scholarly journals Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index

2021 ◽  
Vol 10 (24) ◽  
pp. 5836
Author(s):  
Nicholas H. B. Schräder ◽  
Eva W. H. Korte ◽  
José C. Duipmans ◽  
Roy E. Stewart ◽  
Maria C. Bolling ◽  
...  
Keyword(s):  
Epidermolysis Bullosa ◽  
Clinical Care ◽  
Multidisciplinary Care ◽  
Skin Condition ◽  
Patient Needs ◽  
Patient Benefit ◽  
Ancillary Data ◽  
Treatment Benefits ◽  
Insight Into

Epidermolysis bullosa (EB) is a genetic blistering skin condition for which no cure exists. Symptom alleviation and quality of life are therefore central to EB care. This study aimed to gain insight into EB patient needs and benefits from current clinical care. Two questionnaires were administered cross-sectionally to adult EB patients at the Dutch expertise centre for blistering diseases. Patient needs and benefits were analyzed using the patient benefit index survey (PBI-S). Ancillary data were compiled pertaining to self-reported EB severity, pain and pruritus, as well as current and previous treatments. In total, 104 participants were included (response rate 69.8%). Sixty-eight participants comprised the analyzed cohort (n = 36 omitted from analysis). The needs given the highest importance were to get better skin quickly (64.7%) and to be healed of all skin alterations (61.8%). A positive correlation between pain and EB severity and the importance of most needs was observed. Minimal clinically important differences within the PBI-S, relating to reported benefits from clinical care, were reported by 60.3% of the cohort. This study highlights a discrepancy between patient needs and feasible treatment outcomes. Utilizing the PBI-S in conjunction with well-established multidisciplinary care may catalyze the process of tailoring treatments to the needs of individual patients.

scholarly journals Nutritional, Gastrointestinal and Endo-Metabolic Challenges in the Management of Children with Spinal Muscular Atrophy Type 1

Nutrients ◽  
2021 ◽  
Vol 13 (7) ◽  
pp. 2400
Author(s):  
Antonio Corsello ◽  
Lorenzo Scatigno ◽  
Martina Chiara Pascuzzi ◽  
Valeria Calcaterra ◽  
Dario Dilillo ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Clinical Care ◽  
Palliative Therapy ◽  
Childhood And Adolescence ◽  
Spinal Muscular Atrophy Type ◽  
History Of ◽  
Insight Into

The management of patients with spinal muscular atrophy type 1 (SMA1) is constantly evolving. In just a few decades, the medical approach has switched from an exclusively palliative therapy to a targeted therapy, transforming the natural history of the disease, improving survival time and quality of life and creating new challenges and goals. Many nutritional problems, gastrointestinal disorders and metabolic and endocrine alterations are commonly identified in patients affected by SMA1 during childhood and adolescence. For this reason, a proper pediatric multidisciplinary approach is then required in the clinical care of these patients, with a specific focus on the prevention of most common complications. The purpose of this narrative review is to provide the clinician with a practical and usable tool about SMA1 patients care, through a comprehensive insight into the nutritional, gastroenterological, metabolic and endocrine management of SMA1. Considering the possible horizons opened thanks to new therapeutic frontiers, a nutritional and endo-metabolic surveillance is a crucial element to be considered for a proper clinical care of these patients.

scholarly journals Nutritional, Gastrointestinal and Endo-Metabolic Traps in the Management of Children with Spinal Muscular Atrophy Type 1

2021 ◽  
Author(s):  
Antonio Corsello ◽  
Lorenzo Scatigno ◽  
Martina Chiara Pascuzzi ◽  
Valeria Calcaterra ◽  
Dario Dilillo ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Clinical Care ◽  
Palliative Therapy ◽  
Childhood And Adolescence ◽  
Spinal Muscular Atrophy Type ◽  
History Of ◽  
Insight Into

The management of patients with spinal muscular atrophy type 1 (SMA1) is constantly evolving. In just a few decades the medical approach has switched from an exclusively palliative therapy to a targeted therapy, transforming the natural history of the disease, improving survival time and quality of life, and creating new challenges and goals. Many nutritional problems, gastrointesti-nal disorders and metabolic and endocrine alterations are commonly identified in patients af-fected by SMA1 during childhood and adolescence. For this reason, a proper pediatric multidis-ciplinary approach is then required in the clinical care of these patients, with a specific focus on the prevention of most common complications. The purpose of this narrative review is to provide the clinician with a practical and usable tool about SMA1 patients care, through a comprehensive insight into the nutritional, gastroenterological, metabolic and endocrine management of SMA1. Considering the possible horizons opened thanks to new therapeutic frontiers, a nutritional and endo-metabolic surveillance is a crucial element to be considered for a proper clinical care of these patients.

Thalassaemia in children: from quality of care to quality of life

2015 ◽  
Vol 100 (11) ◽  
pp. 1051-1057 ◽  
Author(s):  
Ali Amid ◽  
Antoine N Saliba ◽  
Ali T Taher ◽  
Robert J Klaassen
Keyword(s):  
Quality Of Life ◽  
Iron Status ◽  
Clinical Care ◽  
Iron Chelation ◽  
Multidisciplinary Care ◽  
Developed Countries ◽  
Limited Resources ◽  
Future Research ◽  
Safe Blood

Over the past few decades, there has been a remarkable improvement in the survival of patients with thalassaemia in developed countries. Availability of safe blood transfusions, effective and accessible iron chelating medications, the introduction of new and non-invasive methods of tissue iron assessment and other advances in multidisciplinary care of thalassaemia patients have all contributed to better outcomes. This, however, may not be true for patients who are born in countries where the resources are limited. Unfortunately, transfusion-transmitted infections are still major concerns in these countries where paradoxically thalassaemia is most common. Moreover, oral iron chelators and MRI for monitoring of iron status may not be widely accessible or affordable, which may result in poor compliance and suboptimal iron chelation. All of these limitations will lead to reduced survival and increased thalassaemia-related complications and subsequently will affect the patient's quality of life. In countries with limited resources, together with improvement of clinical care, strategies to control the disease burden, such as public education, screening programmes and appropriate counselling, should be put in place. Much can be done to improve the situation by developing partnerships between developed countries and those with limited resources. Future research should also particularly focus on patient's quality of life as an important outcome of care.

Gastroesophageal Reflux Disease: A Gastroenterologist's Perspective

2011 ◽  
Vol 21 (3) ◽  
pp. 89-99
Author(s):  
Michael F. Vaezi
Keyword(s):  
Gastroesophageal Reflux Disease ◽  
Gastroesophageal Reflux ◽  
Reflux Disease ◽  
Best Practice ◽  
Clinical Care ◽  
Diagnostic Testing ◽  
Diagnostic Tools ◽  
Swallowing Function ◽  
Atypical Symptoms

Gastroesophageal reflux disease (GERD) is a commonly diagnosed condition often associated with the typical symptoms of heartburn and regurgitation, although it may present with atypical symptoms such as chest pain, hoarseness, chronic cough, and asthma. In most cases, the patient's reduced quality of life drives clinical care and diagnostic testing. Because of its widespread impact on voice and swallowing function as well as its social implications, it is important that speech-language pathologists (SLPs) understand the nature of GERD and its consequences. The purpose of this article is to summarize the nature of GERD and GERD-related complications such as GERD-related peptic stricture, Barrett's esophagus and adenocarcinoma, and laryngeal manifestations of GERD from a gastroenterologist's perspective. It is critical that SLPs who work with a multidisciplinary team understand terminology, diagnostic tools, and treatment to ensure best practice.

Treatment of painful bone metastases in prostate and breast cancer patients with the therapeutic radiopharmaceutical rhenium-188-HEDP

2016 ◽  
Vol 55 (05) ◽  
pp. 188-195 ◽  
Author(s):  
Floor Overbeek ◽  
John de Klerk ◽  
Pieternel Pasker-de Jong ◽  
Alexandra van den Berk ◽  
Rob ter Heine ◽  
...  
Keyword(s):  
Bone Metastases ◽  
Clinical Benefit ◽  
Response Rate ◽  
Clinical Care ◽  
Pain Response ◽  
Pain Palliation ◽  
Breast Cancer Patients ◽  
Routine Clinical Care ◽  
Painful Bone Metastases

Summary Aim: Rhenium-188-HEDP (188Re-HEDP) is an effective radiopharmaceutical for the palliative treatment of osteoblastic bone metastases. However, only limited data on its routine use are available and its effect on quality of life (QoL) has not been studied. Therefore, we evaluated the clinical benefit of 188Re-HEDP in routine clinical care. Patients and methods: Prostate or breast cancer patients with painful bone metastases receiving 188Re-HEDP as a routine clinical procedure were eligible for evaluation. Clinical benefit was assessed in terms of efficacy and toxicity. Pain palliation and QoL were monitored using the visual analogue scale (VAS), corrected for opioid intake, and the EORTC QLQ-C30 Global health status/QoL-scale. Thrombocyte and leukocyte nadirs were used to assess haematological toxicity. Results: 45 and 47 patients were evaluable for pain palliation and QoL, respectively. After a single injection of 188Re-HEDP, the overall pain response rate was 69% and mean VAS-scores decreased relevantly and significantly (p < 0.05). Repeated treatment resulted in similar pain response. The overall QoL response rate was 68% and mean Global health status/QoL-scores increased relevantly and significantly. Haematological side effects were mild and transient. Conclusion: The clinically relevant response on pain and quality of life and the limited adverse events prove clinical benefit of treatment with 188Re-HEDP and support its use in routine clinical care. Its effectiveness appears comparable to that of external beam radiotherapy.

Attitudes of Elderly People in the South Moravian Region to Further Education

2014 ◽  
Vol 4 (2) ◽  
pp. 54-78
Author(s):  
Petr Adamec ◽  
Marián Svoboda
Keyword(s):  
Quality Of Life ◽  
Elderly People ◽  
Further Education ◽  
The South ◽  
The Third ◽  
Interesting Insight ◽  
Survey Results ◽  
Insight Into ◽  
Demographic Features

This paper deals with the results of sociological survey focused on identification of the attitudes of elderly people to further education. The research was carried out in September 2010. Experience of elderly people with further education, their readiness (determination) for further education as well as their motivation and barriers in further education were also subjects of this research. Detecting elderly population’s awareness of universities of the third age and finding out their further education preferences were an integral part of the research. Research sample consisted of citizens over 55 years living in the South Moravian region. The survey results are structured by socio-demographic features e.g.: age, sex, educational attainment etc. and provide an interesting insight into the attitudes of the target group to one of the activities that contributes to improvement of their quality of life.

Quality of Life for Children with Persistent Sinonasal Symptoms

2003 ◽  
Vol 128 (1) ◽  
pp. 17-26 ◽  
Author(s):  
David J. Kay ◽  
Richard M. Rosenfeld
Keyword(s):  
Quality Of Life ◽  
Clinical Care ◽  
Longitudinal Change ◽  
Good Test ◽  
Routine Clinical Care ◽  
Before And After ◽  
The Mean ◽  
The Individual ◽  
Sinonasal Symptoms

OBJECTIVE: The goal was to validate the SN-5 survey as a measure of longitudinal change in health-related quality of life (HRQoL) for children with persistent sinonasal symptoms. DESIGN AND SETTING: We conducted a before and after study of 85 children aged 2 to 12 years in a metropolitan pediatric otolaryngology practice. Caregivers completed the SN-5 survey at entry and at least 4 weeks later. The survey included 5 symptom-cluster items covering the domains of sinus infection, nasal obstruction, allergy symptoms, emotional distress, and activity limitations. RESULTS: Good test-retest reliability ( R = 0.70) was obtained for the overall SN-5 score and the individual survey items ( R ≥ 0.58). The mean baseline SN-5 score was 3.8 (SD, 1.0) of a maximum of 7.0, with higher scores indicating poorer HRQoL. All SN-5 items had adequate correlation ( R ≥ 0.36) with external constructs. The mean change in SN-5 score after routine clinical care was 0.88 (SD, 1.19) with an effect size of 0.74 indicating good responsiveness to longitudinal change. The change scores correlated appropriately with changes in related external constructs ( R ≥ 0.42). CONCLUSIONS: The SN-5 is a valid, reliable, and responsive measure of HRQoL for children with persistent sinonasal symptoms, suitable for use in outcomes studies and routine clinical care.

scholarly journals Comparisons among rainbow trout, Oncorhynchus mykiss, populations of maternal transcript profile associated with egg viability

BMC Genomics ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Gregory M. Weber ◽  
Jill Birkett ◽  
Kyle Martin ◽  
Doug Dixon ◽  
Guangtu Gao ◽  
...  
Keyword(s):  
Rainbow Trout ◽  
Egg Quality ◽  
Transcript Abundance ◽  
Differentially Expressed ◽  
Rainbow Trout Oncorhynchus Mykiss ◽  
Analysis Group ◽  
Maternal Transcripts ◽  
Single Batch ◽  
Insight Into

Abstract Background Transcription is arrested in the late stage oocyte and therefore the maternal transcriptome stored in the oocyte provides nearly all the mRNA required for oocyte maturation, fertilization, and early cleavage of the embryo. The transcriptome of the unfertilized egg, therefore, has potential to provide markers for predictors of egg quality and diagnosing problems with embryo production encountered by fish hatcheries. Although levels of specific transcripts have been shown to associate with measures of egg quality, these differentially expressed genes (DEGs) have not been consistent among studies. The present study compares differences in select transcripts among unfertilized rainbow trout eggs of different quality based on eyeing rate, among 2 year classes of the same line (A1, A2) and a population from a different hatchery (B). The study compared 65 transcripts previously reported to be differentially expressed with egg quality in rainbow trout. Results There were 32 transcripts identified as DEGs among the three groups by regression analysis. Group A1 had the most DEGs, 26; A2 had 15, 14 of which were shared with A1; and B had 12, 7 of which overlapped with A1 or A2. Six transcripts were found in all three groups, dcaf11, impa2, mrpl39_like, senp7, tfip11 and uchl1. Conclusions Our results confirmed maternal transcripts found to be differentially expressed between low- and high-quality eggs in one population of rainbow trout can often be found to overlap with DEGs in other populations. The transcripts differentially expressed with egg quality remain consistent among year classes of the same line. Greater similarity in dysregulated transcripts within year classes of the same line than among lines suggests patterns of transcriptome dysregulation may provide insight into causes of decreased viability within a hatchery population. Although many DEGs were identified, for each of the genes there is considerable variability in transcript abundance among eggs of similar quality and low correlations between transcript abundance and eyeing rate, making it highly improbable to predict the quality of a single batch of eggs based on transcript abundance of just a few genes.

scholarly journals Rapid Deployment of an Algorithm to Triage Dental Emergencies During COVID-19 Pandemic

2021 ◽  
Author(s):  
Sharon C Perelman ◽  
Steven Erde ◽  
Lynda Torre ◽  
Tunaidi Ansari
Keyword(s):  
Rapid Development ◽  
The United States ◽  
Healthcare Systems ◽  
Patient Needs ◽  
Columbia University ◽  
Dental Infection ◽  
Screening Algorithm ◽  
Rapid Deployment ◽  
Medical Infrastructure ◽  
Insight Into

Abstract COVID-19 quickly immobilized healthcare systems in the United States during the early stages of the outbreak. While much of the ensuing response focused on supporting the medical infrastructure, Columbia University College of Dental Medicine pursued a solution to triage and safely treat patients with dental emergencies amidst the pandemic. Considering rapidly changing guidelines from governing bodies, dental infection control protocols and our clinical faculty's expertise, we modeled, built, and implemented a screening algorithm, which provides decision support as well as insight into COVID-19 status and clinical comorbidities, within a newly integrated Electronic Health Record (EHR). Once operationalized, we analyzed the data and outcomes of its utilization and found that it had effectively guided providers in triaging patient needs in a standardized methodology. This article describes the algorithm's rapid development to assist faculty providers in identifying patients with the most urgent needs, thus prioritizing treatment of dental emergencies during the pandemic.

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