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Background: Realistic cancer treatment goals should be used by health care professionals and communicated to patients, families, and the public. The current nomenclature on this subject is outdated and has not been changed since the advent of modern oncology in the middle of the 20th century. Methods: Based on the literature we propose a three-tier system composed of curative, palliative, and potentially life-prolonging (PLP) therapies, instead of the current two-tier system of only curative and palliative treatment. Results: The new system introduces the notion of prolonged survival. Furthermore, the negative connotation linked to palliative care is also eliminated in this setting. Conclusion: The current terminology used to describe cancer treatment goals has not been updated since the mid-20th century and it is time for a more modern approach. We propose a three-tier system: (1) curative treatment, (2) palliative care, and (3) potentially life-prolonging therapy. [ABSTRACT FROM AUTHOR]

Juvenile idiopathic arthritis (JIA) is a chronic arthritis of unknown cause that develops in patients younger than 16 years of age and persists for at least 6 weeks. It is an important cause of short- and long-term physical and mental impairments in children. The goal of treatment for JIA is remission. A T2T (treatment-to-target) has been proposed and practiced as a means of achieving remission. The method of evaluating the disease activity of JIA depends on the disease type. For systemic JIA, disease activity is determined by comprehensively considering joint findings, systemic inflammatory findings, changes in inflammatory and synovitis markers, imaging findings, and other factors. For articular JIA other than systemic JIA, the Juvenile Arthritis Disease Activity Score (JADAS-27) is used to evaluate disease activity. The CHAQ (Childhood Health Assessment Questionnaire) and the Japanese version of the modified Rankin Scale (mRS) are mainly used to assess the physical function and ADL. The CHAQ is a global standard assessment method with the advantage that it can be transitioned to the HAQ used in adults, making it useful for international comparisons. The mRS is used to classify the severity of JIA as a chronic disease, and is an indispensable evaluation method in the specific disease procedure in Japan. It is necessary to have pediatric-specific knowledge of growth and development and routine childhood immunizations and to consider transition support tailored to the patient's situation. Ultimately, the goal is to foster the patient's independence and to provide an uninterrupted follow-up in the adult care department. Continuous follow-up will be provided during the schooling (and later, employment) period, and the relationship with the patient will be tailored to their developmental stage. It is also important to understand and communicate the importance of contraception and the drugs that cannot be used during pregnancy. [ABSTRACT FROM AUTHOR]

Introduction: Medications used to treat type 2 diabetes (T2D) often require dose escalation to optimize effectiveness. Physician and patient perceptions of treatment characteristics of T2D medications have previously been examined, but little is known about perceptions of escalation to the optimal dose for each patient. This study examined physicians' perceptions of dose escalation for medications used to treat T2D. Methods: Data on dose escalation and other factors influencing decision-making for treatment of T2D were collected via an online survey of endocrinologists and primary care physicians in the USA. Results: The sample included 501 physicians (348 primary care physicians and 153 endocrinologists). Dose escalation was not frequently considered by physicians as a primary factor keeping patients' from reaching treatment goals (mentioned as a factor by only 7.6% of the sample) or a barrier to prescribing T2D medication (16.2%). Factors more likely to keep patients from reaching treatment goals included an unhealthy diet (86.6%) and medication adherence (77.4%). The most common reasons that physicians reported for escalating dose levels were the need for better glycemic control (reported by 89.8% of the sample), ability to decrease the total number of medications by increasing the dose of one medication (39.9%), and the need for the patient to lose weight (39.3%). Data reported by primary care physicians and endocrinologists followed similar patterns. Conclusions: Although common with T2D treatments, escalating the dose of T2D medication was not perceived by physicians to be a significant barrier to attaining treatment goals or prescribing medication. Multiple factors contribute to the decision to escalate the dose of T2D medication. Plain Language Summary: In early phases of initiating medication treatment for a patient with type 2 diabetes (T2D), it is common for physicians to increase from a lower initial dose to a higher end dose to maximize treatment benefit. This process is known as dose escalation. The purpose of this study was to examine physicians' perceptions of dose escalation for medications used to treat T2D. An online survey was designed to identify reasons why physicians in the US may choose to escalate or not escalate a dose of medication for T2D. In addition, physicians were asked about factors that keep patients from reaching treatment goals to identify whether the requirement for dose escalation is perceived to be a common barrier to successful treatment. The sample included 501 physicians (348 primary care, 153 endocrinologists). Dose escalation was not frequently considered to be a primary factor keeping patients' from reaching treatment goals or a barrier to prescribing medication for T2D. Dose escalation decisions are complex, driven by a range of factors such as glycemic control medication tolerability, the patient's body mass index, treatment guidelines, comorbidities, characteristics of the patient's entire treatment regimen, and potential cardiovascular benefits. [ABSTRACT FROM AUTHOR]

Juvenile idiopathic arthritis (JIA) is a chronic arthritis of unknown cause that develops in patients younger than 16 years of age and persists for at least 6 weeks. It is an important cause of short- and long-term physical and mental impairments in children. The goal of treatment for JIA is remission. A T2T (treatment-to-target) has been proposed and practiced as a means of achieving remission. The method of evaluating the disease activity of JIA depends on the disease type. For systemic JIA, disease activity is determined by comprehensively considering joint findings, systemic inflammatory findings, changes in inflammatory and synovitis markers, imaging findings, and other factors. For articular JIA other than systemic JIA, the Juvenile Arthritis Disease Activity Score (JADAS-27) is used to evaluate disease activity. The CHAQ (Childhood Health Assessment Questionnaire) and the Japanese version of the modified Rankin Scale (mRS) are mainly used to assess the physical function and ADL. The CHAQ is a global standard assessment method with the advantage that it can be transitioned to the HAQ used in adults, making it useful for international comparisons. The mRS is used to classify the severity of JIA as a chronic disease, and is an indispensable evaluation method in the specific disease procedure in Japan. It is necessary to have pediatric-specific knowledge of growth and development and routine childhood immunizations and to consider transition support tailored to the patient’s situation. Ultimately, the goal is to foster the patient’s independence and to provide an uninterrupted follow-up in the adult care department. Continuous follow-up will be provided during the schooling (and later, employment) period, and the relationship with the patient will be tailored to their developmental stage. It is also important to understand and communicate the importance of contraception and the drugs that cannot be used during pregnancy.

Research on acceptability of non-abstinence goals has focused primarily on addiction counsellors and lacks representation of clinical social workers (SWs). We examined whether acceptability of non-abstinence varied as a function of a client's severity of substance use disorder (SUD) diagnosis (severe vs. moderate), finality of an outcome goal (intermediate vs. final), and type of substance (e.g. alcohol, cannabis) among clinical SWs in the US (United States). We surveyed a geographically representative sample of 309 SWs (Mean age = 48.9; Female = 76%; Mean years practicing = 14.6). For tobacco, alcohol and cannabis use, most SWs (51%–84%) rated non-abstinence as an acceptable goal regardless of finality of the outcome goal for clients with a moderate SUD; but smaller proportions rated non-abstinence acceptable for clients with severe SUD regardless of drug type or finality of the goal. Higher ratings of acceptability of non-abstinence were significantly associated with younger age and lower drug use stigma. Supporting clients who pursue non-abstinence is consistent with the values of the Social Work (SW) profession (e.g. self-determination, social justice, dignity/self-worth, importance/centrality of human relationships). Thus, SW education and training programmes should bolster understanding SUD recovery can take multiple pathways, should be self-directed and empowering, and may be defined in terms of improved health and wellness. [ABSTRACT FROM AUTHOR]

Purpose: Post-operative outcome after total knee arthroplasty (TKA) in the treatment of end-stage osteoarthritis correlates strongly with pre-operative impairment-driven patient treatment goals. However, a clinical tool for measuring patient treatment goals in correlation to impairments is still missing, which impedes patient-oriented indication in TKA. Methods: Patients scheduled for TKA were recruited in four German hospitals. All patients were handed the INDICATE Knee Score pre-operatively. The score contains 31 treatment goals with respective impairments, subdivided into seven categories. They were asked to rank all treatment goals and impairments on a 3-point scale. Treatment goals and impairments were then checked for frequency of occurrence. Correlation of goal and impairment was tested. Analysis for associations of treatment goals and different cohort characteristics (age, sex, BMI) was conducted. Results: 1.298 patients were included in the study. Seven treatment goals were categorised as "main goal" from more than 90% of all patients ("knee pain", "range of motion", "walking distance", "overall physical function", "climbing stairs", "quality of life", "implant survival"). Comparing age groups, there were significant associations towards higher expectations regarding working, physical and sports related treatment goals in younger patients (< 65y) ("ability to work" (P ≤.001), "sports activities" (P ≤.001), "sex life" (P ≤.001), "dependence on help of others" (P =.015), "preventing secondary impairment" (P =.03), "dependence on walking aids" (P =.005)). Higher BMI resulted in increasing relevance of "weight reduction" (P ≤.001), "climbing stairs" (P =.039) "global health status" (P =.015) and "long standing" (P =.007) as a "main goal". Analysis for differences in treatment goals regarding sex showed women choosing more treatment goals as "main goals" than men. Conclusion: Seven treatment goals which were expected by > 90% in our collective can be classified as general treatment goals for TKA. Demographic factors (age, sex, BMI) were significantly associated with patients' expectations for TKA. We conclude physicians should clearly assess their patients' demands prior to TKA to maximise post-operative outcome. Level of evidence: Prognostic Level III. [ABSTRACT FROM AUTHOR]

A study published in Modern Rheumatology examined the factors influencing the achievement of treatment goals for osteoporosis in women with rheumatoid arthritis (RA) who were receiving denosumab. The study found that T-scores at the lumbar spine, total hip, and femoral neck were significantly associated with treatment goal achievement. The study also suggested that therapeutic drugs for RA did not impact the effectiveness of denosumab treatment for osteoporosis. However, the study had limitations due to a small sample size, and further research with larger sample sizes is necessary. The document is a table presenting data on various factors related to osteoporosis and rheumatoid arthritis, comparing two groups of patients based on age, body weight, prior osteoporosis treatment, disease duration, and medical measurements. The data is presented objectively, without additional information or analysis. The authors and their affiliations are listed at the end of the document. [Extracted from the article]

• For this study data from a randomized trial performed in 28 patients with FD were used. • Friedewald, Martin-Hopkins, direct assay and gels to determine LDL-C were compared to UC. • All methods over- or underestimated LDL-C concentrations compared to UC. • UC probably overestimates LDL-C concentrations as well. • Non-HDL-C measured by standard assays performed well compared to non-HDL-C by UC. To compare LDL-C concentrations using the Friedewald formula, the Martin-Hopkins formula, a direct assay and polyacrylamide gradient gel electrophoresis (PGGE) to the reference standard density gradient ultracentrifugation in patients with Familial Dysbetalipoproteinemia (FD) patients. We also compared non-HDL-cholesterol concentrations by two methods. For this study data from 28 patients with genetically confirmed FD from the placebo arm of the EVOLVE-FD trial were used. Four different methods for determining LDL-C were compared with ultracentrifugation. Non-HDL-C was measured with standard assays and compared to ultracentrifugation. Correlation coefficients and Bland-Altman plots were used to compare the methods. Mean age of the 28 FD patients was 62 ± 9 years, 43 % were female and 93 % had an ɛ2ɛ2 genotype. LDL-C determined by Friedewald (R2 = 0.62, p <0.01), Martin-Hopkins (R2 = 0.50, p = 0.01) and the direct assay (R2 = 0.41, p = 0.03) correlated with density gradient ultracentrifugation. However, Bland-Altman plots showed considerable over- or underestimation by the four methods compared to ultracentrifugation. Non-HDL-C showed good correlation and agreement. In patients with FD, all four methods investigated over- or underestimated LDL-C concentrations compared with ultracentrifugation. In contrast, standard non-HDL-C assays performed well, emphasizing the use of non-HDL-C in patients with FD. [ABSTRACT FROM AUTHOR]

The population‐based Understanding Psoriatic Disease Leveraging Insights for Treatment (UPLIFT) survey was designed to better understand patient and dermatologist perceptions of the disease burden of psoriasis (PsO) and their treatment expectations. UPLIFT was a cross‐sectional, quantitative, online survey conducted in Europe, North America, and Japan between 2 March and 3 June 2020. In Japan, 391 patients reporting a diagnosis of PsO and/or psoriatic arthritis (PsA) were surveyed (75% had PsO alone, 23% had PsO and PsA, and 2% had PsA alone). Self‐reported body surface area (BSA) data were available for 309 Japanese patients, with the majority (80%) reporting PsO‐involved BSA ≤3 palms. Current symptoms of PsO were rated as moderate or severe by 43% of Japanese patients with BSA ≤3 palms, and severe by 44% of patients with BSA 4–10 palms. PsO frequently occurred in ≥1 special areas, most commonly the scalp in 76% of Japanese patients with BSA ≤3 palms, and ≥90% of those with BSA ≥4 palms. Furthermore, musculoskeletal symptoms in 42% of patients with PsO alone were suggestive of PsA. Whereas Japanese patients with BSA ≤3 palms mainly reported receiving topical therapy alone (34%) or no treatment (32%), 64% patients with BSA 4–10 palms reported receiving systemic therapy. Overall, 21% of Japanese patients with self‐perceived mild symptoms of PsO and 48% of patients with special area involvement experienced at least a moderate impact of disease on quality of life (Dermatology Life Quality Index score >5). Moreover, patients and dermatologists differed in their perceptions of determinants of PsO severity and treatment, and office visit discussions. In general, these findings from the Japanese subgroup of the UPLIFT survey demonstrated that a high proportion of patients perceived their symptoms to be moderate or severe irrespective of the level of skin involvement, suggesting a persistent unmet treatment need. [ABSTRACT FROM AUTHOR]

Abstract Background Follow-up care is crucial but challenging for disease management particularly in rural areas with limited healthcare resources and clinical capacity, yet few studies have been conducted from the perspective of rural primary care physicians (PCPs). We assessed the frequency of follow-up care delivered by rural PCPs for hypertension and type 2 diabetes – the two most common long-term conditions. Methods We conducted a multi-centre, self-administered survey study built upon existing general practice course programmes for rural PCPs in four provinces. Information on follow-up care delivery were collected from rural PCPs attending centralised in-class teaching sessions using a set of close-ended, multiple choice questions. Binary logistic regression analysis was performed to examine physician-level factors associated with non-attainment of the target frequency of follow-up care for hypertension and type 2 diabetes, respectively. The final sample consisted of rural PCPs from 52 township-level regions. The Complex Samples module was used in the statistical analysis to account for the multistage sample design. Results The overall response rate was 91.4%. Around one fifth of PCPs in rural practices did not achieve the target frequency of follow-up care delivery (18.7% for hypertension; 21.6% for type 2 diabetes). Higher education level of physicians, increased volume of daily patients seen, and no provision of home visits were risk factors for non-attainment of the target frequency of follow-up care for both conditions. Moreover, village physicians with less working experiences tended to have less frequent follow-up care delivery in type 2 diabetes management. Conclusions Efforts that are solely devoted to enhancing rural physicians’ education may not directly translate into strong motivation and active commitment to service provision given the possible existence of clinical inertia and workload-related factors. Risk factors identified for target non-attainment in the follow-up care delivery may provide areas for capacity building programmes in rural primary care practice.

The article contain the main challenges meet doctors in real practice using biologics for treatment patients with psoriasis.Different aspects and concerns have been analised from new point of view: the aims of Bx therapy (incl. control of remission, personalized goals, etc.) primary and secondary responses, immunogenicity, treatment survival, retention rate or long lasting regain of response in psoriasis patients treated by biologics.

Background: Follow-up care is crucial but challenging for disease management particularly in rural areas with limited healthcare resources and clinical capacity, yet few studies have been conducted from the perspective of rural primary care physicians (PCPs). We assessed the frequency of follow-up care delivered by rural PCPs for hypertension and type 2 diabetes – the two most common long-term conditions. Methods: We conducted a multi-centre, self-administered survey study built upon existing general practice course programmes for rural PCPs in four provinces. Information on follow-up care delivery were collected from rural PCPs attending centralised in-class teaching sessions using a set of close-ended, multiple choice questions. Binary logistic regression analysis was performed to examine physician-level factors associated with non-attainment of the target frequency of follow-up care for hypertension and type 2 diabetes, respectively. The final sample consisted of rural PCPs from 52 township-level regions. The Complex Samples module was used in the statistical analysis to account for the multistage sample design. Results: The overall response rate was 91.4%. Around one fifth of PCPs in rural practices did not achieve the target frequency of follow-up care delivery (18.7% for hypertension; 21.6% for type 2 diabetes). Higher education level of physicians, increased volume of daily patients seen, and no provision of home visits were risk factors for non-attainment of the target frequency of follow-up care for both conditions. Moreover, village physicians with less working experiences tended to have less frequent follow-up care delivery in type 2 diabetes management. Conclusions: Efforts that are solely devoted to enhancing rural physicians' education may not directly translate into strong motivation and active commitment to service provision given the possible existence of clinical inertia and workload-related factors. Risk factors identified for target non-attainment in the follow-up care delivery may provide areas for capacity building programmes in rural primary care practice. [ABSTRACT FROM AUTHOR]

Introduction: We examined differences in social and sociodemographic characteristics and treatment goals between people with primary alcohol use disorder (AUD) versus those with a primary drug use disorder receiving inpatient treatment for a substance use disorder (SUD). Methods: A national census utilizing a cross sectional design included 56 of 60 specialized inpatient SUD treatment clinics in Norway and all patients receiving treatment on a specific date (responserate = 70%). Data on substance use, social and sociodemographic characteristics, and patient-reported treatment goals were collected. Patients were classified as having primary AUD or a drug use disorder based on the main SUD diagnosis relevant to the treatment episode. Results: The analytic sample included 1093 patients. Patients with primary AUD (n = 362) were more often older, had a higher educational level and income from work, and lived in permanent housing compared with patients with a drug use disorder (n = 731). Patients with AUD were more likely to have good relationships with friends. The higher frequency of reported reduced substance use (versus quitting substance use) as the treatment goal among AUD patients disappeared when controlled for sociodemographic factors. Conclusions: Knowledge about the different characteristics of inpatients with AUD versus a drug use disorder is relevant when conducting research involving the SUD treatment population and for facilitating treatment. The lower frequency of perceived support from friends among patients with a drug use disorder suggests a need for targeted efforts in (re)building supportive social relationships for inpatients being treated for SUD.

Introduction There are many possible treatment goals for patients with schizophrenia. Two major perspectives on treatment goals are the patient's and the physician's perspective. Patient-centered treatment mandates that an individual patient's treatment goals are taken into account when treatment is planned. In this narrative review, we address the commonalities and differences of the patient's and physician's perspectives. Methods We searched for literature on treatment goals for patients with schizophrenia from the last 10 years. Results Fifty-two relevant records were identified, 4 of which directly compare patient's and physician's perspectives. Two further articles used the same set of goals to ask patients or physicians for their assessment. Discussion Agreement between patients and physicians regarding valuation of treatment goals was high. However, physicians tended to put more emphasis on the classical "textbook" goals of symptom resolution and functioning, while patients stressed well-being and quality of life more. Results on treatment goals from patients are difficult to generalize, since recruiting representative patient samples is challenging and patient subgroups may have differing priorities. [ABSTRACT FROM AUTHOR]

The long-term goal of asthma management is to achieve disease control, comprising the assessment of 2 main domains: (1) symptom control and (2) future risk of adverse outcomes. Decades of progress in asthma management have correlated with increasingly ambitious disease control targets. Moreover, the introduction of precision medicines, such as biologics, has further expanded the limits of what can be achieved in terms of disease control. It is now believed that clinical remission, a term rarely associated with asthma, may be an achievable treatment goal. An expert framework published in 2020 took the first step toward developing a commonly accepted definition of clinical remission in asthma. However, there remains a widespread discussion about the clinical parameters and thresholds that should be included in a standardized definition of clinical remission. This review aims to discuss on-treatment clinical remission as an aspirational outcome in asthma management, drawing on experiences from other chronic diseases where remission has long been a goal. We also highlight the integral role of shared decision-making between patients and health care professionals and the need for a common understanding of the individual patient journey to remission as foundational elements in reducing disease burden and improving outcomes for patients with asthma., (Copyright © 2024. Published by Elsevier Inc.)

Background Patients with malignant gliomas have a poor prognosis. However, little is known about patients' and caregivers' understanding of the prognosis and the primary treatment goal. Methods We conducted a prospective study in patients with newly diagnosed malignant gliomas (N = 72) and their caregivers (N = 55). At 12 weeks after diagnosis, we administered the Prognosis and Treatment Perceptions Questionnaire to assess understanding of prognosis and the Hospital Anxiety and Depression Scale to evaluate mood. We used multivariable regression analyses to explore associations between prognostic understanding and mood and McNemar tests to compare prognostic perceptions among patient-caregiver dyads (N = 48). Results A total of 87.1% (61/70) of patients and 79.6% (43/54) of caregivers reported that it was "very" or "extremely" important to know about the patient's prognosis. The majority of patients (72.7%, [48/66]) reported that their cancer was curable. Patients who reported that their illness was incurable had greater depressive symptoms (B = 3.01, 95% CI, 0.89-5.14, P =.01). There was no association between caregivers' prognostic understanding and mood. Among patient-caregiver dyads, patients were more likely than caregivers to report that their primary treatment goal was cure (43.8% [21/48] vs 25.0% [12/48], P =.04) and that the oncologist's primary goal was cure (29.2% [14/48] vs 8.3% [4/48], P =.02). Conclusions Patients with malignant gliomas frequently hold inaccurate perceptions of the prognosis and treatment goal. Although caregivers more often report an accurate assessment of these metrics, many still report an overly optimistic perception of prognosis. Interventions are needed to enhance prognostic communication and to help patients cope with the associated distress. [ABSTRACT FROM AUTHOR]

Introduction: Hypertension and dyslipidaemia are major risk factors for cardiovascular diseases and achieving treatment goals mitigates such risks. This study determined demographic and medication-related factors associated with blood pressure (BP) goal attainment in patients with concomitant hypertension and dyslipidaemia.Methods: This paper is a sub-analysis of the Lipid HEALTH study, a questionnaire survey focusing on adult Asian patients with both dyslipidaemia and hypertension. An interviewer-administered questionnaire was used to obtain demographic and clinical information. Laboratory and prescription data was retrieved from electronic health records. BP goals were defined by international guidelines. Data was audited and analysed, and logistic regression analysis was used to identify factors determining BP goal attainment.Results: Among the 851 included patients, 49.7% attained their BP goals. 37.0% were on monotherapy, 57.6% on ≥ 2 BP-lowering medications, and 5.4% had no pharmacologic treatment. Among those on pharmacotherapy, 51.2% failed to achieve BP goals. Calcium channel blockers were the most frequently prescribed medications. Attainment of BP goals was significantly associated with: not having Type 2 diabetes mellitus (odds ratio [OR] 2.27, 95% confidence interval [CI] 1.61-3.13); attaining low-density lipoprotein cholesterol goal (OR 2.02, 95% CI 1.45-2.81); being solely on dietary control (OR 2.19, 95% CI 1.09-4.39); and receiving monotherapy (OR 1.71, 95% CI 1.18-2.48).Conclusion: BP treatment goals were attained by half of the patients with dyslipidaemia and hypertension, and half of those on pharmacotherapy. Type 2 diabetes mellitus and low-density lipoprotein cholesterol control were significantly associated with BP goal attainment. [ABSTRACT FROM AUTHOR]

People with psychosis benefit enormously from Cognitive Behavioural Therapy for Insomnia (CBTI), although some variability exists in treatment outcomes. While recent efforts have focused on profiling sleep at treatment initiation, an alternative methodological approach involves using treatment response as a starting point to better understand what constitutes an effective treatment. This study used Grade of Membership analysis (GoM) to estimate the occurrence of unique treatment outcomes and associated patient characteristics. Outcome measures included changes in sleep (self-reported latency, efficiency, duration, quality), functional outcomes (daytime dysfunction, negative mood, need for sleep medication) and treatment goal, collected in 50 individuals with a psychotic disorder and insomnia who underwent CBT-I treatment. Three distinct profiles were identified: (1) Strong responders, who met their treatment goals and showed broad improvements in both sleep and functional domains; (2) Partial responders, who showed sleep improvements (particularly in total sleep time), without noticeable gains in function, and who were predominantly female; and (3) Non-responders who showed little treatment response and failed to meet their treatment goals. This group was also more likely to have greater psychopathology (high levels of negative mood and psychotic symptoms, and antipsychotic medication dosage). These findings suggest that (i) CBT-I can serve to improve sleep and daytime function (although sleep can improve independently from function), (ii) client-elicited treatment goals are a key predictor of CBT-I response, (iii) other important variables associated with treatment response include sleep profile, clinical acuity, and sex. [ABSTRACT FROM AUTHOR]

Background: To assess the efficacy and safety of lipid-lowering treatment in children with heterozygous familial hypercholesterolemia (HeFH) aged ≤12 years attending a tertiary hospital-based outpatient lipid clinic. Methods: Data in 318 children from the University Hospital of Ioannina (Northwestern Greece) Outpatient Lipid Clinic Project for Children and Adolescents with Dyslipidemia from March 2009 to December 2018 were analyzed. We assessed the efficacy and safety treatment alongside any possible predictors of the achievement of the treatment target. Results: Of 318 children with hyperlipidemia, 72 were diagnosed having HeFH based on clinical criteria and genetic confirmation. Compared with non-familial hypercholesterolemia (non-FH) children, those with FH had a higher occurrence of positive family history of premature cardiovascular disease, and higher levels of total, low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apoB) and lipoprotein (a) (Lp(a)). Treatment regimens included either atorvastatin 10–20 mg/day, rosuvastatin 5–10 mg/day, pitavastatin 2–4 mg/day monotherapy or in combination with ezetimibe. The treatment goal of LDL-C (<135 mg/dL, 3.5 mmol/L) was achieved in 69% of children treated. The achievement of the treatment targets correlated positively with male sex and inversely with the Dutch Lipid Clinic Network Score, baseline total, LDL-C and apoB levels. No clinically significant changes in liver or muscle-related laboratory tests were reported; no effect on growth or sexual maturation was noted. Conclusions: This study confirms that lipid-lowering treatment in HeFH children initiated in the setting of a specialized tertiary hospital-based outpatient lipid clinic is efficacious and safe. Children of male sex and low baseline lipid values had a better achievement of treatment target. [ABSTRACT FROM AUTHOR]

Introduction: Although rapid evolution over the past few years in advanced soft tissue sarcoma (STS) management has not been without its challenges, it has brought clarity in several areas. Areas covered: This article summarizes the proceedings of the third edition of the Soft Tissue Sarcoma: Evidence and Experience symposium held March 2019 in Madrid, Spain. An update is provided of current approaches to advanced STS management. Case studies illustrate the role of trabectedin in advanced STS management. Expert opinion: First-line treatment of advanced STS requires distinct therapeutic strategies depending on goal: tumor shrinkage or tumor control. Since all sarcoma patients benefit from active treatment irrespective of age or line of therapy, oncologists have a duty to offer active systemic therapies unless the patient is unfit for treatment or chooses to end active treatment. Beyond the first line, histology becomes increasingly relevant for treatment selection. Agents with activity in specific sarcoma subtypes have been identified. Rare tumors represent a substantial medical need requiring strong international collaboration between research groups, pharmaceutical companies, regulatory agencies, and patients to identify active drugs per subtype. Multidisciplinary care in an expert sarcoma center is the primary means of reducing morbidity and mortality in patients with sarcoma. [ABSTRACT FROM AUTHOR]

Background: Psoriasis is a chronic inflammatory skin disorder with marked impact on quality of life. In view of the well-recognized need to integrate the patient perspective in psoriasis care, treatment goals which originate entirely from the experiences of the patients are of utmost importance. Objective: To explore treatment goals in patients with psoriasis that originate entirely from the patient perspective. Methods: Data was generated using face-to-face in-depth interviews with 15 Dutch psoriasis patients, which were analyzed using template analysis resulting in 2 first-level, 10 second-level, and 14 third-level themes. Results: A wide variety of treatment goals was reported. Almost all patients reported their 'ultimate' treatment goal would be achieving total skin clearance. Barriers in achieving treatment goals included the fact that psoriasis is a chronic disease with no cure available, lack of effectiveness of current treatment options and side effects of treatments of which the treatment effect does not compensate. Conclusion: There is a great variety and inter-individual difference in treatment goals. Therefore, we recommend to define individualized patient-defined treatment goals, as every patient with psoriasis requires a tailor-made treatment program. [ABSTRACT FROM AUTHOR]

In 2011 a European consensus defined the target treatment of psoriasis, with PASI 75 as the main objective and had been adopted in guidelines around the world. In those 9 years the progress of the medications to treat psoriasis have been able to obtain an almost clear skin. Current literature promotes PASI 90, PASI absolute and patient reported outcomes as the goal of recent treatments. PASI 90 and PASI absolute <3 have a strong relationship with DLQI 0-1 and permit to measure the satisfaction of patients and doctors with the psoriasis treatments. This is the moment to move forward to current and realistic goals. [ABSTRACT FROM AUTHOR]

There is limited research on the perceptions of substance use (SU) and treatment goals among patients with substance use disorders (SUD) and attention deficit hyperactivity disorder (ADHD). We investigate whether SU perceptions and treatment goals were different among SUD patients with (SUD+ADHD) and without (SUD-ADHD) ADHD. Twelve SUD+ADHD patients (39.5 ± 8.5 years, 10 men), and 10 age- and substance severity matched SUD-ADHD patients (34.0 ± 10.0 years, six men), consecutively recruited between February 2010 and July 2012 were interviewed during the course of their SUD treatment. Interview data were analyzed using content analysis. The perceived positive aspects of SU were self-regulation of physical health, behavior, feelings, reasoning/thoughts, and the rewarding effects. The perceived negative aspects of SU included consequences on physical health, behavior, feelings, and reasoning/thoughts. SUD+ADHD patients less frequently linked SU to physical health and more frequently perceived SU as helpful to self-regulate their behavior. Four treatment goals categories emerged: total abstinence, conditional abstinence, substance reduction, and unspecified. SUD+ADHD patents less frequently chose total abstinence, and when using more than one substance, they commonly chose variable goals. In contrast, SUD-ADHD patients chose more similar goals. SUD+ADHD patients showed a more complex relation to their SUD than SUD-ADHD patients.

Clinical guidelines for pulmonary hypertension (PH) recommend shared decision-making and individualized treatment. However, patient perspectives on PH treatment goals, preference toward a decision-making style of treatment, and adoption of shared decision-making remain unclear. This cross-sectional questionnaire-based study assessed the patients' preferred and actual participation role in treatment decision-making, rated on 5 scales (ranging from passive [patients leave all decisions to physicians] to active [patients make the decision after physicians show patients several options]) and evaluated the concordance between preferred and actual participation roles. The important factors underlying patients' perspectives in treatment decision-making (i.e., prognosis; symptom, financial, family, and social burdens; patient values; and physician recommendation) were evaluated. Univariate logistic regression analysis was performed to determine the patients with a positive preference toward "physician recommendation" in treatment decision-making. Among 130 patients with PH (median age: 58 years; mean pulmonary arterial pressure: 23 mm Hg; 27.7% were males), 59.2% preferred that "physicians make the decision regarding treatment after showing patients therapeutic options (i.e., intermediate between passive and active roles)." The patient-preferred and actual participation roles in decision-making had moderate agreement (Cohen's kappa = 0.46). The most important factor in treatment decisions was "symptom burden reduction" (93.8%). Although 85.0% of patients chose "physician recommendation" as an important factor, 49.6% chose "alignment with my values." The determinants of patients who chose "physician recommendation" were less severe hemodynamics and better functional capacity. In conclusion, patients with PH preferred that the "physicians make the decision after showing patients therapeutic options" and prioritized physician recommendation over their values., Competing Interests: Declaration of Competing Interest The authors have no competing interests to declare., (Copyright © 2023 Elsevier Inc. All rights reserved.)

Alcohol is a major cause of pre-mature death and individual suffering worldwide, and the importance of diagnosing and treating AUD cannot be overstated. Given the global burden and the high attributable factor of alcohol in a vast number of diseases, the need for additional interventions and the development of new medicines is considered a priority by the World Health Organization (WHO). As of today, AUD is severely under-treated with a treatment gap nearing 90%, strikingly higher than that for other psychiatric disorders. Patients often seek treatment late in the progress of the disease and even among those who seek treatment only a minority receive medication, mirroring the still-prevailing stigma of the disease, and a lack of access to effective treatments, as well as a reluctance to total abstinence. To increase adherence, treatment goals should focus not only on maintaining abstinence, but also on harm reduction and psychosocial functioning. A personalised approach to AUD treatment, with a holistic view, and tailored therapy has the potential to improve AUD treatment outcomes by targeting the heterogeneity in genetics and pathophysiology, as well as reason for, and reaction to drinking. Also, the psychiatric co-morbidity rates are high in AUD and dual diagnosis can worsen symptoms and influence treatment response and should be considered in the treatment strategies., (Copyright © 2024. Published by Elsevier Inc.)

Copyright of Interní Medicína pro Praxi is the property of SOLEN sro and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

Objective: To evaluate the impact of a 0.2% reduction in glycated hemoglobin (HbA1c) on treatment intensification, poor HbA1c control and HbA1c goal attainment in patients with type 2 diabetes mellitus (T2DM) initiated on a sodium glucose co-transporter 2 (SGLT2) inhibitor (SGLT2i).Methods: IQVIATM Health Plan Claims Data - US and IQVIATM Ambulatory EMR Data - US databases (29 October 2012-31 March 2016) were used to identify adults with T2DM initiated on an SGLT2i (index date) who had HbA1c measurements pre- and post-index, and ≥6 months of eligibility pre-index (baseline). HbA1c change was defined as the difference between the first post-index and the last pre-index measurements. Cox regression models were used to assess treatment intensification, poor HbA1c control (i.e. HbA1c > 9%, among patients <9% at baseline) and goal attainment (HbA1c < 7%, <8%; among patients with HbA1c above goal at baseline) adjusting for HbA1c change and baseline characteristics. Patients were observed up to one year after the first HbA1c measurement or end of eligibility. Hazard ratios (HRs) and 95% confidence intervals (CIs) were reported.Results: A total of 938 patients (mean age 54.9, 42.5% female, mean HbA1c 8.5%) were selected. Following SGLT2i initiation, each 0.2% reduction in HbA1c levels was associated with a decreased risk of treatment intensification (HR [95% CI] = 0.90 [0.86-0.92]), a decreased likelihood of reaching HbA1c > 9% (HR [95% CI] = 0.85 [0.79-0.88]) and higher likelihoods of achieving a treatment goal of HbA1c < 7% (HR [95% CI] = 1.17 [1.12-1.21]) and HbA1c < 8% (HR [95% CI] = 1.08 [1.04-1.10]).Conclusions: In T2DM patients, each HbA1c reduction of 0.2% following the initiation of an SGLT2i was associated with a significant positive impact on treatment intensification and HbA1c goal attainment. [ABSTRACT FROM AUTHOR]

Objective: This study explores the views of healthcare professionals regarding care and treatment goals in irreversible terminal delirium and their effect on patients and caregivers.Method: We conducted a qualitative interview study of healthcare professionals (palliative care physician, oncologist, psycho-oncologist, and clinical psychologist) engaged in the treatment of terminally ill cancer patients. We assessed the views of healthcare workers regarding treatment goals in terminal delirium and their effect on patients and their families.Result: Of the 21 eligible healthcare professionals, 20 agreed to participate in this study. Three of the professionals had experience with treating terminal delirium as family caregivers. We identified five important aspects of treatment goals in terminal delirium based on the views of healthcare professionals: (1) adequate management of symptoms/distress, (2) ability to communicate, (3) continuity of self, (4) provision of care and support to families, and (5) considering a balance (between symptom alleviation and maintaining communication; between symptom alleviation and family preparations for the death of patients; balance between specific treatment goals for delirium and general treatment goals).Significance Of Results: According to the views of healthcare workers questioned in this study, goals of care and treatment in terminal delirium are multidimensional and extend beyond simply controlling patient symptoms. [ABSTRACT FROM AUTHOR]

There is limited research on the perceptions of substance use (SU) and treatment goals among patients with substance use disorders (SUD) and attention deficit hyperactivity disorder (ADHD). We investigate whether SU perceptions and treatment goals were different among SUD patients with (SUD+ADHD) and without (SUD-ADHD) ADHD. Twelve SUD+ADHD patients (39.5 ± 8.5 years, 10 men), and 10 age- and substance severity matched SUD-ADHD patients (34.0 ± 10.0 years, six men), consecutively recruited between February 2010 and July 2012 were interviewed during the course of their SUD treatment. Interview data were analyzed using content analysis. The perceived positive aspects of SU were self-regulation of physical health, behavior, feelings, reasoning/thoughts, and the rewarding effects. The perceived negative aspects of SU included consequences on physical health, behavior, feelings, and reasoning/thoughts. SUD+ADHD patients less frequently linked SU to physical health and more frequently perceived SU as helpful to self-regulate their behavior. Four treatment goals categories emerged: total abstinence, conditional abstinence, substance reduction, and unspecified. SUD+ADHD patents less frequently chose total abstinence, and when using more than one substance, they commonly chose variable goals. In contrast, SUD-ADHD patients chose more similar goals. SUD+ADHD patients showed a more complex relation to their SUD than SUD-ADHD patients. [ABSTRACT FROM AUTHOR]

To assess the goals of gout treatment from a patient perspective, a convenience sample of consecutive patients with doctor-diagnosed gout seen at a community-based outpatient clinic were invited. Sex-stratified nominal groups were conducted until saturation was achieved. Responses were collected verbatim, discussed, and rank-ordered by each participant. Thirty-six patients with doctor-diagnosed gout participated in 12 nominal groups: 6 male only, 5 female only, and 1 group with both. Mean age was 61.9 years (SD, 12.3); mean gout duration was 13.3 years (SD, 12.5); 53% were men, 64% African-American, 42% retired, 47% currently married, 87% were using either allopurinol and/or febuxostat, and 40% had had no gout flares in the last 6 months. The top 5 treatment goals accounted for 91% of all votes and included the following: (1) prevent and better manage flare-ups and improve function (25%), (2) eliminate flare-ups/disease remission (30%), (3) diet and activity modification/lifestyle change (13%), (4) patient education and public awareness (12%), and (5) medication management and minimization of side effects (11%). When examining the top-rated concern for each nominal group, the first two goals were nominated by four groups each, diet/activity modification and medication management by 1 group each, and patient education by 3 groups. There were no differences evident by sex in top-ranked treatment goal. People with gout identified and rank-ordered treatment goals relevant to them. Providers of gout care need to be cognizant of these goals. Disease management concordant with these treatment goals might lead to a more satisfied, informed patient. [ABSTRACT FROM AUTHOR]

Objective: Accurate understanding of the extent of disease, treatment goal, and prognosis is a prerequisite for patients with cancer and their caregivers to make informed decision. We sought to evaluate patients' and family caregivers' understanding of the cancer stage, treatment goal, and chance of cure taking their own physician's evaluation as reference.Methods: A national survey was performed with 750 patient-caregiver dyads (75.5% participation rate) recruited by 134 oncologists in 13 cancer centers (93% participation rate) in South Korea. Both patients and caregivers were asked to report their knowledge of patient's cancer stage, treatment goal, and chance of cure. Concordance was evaluated with percentage agreement and weighted κ, and predictors of discordance of patient and caregiver's response with that of physician's were explored by multivariate regression analyses with mixed effect model.Results: The agreement rates between patient-physician and caregiver-physician were 63.0% and 65.9% for disease stage, 69.0% and 70.0% for treatment goal, and 41.4% and 45.1% for chance of cure. When discordance occurs, patients and caregivers often had an optimistic view. Distant stage, older age, female sex, and absence of depression were associated with optimistic view of patients, and there was significant between-physician variance for all 3 outcomes.Conclusion: The discordance revealed that our study suggests the need for better communication between physician and patients, as well as the caregivers. Research is needed to develop a comprehensive strategy to improve the understanding of the patients and family so that treatment decisions are made based on realistic estimation. [ABSTRACT FROM AUTHOR]

Social cognitive theory suggests when individuals select their own goals, they work harder to achieve them and experience increases in self-efficacy. Research in adults with alcohol use disorder supports the utility of treatment goal choice in predicting longitudinal outcomes; a total abstinence (TA) goal choice has been associated with better clinical outcomes versus a controlled use goal choice. Research on goal choice in adolescent substance users has not been reported. Data from 110 adolescents were collected upon admission to outpatient substance use disorder treatment. Hierarchical linear regressions tested baseline goal choice as a predictor of drinking outcomes at 6-, 12-, and 24-month follow-ups. Goal choice significantly predicted drinking outcomes at 12-month follow-up, but not at 6- or 24-month time points; TA was associated with better clinical outcomes. These findings suggest that goal choice may have clinical utility as a predictor of alcohol use disorder clinical course in adolescents. [ABSTRACT FROM PUBLISHER]

Multiple sclerosis (MS) is an autoimmune demyelinating disease of the central nervous system, and the most common cause of nontraumatic disability in young adults. Most patients have a relapsing-remitting course, and roughly half of them will eventually enter a degenerative progressive phase, marked by gradual accrual of disability over time in the absence of relapses. Early initiation of treatment has delayed the onset of disability progression. Thus, there is increased interest in treating to target in MS, particularly targeting no evidence of disease activity. This review will describe the most common treatment goals in MS: the Rio scores, disease-free survival, and no evidence of disease activity. We will also cover how well current disease-modifying therapies achieve no evidence of disease activity, and discuss future options for improving MS treatment targets. [ABSTRACT FROM AUTHOR]

Background: Pulmonary arterial hypertension (PAH) guidelines suggest that achieving a low-risk profile should be the treatment goal. Our aim was to assess a risk assessment strategy based on three non-invasive variables from the ESC/ERS 2015 guidelines in a Latin American cohort., Methods: 92 incident patients (mean [SD] age 47, 77% female, 53% idiopathic PAH) were included in this retrospective, multicenter study. Patients were stratified at baseline and at early follow-up, within the first year, using three non-invasive variables (WHO functional class, 6-minute walking distance, BNP/NT-proBNP) from the ESC/ERS 2015 risk assessment instrument. Median (IQR) follow-up was 3.11 years (3.01 years)., Results: At baseline assessment, 25% of patients were at low risk, 61.9% at intermediate-risk, and 13% at high-risk. At early follow-up (median 9.5 months), 56.5% of patients were at low-risk, 40.2% at intermediate-risk, and 3.2% at high-risk (p<0.001 vs. baseline). According to risk stratification at early follow-up, one, three and five-year overall survival was 100% in the low-risk group (no deaths at five-year follow-up), and 100%, 84% (95% CI: 72-98%), and 66% (95% CI: 48-90%) respectively in the intermediate-risk group, p = 0.0003. Mortality in the high-risk patients at early follow-up was 1/3 (33.3%). One, three, and five-year event-free survival (death or transplant or first hospitalization due to worsening PAH) based on early follow-up risk assessment was higher in the low-risk group, p = 0.0003., Conclusion: Our study validates a risk assessment strategy based on three non-invasive variables and confirms that early achievement of a low-risk profile should be the treatment goal., Competing Interests: Declaration of Competing Interest None., (Copyright © 2022 The Authors. Published by Elsevier Masson SAS.. All rights reserved.)

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Background Dyslipidemia is the primary risk factor for arthrosclerosis. It is the most common chronic disease among the multiethnic Asian population in Singapore. Local national health survey has shown ethnic variability in achieving control of dyslipidemia. Objectives This study aimed to determine the proportion of patients in primary care, who achieved their low-density lipoprotein (LDL)-cholesterol treatment goals, stratified by the local major ethnic groups. It also evaluated the factors that affected their dyslipidemia control, including diet, exercise and medication usage. Methods Research assistants administered questionnaires on adult patients with physician-diagnosed dyslipidemia to determine their views on diet, exercise, and medications in this cross-sectional study in 2 local primary care clinics. Their lipid profiles were retrieved from their laboratory reports in their electronic health records. Chi-square and Fisher exact tests were used for the categorical demographics and questionnaire variables, ( P < .05: statistically significant). Logistic regression was performed using these significant variables to determine the adjusted odds of the ethnic groups. Results A total of 1093 eligible patients completed the questionnaires. The proportion of Chinese, Malay, and Indian patients who achieved LDL-cholesterol goals was 78.3%, 67.9%, and 68.5%, respectively. Among those who self-reported taking their favorite cholesterol-rich food occasionally when their cholesterol became controlled, 35.8% Indians failed to achieve treatment goals, compared to 20.1% Chinese and 30.9% Malay patients. Regular medication adherence was associated with 81.8% Chinese, 69.0% Malay, and 69.7% Indian reaching treatment goals. Conclusions More Chinese met LDL-cholesterol treatment goals compared to Malays and Indians. Lipid-lowering medications enabled but smoking hindered their achievement of these treatment goals. [ABSTRACT FROM AUTHOR]