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BACKGROUND: This study aimed to examine the early experience of nusinersen for spinal muscular atrophy (SMA) from the patient and caregiver perspective. METHODS: A 54-item online survey was administered to adult patients and caregivers of pediatric patients diagnosed with SMA. RESULTS: Overall, respondents (56 patients and 45 caregivers) were satisfied with nusinersen. Satisfaction was highest on changes in energy, stamina, and motor function and lowest on treatment administration and overall time commitment. Differences were noted for treatment effect sustained over time as reported by adult patients vs caregivers reporting on behalf of pediatric patients. Respondents reported insurance approval as a key barrier to access, particularly among adult patients. CONCLUSIONS: Despite therapeutic advances, there remain significant unmet needs for SMA. Challenges with administration and barriers to access potentially limit the number of patients treated or delay treatment. Continued efforts are needed to develop more treatment options and to improve access to treatments.
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Cuidadores , Atrofia Muscular Espinal/tratamento farmacológico , Oligonucleotídeos/uso terapêutico , Satisfação do Paciente , Atividades Cotidianas , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Gastos em Saúde , Humanos , Lactente , Injeções Espinhais , Cobertura do Seguro , Seguro Saúde , Masculino , Pessoa de Meia-Idade , Atrofia Muscular Espinal/fisiopatologia , Oligonucleotídeos/economia , Inquéritos e Questionários , Fatores de Tempo , Resultado do Tratamento , Adulto JovemRESUMO
Purpose: This study was intended to characterize the impact of meibomian gland dysfunction (MGD) on patients' quality of life. Methods: In this prospective, multicenter, noninterventional clinical study (NCT01979887), eligible individuals (age ≥40 years; absence of uncontrolled ocular/systemic disease) were categorized, based on composite grading of ocular symptoms, Schirmer score, and meibum quality, into (1) non-MGD, (2) mild/moderate MGD, or (3) severe MGD cohorts. The MGD Impact Questionnaire (MGD IQ), a 10-item patient-reported outcome measure, was self-administered at clinic visit on day 1, and readministered on day 22 to assess intervisit agreement regarding MGD IQ responses. Results: In total, 75 subjects were assigned to the study cohorts (25 per cohort). Across cohorts, MGD IQ item scores rose incrementally with increasing MGD severity. The severe MGD cohort experienced greater difficulty with reading and performance of leisure activities, greater time on eye care, and greater bother with eye care and eye appearance than the mild/moderate MGD cohort (all P < 0.05). Compared with the non-MGD cohort, the mild/moderate MGD cohort had greater difficulty working on computer, whereas the severe MGD cohort had greater difficulty reading, driving, and performing leisure activities, more frequent difficulty with outdoor activities, more time on eye care, and greater bother with eye care (all P < 0.05). Intervisit agreement between MGD IQ responses was fair to moderate (weighted kappa statistic 0.33â0.58). Conclusions: Vision-related activities are negatively impacted by increasing severity of MGD. The MGD IQ instrument can help characterize disease severity and amplify the patient's voice in patient-centric clinical research. ClinicalTrials.gov NCT01979887.
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Síndromes do Olho Seco , Disfunção da Glândula Tarsal , Adulto , Humanos , Síndromes do Olho Seco/diagnóstico , Disfunção da Glândula Tarsal/diagnóstico , Disfunção da Glândula Tarsal/terapia , Glândulas Tarsais , Estudos Prospectivos , Qualidade de Vida , LágrimasRESUMO
OBJECTIVE: To evaluate the psychometric properties of the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) and Visual Function Questionnaire Utility Index (VFQ-UI) in patients with non-infectious intermediate and posterior uveitis. METHODS: Secondary analysis of pooled data from a 26-week, multicenter, masked, randomized, sham-controlled Phase 3 clinical trial. Health-related quality of life was assessed using the NEI VFQ-25, the EQ-5D, and SF-36. Internal consistency reliability, reproducibility, convergent validity, and known groups of BCVA and vitreous haze severity were assessed. Clinically significant difference was assessed using anchor-based and distribution-based methods. RESULTS: The study included 224 subjects with non-infectious intermediate (80.4 %) or posterior uveitis (19.6 %). The NEI VFQ-25 and the VFQ-UI demonstrated good internal consistency (Cronbach's alpha 0.87-0.94) and test-retest reliability (ICCs 0.58-0.88). Spearman's product-moment rank correlations between the NEI VFQ-25 and VFQ-UI scores and the SF-6D, EQ-5D, and BCVA ranged from small to moderate. There was a significant association between visual functioning and known groups of visual acuity (p < 0.05). Clinical significance, using the anchor-based method (difference between visual acuity groups ≥10-<15 letter better and no change), was 10.2 for change from baseline to week 26 for the NEI VFQ-25 composite score and 0.05 for the VFQ-UI. Using the distribution-based method, the clinical significance was 3.86 for the composite score and 0.04 for the VFQ-UI. CONCLUSION: The NEI VFQ-25 and the VFQ-UI are reliable and valid measures of vision-related functioning and preference-based status in patients with non-infectious intermediate and posterior uveitis.
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Psicometria/métodos , Qualidade de Vida , Inquéritos e Questionários/normas , Uveíte/fisiopatologia , Adolescente , Feminino , Nível de Saúde , Inquéritos Epidemiológicos , Humanos , Masculino , Pessoa de Meia-Idade , National Eye Institute (U.S.) , Avaliação de Resultados da Assistência ao Paciente , Reprodutibilidade dos Testes , Perfil de Impacto da Doença , Estados Unidos , Acuidade VisualRESUMO
This multicenter, retrospective study compared clinical outcomes and healthcare resource use in patients who underwent dual-plane (DP) or prepectoral (PP) implant-based breast reconstruction (IBR) after mastectomy in the United States. Methods: Medical records were selected for patients at five sites undergoing immediate one-stage direct-to-implant (first hospitalization) or two-stage IBR (first and second hospitalization) using either DP or PP. Inverse probability of treatment weighting was used to adjust for potential confounders. Complications and healthcare resource use were assessed with logistic regression; pain severity was assessed with ordinary least-squares regression. Results: After inverse probability of treatment weighting, data from 255 patients (DP = 130, PP = 125) and 441 breasts (DP = 226, PP = 215) were analyzed. Mean pain severity scores were lower with PP versus DP immediately after IBR for first (P = 0.0002) and second hospitalizations (P = 0.0145), and before discharge for first (P < 0.0001) and second hospitalizations (P = 0.0002). A greater proportion of PP versus DP patients had a shorter hospital length of stay (≤ 23 hours) for first hospitalization (P = 0.0052); proportions were similar for second hospitalization (P = 0.5499). Intravenous narcotics were prescribed less frequently to PP versus DP patients during first (61.1% versus 69.8%, respectively; P = 0.1486) and second (37.5% versus 55.3%, respectively; P = 0.0172) hospitalizations. Complication rates were low in both groups after first hospitalization discharge (DP: 13.6%, PP: 12.5%, P = 0.7225). Conclusion: This retrospective study suggests that the PP technique in IBR may offer benefits related to clinical outcomes and health resource utilization; however, larger studies, including randomized controlled trials, are needed to confirm.
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BACKGROUND: Few studies have described improvement in health-related quality of life (HRQOL) associated with opioid dependence treatment with buprenorphine (ODT-B). OBJECTIVE: To evaluate HRQOL changes in domain scores, physical and mental component summaries, and health utilities (HUs) associated with ODT-B using the Short Form 36 (SF-36). METHODS: We assessed HRQOL changes in a substudy of a pharmacokinetic study that compared buprenorphine oral tablet and liquid dosage formulations over 16 weeks. Individuals, aged 18-65 years, were screened for opioid dependence. They were excluded if they would not agree to birth control or had a serious medical condition. Subjects received psychosocial counseling and weekly group therapy. The SF-36 was administered upon enrollment and at 4-week intervals. We used the SF-6D to estimate HUs. We performed intention to treat (ITT) analyses based on the last observation available for each subject. Paired t tests of each domain and HU, limited to remaining patients at each 4-week interval, were also conducted. RESULTS: Of 96 subjects enrolled, cumulative dropouts over time resulted in 80, 69, 59, and 44 subjects remaining at 4, 8, 12, and 16 weeks. There were no significant differences in opioid-positive urines, dropout rates, or dosage changes between formulations. In the ITT analyses, HRQOL improvements over time were bodily pain (62.1 vs. 69.1, P = 0.017), vitality (49.8 vs. 56.5, P = 0.001), mental health (59.9 vs. 66.0, P = 0.001), social function (66.4 vs. 74.7, P = 0.001), role emotional (59.4 vs. 71.9, P = 0.003), role physical (60.9 vs. 70.6, P = 0.005), and mental component summary (41.9 vs. 45.4, P<0.001). HU scores also improved (0.674 vs. 0.715, P = 0.001). Results from paired t tests, with only concurrently enrolled patients, showed similar improvements from baseline to 4, 8, 12, or 16 weeks. CONCLUSION: Buprenorphine, accompanied with psychosocial counseling, was associated with improved HRQOL and HUs.
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Buprenorfina/uso terapêutico , Nível de Saúde , Antagonistas de Entorpecentes/uso terapêutico , Transtornos Relacionados ao Uso de Opioides/tratamento farmacológico , Transtornos Relacionados ao Uso de Opioides/fisiopatologia , Qualidade de Vida , Adolescente , Adulto , Emoções , Feminino , Seguimentos , Humanos , Relações Interpessoais , Masculino , Saúde Mental , Pessoa de Meia-Idade , Ensaios Clínicos Controlados Aleatórios como Assunto , Resultado do Tratamento , Adulto JovemRESUMO
PURPOSE: To characterize newly diagnosed primary open-angle glaucoma (OAG) patients and to describe their treatment journey in United States clinical practice according to the use of topical therapy, laser trabeculoplasty, and surgical procedures. DESIGN: Retrospective claims database study. PARTICIPANTS: Patients with at least 2 diagnoses of OAG 7 days or more apart and within 1 year, with the first (index) diagnosis in 2010, at least 30 months of continuous enrollment before index diagnosis with no OAG diagnosis or medication (exception for ocular hypertension diagnosis), and 48 months of continuous enrollment. METHODS: Analysis of United States healthcare insurance claims database (July 2007-December 2014). MAIN OUTCOME MEASURES: Treatment patterns and ophthalmology visits were evaluated over 48 months in 4 cohorts based on initial therapy after the index diagnosis: (1) drug monotherapy, (2) combination drug therapy, (3) glaucoma procedure, or (4) no claims for treatment. Treatment modification was defined as an addition to or change in drug therapy or procedure. RESULTS: In total, 83.0% of patients (5120/6172) began a drug therapy (69.5%) or underwent a procedure initially (13.5%); topical prostaglandin analogs (n = 2887/5120 [56.4%]) and laser trabeculoplasty (n = 705/5120 [13.8%]) were the most common. During the 4-year follow-up, 58.3% of patients (2109/3620) who began drug monotherapy experienced no further treatment modification. Over this period, 43.8% of patients who began treatment (2242/5120) experienced a treatment modification to the first treatment. Two thirds (1505/2242 [67.1%]) of these patients subsequently underwent a third treatment modification. Ophthalmology visits declined over time regardless of initial therapy, with the greatest decrease among the untreated and first-treatment procedure cohorts. CONCLUSIONS: The high rates of 2 or 3 treatment modifications over the 4-year period suggest an unmet need for glaucoma therapies with durable and predictable actions.
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Glaucoma de Ângulo Aberto , Hipertensão Ocular , Trabeculectomia , Glaucoma de Ângulo Aberto/diagnóstico , Humanos , Pressão Intraocular , Hipertensão Ocular/cirurgia , Estudos Retrospectivos , Estados Unidos/epidemiologiaRESUMO
Microchimerism is the presence of cells from one individual in another genetically distinct individual. Pregnancy is the main cause of natural microchimerism through transplacental bi-directional cell trafficking between mother and fetus. In addition to a variety of cell-free substances, it is now well-recognized that some cells are also exchanged in pregnancy. Furthermore, it is now known that microchimerism persists decades later both in mother and in her progeny. The consequences of pregnancy-related microchimerism are under active investigation. However, many authors have suggested a close relationship linking fetal microchimerism and the development of autoimmune diseases. Fetal microchimerism is emerging as a potential contributing factor in certain diseases, including cancer. Parallel studies in animal and human pregnancy suggest that microchimeric fetal cells play a role in wound healing. Role of these microchimeric cells in human health and disease is discussed here.
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INTRODUCTION: Micronuclei (MNs) are extranuclear cytoplasmic DNA bodies which are induced in cells by numerous genotoxic agents that damage chromosome. The MN assay in exfoliated buccal cells is a useful and minimally invasive method for monitoring genetic damage. AIM: The aim of present study was to detect and assess MNs in oral exfoliated cells in patients diagnosed with leukoplakia with dysplasia, oral submucous fibrosis (OSMF) and oral squamous cell carcinoma (OSCC) using special stains and to determine the most appropriate staining technique for the evaluation of MNs along with a comparative evaluation of MNs with histological grading. MATERIALS AND METHODS: The study was conducted in the Department of Oral Pathology and Microbiology, CDCRI, Rajnandgaon, and a total of 45 subjects were included in the study who were subsequently divided into three groups (15 each). Four smears were obtained from each subject which were taken from the lesional tissue and stained simultaneously. ANALYSIS: The results were analyzed via Statistical Package for the Social Sciences, version 23.0 (SPSS). RESULTS: The results confirmed the association of MNs with genotoxic agents and showed an elevated number in OSCC followed by OSMF and leukoplakia. The frequency also increased with the severity of the lesion. Besides this, Papanicolaou (PAP) stain was found to be the most suitable stain for detection of MNs. CONCLUSION: Based on the above pretext, we can conclude that PAP stain was the most suitable stain for valuation of MNs and that the MN assay holds promise as a specific biomarker of genotoxicity, for screening of oral cancer and can be used as a prognostic indicator.
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BACKGROUND: Eosin is most widely used synthetic dye belonging to the xanthene group. These dyes are efficient but are hazardous to human and animal health. With the increasing awareness of a green earth, it is advisable to use more of eco-friendly and biodegradable material which can be effectively achieved by the use of natural dyes obtained from plants and other natural sources. Turmeric, available as Curcuma longa (domestic), has long been in use in the subcontinent as a spice and flavoring agent in most food preparations. Its health benefit as a natural antibiotic and anti-inflammatory has been successfully established by several researchers. The intense yellow color imparted by turmeric inspired us to explore its efficacy as a potential alternative for eosin in routine histopathological procedures. AIM: The aim of this was to explore the efficacy of turmeric extract as a stand-alone counterstain for hematoxylin and its comparative assessment with routine H and E staining. MATERIALS AND METHODS: The rhizomes of C. longa were cut into small pieces, dried and milled. This powder was dissolved into alcohol and centrifuged using a centrifugal machine. The supernatant was then collected with the help of micropipette. This supernatant was used as a counterstain for hematoxylin. RESULTS: The data were analyzed using Mann-Whitney U test with Statistical Package for the Social Sciences version 15.0 (SPSS Inc.,). The P value obtained was statistically insignificant (P > 0.05). CONCLUSION: Although eosin is the most efficient counterstain for hematoxylin, turmeric can also be used as an alternative for eosin.
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OBJECTIVE: To compare vision-related functioning and health-related quality of life of patients with noninfectious intermediate or posterior uveitis with those of the US general population and normal-vision reference groups. METHODS: Secondary analysis of health-related quality of life measures administered at baseline to patients with noninfectious intermediate or posterior uveitis participating in the HURON trial, a 26-week, multicenter, masked, randomized, sham-controlled trial of a dexamethasone intravitreal implant (n=224) was performed. Patient-reported outcome measures included the National Eye Institute Visual Function Questionnaire-25, the 36-Item Short-Form Health Survey, the Short Form-6 Dimensions, and the EuroQol-5D. The National Eye Institute Visual Function Questionnaire-25 scores from the HURON uveitis population were compared with published National Eye Institute Visual Function Questionnaire-25 scores from a normal-vision reference group (n=122). The 36-Item Short-Form Health Survey, Short Form-6 Dimensions, and EuroQol-5D scores were compared with the US general population using data from the National Health Measurement Study (n=3844) and the Medical Expenditure Panel Survey (n=955). RESULTS: Compared with a normal-vision population, the HURON uveitis population had clinically significant impairments across all National Eye Institute Visual Function Questionnaire-25 subscales and the composite score, with all subscale score differences exceeding 10 points (P< .001). The HURON uveitis population had significantly lower 36-Item Short-Form Health Survey mental component summary and Short Form-6 Dimensions scores compared with a US general population sample (P< .001). No significant differences were found for the 36-Item Short-Form Health Survey physical component summary and EuroQol-5D scores between the uveitis and US general population samples. CONCLUSIONS: Compared with the US general population and normal-vision reference groups, noninfectious intermediate or posterior uveitis results in meaningful reductions in mental health outcomes, health-related quality of life, and vision-related functioning. TRIAL REGISTRATION: clinicaltrials.gov Identifier:NCT00333814.
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Dexametasona/administração & dosagem , Glucocorticoides/administração & dosagem , Avaliação de Resultados em Cuidados de Saúde , Qualidade de Vida , Perfil de Impacto da Doença , Uveíte Intermediária/tratamento farmacológico , Uveíte Posterior/tratamento farmacológico , Adulto , Método Duplo-Cego , Implantes de Medicamento , Feminino , Nível de Saúde , Humanos , Masculino , Pessoa de Meia-Idade , Satisfação do Paciente , Inquéritos e Questionários , Resultado do Tratamento , Uveíte Intermediária/fisiopatologia , Uveíte Posterior/fisiopatologia , Acuidade Visual/fisiologiaRESUMO
PURPOSE: To evaluate the effect of a single treatment with dexamethasone intravitreal implant (DEX implant) on patient-reported visual functioning in patients with noninfectious intermediate or posterior uveitis. METHODS: Patient eyes with noninfectious intermediate or posterior uveitis were randomized to a single treatment with DEX implant 0.70 mg (n=77), DEX implant 0.35 mg (n=76), or a sham procedure (n=76) and followed for 26 weeks. Vision-related functioning was measured using the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) assessed at baseline and at weeks 8, 16, and 26 or early exit. Analysis of covariance and mixed model analysis of covariance were used to compare vision-related functioning between the DEX implant 0.70 and 0.35 mg groups and the sham group. RESULTS: By 8 weeks, the DEX implant 0.70 mg group demonstrated significant improvements in NEI VFQ-25 subscales near vision (P=0.031), distance vision (P=0.023), peripheral vision (P=0.045), vision-specific social functioning (P=0.019), and the NEI VFQ-25 composite score (P=0.007) compared with sham. After 26 weeks, the DEX implant 0.70 mg group reported significant improvements in NEI VFQ-25 subscales distance vision (P=0.003), vision-specific role difficulties (P=0.038), vision-specific dependency (P=0.017), vision-specific social functioning (P=0.009), vision-specific mental health (P=0.036), and the composite score (P=0.001) compared with sham. CONCLUSIONS: In patients with noninfectious intermediate or posterior uveitis receiving a single treatment of DEX implant 0.70 mg, significant and clinically meaningful improvements in patient-reported visual functioning were observed as early as week 8 and were maintained over 26 weeks. (ClinicalTrials.gov number, NCT00333814.).
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Anti-Inflamatórios/administração & dosagem , Dexametasona/administração & dosagem , Uveíte Posterior/tratamento farmacológico , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Análise de Variância , Implantes de Medicamento , Feminino , Humanos , Injeções Intravítreas , Masculino , Pessoa de Meia-Idade , Inquéritos e Questionários , Uveíte Posterior/fisiopatologia , Acuidade Visual/fisiologia , Adulto JovemRESUMO
OBJECTIVES: To assess pharmacy students' knowledge, attitudes, and evaluation of direct-to-consumer advertising (DTCA). METHODS: A cross sectional, self-administered, 106-item survey instrument was used to assess first, second, and third professional year pharmacy students' knowledge about DTCA regulations, attitudes toward DTCA, and evaluation of DTC advertisements with different brief summary formats (professional labeling and patient labeling) and in different media sources (print and television). RESULTS: One hundred twenty (51.3%) of the 234 students enrolled participated in the study. The mean percentage knowledge score was 48.7% +/- 12.5%. Based on the mean scores per item, pharmacy students had an overall negative attitude toward DTC advertisements. Students had an overall negative attitude toward television and print advertisements using the professional labeling format but an overall positive attitude toward the print advertisement using the patient labeling format. CONCLUSIONS: Lectures discussing DTC advertising should be included in the pharmacy curriculum.
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Publicidade/métodos , Informação de Saúde ao Consumidor/métodos , Conhecimentos, Atitudes e Prática em Saúde , Estudantes de Farmácia , Publicidade/economia , Informação de Saúde ao Consumidor/economia , Estudos Transversais , Indústria Farmacêutica/economia , Indústria Farmacêutica/métodos , Feminino , Humanos , MasculinoRESUMO
OBJECTIVE: To describe New Mexico pharmacists' knowledge, attitudes, and beliefs toward the prescribing of oral emergency contraception (EC) in their practices. DESIGN: Cross-sectional study. SETTING: New Mexico in January through March 2004. POTENTIAL PARTICIPANTS: New Mexico pharmacists. INTERVENTIONS: Questionnaire containing 74 items. MAIN OUTCOME MEASURE(S): Mean scores were calculated for individual knowledge items, overall knowledge scores, and attitude/belief items. Knowledge and attitude/belief scores were compared across demographic variables using t tests, ANOVA, and chi-square analyses. RESULTS: Of 1392 deliverable questionnaires, 555 (40%) were returned and 523 (38%) were usable; 136 contained written comments. Pharmacists had overall knowledge scores of 71.2% +/- 11.3%. Pharmacists who had participated in a state-approved EC prescribing training program and had time in their practice setting to prescribe EC had significantly higher knowledge scores. Mean scores indicated that pharmacists have positive attitudes and beliefs toward prescribing EC. Overall, 40% of respondents indicated that they would like to become certified to prescribe EC. Pharmacists who agreed that they would like to be certified to prescribe EC were significantly more likely to be male, non-Hispanic, non-Christian, to have liberal or moderate political views, and to indicate that they had employer/manager approval, time, and privacy in their practice setting to prescribe EC. CONCLUSION: New Mexico pharmacists have positive attitudes/beliefs toward EC prescribing; however, their knowledge in this area is average. Although religious, moral, and political views influence pharmacists' willingness to prescribe EC, factors such as education and practice environment must be addressed if more pharmacists are to accept this EC prescriptive authority.