Objective: To examine reasons why Multiple Sclerosis (MS) patients may not be adherent to disease-modifying therapies (DMTs). Also, to determine patient perceptions of MS as a disease and DMTs as a source of treatment, and compare these between patients with prior DMT experience and those who were DMT-naïve. Finally, to assess the level of MS symptoms reported in patients taking DMTs, and compare these between DMT users and non-users. Methods: Patients with MS who were affiliated with a regional health plan at any point between January 2005 and December 2010 were asked to fill out a survey, the Multiple Sclerosis Medication Questionnaire (MSMQ). For study purposes, non-adherence was defined as missing any doses in the previous 28 days by the patient. In addition, the MSMQ examined reasons why MS patients do not initiate or discontinue DMT use. For statistical analyses, chi-square tests were performed to detect differences and t-tests and Mann-Whitney U tests to confirm the results. Results: A total of 197 surveys were returned, of which 105 (53.3%) patients were currently on a DMT. Thirty-two (30.5%) of the DMT users were considered non-adherent. Of the non-adherent respondents, the most frequent reason for non-adherence that was at least moderately important was being "too busy" (n=13/29, 44.8%). Amongst patients who were not using a DMT, the most common barrier to DMT use was related to possible side effects of treatment (n=46/79, 58.2%). Analyzing the statements regarding barriers to DMTs revealed significant differences in the proportion of agreement regarding physician's lack of advocacy for DMT use between DMT-naïve patients and those who discontinued DMT use (44.7% vs. 17.1%, respectively, p[less than]0.01), as well as for dislike for using needles (24.3% vs. 46.3%, respectively, p=0.043). In terms of MS symptoms, patients using a DMT generally reported the symptoms posed less of a problem, although significant differences were not seen in chi-square analyses. Conclusion: The injectable nature of most DMTs seems to cause varying degrees of discomfort in MS patients, which may contribute to non-adherence. Reasons given by MS patients for DMT non-adherence in the MSMQ mirror the literature regarding this topic. MS patients who are not currently on DMT may not seek or remain on treatment for various reasons. It appears certain perceptions regarding MS and DMTs are associated with potential DMT use. / text

No description available.

Rheumatoid arthritis (RA) is a chronic inflammatory disease affecting 1% of the population. The aetiology of rheumatoid arthritis is unknown, although there are multiple postulated theories. In 1950, Philip Hench won the Nobel prize for treating patients with rheumatoid arthritis with cortisone. He also treated 6 patients with adrenocorticotropic hormone (ACTH) with good results. ACTH is a melanocortin. The melanocortin system describes the five melanocortin receptors, their ligands, agonists and antagonists and the accessory proteins. The aim of this study was to explore the melanocortin receptors in rheumatoid arthritis synovium. Methods HA-tagged stable cell lines were created for MC1R, MC3R and MC5R. Multiple antibodies were tested for their utility using Western Blot, immunohistochemistry and flow cytometry. Samples of synovium from 28 patients with RA were tested using RTPCR for the presence of MC1R and MC3R. Gene expression was correlated with clinical characteristics, cytokine (RTPCR) expression and immunohistochemical score. Results The stable cell lines expressed MC1R, MC3R and MC5R respectively. Of the antibodies tested none were found to be of utility in detecting MC1R or MC3R .The MC1R RQ values in rheumatoid synovium appear to split into two groups, high and low. The medians of the two groups are significantly different (p=0.0005). There is almost a 5 cycle, or 64 fold, difference in gene expression between the medians of the two groups (1.59 v 6.23). Of note no MC3R positive samples were CD138 high (i.e. no MC3R positive samples had a significant plasma cell infiltrate) (p=0.006). Categorical analysis using Fishers Exact test revealed an association between MC1R high samples and CD68 lining high scores, (i.e. MC1R high samples also had a high macrophage score in the lining of the sample) (p=0.02). MC1R low samples were associated with not being on combination therapy, 15 this did not quite reach significance (p=0.07). Linear regression analysis confirmed these associations for MC1R. PCA analysis did not show any grouping of samples according to any of the variables tested, likely due to sample size. Conclusion MC1R and MC3R are found in human synovium. Current commercial antibodies are not of utility in detecting MC1R or MC3R. Synovial samples can be split into high and low MC1R gene expression groups. MC3R was either present or absent. High expression of MC1R was associated with a high macrophage score and MC3R expression was associated with a low plasma cell score. MC1R and MC3R expression in RA synovium could be used as biomarkers of disease state or severity as well as a target for therapy.

PURPOSE: Sickle cell disease (SCD) is an inherited blood disorder that affects the hemoglobin protein of red blood cells and has a significant impact on morbidity, mortality, and quality of life. Hydroxyurea has been FDA approved since 1998 as a disease-modifying therapy for SCD. However, hydroxyurea has not been optimally utilized for those with SCD. The purpose of this study was to evaluate reasons for hydroxyurea use, from the perspectives of providers, adults with SCD, and parents/caregivers of children with SCD, as well as perceived barriers to its use. We examined indications and reasons for being “on hydroxyurea,” defined by patients as currently taking hydroxyurea, and reported on pain frequency, perceptions of barriers, hydroxyurea adherence, and health care access for patients with SCD who were either on and not on hydroxyurea. METHODS: We conducted a cross sectional analysis of data collected within the Pacific Sickle Cell Regional Collaborative (PSCRC), a consortium of nine western U.S. states. Individuals were eligible for this study if they 1) had a confirmed diagnosis of SCD, 2) were followed at one of the PSCRC sites, and 3) were eligible for hydroxyurea therapy. Parents/caregivers of children with SCD less than 18 years and adults with SCD 18 years and older completed a brief survey about hydroxyurea use, indications, side effects, pain frequency, number of hospital and emergency department (ED) admissions per year, and individual and family perceptions of barriers to hydroxyurea use. Participants completed a follow-up survey annually, but we reported only on baseline data. Data collection occurred between February 2016 and May 2018. RESULTS: Individuals with SCD (n = 413) included 1) children (n=178; 6.7 ± 3.4 years), 2) adolescents (n=66; 15.0 ± 1.4 years), 3) young adults (n=57; 21.4 ± 2.6 years), and 4) adults (n=112; 39.2 ± 10.6 years). The majority were predominantly female (51.6%), African American (93.2%), and had HgbSS (74.1%) genotype. The majority of children (65.2%), adolescents (62.1%), and young adults (54.4%) were on hydroxyurea; fewer adults (39.3%) were on hydroxyurea. The majority with HgbSS (65.5%) were adherent to hydroxyurea. There was no significant difference in hospitalizations for pain, ED visits, and pain severity in the previous 12 months between individuals who were and were not on hydroxyurea, and between individuals who were and were not adherent to hydroxyurea. For those with a current prescription for hydroxyurea, the majority (66.5%) were receiving hydroxyurea for recurrent pain episodes or acute chest syndrome (19.9%). Hydroxyurea was discontinued because of patient/family preference (34.5%), chronic transfusions (31.1%), and side effects (24.1%). Patients prescribed hydroxyurea for empiric use (n=21) had fewer hospitalizations for pain, ED visits, and severe pain interfering with daily activities. The major barriers to hydroxyurea use, from the perspective of individuals with SCD or their caregivers, were 1) forgetting to take the medicine (19.4%), 2) worried about side effects (16.4%), and 3) lack of knowledge about hydroxyurea (13.6%). Fewer young adults (49.1%) and adults (50.0%) had primary care providers than children (78.1%) and adolescents (65.2%). CONCLUSIONS: Barriers to hydroxyurea use persist with emerging solutions to alleviate these barriers. For this sample, while hydroxyurea prescription rates by sickle cell specialists were similar to what has been seen in some other studies, neither hydroxyurea use nor adherence were associated with decreased frequency of hospitalizations for pain, ED visits, and severe acute pain episodes in the previous 12 months. Future studies need to evaluate hydroxyurea prescription patterns, duration on hydroxyurea, and adherence to hydroxyurea. Healthcare providers are recommended to prescribe hydroxyurea for eligible individuals who may benefit from it, such as those HgbSS or HgbS-β0 thalassemia genotype, and prescribe for empiric use to minimize complications. Provider and patient education about hydroxyurea could reduce common barriers experienced by individuals with SCD. It is important to customize educational resources to specific concerns for different age groups. Individuals 18 years and older with SCD have been documented with more ED visits and hospitalizations due to pain, most likely because they did not have a primary care provider and an adult hematologist with expertise in SCD. Future studies need to evaluate whether primary care providers who receive SCD education may promote hydroxyurea use and adherence. Dedicating time and resources for shared decision making between providers and patients/families can address concerns about hydroxyurea and increase patient/family confidence when deciding about hydroxyurea. As more disease-modifying therapies become available for individuals with SCD, strategies for shared decision making facilitate standardization and optimize the use of hydroxyurea and emerging therapies.

No description available.