recruitment success of this study underscores the feasibility of involving people living with achalasia in intervention research. However, with a retention rate of only 50% at follow-up, it is evident that future studies should consider recruiting a larger baseline sample to ensure the target is achieved. The study acknowledges that the sample size was small and limits the generalisability of the findings. However, this is a characteristic typical of feasibility studies; therefore, the study is designed to assess the feasibility of recruitment and implementing a co-designed intervention rather than drawing conclusions on its efficacy. The positive outcomes of the co-designed intervention highlights the importance of user involvement in developing interventions. The intervention demonstrated the potential to support people living with achalasia in eating in a social setting. The co-designed intervention has significant practical implications by providing healthcare professionals and support groups with a feasible, effective method to enhance the social eating experience of people living with achalasia, potentially improving their overall quality of life.

Interleukin 17 as a central component of the pathogenesis of pain associated with immunoinflammatory process: A new “target” of pharmacotherapy

Modern pathogenetic therapy of inflammatory rheumatic diseases (IRD) is aimed not only at reducing disease activity (although achieving remission and low disease activity remains the main goal of treatment), but also at eliminating as quickly and completely as possible the main symptoms that cause a decrease in the quality of life of patients. Particular importance is attached to effective control of chronic pain – the main and most distressing manifestation of IRD. To solve this problem, the pathogenesis of chronic pain in IRD continues to be actively studied, aimed at finding new ”targets” of pharmacotherapy. Thus, the role of central sensitization (CS) and comorbid fibromyalgia in the formation of clinical manifestations of IRD is now clearly proven. Signs of CS, depending on the instrument of its detection, are determined in 20–40% of patients with rheumatoid arthritis (RA), psoriatic arthritis (PsA) and axial spondyloarthritis (AxSpA).Interleukin (IL) 17 plays a fundamental role in the development of chronic pain in IIRD. This cytokine takes a leading position in the development of the ”cytokine cascade”, inducing the synthesis of various cytokines and chemokines, as well as chemotaxis and activation of neutrophils and T cells. Induction of synthesis of inflammatory mediators (including prostaglandin E2) determines the role of IL-17 in activation of nociceptors and their sensitization. IL-17 also takes an active part in neuroimmune interactions by activating glia cells and affecting receptors present on the membrane of neurons of the posterior horns of the spinal cord. This defines the role of IL-17 as one of the inductors of CS development. Pharmacologic blockade of IL-17 is a known pathway to suppress the activity of IIRPs such as PsA and AxSpA. However, this mechanism also allows for significant effects on chronic pain. In particular, the IL-17 inhibitor ixekizumab has shown high analgesic potential in a series of studies in PsA and AxSpA (SPIRIT-P1 and SPIRIT-P2, COAST V and COAST W). It is important to note that this drug demonstrated a very rapid analgesic effect: pain intensity was significantly reduced already 7 days after the first injection. These data suggest a specific effect of ixekizumab on the nociceptive system, independent of the anti-inflammatory effect. This fact allows us to consider ixekizumab as a drug of choice for the treatment of patients with PsA and AxSpA who experience severe pain and have signs of CS and fibromyalgia.