| 1. |
- Mercuri, E., et al.
(författare)
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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- 2020
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
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Tidskriftsartikel (refereegranskat)abstract
- Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p <= 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
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| 2. |
- Bushby, Katharine, et al.
(författare)
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Ataluren treatment of patients with nonsense mutation dystrophinopathy.
- 2014
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Ingår i: Muscle & nerve. - : Wiley. - 1097-4598 .- 0148-639X. ; 50:4, s. 477-87
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Tidskriftsartikel (refereegranskat)abstract
- Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of premature stop codons in nonsense mutation (nm) genetic disorders.
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| 3. |
- Henricson, E. K., et al.
(författare)
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The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures
- 2013
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Ingår i: Muscle & Nerve. - : Wiley. - 0148-639X. ; 48:1, s. 55-67
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Tidskriftsartikel (refereegranskat)abstract
- Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history studies. Methods: The Cooperative Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 DMD males, ages 2-28 years. A comprehensive battery of measures was obtained. Results: A novel composite functional milestone scale scale showed clinically meaningful mobility and upper limb abilities were significantly preserved in GC-treated adolescents/young adults. Manual muscle test (MMT)-based calculations of global strength showed that those patients <10 years of age treated with steroids declined by 0.4 +/- 0.39 MMT unit/year, compared with -0.4 +/- 0.39 MMT unit/year in historical steroid-naive subjects. Pulmonary function tests (PFTs) were relatively preserved in steroid-treated adolescents. The linearity and magnitude of decline in measures were affected by maturational changes and functional status. Conclusions: In DMD, long-term use of GCs showed reduced strength loss and preserved functional capabilities and PFTs compared with previous natural history studies performed prior to the widespread use of GC therapy.
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| 9. |
- Mercuri, E., et al.
(författare)
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Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis
- 2023
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Ingår i: Journal of Neurology. - 0340-5354. ; 270, s. 3896-3913
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Tidskriftsartikel (refereegranskat)abstract
- ObjectiveStrategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, international, multicenter registry of real-world ataluren use in individuals with nonsense mutation Duchenne muscular dystrophy (nmDMD) in clinical practice. This updated interim report (data cut-off: January 31, 2022), describes STRIDE patient characteristics and ataluren safety data, as well as the effectiveness of ataluren plus standard of care (SoC) in STRIDE versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS).MethodsPatients are followed up from enrollment for at least 5 years or until study withdrawal. Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established predictors of disease progression.ResultsAs of January 31, 2022, 307 patients were enrolled from 14 countries. Mean (standard deviation [SD]) ages at first symptoms and at genetic diagnosis were 2.9 (1.7) years and 4.5 (3.7) years, respectively. Mean (SD) duration of ataluren exposure was 1671 (56.8) days. Ataluren had a favorable safety profile; most treatment-emergent adverse events were mild or moderate and unrelated to ataluren. Kaplan-Meier analyses demonstrated that ataluren plus SoC significantly delayed age at loss of ambulation by 4 years (p < 0.0001) and age at decline to %-predicted forced vital capacity of < 60% and < 50% by 1.8 years (p = 0.0021) and 2.3 years (p = 0.0207), respectively, compared with SoC alone.ConclusionLong-term, real-world treatment with ataluren plus SoC delays several disease progression milestones in individuals with nmDMD. NCT02369731; registration date: February 24, 2015.
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| 12. |
- Egerod, I., et al.
(författare)
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The patient experience of intensive care: A meta-synthesis of Nordic studies
- 2015
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Ingår i: International Journal of Nursing Studies. - : Elsevier BV. - 0020-7489 .- 1873-491X. ; 52:8, s. 1354-1361
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Tidskriftsartikel (refereegranskat)abstract
- Background: Sedation practices in the intensive care unit have evolved from deep sedation and paralysis toward lighter sedation and better pain management. The new paradigm of sedation has enabled early mobilization and optimized mechanical ventilator weaning. Intensive care units in the Nordic countries have been particularly close to goals of lighter or no sedation and a more humane approach to intensive care. Objectives: The aim of our study was to systematically review and reinterpret newer Nordic studies of the patient experience of intensive care to obtain a contemporary description of human suffering during life-threatening illness. Design: We conducted a meta-synthesis in which we collected, assessed, and analyzed published qualitative studies with the goal of synthesizing these findings into a new whole. Analysis was based on the scientific approach of Gadamerian hermeneutics. Methods: We performed a literature search of qualitative studies of the patient experience of intensive care based on Nordic publications in 2000-2013. We searched the following databases: PubMed, CINAHL, Scopus, and PsycINFO. Each original paper was assessed by all authors using the Critical Appraisal Skills Program instrument for qualitative research. We included 22 studies, all of which provided direct patient quotes. Results: The overarching theme was identified as: The patient experience when existence itself is at stake. We constructed an organizing framework for analysis using the main perspectives represented in the included studies: body, mind, relationships, and ICU-environment. Final analysis and interpretation resulted in the unfolding of four themes: existing in liminality, existing in unboundedness, existing in mystery, and existing on the threshold. Conclusions: Our main finding was that human suffering during intensive care is still evident although sedation is lighter and the environment is more humane. Our interpretation suggested that patients with life-threatening illness descend into a liminal state, where they face the choice of life or death. Caring nurses and family members play an important role in assisting the patient to transition back to life. (C) 2015 Elsevier Ltd. All rights reserved.
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| 13. |
- Hagiwara, Magnus, et al.
(författare)
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The effect of a Computerised Decision Support System (CDSS) on compliance with the prehospital assessment process: results of an interrupted time-series study
- 2014
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Ingår i: BMC Medical Informatics and Decision Making. - : Springer Science and Business Media LLC. - 1472-6947. ; 14:1
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Tidskriftsartikel (refereegranskat)abstract
- Background: Errors in the decision-making process are probably the main threat to patient safety in the prehospital setting. The reason can be the change of focus in prehospital care from the traditional "scoop and run" practice to a more complex assessment and this new focus imposes real demands on clinical judgment. The use of Clinical Guidelines (CG) is a common strategy for cognitively supporting the prehospital providers. However, there are studies that suggest that the compliance with CG in some cases is low in the prehospital setting. One possible way to increase compliance with guidelines could be to introduce guidelines in a Computerized Decision Support System (CDSS). There is limited evidence relating to the effect of CDSS in a prehospital setting. The present study aimed to evaluate the effect of CDSS on compliance with the basic assessment process described in the prehospital CG and the effect of On Scene Time (OST). Methods: In this time-series study, data from prehospital medical records were collected on a weekly basis during the study period. Medical records were rated with the guidance of a rating protocol and data on OST were collected. The difference between baseline and the intervention period was assessed by a segmented regression. Results: In this study, 371 patients were included. Compliance with the assessment process described in the prehospital CG was stable during the baseline period. Following the introduction of the CDSS, compliance rose significantly. The post-intervention slope was stable. The CDSS had no significant effect on OST. Conclusions: The use of CDSS in prehospital care has the ability to increase compliance with the assessment process of patients with a medical emergency. This study was unable to demonstrate any effects of OST.
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| 19. |
- Magnusson, B.M., et al.
(författare)
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Intra- and inter-individual variability in the development of erythema after application of methyl nicotinate evaluated by polarization spectroscopy imaging
- 2010
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Ingår i: JOURNAL OF PHARMACY AND PHARMACOLOGY, vol 62, issue 6, pp 801-801, ISSN: 002-3573. - : Pharmaceutical Press. ; , s. 801-801
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Konferensbidrag (refereegranskat)abstract
- The concept that the time to onset of erythema after the application of the rubefacient and urticant substance methyl nicotinate (MN) indicates skin barrier competence was introduced 30 years ago. MN produces a dose-dependent erythema on topical application to intact skin, the nature of which is known to be fast moving (in the order of minutes) and variable. Using tissue viability imaging (TiVi) the time course and degree of the reaction can be conveniently followed and analysed. Inter-individual variability can be quite marked but intra-individual variability is less pronounced. At the upper end of provocation (higher doses, more sensitive individuals) urtication can occur, which decreases blood flow by increasing pressure on and thus emptying capillaries. The TiVi system can quantitate urtication and inherent (blanched) skin colour. The utility of MN application in the study of individual barrier function and microvascular reactivity is increased by the use of the TiVi system for collection and analysis of data.
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| 20. |
- Muller, David A., et al.
(författare)
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Innate local response and tissue recovery following application of high density microarray patches to human skin
- 2020
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Ingår i: Scientific Reports. - : NATURE RESEARCH. - 2045-2322. ; 10:1
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Tidskriftsartikel (refereegranskat)abstract
- The development of microarray patches for vaccine application has the potential to revolutionise vaccine delivery. Microarray patches (MAP) reduce risks of needle stick injury, do not require reconstitution and have the potential to enhance immune responses using a fractional vaccine dose. To date, the majority of research has focused on vaccine delivery with little characterisation of local skin response and recovery. Here we study in detail the immediate local skin response and recovery of the skin post high density MAP application in 12 individuals receiving 3 MAPs randomly assigned to the forearm and upper arm. Responses were characterised by clinical scoring, dermatoscopy, evaporimetry and tissue viability imaging (TiVi). MAP application resulted in punctures in the epidermis, a significant transepidermal water loss (TEWL), the peak TEWL being concomitant with peak erythema responses visualised by TiVi. TEWL and TiVi responses reduced over time, with TEWL returning to baseline by 48 h and erythema fading over the course of a 7 day period. As MAPs for vaccination move into larger clinical studies more variation of individual subject phenotypic or disease propensity will be encountered which will require consideration both in regard to reliability of dose delivery and degree of inherent skin response.
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| 21. |
- Nilsson, Gert, 1947-, et al.
(författare)
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Assessment of tissue viability by polarization spectroscopy
- 2008
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Ingår i: Opto-Electronics Review. - : SpringerLink. - 1230-3402 .- 1896-3757. ; 16:3, s. 309-313
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Tidskriftsartikel (refereegranskat)abstract
- A new and versatile method for tissue viability imaging based on polarization spectroscopy of blood in superficial tissue structures such as the skin is presented in this paper. Linearly polarized light in the visible wavelength region is partly reflected directly by the skin surface and partly diffusely backscattered from the dermal tissue matrix. Most of the directly reflected light preserves its polarization state while the light returning from the deeper tissue layers is depolarized. By the use of a polarization filter positioned in front of a sensitive CCD-array, the light directly reflected from the tissue surface is blocked, while the depolarized light returning from the deeper tissue layers reaches the detector array. By separating the colour planes of the detected image, spectroscopic information about the amount of red blood cells (RBCs) in the microvascular network of the tissue under investigation can be derived. A theory that utilizes the differences in light absorption of RBCs and bloodless tissue in the red and green wavelength region forms the basis of an algorithm for displaying a colour coded map of the RBC distribution in a tissue. Using a fluid model, a linear relationship (cc. = 0.99) between RBC concentration and the output signal was demonstrated within the physiological range 0–4%. In-vivo evaluation using transepidermal application of acetylcholine by the way of iontophoresis displayed the heterogeneity pattern of the vasodilatation produced by the vasoactive agent. Applications of this novel technology are likely to be found in drug and skin care product development as well as in the assessment of skin irritation and tissue repair processes and even ultimately in a clinic case situation.
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| 22. |
- Zhou, Lihua, et al.
(författare)
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Factors associated with posttraumatic growth among spouses of women diagnosed with gynaecological cancer: A cross-sectional study
- 2023
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Ingår i: Nursing Open. - : Wiley. - 2054-1058. ; 10:2, s. 630-640
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Tidskriftsartikel (refereegranskat)abstract
- Aim:The aim of this study was to explore the factors that are associated with posttraumatic growth among spouses of women diagnosed with gynaecological cancer. Design:A cross-sectional descriptive study. Methods:A convenience sample of 312 spouses of women diagnosed with gynaecological cancer was recruited from two comprehensive hospitals in China, from March 2018 to March 2020. Demographic characteristics, cancer-related characteristics, posttraumatic growth, perceived social support and coping were assessed using self-reported questionnaires. Descriptive statistics and multiple linear regression analysis were performed. The methods were guided by the STROBE checklist. Results:The mean score of posttraumatic growth was 46.7 (standard deviation = 16.7). The associated factors of posttraumatic growth were spouses' age, perceived social support, problem-focused coping, dysfunctional coping (e.g. denial) and cancer treatment received by partners, which accounted for 34% of total posttraumatic growth score. Patient or public contribution:All participants contributed to the conducting of this study by completing self-reported questionnaires.
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