Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation.

BackgroundMucopolysaccharidosis I (MPS IH) is a lysosomal storage disease treated with hematopoietic cell transplantation (HCT) because it stabilizes cognitive deterioration, but is insufficient to alleviate all somatic manifestations. Intravenous laronidase improves somatic burden in attenuated MPS I. It is unknown whether laronidase can improve somatic disease following HCT in MPS IH. The objective of this study was to evaluate the effects of laronidase on somatic outcomes of patients with MPS IH previously treated with HCT.MethodsThis 2-year open-label pilot study of laronidase included ten patients (age 5-13 years) who were at least 2 years post-HCT and donor engrafted. Outcomes were assessed semi-annually and compared to historic controls.ResultsThe two youngest participants had a statistically significant improvement in growth compared to controls. Development of persistent high-titer anti-drug antibodies (ADA) was associated with poorer 6-min walk test (6MWT) performance; when patients with high ADA titers were excluded, there was a significant improvement in the 6MWT in the remaining seven patients.ConclusionsLaronidase seemed to improve growth in participants <8 years old, and 6MWT performance in participants without ADA. Given the small number of patients treated in this pilot study, additional study is needed before definitive conclusions can be made

Perceived barriers and facilitators to physical activity and functional limitations among older adults

Lower physical function, mobility disability and the possibility of subsequent physical disabilities are major public health issues due to the rapid and continuing growth of the older adult population. In addition, the risk for mobility disability and physical disability increases with age. Longer life expectancy, rapid population growth, and low physical activity participation rates among older adults justify the need for better understanding of perceived barriers to and facilitators of physical activity. However, perceived barriers and facilitators, modifiable intra- and extra-individual mechanisms in the disablement pathway, remain underexplored. This dissertation aimed to provide novel insight into the associations between perceived barriers and facilitators, physical activity and related programs, and functional limitations among older adults. First, there are a range of factors that contribute to engagement in lifestyle programs aimed at mobility disability prevention for sedentary older adults, and these factors may be related to the type of program. Next, barriers may attenuate the effect of a structured physical activity program aimed at reducing major mobility disability. Finally, evidence was provided supporting the importance of considering social support as a specific barrier/facilitator in interventions aimed at improving physical function. Low levels of social support over time may increase the risk for declining physical function and subsequent disability development compared with those reporting moderate or high levels of social support. This research provides novel evidence that perceived barriers and facilitators are important to consider as they play key roles in the ongoing disablement pathway toward physical disability in older adults. This could inform the development and translation of future intervention efforts aimed at mobility disability reduction and physical disability prevention. Dissemination of feasible, sustainable and low cost programs for older adults is an important public health issue currently gaining more attention. This research took steps toward understanding the complexities of the disablement pathway and where we might be able to intervene to reduce the negative impact of these outcomes on the individual, health care system, and society. Ultimately, generating information to assist public health and health care professionals in addressing functional decline and disability

Effectiveness of mobile-phone short message service (SMS) reminders for ophthalmology outpatient appointments: Observational study

Abstract Background Non-attendance for hospital outpatient appointments is a significant problem in many countries. It causes suboptimal use of clinical and administrative staff and financial losses, as well as longer waiting times. The use of Short Message Service (SMS) appointment reminders potentially offers a cost-effective and time-efficient strategy to decrease non-attendance and so improve the efficiency of outpatient healthcare delivery. Methods An SMS text message was sent to patients with scheduled appointments between April and September 2006 in a hospital ophthalmology department in London, reminding them of their appointments. This group acted as the intervention group. Controls were patients with scheduled ophthalmology appointments who did not receive an SMS or any alternative reminder. Results During the period of the study, 11.2% (50/447) of patients who received an SMS appointment reminder were non-attenders, compared to 18.1% (1720/9512) who did not receive an SMS reminder. Non-attendance rates were 38% lower in patients who received an SMS reminder than in patients who did not receive a reminder (RR of non-attendance = 0.62; 95% CI = 0.48 – 0.80). Conclusion The use of SMS reminders for ophthalmology outpatient appointments was associated with a reduction of 38% in the likelihood of patients not attending their appointments, compared to no appointment reminder. The use of SMS reminders may also be more cost-effective than traditional appointment reminders and require less labour. These findings should be confirmed with a more rigorous study design before a wider roll-out.</p

Food Waste to Bio-Products

The goal of this project was to design and evaluate a project for the collection and processing of food waste and spent oil in Philadelphia. The project was designed to handle 5% of the total commercial waste generated in Philadelphia. This amounted to approximately 9,700 tons/year of food waste and 73,000 gallons/year of spent oil. The process was designed to utilize a BIOFerm™ Dry Fermentation Digestion System. Following the digestion, the biogas produced is passed through a Caterpillar CG132-12 Generator Set, producing electricity to be sold back to the local grid. The digestate from the anaerobic digestion is used to produce compost, providing an additional revenue stream. In addition to handling the solid food waste, the project is designed to convert the collected spent oil into biodiesel using prepackaged processing units by Springboard Biodiesel. The facility is anticipated to annually produce 2,541 tons of biogas, 5,184,000 kWh of electricity, 14,756 tons of compost, and 59,616 gallons of biodiesel. A rigorous profitability analysis was conducted in order to project cash flows for fifteen years. The total capital investment of the plant is $5.6MM and the expected NPV of the project is -($682,000). The estimated IRR of the project is 12% and the 3-year ROI is 7%. Given the project’s negative NPV, our recommendation is to adopt such a process solely for environmentally beneficial waste management purposes. A key takeway is that in order for such a project to be profitable it would need to target more than just 5% of the total commercial food waste produced

Diaphragm position can be accurately estimated from the scattering of a parallel transmit RF coil at 7 T.

PurposeTo evaluate the use of radiofrequency scattering of a parallel transmit coil to track diaphragm motion. MethodsMeasurements made during radiofrequency excitation on an 8-channel parallel transmit coil by the directional couplers of the radiofrequency safety monitor were combined and converted into diaphragm position. A 30-s subject-specific calibration with an MRI navigator was used to determine a diaphragm estimate from each directional-coupler measure. Seven healthy volunteers were scanned at 7 T, in which images of the diaphragm were continuously acquired and directional couplers were monitored during excitation radiofrequency pulses. The ability to detect coughing was evaluated in one subject. The method was implemented on the scanner and evaluated for diaphragm gating of a free-breathing cardiac cine. ResultsSix of the seven scans were successful. In these subjects, the root mean square difference between MRI and scattering estimation of the superior–inferior diaphragm position was 1.4 ± 0.5 mm. On the scanner, the position was calculated less than 2 ms after every radiofrequency pulse. A prospectively gated (echocardiogram and respiration) high-resolution free-breathing cine showed no respiratory artifact and sharp blood-myocardium definition. ConclusionsTransmit coil scattering is sensitive to diaphragm motion and provides rapid, quantitative, and accurate monitoring of respiration

Effects of antiplatelet therapy on stroke risk by brain imaging features of intracerebral haemorrhage and cerebral small vessel diseases: subgroup analyses of the RESTART randomised, open-label trial

Background Findings from the RESTART trial suggest that starting antiplatelet therapy might reduce the risk of recurrent symptomatic intracerebral haemorrhage compared with avoiding antiplatelet therapy. Brain imaging features of intracerebral haemorrhage and cerebral small vessel diseases (such as cerebral microbleeds) are associated with greater risks of recurrent intracerebral haemorrhage. We did subgroup analyses of the RESTART trial to explore whether these brain imaging features modify the effects of antiplatelet therapy

Unambiguous detection of cardiac Pi using long TM 31P STEAM

Synopsis Inorganic Phosphate is a resonance that holds important information on the metabolic state of tissues. From its resonance frequency, intracellular pH can be derived. The ratio of P to PCr or ATP are also important markers. Unlike in other tissues, myocardial P is frequently hidden underneath blood DPG signals. Using STEAM's T delay to be one cardiac cycle, blood-pool originating signals are gone and the Pi resonance is clearly visible. In 3 subjects, P signal was detected and quantified. The signal was around 4.89±0.02ppm, corresponding to a pH of 7.08±0.02. This is a breakthrough for the investigation of cardiac metabolism.This work was supported by the Austrian Science Fund (FWF) project J 4043 as well as by the Sir Henry Dale Fellowship from the Wellcome Trust and the Royal Society (098436/Z/12/Z)

A multitude of modes: considering ‘blended learning’ in context

Discussions of mode in higher education tend to contrast face-to-face teaching with distance education, while acknowledging that somewhere in between these poles, some students are engaged in a somewhat mysterious practice of ‘blended learning’. Blended learning is generally taken to indicate that a combination of face-to-face time in the classroom with the use of digital/online platforms is in use. While the question of whether blended learning is well-understood or well-defined remains unanswered, it can nonetheless be described as the ‘new normal’. This session considers transformation in educational delivery mode in the context of the results of an institutional review of technology-enhanced learning, undertaken at Birkbeck, University of London in 2018. An overview of the method and results of the review will be discussed, alongside consideration of the extent to which these findings are more widely applicable. Birkbeck’s mission has been to create opportunities to study for groups who are usually excluded by conventional modes of provision. However, where ‘distance’ mode teaching acts to take location out of the equation, Birkbeck has historically opened access through its timetable. Birkbeckstudents give up the semblance of a normal life to dedicate many of their evenings to the classroom. But perhaps this has obscured the extent to which student life is increasingly the life of the computer, as well as of the mind. Evening, face-to-face mode teaching, while seen to a great extent as the college’s ‘USP’, is only one element of the ‘student experience’ and digital practices have quietly gone mainstream. These results have thrown into question whether ‘face-to-face’ mode truly exists at Birkbeck any more. Furthermore, if blended learning has become an umbrella term that covers a multitude of modes, it seems that much closer attention is now due to the nature and diversity of ‘blends’

Metabolic Costs of a 58-minute Multi-Intensity Exercise Session with and Without Music and Cueing

International Journal of Exercise Science 13(2): 358-365, 2020. It is unclear if the presence or absence of music and cueing influence total energy expenditure (TEE) during a multi-intensity exercise program. The purpose of this study was to determine the difference between TEE with or without music and cueing during a 58-minute exercise session using heart rate estimation (HRe) and indirect calorimetry (IC). Using a randomized crossover design, 22 participants (6 males; 16 females; 27.64 ± 10.33 yrs.) were randomized into two groups (Group A = 11; Group B = 11). All participants performed the same 58-minute exercise session under two conditions: with music (WM) and without music and cueing (WOM). TEE was obtained through the Activio heart rate system for all 22 participants. TEE and excess post-exercise oxygen consumption (EPOC) were also obtained in a subset of eight participants (4 males; 4 females; 28.25 ± 5.9 yrs.) via IC through a ParvoMedics metabolic cart. Paired samples t-tests were performed to compare TEE between conditions using HRe and IC. Statistical analysis was performed using IBM Analytics, SPSS v24 with significance set at p\u3c 0.05. A significant difference (p= 0.008) was found between TEE WM and WOM using IC (475.74 ± 98.50 vs. 429.37 ± 121.42), but not between TEE WM and WOM using HRe (p= 2.04; 482.67 ± 151.79 vs. 452.90 ± 164.59). The presence of music and cueing increased TEE when monitored via IC, but not when measured via wearable heart rate technology. Music and cueing does aid in additional caloric expenditure

Implementation of the Joint Duty Program at CIA: Analysis and Recommendations

This report assesses the strengths and weaknesses of the Joint Duty (JD) program as it is currently implemented at the CIA. Over the course of approximately six weeks, the Bush School 2012 Capstone interviewed - in-person and by phone - 160 CIA employees who completed a JD assignment. In assessing the qualitative and quantitative responses reported by personnel, we conclude that employees find value in the program, are well-integrated within their host agency, and achieve the program's mission of increasing employees' knowledge of other Intelligence Community agencies. Weaknesses hindering the program include: a disproportionately high number of employees choosing assignments at the Office of Director of National Intelligence (ODNI) and the National Reconnaissance Agency (NRO); insufficient manager guidance in selecting career-relevant assignments; failure to comply with regulations tasking agencies to maintain contact with their JD employees; employees experiencing significant difficulty returning to the CIA upon completion of their assignment; and inconsistent views regarding whether the program aids promotion potential.The Center for the Study of Intelligenc