Endocrine complications in patients of beta thalassemia major in a tertiary care hospital in Pakistan

OBJECTIVE: To determine the major endocrine complications present in patients of Thalassemia major presenting to a tertiary care center in Karachi, Pakistan. METHODS: Records of all thallasemia major patients at a Haematology Department in a tertiary care hospital of Karachi were retrospectively reviewed from May to August 2009 with specific focus on endocrine data and investigations. The patients\u27 data was recorded in a questionnaire and analyzed using SPSS v.17, frequencies and percentages along with corresponding 95% confidence interval were computed. RESULTS: Our data revealed that a significantly small percentage of those under care received regular endocrine follow-up. Male hypo-gonadal abnormalities had the highest probability; 100% of the tested patients had decreased levels of testosterone, while 95.2% had raised serum creatinine levels. Parathyroid dysfunction was noted in 40% of the patients. Of those screened 29.4% had blood glucose levels in the diabetic range and 11.8% of the tested patients had reduced levels of FT4. CONCLUSION: A high frequency at endocrine dysfunctions seen in thalasaemia patients included: male hypogonadism, parathyroid dysfunction, deranged blood glucose and FT4 levies

Standardized observation of neighbourhood disorder: does it work in Canada?

<p>Abstract</p> <p>Background</p> <p>There is a growing body of evidence that where you live is important to your health. Despite numerous previous studies investigating the relationship between neighbourhood deprivation (and structure) and residents' health, the precise nature of this relationship remains unclear. Relatively few investigations have relied on direct observation of neighbourhoods, while those that have were developed primarily in US settings. Evaluation of the transferability of such tools to other contexts is an important first step before applying such instruments to the investigation of health and well-being. This study evaluated the performance of a systematic social observational (SSO) tool (adapted from previous studies of American and British neighbourhoods) in a Canadian urban context.</p> <p>Methods</p> <p>This was a mixed-methods study. Quantitative SSO ratings and qualitative descriptions of 176 block faces were obtained in six Toronto neighbourhoods (4 low-income, and 2 middle/high-income) by trained raters. Exploratory factor analysis was conducted with the quantitative SSO ratings. Content analysis consisted of independent coding of qualitative data by three members of the research team to yield common themes and categories.</p> <p>Results</p> <p>Factor analysis identified three factors (physical decay/disorder, social accessibility, recreational opportunities), but only 'physical decay/disorder' reflected previous findings in the literature. Qualitative results (based on raters' fieldwork experiences) revealed the tool's shortcomings in capturing important features of the neighbourhoods under study, and informed interpretation of the quantitative findings.</p> <p>Conclusions</p> <p>This study tested the performance of an SSO tool in a Canadian context, which is an important initial step before applying it to the study of health and disease. The tool demonstrated important shortcomings when applied to six diverse Toronto neighbourhoods. The study's analyses challenge previously held assumptions (e.g. social 'disorder') regarding neighbourhood social and built environments. For example, neighbourhood 'order' has traditionally been assumed to be synonymous with a certain degree of homogeneity, however the neighbourhoods under study were characterized by high degrees of heterogeneity and low levels of disorder. Heterogeneity was seen as an appealing feature of a block face. Employing qualitative techniques with SSO represents a unique contribution, enhancing both our understanding of the quantitative ratings obtained and of neighbourhood characteristics that are not currently captured by such instruments.</p

Development of a framework for effective community engagement in Ontario, Canada

Objectives This study aims to: (1) conceptualize effective community engagement (CE) within the Local Health Integration Networks (LHIN) of Ontario, Canada, (2) develop an effective CE framework and (3) compare the CE framework to public engagement (PE) frameworks and assess their relevance to the LHIN context.Methods We implemented "concept mapping," a participatory, mixed-methods approach, with LHIN staff. The method includes three components: (1) "Brainstorming" where participants generated statements describing effective CE; (2) "Sorting and Rating" where participants grouped statements and rated their relative importance to generate a "cluster map" and (3) "Mapping" where participants reviewed and named the clusters to yield the effective CE framework.Results Staff generated a list of 64 statements. Participants reviewed the cluster map to generate six categories for the framework: (1) collaboration, (2) accessibility, (3) accountability, (4) education, (5) principles and (6) organizational capacity.Conclusions The effective CE framework overlaps with PE frameworks with two caveats. First, applications of PE evaluation frameworks to the LHINs may yield incomplete results given that partnerships with service providers and organizations are integral to CE strategies. Second, "organizational capacity," largely not captured within existing frameworks, must be integrated as a feature given that emerging research identifies its importance to the successful implementation of engagement strategies.Evaluation Community engagement Frameworks Mixed methods research Concept mapping Health care authorities

Are residents of downtown Toronto influenced by their urban neighbourhoods? Using concept mapping to examine neighbourhood characteristics and their perceived impact on self-rated mental well-being

Abstract Background There is ample evidence that residential neighbourhoods can influence mental well-being (MWB), with most studies relying on census or similar data to characterize communities. Few studies have actively investigated local residents’ perceptions. Methods Concept mapping was conducted with residents from five Toronto neighbourhoods representing low income and non-low income socio-economic groups. These residents participated in small groups and attended two sessions per neighbourhood. The first session (brainstorming) generated neighbourhood characteristics that residents felt influenced their MWB. A few weeks later, participants returned to sort these neighbourhood characteristics and rate their relative importance in affecting residents’ ‘good’ and ‘poor’ MWB. The data from the sorting and rating groups were analyzed to generate conceptual maps of neighbourhood characteristics that influence MWB. Results While agreement existed on factors influencing poor MWB (regardless of neighbourhood, income, gender and age), perceptions related to factors affecting good MWB were more varied. For example, women were more likely to rank physical beauty of their neighbourhood and range of services available as more important to good MWB, while men were more likely to cite free access to computers/internet and neighbourhood reputation as important. Low-income residents emphasized aesthetic attributes and public transportation as important to good MWB, while non-low-income residents rated crime, negative neighbourhood environment and social concerns as more important contributors to good MWB. Conclusion These findings contribute to the emerging literature on neighbourhoods and MWB, and inform urban planning in a Canadian context.</p

Are residents of downtown Toronto influenced by their urban neighbourhoods? Using concept mapping to examine neighbourhood characteristics and their perceived impact on self-rated mental well-being

Abstract Background There is ample evidence that residential neighbourhoods can influence mental well-being (MWB), with most studies relying on census or similar data to characterize communities. Few studies have actively investigated local residents’ perceptions. Methods Concept mapping was conducted with residents from five Toronto neighbourhoods representing low income and non-low income socio-economic groups. These residents participated in small groups and attended two sessions per neighbourhood. The first session (brainstorming) generated neighbourhood characteristics that residents felt influenced their MWB. A few weeks later, participants returned to sort these neighbourhood characteristics and rate their relative importance in affecting residents’ ‘good’ and ‘poor’ MWB. The data from the sorting and rating groups were analyzed to generate conceptual maps of neighbourhood characteristics that influence MWB. Results While agreement existed on factors influencing poor MWB (regardless of neighbourhood, income, gender and age), perceptions related to factors affecting good MWB were more varied. For example, women were more likely to rank physical beauty of their neighbourhood and range of services available as more important to good MWB, while men were more likely to cite free access to computers/internet and neighbourhood reputation as important. Low-income residents emphasized aesthetic attributes and public transportation as important to good MWB, while non-low-income residents rated crime, negative neighbourhood environment and social concerns as more important contributors to good MWB. Conclusion These findings contribute to the emerging literature on neighbourhoods and MWB, and inform urban planning in a Canadian context

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

Introduction: Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier for the treatment of patients with spinal muscular atrophy (SMA). The JEWELFISH study (NCT03032172) was designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of risdiplam in previously treated pediatric and adult patients with types&nbsp;1–3 SMA. Here, an analysis was performed after all patients had received at least 1&nbsp;year of treatment with risdiplam. Methods: Patients with a confirmed diagnosis of 5q-autosomal recessive SMA between the ages of 6&nbsp;months and 60&nbsp;years were eligible for enrollment. Patients were previously enrolled in the MOONFISH study (NCT02240355) with splicing modifier RG7800 or treated with olesoxime, nusinersen, or onasemnogene abeparvovec. The primary objectives of the JEWELFISH study were to evaluate the safety and tolerability of risdiplam and investigate the PK after 2&nbsp;years of treatment. Results: A total of 174 patients enrolled: MOONFISH study (n = 13), olesoxime (n = 71 patients), nusinersen (n = 76), onasemnogene abeparvovec (n = 14). Most patients (78%) had three SMN2 copies. The median age and weight of patients at enrollment was 14.0&nbsp;years (1–60&nbsp;years) and 39.1&nbsp;kg (9.2–108.9&nbsp;kg), respectively. About 63% of patients aged 2–60&nbsp;years had a baseline total score of less than 10 on the Hammersmith Functional Motor Scale–Expanded and 83% had scoliosis. The most common adverse event (AE) was upper respiratory tract infection and pyrexia (30 patients each; 17%). Pneumonia (four patients; 2%) was the most frequently reported serious AE (SAE). The rates of AEs and SAEs per 100 patient-years were lower in the second 6-month period compared with the first. An increase in SMN protein was observed in blood after risdiplam treatment and was comparable across all ages and body weight quartiles. Conclusions: The safety and PD of risdiplam in patients who were previously treated were consistent with those of treatment-naïve patients