| 1. |
- Carlsson, Per, 1951-, et al.
(författare)
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Prioritering av läkemedel för behandling av patienter med sällsynta sjukdomar
- 2014
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Ingår i: Läkemedel för djur, maskinell dos och sällsynta tillstånd. - Stockholm : Fritzes. - 9789138242070 ; , s. 639-699
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Bokkapitel (övrigt vetenskapligt/konstnärligt)abstract
- I vilken utsträckning kan särskilda hänsyn tas vid prioritering av särläkemedel och läkemedel för behandling av sällsynta tillstånd? Bör man i vissa speciella situationer kunna acceptera sämre kostnadseffektivitet och lägre krav på vetenskapligt underlag? Om så, vad är det för villkor/kriterier som då bör vara uppfyllda? Hur stämmer ett sådant undantag med den etiska plattformen för prioriteringar? Finns det andra argument som talar för eller mot en särbehandling av sällsynta tillstånd? Detta är frågor som vi diskuterar i denna rapport som tagits fram på uppdrag av Läkemedels- och apoteksutredningen.I rapporten konstaterar vi att särläkemedel är en relativt heterogen grupp av läkemedel och att det finns andra läkemedel med motsvarande egenskaper som inte ansökt eller kunnat ansöka om status som särläkemedel. Ur prioriteringssynpunkt är det därför mer relevant att fokusera på alla typer av läkemedel, som används för sällsynta tillstånd, när vi diskuterar vilka principer för prioriteringar som bör gälla.Utifrån litteratur och erfarenheter i andra länder, som formulerat kriterier för en särskild hantering av läkemedel vid sällsynta sjukdomar, drar vi den preliminära slutsatsen att samhället bör kunna betala mer per hälsovinst (kostnad per kvalitetsjusterat levnadsår (QALY)) och acceptera lägre krav på vetenskapligt underlag vid prioritering av läkemedel för behandling av sällsynta sjukdomar om samtliga följande villkor är uppfyllda:att behandlingen har en hög kostnad per hälsovinst som en konsekvens av att den omfattar endast få patienter,att det rör sig om ett tillstånd med mycket stor svårighetsgrad,att det behandlingsalternativ som övervägs på goda grunder ska antas ha en väsentlig effekt,att det inte finns någon alternativ behandling med en väsentlig effekt som förväntas förebygga, bota, fördröja försämring eller lindra det aktuella tillståndet.Om dessa kriterier anses uppfyllda kan läkemedlet bedömas i relation till ett förhöjt tröskelvärde för kostnadseffektivitet. Hur högt detta värde kan vara för att anses rimligt tar vi inte ställning till i rapporten, men en viktig ståndpunkt är att det måste finnas en övre gräns för hur hög kostnad per QALY samhället kan acceptera. Detta bör gälla även om ovanstående kriterier är uppfyllda, annars riskerar det uppstå oacceptabla undanträngningseffekter av andra prioriterade åtgärder. Även lägre krav på vetenskapligt underlag skakunna accepteras....
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| 2. |
- Carlsson, Per, et al.
(författare)
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Prioritering och finansiering av läkemedel för behandling av patienter med sällsynta sjukdomar
- 2012
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Rapport (övrigt vetenskapligt/konstnärligt)abstract
- Den pågående Läkemedels- och apoteksutredningen har i ett tilläggsdirektiv fått i uppdrag att analysera behovet av särskilda lösningar vid beslut om subventionering för särläkemedel. Ett särläkemedel är ett läkemedel som uppfyller vissa villkor och därmed omfattas av ett särskilda stimulansåtgärder innan godkännande av läkemedelsmyndighet samt möjlighet, men inte rätt, till ensamrätt på marknaden under 10 år. Till villkoren hör bl.a. att läkemedlet är avsett för att diagnostisera, förebygga eller behandla livshotande tillstånd eller tillstånd med kronisk funktionsnedsättning och som högst 5 av 10 000 personer i gemenskapen lider av vid ansökningstillfället.
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| 3. |
- Ekedahl, Anders, 1949-, et al.
(författare)
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Patients' information on the prescribed current treatment
- 2012
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Ingår i: Journal of Pharmaceutical Health Services Research. - : John Wiley & Sons. - 1759-8885 .- 1759-8893. ; 3:2, s. 79-84
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Tidskriftsartikel (refereegranskat)abstract
- Objective To investigate how many patients, with five or more prescriptions in the Swedish National Prescription Repository (NPR) had received (1) the Medication List (ML) from the electronic medical record and (2) the Prescription List (PL) on prescriptions stored in the NPR and how these lists were used.Methods Patients> 18 years of age, with five or more prescriptions stored electronically in the NPR, collecting medicines on prescription for themselves at nine pharmacies all around Sweden were interviewed about their knowledge of, and if they had received, the ML, the PL and/or the pharmacy record (PR) on medicines purchased on prescription during the last 15 months, and what documentation they used to track their current, prescribed treatment.Key findings A total of 777 consensual patients (485 women) were interviewed. Of these, 32% were aware of the ML, but only 13% had received the MLat least once a year, and only 9% used the MLto track their current, prescribed medicines. Virtually all patients (99%) had received the PL at every pharmacy dispensing and 68% said they used the PL as documentation on their current, prescribed medicines. 52% used the dispensed packs to track their medication and 10% said they used a handwritten list. Only few patients were aware of the PR.Conclusion Our study shows that patients’ access to information on their prescribed medicines is not in accordance with intentions in health care. The risk for errors in medicine use at home is substantial.
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| 4. |
- Fanidi, Anouar, et al.
(författare)
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Circulating Folate, Vitamin B6, and Methionine in Relation to Lung Cancer Risk in the Lung Cancer Cohort Consortium (LC3)
- 2018
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Ingår i: Journal of the National Cancer Institute. - : Oxford University Press. - 0027-8874 .- 1460-2105. ; 110:1
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Tidskriftsartikel (refereegranskat)abstract
- Background: Circulating concentrations of B vitamins and factors related to one-carbon metabolism have been found to be strongly inversely associated with lung cancer risk in the European Prospective Investigation into Cancer and Nutrition (EPIC) study. The extent to which these associations are present in other study populations is unknown.Methods: Within 20 prospective cohorts from the National Cancer Institute Cohort Consortium, a nested case-control study was designed including 5364 incident lung cancer case patients and 5364 control subjects who were individually matched to case patients by age, sex, cohort, and smoking status. Centralized biochemical analyses were performed to measure circulating concentrations of vitamin B6, folate, and methionine, as well as cotinine as an indicator of recent tobacco exposure. The association between these biomarkers and lung cancer risk was evaluated using conditional logistic regression models.Results: Participants with higher circulating concentrations of vitamin B6 and folate had a modestly decreased risk of lung cancer risk overall, the odds ratios when comparing the top and bottom fourths (OR 4vs1 ) being 0.88 (95% confidence interval [CI] = 0.78 to 1.00) and 0.86 (95% CI = 0.74 to 0.99), respectively. We found stronger associations among men (vitamin B6: OR 4vs1 = 0.74, 95% CI = 0.62 to 0.89; folate: OR 4vs1 = 0.75, 95% CI = 0.61 to 0.93) and ever smokers (vitamin B6: OR 4vs1 = 0.78, 95% CI = 0.67 to 0.91; folate: OR 4vs1 = 0.87, 95% CI = 0.73 to 1.03). We further noted that the association of folate was restricted to Europe/Australia and Asia, whereas no clear association was observed for the United States. Circulating concentrations of methionine were not associated with lung cancer risk overall or in important subgroups.Conclusions: Although confounding by tobacco exposure or reverse causation cannot be ruled out, these study results are compatible with a small decrease in lung cancer risk in ever smokers who avoid low concentrations of circulating folate and vitamin B6.
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| 5. |
- Fanidi, Anouar, et al.
(författare)
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Is high vitamin B12 status a cause of lung cancer?
- 2019
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Ingår i: International Journal of Cancer. - : John Wiley & Sons. - 0020-7136 .- 1097-0215. ; 145:6, s. 1499-1503
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Tidskriftsartikel (refereegranskat)abstract
- Vitamin B supplementation can have side effects for human health, including cancer risk. We aimed to elucidate the role of vitamin B12 in lung cancer etiology via direct measurements of pre‐diagnostic circulating vitamin B12 concentrations in a nested case–control study, complemented with a Mendelian randomization (MR) approach in an independent case–control sample. We used pre‐diagnostic biomarker data from 5183 case–control pairs nested within 20 prospective cohorts, and genetic data from 29,266 cases and 56,450 controls. Exposures included directly measured circulating vitamin B12 in pre‐diagnostic blood samples from the nested case–control study, and 8 single nucleotide polymorphisms associated with vitamin B12 concentrations in the MR study. Our main outcome of interest was increased risk for lung cancer, overall and by histological subtype, per increase in circulating vitamin B12 concentrations. We found circulating vitamin B12 to be positively associated with overall lung cancer risk in a dose response fashion (odds ratio for a doubling in B12 [ORlog2B12] = 1.15, 95% confidence interval (95%CI) = 1.06–1.25). The MR analysis based on 8 genetic variants also indicated that genetically determined higher vitamin B12 concentrations were positively associated with overall lung cancer risk (OR per 150 pmol/L standard deviation increase in B12 [ORSD] = 1.08, 95%CI = 1.00–1.16). Considering the consistency of these two independent and complementary analyses, these findings support the hypothesis that high vitamin B12 status increases the risk of lung cancer.
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| 6. |
- Haghighi, Mona, et al.
(författare)
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A Comparison of Rule-based Analysis with Regression Methods in Understanding the Risk Factors for Study Withdrawal in a Pediatric Study
- 2016
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Ingår i: Scientific Reports. - : Springer Science and Business Media LLC. - 2045-2322. ; 6
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Tidskriftsartikel (refereegranskat)abstract
- Regression models are extensively used in many epidemiological studies to understand the linkage between specific outcomes of interest and their risk factors. However, regression models in general examine the average effects of the risk factors and ignore subgroups with different risk profiles. As a result, interventions are often geared towards the average member of the population, without consideration of the special health needs of different subgroups within the population. This paper demonstrates the value of using rule-based analysis methods that can identify subgroups with heterogeneous risk profiles in a population without imposing assumptions on the subgroups or method. The rules define the risk pattern of subsets of individuals by not only considering the interactions between the risk factors but also their ranges. We compared the rule-based analysis results with the results from a logistic regression model in The Environmental Determinants of Diabetes in the Young (TEDDY) study. Both methods detected a similar suite of risk factors, but the rule-based analysis was superior at detecting multiple interactions between the risk factors that characterize the subgroups. A further investigation of the particular characteristics of each subgroup may detect the special health needs of the subgroup and lead to tailored interventions.
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| 7. |
- Hallberg, Inger, 1956, et al.
(författare)
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Phases in development of an interactive mobile phone-based system to support self-management of hypertension
- 2014
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Ingår i: Integrated blood pressure control. - : Dove Medical Press. - 1178-7104. ; 7, s. 19-28
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Tidskriftsartikel (refereegranskat)abstract
- Hypertension is a significant risk factor for heart disease and stroke worldwide. Effective treatment regimens exist; however, treatment adherence rates are poor (30%–50%). Improving self-management may be a way to increase adherence to treatment. The purpose of this paper is to describe the phases in the development and preliminary evaluation of an interactive mobile phone-based system aimed at supporting patients in self-managing their hypertension. A person-centered and participatory framework emphasizing patient involvement was used. An interdisciplinary group of researchers, patients with hypertension, and health care professionals who were specialized in hypertension care designed and developed a set of questions and motivational messages for use in an interactive mobile phone-based system. Guided by the US Food and Drug Administration framework for the development of patient-reported outcome measures, the development and evaluation process comprised three major development phases (1, defining; 2, adjusting; 3, confirming the conceptual framework and delivery system) and two evaluation and refinement phases (4, collecting, analyzing, interpreting data; 5, evaluating the self-management system in clinical practice). Evaluation of new mobile health systems in a structured manner is important to understand how various factors affect the development process from both a technical and human perspective. Forthcoming analyses will evaluate the effectiveness and utility of the mobile phone-based system in supporting the self-management of hypertension.
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| 8. |
- Hellqvist, Henrik, et al.
(författare)
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Estimation of aortic stiffness by finger photoplethysmography using enhanced pulse wave analysis and machine learning
- 2024
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Ingår i: Frontiers in Cardiovascular Medicine. - : Frontiers Media SA. - 2297-055X. ; 11
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Tidskriftsartikel (refereegranskat)abstract
- Introduction: Aortic stiffness plays a critical role in the evolution of cardiovascular diseases, but the assessment requires specialized equipment. Photoplethysmography (PPG) and single-lead electrocardiogram (ECG) are readily available in healthcare and wearable devices. We studied whether a brief PPG registration, alone or in combination with single-lead ECG, could be used to reliably estimate aortic stiffness. Methods: A proof-of-concept study with simultaneous high-resolution index finger recordings of infrared PPG, single-lead ECG, and finger blood pressure (Finapres) was performed in 33 participants [median age 44 (range 21–66) years, 19 men] and repeated within 2 weeks. Carotid–femoral pulse wave velocity (cfPWV; two-site tonometry with SphygmoCor) was used as a reference. A brachial single-cuff oscillometric device assessed aortic pulse wave velocity (aoPWV; Arteriograph) for further comparisons. We extracted 136 established PPG waveform features and engineered 13 new with improved coupling to the finger blood pressure curve. Height-normalized pulse arrival time (NPAT) was derived using ECG. Machine learning methods were used to develop prediction models. Results: The best PPG-based models predicted cfPWV and aoPWV well (root-mean-square errors of 0.70 and 0.52 m/s, respectively), with minor improvements by adding NPAT. Repeatability and agreement were on par with the reference equipment. A new PPG feature, an amplitude ratio from the early phase of the waveform, was most important in modelling, showing strong correlations with cfPWV and aoPWV (r = −0.81 and −0.75, respectively, both P < 0.001). Conclusion: Using new features and machine learning methods, a brief finger PPG registration can estimate aortic stiffness without requiring additional information on age, anthropometry, or blood pressure. Repeatability and agreement were comparable to those obtained using non-invasive reference equipment. Provided further validation, this readily available simple method could improve cardiovascular risk evaluation, treatment, and prognosis.
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| 9. |
- Hillier, Ladeana W, et al.
(författare)
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Sequence and comparative analysis of the chicken genome provide unique perspectives on vertebrate evolution
- 2004
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Ingår i: Nature. - 0028-0836 .- 1476-4687. ; 432:7018, s. 695-716
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Tidskriftsartikel (refereegranskat)abstract
- We present here a draft genome sequence of the red jungle fowl, Gallus gallus. Because the chicken is a modern descendant of the dinosaurs and the first non-mammalian amniote to have its genome sequenced, the draft sequence of its genome--composed of approximately one billion base pairs of sequence and an estimated 20,000-23,000 genes--provides a new perspective on vertebrate genome evolution, while also improving the annotation of mammalian genomes. For example, the evolutionary distance between chicken and human provides high specificity in detecting functional elements, both non-coding and coding. Notably, many conserved non-coding sequences are far from genes and cannot be assigned to defined functional classes. In coding regions the evolutionary dynamics of protein domains and orthologous groups illustrate processes that distinguish the lineages leading to birds and mammals. The distinctive properties of avian microchromosomes, together with the inferred patterns of conserved synteny, provide additional insights into vertebrate chromosome architecture.
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| 10. |
- Jansson, Johan, et al.
(författare)
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Adaptive stabilized finite element framework for simulation of vocal fold turbulent fluid-structure interaction
- 2013
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Ingår i: Proceedings of Meetings on Acoustics. - : Acoustical Society of America (ASA). ; , s. 1-9
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Konferensbidrag (refereegranskat)abstract
- As a step toward building a more complete model of voice production mechanics, we assess the feasibility of a fluid-structure simulation of the vocal fold mechanics in the Unicorn incompressible Unified Continuum framework. The Unicorn framework consists of conservation equations for mass and momentum, a phase function selecting solid or fluid constitutive laws, a convection equation for the phase function and moving mesh methods for tracking the interface, and discretization through an adaptive stabilized finite element method. The framework has been validated for turbulent flow for both low and high Reynolds numbers and has the following features: implicit turbulence modeling (turbulent dissipation only occurs through numerical stabilization), goal-oriented mesh adaptivity, strong, implicit fluid-structure coupling and good scaling on massively parallel computers. We have applied the framework for turbulent fluid-structure interaction simulation of vocal folds, and present initial results. Acoustic quantities have been extracted from the framework in the setting of an investigation of a configuration approximating an exhaust system with turbulent flow around a flexible triangular steel plate in a circular duct. We present some results of the investigation as well as results of the framework applied to other problems.
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