scholarly journals Oxytocin and Naltrexone Successfully Treat Hypothalamic Obesity in a Boy Post-Craniopharyngioma Resection

2017 ◽  
Vol 103 (2) ◽  
pp. 370-375 ◽  
Author(s):  
Eugenie A Hsu ◽  
Jennifer L Miller ◽  
Francisco A Perez ◽  
Christian L Roth
Keyword(s):  
Parental Monitoring ◽  
Animal Studies ◽  
Phase 1 ◽  
Poor Quality ◽  
Free Access ◽  
Phase 2 ◽  
Z Score ◽  
Hypothalamic Obesity ◽  
Food Seeking

Abstract Context Hypothalamic obesity, a treatment-resistant condition common to survivors of craniopharyngioma (CP), is strongly associated with a poor quality of life in this population. Oxytocin (OT), a hypothalamic neuropeptide, has been shown to play a role in the regulation of energy balance and to have anorexigenic effects in animal studies. Naltrexone (NAL), an opiate antagonist, has been shown to deter hedonic eating and to potentiate OT’s effects. Design In this parent-observed study, we tested the administration of intranasal OT for 10 weeks (phase 1), followed by a combination of intranasal OT and NAL for 38 weeks (phase 2) in a 13-year-old male with confirmed hypothalamic obesity and hyperphagia post-CP resection. Treatment resulted in 1) reduction in body mass index (BMI) z score from 1.77 to 1.49 over 10 weeks during phase 1; 2) reduction in BMI z score from 1.49 to 0.82 over 38 weeks during phase 2; 3) reduced hyperphagia during phases 1 and 2; 4) continued hedonic high-carbohydrate food-seeking in the absence of hunger during phases 1 and 2; and 5) sustained weight reduction during decreased parental monitoring and free access to unlocked food in the home during the last 10 weeks of phase 2. Conclusion This successful intervention of CP-related hypothalamic obesity and hyperphagia by OT alone and in combination with NAL is promising for conducting future studies of this treatment-recalcitrant form of obesity.

scholarly journals Longitudinal Impact of APRNs on Nursing Home Quality Measures in the Missouri Quality Initiative

2021 ◽  
Author(s):  
Marilyn Rantz ◽  
G. F. Petroski ◽  
L. L. Popejoy ◽  
A. A. Vogelsmeier ◽  
K. E. Canada ◽  
...  
Keyword(s):  
Quality Of Care ◽  
Phase 1 ◽  
Quality Measures ◽  
The State ◽  
Full Time ◽  
Improve Quality ◽  
Phase 2 ◽  
Intervention Phase ◽  
Tract Infections

Abstract Objectives To measure the impact of advanced practice nurses (APRNs) on quality measures (QM) scores of nursing homes (NHs) in the CMS funded Missouri Quality Initiative (MOQI) that was designed to reduce avoidable hospitalizations of NH residents, improve quality of care, and reduce overall healthcare spending. Design A four group comparative analysis of longitudinal data from September 2013 thru December 2019. Setting NHs in the interventions of both Phases 1 (2012–2016) and 2 (2016–2020) of MOQI (n=16) in the St. Louis area; matched comparations in the same counties as MOQI NHs (n=27); selected Phase 2 payment intervention NHs in Missouri (n=24); NHs in the remainder of the state (n=406). Participants NHs in Missouri Intervention: Phase 1 of The Missouri Quality Initiative (MOQI), a Centers for Medicare and Medicaid (CMS) Innovations Center funded research initiative, was a multifaceted intervention in NHs in the Midwest, which embedded full-time APRNs in participating NHs to reduce hospitalizations and improve care of NH residents. Phase 2 extended the MOQI intervention in the original intervention NHs and added a CMS designed Payment Intervention; Phase 2 added a second group of NHs to receive the Payment. Intervention Only. Measurements Eight QMs selected by CMS for the Initiative were falls, pressure ulcers, urinary tract infections, indwelling catheters, restraint use, activities of daily living, weight loss, and antipsychotic medication use. For each of the monthly QMs (2013 thru 2019) an unobserved components model (UCM) was fitted for comparison of groups. Results The analysis of QMs reveals that that the MOQI Intervention + Payment group (group with the embedded APRNs) outperformed all comparison groups: matched comparison with neither intervention, Payment Intervention only, and remainder of the state. Conclusion These results confirm the QM analyses of Phase 1, that MOQI NHs with full-time APRNs are effective to improve quality of care.

scholarly journals Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects with Hemophilia A and B with Inhibitors Assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 23-24
Author(s):  
Sylvia Von Mackensen ◽  
Pratima Chowdary ◽  
Sarah Mangles ◽  
Qifeng Yu ◽  
Baisong Mei ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Hemophilia A ◽  
Phase 1 ◽  
Phase 2 ◽  
Publicly Traded ◽  
Advisory Committees ◽  
Related Quality ◽  
Health Related ◽  
Dose Dependent

Background: Fitusiran, an investigational RNA interference treatment for people with hemophilia A or B (PwH), with or without inhibitors, has shown dose-dependent lowering of antithrombin, increase in thrombin generation, and decrease in bleeding frequency in clinical trials. The novel mechanism of action and long pharmacodynamic effect enables once-monthly subcutaneous administration. This sustained hemostatic protection and less burdensome administration may improve patient-reported outcomes (PRO). Objective: To evaluate changes in PRO in terms of patient-relevant improvements in health-related quality of life (HRQoL) in PwH with inhibitors (PwHI) on prophylactic fitusiran treatment. Methods: Fitusiran was evaluated in a phase 1 dose-escalation study (NCT02035605) followed by a phase 2 open-label extension (OLE) study (NCT02554773) with monthly subcutaneous fixed doses of 50 mg or 80 mg. HRQoL was assessed using the Haem-A-QoL and the EuroQol 5 Dimensions (EQ-5D) questionnaires at baseline and at end of study in a cohort of 17 PwHI (Hemophilia A, n=15; Hemophilia B, n=2) from the phase 1 study. Results: Subjects previously treated on-demand or prophylactically had a mean (standard deviation [SD]) age of 34.6 (10.3) years and a mean (SD) number of bleeding episodes in the 6 months before baseline of 16.6 (10.7). Mean (SD) changes from baseline to end of study (day 84 or later) in Haem-A-QoL total (-9.2 [11.2]) and physical health (−12.3 [15.1]) domain scores suggest clinically meaningful improvement (lower scores indicate better HRQoL). Numeric reduction (i.e., improvement) in all other domains appeared to be dose-dependent (greater improvement in the 80 mg group) (Table 1). Changes in EQ-5D utility and EQ-VAS scores were not clinically meaningful. Further analyses in PwH with and without inhibitors from the phase 2 OLE will be presented. Conclusions: Fitusiran prophylaxis may improve HRQoL - particularly the Haem-A-QoL 'Physical health' domain (painful swelling, joint pain, pain with movement, difficulty walking, and time to get ready) as shown in a cohort of 17 PwHI . Additional analyses from ongoing OLE and phase 3 studies are planned to quantify the patient-relevant changes with fitusiran treatment in all hemophilia patients over time. Disclosures Von Mackensen: Sanofi, Bayer, Sobi, Chugai, Kedrion, Spark: Consultancy; Biotest, Sobi, CSL Behring: Honoraria; Novo Nordisk, Sobi: Research Funding. Chowdary:BioMarin: Honoraria; Bayer, CSL Behring, Freeline, Novo Nordisk, Pfizer and Sobi: Research Funding; Chugai, CSL Behring, Novo Nordisk, Pfizer, Roche, Sobi: Speakers Bureau; Bayer, Chugai, CSL Behring, Freeline, Novo Nordisk, Pfizer, Roche, Sanofi, Shire (Baxalta), Sobi, Spark: Membership on an entity's Board of Directors or advisory committees. Mangles:Roche, Takeda, Novo Nordisk: Consultancy, Membership on an entity's Board of Directors or advisory committees; Sobi, Octapharma, Novo Nordisk, Shire and Roche/Chugai: Other: travel funding. Yu:Sanofi: Other: was an employee and stockholder of Sanofi, at the time of study; Albireo Pharmaceuticals, Inc: Current Employment. Mei:Sanofi: Current Employment, Current equity holder in publicly-traded company. Andersson:Sanofi: Current Employment, Current equity holder in publicly-traded company. Dasmahapatra:Sanofi: Current Employment, Current equity holder in publicly-traded company.

Optimal Thyroid Hormone Replacement

2021 ◽  
Author(s):  
Jacqueline Jonklaas
Keyword(s):  
Combination Therapy ◽  
Animal Studies ◽  
Randomized Clinical Trials ◽  
Phase 1 ◽  
Life Stage ◽  
Hormone Replacement ◽  
Thyroid Hormone Replacement ◽  
The Impact ◽  
Baseline Health

Abstract Hypothyroidism is a common endocrinopathy and levothyroxine is frequently prescribed. Despite the basic tenets of initiating and adjusting levothyroxine being agreed upon, there are many nuances and complexities to consistently maintaining euthyroidism. Understanding the impact of patient weight and residual thyroid function on initial levothyroxine dosage and consideration of age, co-morbidities, TSH goal, life stage, and quality of life as levothyroxine is adjusted can be challenging and continually evolving. As levothyroxine is a life-long medication it is important to avoid risks from periods of overtreatment or undertreatment. For the subset of patients not restored to baseline health with levothyroxine, causes arising from all aspects of the patient’s life (co-existent medical conditions, stressors, lifestyle, psychosocial factors) should be broadly considered. If such factors do not appear to be contributing, and biochemical euthyroidism has been successfully maintained, there may be benefit to a trial of combination therapy with levothyroxine and liothyronine. This is not supported by the majority of randomized clinical trials, but may be supported by other studies providing lower quality evidence and by animal studies. Given this discrepancy, it is important that any trial of combination therapy only be continued as long as a patient benefit is being enjoyed. Monitoring for adverse effects, particularly in older or frail individuals, is necessary and combination therapy should not be utilized during pregnancy. A sustained release liothyronine preparation has completed phase 1 testing and may soon be available for better designed and powered studies assessing whether combination therapy provides superior therapy for hypothyroidism.

scholarly journals Can prospective systematic reviews of animal studies improve clinical translation?

2020 ◽  
Vol 18 (1) ◽  
Author(s):  
Pandora Pound ◽  
Merel Ritskes-Hoitinga
Keyword(s):  
Clinical Trials ◽  
Systematic Reviews ◽  
Animal Studies ◽  
Poor Quality ◽  
Human Studies ◽  
Clinical Knowledge ◽  
Safety And Efficacy ◽  
High Quality Evidence ◽  
Animal Data

AbstractSystematic reviews are powerful tools with the potential to generate high quality evidence. Their application to animal studies has been instrumental in exposing the poor quality of these studies, as well as a catalyst for improvements in study design, conduct and reporting. It has been suggested that prospective systematic reviews of animal studies (i.e. systematic reviews conducted prior to clinical trials) would allow scrutiny of the preclinical evidence, providing valuable information on safety and efficacy, and helping to determine whether clinical trials should proceed. However, while prospective systematic reviews allow valuable scrutiny of the preclinical animal data, they are not necessarily able to reliably predict the safety and efficacy of an intervention when trialled in humans. Consequently, they may not reliably safeguard humans participating in clinical trials and might potentially result in lost opportunities for beneficial clinical treatments. Furthermore, animal and human studies are often conducted concurrently, which not only makes prospective systematic reviews of animal studies impossible, but suggests that animal studies do not inform human studies in the manner presumed. We suggest that this points to a confused attitude regarding animal studies, whereby tradition demands that they precede human studies but practice indicates that their findings are often ignored. We argue that it is time to assess the relative contributions of animal and human research in order to better understand how clinical knowledge is actually produced.

scholarly journals An evaluation of foundation doctor training: a mixed-methods study of the impact on workforce well-being and patient care [the Evaluating the Impact of Doctors in Training (EDiT) study]

2013 ◽  
Vol 1 (15) ◽  
pp. 1-208 ◽  
Author(s):  
S Mason ◽  
C O’Keeffe ◽  
A Carter ◽  
R O’Hara ◽  
C Stride
Keyword(s):  
Health Care ◽  
Quality Of Care ◽  
Longitudinal Study ◽  
Phase 1 ◽  
Well Being ◽  
Junior Doctors ◽  
Phase 2 ◽  
Adequate Quality ◽  
The Impact

BackgroundA major reform of junior doctor training was undertaken in 2004–5, with the introduction of foundation training (FT) to address perceived problems with work structure, conditions and training opportunities for postgraduate doctors. The well-being and motivation of junior doctors within the context of this change to training (and other changes such as restrictions in working hours of junior doctors and increasing demand for health care) and the consequent impact upon the quality of care provided is not well understood.ObjectivesThis study aimed to evaluate the well-being of foundation year 2 (F2) doctors in training. Phase 1 describes the aims of delivering foundation training with a focus on the role of training in supporting the well-being of F2 doctors and assesses how FT is implemented on a regional basis, particularly in emergency medicine (EM). Phase 2 identifies how F2 doctor well-being and motivation are influenced over F2 and specifically in relation to EM placements and quality of care provided to patients.MethodsPhase 1 used semistructured interviews and focus groups with postgraduate deanery leads, training leads (TLs) and F2 doctors to explore the strategic aims and implementation of FT, focusing on the specialty of EM. Phase 2 was a 12-month online longitudinal study of F2 doctors measuring levels of and changes in well-being and motivation. In a range of specialties, one of which was EM, data from measures of well-being, motivation, intention to quit, confidence and competence and job-related characteristics (e.g. work demands, task feedback, role clarity) were collected at four time points. In addition, we examined F2 doctor well-being in relation to quality of care by reviewing clinical records (criterion-based and holistic reviews) during the emergency department (ED) placement relating to head injury and chronic obstructive pulmonary disease (COPD).ResultsPhase 1 of the study found that variation exists in how successfully FT is implemented locally; F2 lacks a clearly defined end point; there is a minimal focus on the well-being of F2 doctors (only on the few already shown to be ‘in difficulty’); the ED presented a challenging but worthwhile learning environment requiring a significant amount of support from senior ED staff; and disagreement existed about the performance and confidence levels of F2 doctors. A total of 30 EDs in nine postgraduate medical deaneries participated in phase 2 with 217 foundation doctors completing the longitudinal study. F2 doctors reported significantly increased confidence in managing common acute conditions and undertaking practical procedures over their second foundation year, with the biggest increase in confidence and competence associated with their ED placement. F2 doctors had levels of job satisfaction and anxiety/depression that were comparable to or better than those of other NHS workers, and adequate quality and safety of care are being provided for head injury and COPD.ConclusionsThere are ongoing challenges in delivering high-quality FT at the local level, especially in time-pressured specialties such as EM. There are also challenges in how FT detects and manages doctors who are struggling with their work. The survey was the first to document the well-being of foundation doctors over the course of their second year, and average scores compared well with those of other doctors and health-care workers. F2 doctors are benefiting from the training provided as we found improvements in perceived confidence and competence over the year, with the ED placement being of most value to F2 doctors in this respect. Although adequate quality of care was demonstrated, we found no significant relationships between well-being of foundation doctors and the quality of care they provided to patients, suggesting the need for further work in this area.FundingThe National Institute for Health Research Health Services and Delivery Research programme.

scholarly journals Increased emotional eating during COVID-19 associated with lockdown, psychological and social distress

2020 ◽  
Author(s):  
Cinzia Cecchetto ◽  
Marilena Aiello ◽  
Silvio Ionta ◽  
Claudio Gentili ◽  
Sofia Adelaide Osimo
Keyword(s):  
Binge Eating ◽  
Emotional Eating ◽  
Online Survey ◽  
Phase 1 ◽  
Eating Behaviour ◽  
Phase 2 ◽  
Emotional Wellbeing ◽  
Negative Effects ◽  
The Impact

Due to the spread of COVID 2019, the Italian government imposed a lockdown on the national territory. Initially, citizens were required to stay at home and not to mix with others outside of their household (Phase 1); eventually, some of these restrictions were lifted (Phase 2). To investigate the impact of lockdown on emotional and binge eating, an online survey was conducted to compare measures of self-reported physical (BMI), psychological (Alexithymia), affective (anxiety, stress, and depression) and social (income, workload) state during Phase 1 and Phase 2. Data from 365 Italian residents showed that increased emotional eating was predicted by higher depression, anxiety, quality of personal relationships, and quality of life, while the increase of bingeing was predicted by higher stress. Moreover, we showed that higher alexithymia scores were associated by increased emotional eating and higher BMI scores were associated with both increased emotional eating and binge eating. Finally, we found that from Phase 1 to Phase 2 binge and emotional eating decreased. These data provide evidence of the negative effects of isolation and lockdown on emotional wellbeing, and, relatedly, on eating behaviour.

scholarly journals A Cross-Sectional Examination of the Mental Wellbeing, Coping and Quality of Working Life in Health and Social Care Workers in the UK at Two Time Points of the COVID-19 Pandemic

Epidemiologia ◽  
2021 ◽  
Vol 2 (3) ◽  
pp. 227-242
Author(s):  
Paula McFadden ◽  
Ruth D. Neill ◽  
John Moriarty ◽  
Patricia Gillen ◽  
John Mallett ◽  
...  
Keyword(s):  
Social Care ◽  
Phase 1 ◽  
Working Life ◽  
Phase 2 ◽  
Cross Sectional ◽  
Quality Of Working Life ◽  
Care Workers ◽  
Health And Social Care ◽  
The Uk

As the COVID-19 pandemic continues to evolve around the world, it is important to examine its effect on societies and individuals, including health and social care (HSC) professionals. The aim of this study was to compare cross-sectional data collected from HSC staff in the UK at two time points during the COVID-19 pandemic: Phase 1 (May–July 2020) and Phase 2 (November 2020–January 2021). The HSC staff surveyed consisted of nurses, midwives, allied health professionals, social care workers and social workers from across the UK (England, Wales, Scotland, Northern Ireland). Multiple regressions were used to examine the effects of different coping strategies and demographic and work-related variables on participants’ wellbeing and quality of working life to see how and if the predictors changed over time. An additional multiple regression was used to directly examine the effects of time (Phase 1 vs. Phase 2) on the outcome variables. Findings suggested that both wellbeing and quality of working life deteriorated from Phase 1 to Phase 2. The results have the potential to inform interventions for HSC staff during future waves of the COVID-19 pandemic, other infectious outbreaks or even other circumstances putting long-term pressures on HSC systems.

scholarly journals INDONESIAN CHILDREN’S QUALITY OF LIFE: A CASE STUDY OF RESIDENTS RELOCATION TO FLATS IN JAKARTA PROVINCE

2019 ◽  
Vol 19 (2) ◽  
pp. 61-67
Author(s):  
Rahmah Hida Nurrizka ◽  
Rafiah Maharani
Keyword(s):  
Quality Of Life ◽  
Family Involvement ◽  
Poor Quality ◽  
Provincial Government ◽  
Z Score ◽  
The Family ◽  
Score Method ◽  
Nutrients Intake

Resident relocation to flats policy becomes a strategy of the Jakarta Provincial Government to overcome illegal settlements and slum areas. The relocation is also aimed to improve the quality of life, particularly of the children. This research analyses the determinant factors of the quality of life of children who are moved to the flats due to the relocation. This research uses the Kid-KINDLR questionnaire to collect data of children in the range of age 7-13 years and the Z-score method to process the data and to build an analysis. The Z-score > 0 indicates a good quality of life, while Z-score < 0 is otherwise. The result shows that 48% of the children have a good quality of life and 52% of the children have a poor quality of life (mean = 91.45 and SD = 9.559). There is a positive correlation between the quality of life of children and the pattern of nutrients intake of children (r = 0.053, p = 0.518), the education of the head of the family (r = 0.058, p = 0.478), and the household income (r = 0.070, p = 0.401). Whereas, the negative correlation between the quality of life of children and the number of family members (r = -0.088, p = 0.284). This research argues that to increase the quality of life of children, the facilities in the flats must be built properly for the growth of the children. In addition, family involvement must be improved to provide a high quality of nutrients intake for the children.

THYROXINE UTILIZATION IN LAMBS IN NATURAL AND CONTROLLED ENVIRONMENTS

1970 ◽  
Vol 46 (3) ◽  
pp. 363-367 ◽  
Author(s):  
I. R. FALCONER ◽  
B. MARCHANT
Keyword(s):  
Poor Quality ◽  
Repeated Measurements ◽  
Controlled Environment ◽  
Free Access ◽  
Ambient Temperatures ◽  
Ewe Lambs ◽  
Controlled Environments ◽  
Inadequate Nutrition ◽  
Access To Food

SUMMARY The rate of thyroxine utilization has been measured from the rate of degradation of [125I]thyroxine and the concentration of plasma proteinbound iodine in ewe lambs. Repeated measurements were made between 10 and 60 weeks of age in two groups of lambs, one in conditions including temporarily inadequate nutrition and outdoor ambient temperatures, and the other in a controlled environment with free access to food. The lambs in the controlled environment showed an increase in thyroxine utilization with increasing age throughout the experiment, whereas the 'outdoor lambs' showed a decrease in hormone utilization until after 34 weeks of age. The 'outdoor lambs' also lost bodyweight between the 20th and 26th week of age due to the poor quality of the pasture, and this undernutrition is considered to have caused the lowered rate of thyroxine utilization. After 26 weeks of age the nutrition of these lambs was adequate but thyroxine utilization did not increase until after 34 weeks of age, when the mean environmental temperature was about 5°. It was concluded that undernutrition caused a marked depression in thyroxine utilization of young lambs.

The cognitive effects of modafinil in breast cancer survivors: A randomized clinical trial

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 9004-9004 ◽  
Author(s):  
S. Kohli ◽  
S. G. Fisher ◽  
Y. Tra ◽  
K. Wesnes ◽  
G. R. Morrow
Keyword(s):  
Breast Cancer ◽  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Cancer Patients ◽  
Phase 1 ◽  
Secondary Memory ◽  
Phase 2 ◽  
Breast Cancer Patients ◽  
Memory And Attention

9004 Background: Patients receiving treatment for cancer can experience a diminution of their cognitive abilities which can persist for many years after. The present study examined the potential of modafinil as a cognitive enhancer on memory and attention in a randomized clinical trial in breast cancer patients following completion of treatment. Methods: Breast cancer patients who reported a score of = 2 on the Brief Fatigue Inventory (BFI) an average of 22.8 months following completion of chemotherapy were enrolled. In Phase 1, patients received 200mg modafinil open-label once daily for 4 weeks. In Phase 2, patients with a positive response following Phase 1 were randomized either to an additional 4 weeks of modafinil or to placebo. Tests of memory and attention selected from the Cognitive Drug Research (CDR) computerized cognitive assessment were performed at baseline (before modafinil) and after completing Phase 1 and 2. The difference between scores at baseline and scores at Phase 1 and Phase 2, were subjected to a Wilcoxon's signed rank test. Results: Of the 82 women enrolled, 68 completed all 3 CDR assessments in the study (range=33–83 years, median age=54 years). At the end of Phase 1 (week 4), modafinil had a significant effect on the Speed of Memory Index (p=0.0047) and the Digit Vigilance Test (p=0.0014) but showed no significant effect on Quality of Working Memory (p=0.3528) nor on Power of Attention (p=0.8953). At the end of phase 2 (week 8), the group that continued modafinil demonstrated improved Mean Continuity of Attention (p=0.0002) compared with the placebo group and also demonstrated significantly greater improvements in Quality of Episodic Secondary Memory relative to placebo (p<0.005). This improvement was observed at week 4, and was maintained throughout the study. Finally, continued modafinil demonstrated significantly greater improvement in Speed of Memory relative to placebo (p=0.009). Conclusions: Modafinil provides significant improvements in attention and speed of memory for breast cancer patients following their treatment of cancer. Although further study is needed, these findings suggest that modafinil may enhance quality of life in this patient population. Supported by a grant from Cephalon, Inc and NCI Grants U10-CA37420 and R25- CA102618. No significant financial relationships to disclose.

Sign in / Sign up

Export Citation Format

Share Document