| 1. |
- Bolin, Kristian, et al.
(författare)
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The cost-effectiveness of growth hormone replacement therapy (Genotropin®) in hypopituitary adults in Sweden
- 2013
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Ingår i: Cost Effectiveness and Resource Allocation. - : Springer Science and Business Media LLC. - 1478-7547. ; 11:1
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Tidskriftsartikel (refereegranskat)abstract
- Background: To evaluate the cost-effectiveness of growth hormone (GH) treatment (Genotropin®) compared with no GH treatment in adults with GH deficiency in a Swedish societal setting.Methods: A Markov-type cost-utility simulation model was constructed and used to simulate, for men and women, morbidity and mortality for GH-treated and -untreated individuals over a 20-year period. The calculations were performed using current available prices concerning morbidity-related healthcare costs and costs for Genotropin®. All costs and treatment effects were discounted at 3%. Costs were expressed in Euro (1€ = 9.03 SEK). GH-treated Swedish patients (n = 434) were identified from the KIMS database (Pfizer International Metabolic Database) and untreated patients (n = 2135) from the Swedish Cancer Registry and the Hospital Discharge Registry.Results: The results are reported as incremental cost per quality-adjusted life year (QALY) gained, including both direct and indirect costs for GH-treated versus untreated patients. The weighted sum of all subgroup incremental cost per QALY was €15,975 and €20,241 for men and women, respectively. Including indirect cost resulted in lower cost per QALY gained: €11,173 and €10,753 for men and women, respectively. Key drivers of the results were improvement in quality of life, increased survival, and intervention cost.Conclusions: The incremental cost per QALY gained is moderate when compared with informal thresholds applied in Sweden. The simulations suggest that GH-treatment is cost-effective for both men and women at the €55,371 (SEK 500,000 - the informal Swedish cost-effectiveness threshold) per QALY threshold. © 2013 Bolin et al.; licensee BioMed Central Ltd.
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| 2. |
- Brue, Thierry, et al.
(författare)
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Diabetes in patients with acromegaly treated with pegvisomant : observations from acrostudy
- 2019
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Ingår i: Endocrine. - : SPRINGER. - 1355-008X .- 1559-0100. ; 63:3, s. 563-572
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Tidskriftsartikel (refereegranskat)abstract
- PurposeTo explore the effects of pegvisomant (PEGV) on glucose metabolism in patients with acromegaly within ACROSTUDY, an international, observational, prospective safety surveillance study.MethodsPatients were retrospectively divided into two cohorts, with (DM group) or without diabetes mellitus (no-DM). Parameters of glucose metabolism and IGF-I values were analyzed yearly both cross-sectionally for 4 years (yrs) and longitudinally at 1 and 4-5yrs of PEGV treatment.ResultsAmong 1762 patients, 510 (28.9%) had DM before PEGV start. At cross-sectional analyses, in the DM group mean blood glucose was 140.058.7mg/dl at baseline, 116.4 +/- 44.8mg/dl at year 1 and 120.0 +/- 44.3mg/dl at yr 4. Mean HbA1c was 6.6 +/- 1.2 % at yr 1 vs. 7.0 +/- 1.4 % at baseline. HbA1c was above 6.5% in 61.9% at baseline and ranged from 45.4 to 53.8% at subsequent yearly time points. At the 4-yr longitudinal analysis, in the DM group (n=109), mean blood glucose decreased by 20.2mg/dl at yr 4, mean HbA1c was 7.0 +/- 1.5% at baseline vs. 6.8 +/- 1.4%. Patients achieved IGF-I normalization in 52.1% and 57.4% of cases in the DM and no-DM groups, respectively at 1 year. The mean daily PEGV dose (mg/day) was higher in the DM group (18.2 vs. 15.3) while the absolute change of IGF-I values from baseline was similar in both groups. PEGV was well tolerated in both groups without any unexpected AEs.Conclusions p id=Par4 Patients with DM had a moderate decrease in mean fasting glucose values during PEGV treatment.
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| 3. |
- Freda, Pamela U, et al.
(författare)
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Long-Term Treatment with Pegvisomant as Monotherapy in Patients with Acromegaly : Experience from Acrostudy
- 2015
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Ingår i: Endocrine Practice. - 1530-891X .- 1934-2403. ; 21:3, s. 264-274
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Tidskriftsartikel (refereegranskat)abstract
- Objective: To evaluate use of pegvisomant, a GH receptor antagonist, as monotherapy in ACROSTUDY, a global safety surveillance study set in 14 countries (373 sites).Methods: A descriptive analysis of safety, magnetic resonance imaging (MRI) reading and treatment outcomes in 710 subjects who received at least one pegvisomant dose as monotherapy during and up to 5 years follow-up in ACROSTUDY.Results: Subjects received 5.4 yr. (mean) of pegvisomant and were followed in ACROSTUDY 3.8 yr. (mean). A total of 1255 adverse events were reported in 345 subjects (48.6%). Serious adverse events were reported in 133 (18.7%) subjects including 22 deaths, none of which were attributed to pegvisomant use. Of 670 (94%) subjects with at least one liver function test reported in ACROSTUDY, 8 (1.2%) had reported increases in transaminases > 3X ULN. No liver failure was reported. Based on central MRI reading, 12 of 542 subjects (2.2%) had a confirmed increase or increase/decrease in tumor size. Injection-site reactions were reported in 2.3%. At 5 years of therapy, IGF-1 level was reported normal in 67.5% (mean dose 17.2 mg/day) and elevated in 29.9% (mean dose 19.8 mg/day). Subjects on 20 mg per day or more rose from 36% at 3 years to 41% at 5 years of therapy.Conclusions: ACROSTUDY data indicate that pegvisomant used as sole medical therapy is safe and effective medical treatment for acromegaly. The reported low incidence of pituitary tumor size increase and liver enzyme elevations are reassuring and support the positive benefit-risk of pegvisomant therapy.
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| 4. |
- Kołtowska-Häggström, Maria, 1957-
(författare)
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Quality of Life in Adult Patients with Growth Hormone Deficiency : Bridging the gap between clinical evaluation and health economic assessment
- 2007
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Doktorsavhandling (övrigt vetenskapligt/konstnärligt)abstract
- The goals of this thesis are to evaluate quality of life (QoL) in adult patients with growth hormone deficiency (GHD) in relation to population normative data, to construct a preference-weighted index (utility) from a disease-specific QoL measure and to assess it in a clinical context.The study included samples from the general population and patients with GHD from four European populations: England & Wales, the Netherlands, Spain and Sweden. The country-specific patient cohorts were retrieved from KIMS (Pfizer International Metabolic Database). A questionnaire was developed that contained items from existing QoL questionnaires including, among others, Quality of Life Assessment in Growth Hormone Deficiency in Adults (QoL-AGHDA) and the EQ-5D. The QoL-AGHDA is a disease-specific measure for use in adults with GHD. The EQ-5D is a generic instrument which describes health states for which country-specific preference-based weights are available. Thus, it was possible to generate preference-weighted indices (utilities) based on data generated by both instruments. This thesis reports QoL-AGHDA normative values for the populations of England & Wales, the Netherlands, Spain and Sweden, and confirms the extent of QoL impairment in patients with GHD in comparison with the general population. Long-term GH replacement resulted in sustained improvements in overall QoL towards normative country-specific values, as well in most of the dimensions that were impaired before treatment. For use in health economic evaluations, models for generating utilities (QoL-AGHDAutility) from QoL-AGHDA were developed. It is believed that these models may facilitate medical decision making, given that they provide a tool for obtaining utilities in the absence of directly collected preference-weighted indices.QoL-AGHDAutility effectively monitored treatment effects in patients with GHD. Moreover, this study confirmed a QoL-AGHDAutility deficit before treatment and a gain after starting GH replacement. The novel aspect of the present approach was to apply preference-weighted indices derived from a disease-specific measure to assess QoL in the clinical context, together with patient demographic and clinical characteristics. The robustness of this analysis is reinforced by the fact that utilities in both general and patient populations were generated using the same methodology.
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| 5. |
- Koltowski, Lukasz, et al.
(författare)
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Quality of Life in Patients With ST-Segment Elevation Myocardial Infarction Undergoing Percutaneous Coronary Intervention-Radial Versus Femoral Access (from the OCEAN RACE Trial)
- 2014
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Ingår i: American Journal of Cardiology. - : Elsevier BV. - 0002-9149 .- 1879-1913. ; 114:4, s. 516-521
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Tidskriftsartikel (refereegranskat)abstract
- Numerous studies have compared transradial (TR) versus transfemoral (TF) access for percutaneous coronary intervention (PCI) in patients with acute ST-segment elevation myocardial infarction. These studies have focused on clinical efficacy and safety; yet little is known about the effect of the vessel access on the health-related quality of life (HRQoL). In the present study, patients were randomly assigned to TR (n = 52) or TF (n = 51) access groups. Generic (EQ-5D-3L) and cardiac-specific (Quality of Life Index and MacNew) tools were used to assess HRQoL before PCI and 2 hours and 4 days after PCI. Baseline HRQoL was comparable in both groups and improved after PCI. The mean +/- SD EQ-5D-3L health utility score 2 hours after PCI was 0.46 +/- 0.291 and was higher in the TR group (TR: 0.60 +/- 0.299 versus TF: 0.32 +/- 0.283, p <0.001). Patients in the TR group reported fewer problems with mobility (TR: 71.7% vs TF: 94.4%, p <0.01) and self-care (TR: 62.5% vs TF: 97.2%, p <0.001). At day 4, fewer patients reported problems with anxiety and/or depression in the TR group than in the TF group (TR: 42.9% vs TF: 75.0%, p <0.001); no differences between groups in other measures were observed (Quality of Life Index and MacNew). The N-terminal of the prohormone brain natriuretic peptide levels were inversely correlated with EQ-5D-3L visual analog scale (r = -0.348, p <0.05) and EQ-5D-3L health utility score (r = -0.322, p <0.05). There was a correlation between in-hospital mortality and 2 MacNew domains: physical (r = -0.329, p <0.05) and emotional (r = -0.374, p <0.01). In conclusion, radial access should be the preferred approach in patients with ST-segment elevation myocardial infarction undergoing PCI when considering HRQoL. Radial access is associated with fewer problems with mobility and self-care and better psychological outcome after PCI.
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| 6. |
- Kreitschmann-Andermahr, Ilonka, et al.
(författare)
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Diagnosis and management of acromegaly : the patient's perspective
- 2016
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Ingår i: Pituitary. - : Springer Science and Business Media LLC. - 1386-341X .- 1573-7403. ; 19:3, s. 268-276
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Tidskriftsartikel (refereegranskat)abstract
- PURPOSE:Early diagnosis is a success factor for the prevention of long-term comorbidity and premature death in patients with acromegaly, but large-scale data on the diagnostic process and disease management are scarce. Therefore, we aimed to evaluate the diagnostic process, implementation of treatment and changes in life situation in patients with acromegaly, focusing on sex-specific differences.METHODS:Non-interventional patient-reported outcome study. 165 patients with clinically and biochemically proven acromegaly were questioned about the diagnostic process and utilization of health care by means of a self-developed standardized postal survey including questions on acromegaly symptoms experienced before diagnosis, number and specialty of consulted doctors, time to diagnosis and aftercare.RESULTS:The diagnostic process took 2.9 (SD 4.53) years, during which 3.4 (SD 2.99) physicians were consulted. Women waited longer [4.1 (SD 5.53) years] than men [1.6 (SD 2.69) years; p = 0.001] for the correct diagnosis, and consulted more doctors in the process [4.0 (SD 2.99) vs. 2.7 (SD 2.84) doctors, p < 0.001, respectively]. In 48.5 % of patients, acromegaly was diagnosed by an endocrinologist (men: 45.1 %; women: 52.4 %). Overall disease duration from symptom onset until last surgery was 5.5 (SD 6.85) years, with no sex differences. A change in employment status was the most commonly reported event after diagnosis and a quarter of the patients stated that the illness had changed their lives.CONCLUSIONS:Our findings confirm the urgent need to increase awareness of the clinical manifestation of acromegaly to facilitate an earlier diagnosis of the disease and to provide diagnostic equality across the sexes.
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| 7. |
- Kreitschmann-Andermahr, Ilonka, et al.
(författare)
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Support Needs of Patients with Cushing's Disease and Cushing's Syndrome : Results of a Survey Conducted in Germany and the USA
- 2018
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Ingår i: International Journal of Endocrinology. - : Hindawi Publishing Corporation. - 1687-8337 .- 1687-8345.
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Tidskriftsartikel (refereegranskat)abstract
- Background. Cushing's disease (CD) and Cushing's syndrome (CS) are chronic illnesses, characterized by symptoms of prolonged hypercortisolism, which often changes to hypocortisolism after successful treatment. In view of the high disease burden of CD/CS patients and long-term impaired quality of life, the present survey was conducted to gain information about subjective illness distress and patients' specific needs in terms of supportive measures beyond medical interventions. Patients and Methods. Cross-sectional questionnaire study including patients with CD treated in 2 German neurosurgical tertiary referral centers and CD/CS patient members of a US-based patient support group completed a survey inquiring about disease burden, coping strategies, and support needs. Additionally, the degree of interest in different offers, e.g., internet-based programs and seminars, was assessed. Results. 84 US and 71 German patients answered the questionnaire. Patients in both countries indicated to suffer from Cushing-related symptoms, reduced performance, and psychological problems. 48.8% US patients and 44.4% German patients stated that good medical care and competent doctors helped them the most in coping with the illness. US patients were more interested in support groups (p = 0.035) and in courses on illness coping (p = 0.008) than the German patients, who stated to prefer brochures (p = 0.001). 89.3% of US patients would attend internet-based programs compared to 75.4% of German patients (p = 0.040). There were no differences between groups for the preferred duration of and the willingness to pay for such a program, but US patients would travel longer distances to attend a support meeting (p = 0.027). Conclusion. Patients in both countries need skilled physicians and long-term medical care in dealing with the effects of CD/CS, whereas other support needs differ between patients of both countries. The latter implies that not only disease-specific but also culture-specific training programs would need to be considered to satisfy the needs of patients in different countries.
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| 8. |
- Merks, Piotr, et al.
(författare)
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Comparison of factors influencing patient choice of community pharmacy in Poland and in the UK, and identification of components of pharmaceutical care
- 2014
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Ingår i: Patient Preference and Adherence. - 1177-889X. ; 8, s. 715-726
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Tidskriftsartikel (refereegranskat)abstract
- Background: Several factors, which are components of pharmaceutical care, can influence a patient's choice of a community pharmacy store and contribute to frequent visits to the same pharmacy. Objectives: To compare factors that influence a patient's choice of pharmacy in Poland and in the UK, to identify which of them are components of pharmaceutical care, and to relate them to patient loyalty to the same pharmacy. Methods: A self-administered, anonymous questionnaire was distributed to clients visiting pharmacies in Poland and the UK January- August2011. Comparisons were performed using chi-square tests and logistic regression. All statistical analyses were performed using SPSS 20.0. Results: The response rate was 55.6% (n= 417/750; 36 pharmacies) and 54.0% (n= 405/750; 56 pharmacies) in Poland and in the UK, respectively. The most frequently reported factors, as defined by a percentage of responders, were in Poland: 1) location ( 84%); 2) professional and high-quality of service (82%); 3) good price of medicines (78%); and 4) promotions on medicines (66%). In the UK, the most commonly reported factors were: 1) professional and high quality of service (90%); 2) location (89%); 3) good advice received from the pharmacist (86%); and 4) option of discussing and consulting all health issues in a consultation room (80%). Good advice and an option of discussing personal concerns with a pharmacist are components of pharmaceutical care. Thirty-eight percent of patients in Poland and 61% in the UK declared visiting the same pharmacy. Conclusion: Components of pharmaceutical care are important factors influencing the patient's choice of pharmacy in the UK and, to a lesser degree, in Poland. Additionally, more patients in the UK than in Poland are committed to a single pharmacy. Therefore, implementing the full pharmaceutical care in Poland may contribute to an increase in patient loyalty and thus strengthen competitiveness of pharmacy businesses.
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| 9. |
- Mitra, M. Tanya, et al.
(författare)
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Social, educational and vocational outcomes in patients with childhood-onset and young-adult-onset growth hormone deficiency
- 2017
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Ingår i: Clinical Endocrinology. - : Wiley. - 0300-0664 .- 1365-2265. ; 86:4, s. 526-533
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Tidskriftsartikel (refereegranskat)abstract
- Objective Hypopituitarism diagnosed in childhood, adolescence and young adulthood has the potential to affect growth and somatic development. Less is known about the impact of such a diagnosis on other aspects of development. Design An analysis of the KIMS database (Pfizer International Metabolic Database) was performed to explore social, educational and vocational outcomes of adult patients diagnosed in childhood, adolescence and young adulthood compared with adult-onset controls. Patients A total of 2952 adult patients diagnosed with hypothalamic pituitary conditions before the age of 25 were divided into two groups: childhood-onset [<16 years (CO)] (n = 1782) and young-adult-onset [16 to <25 years (YAO)] (n = 1170). A total of 1617 adult patients diagnosed with a nonfunctioning pituitary adenoma at the age of 25 or older formed the adult-onset control group (AO). Measurements KIMS Patient Life Situation Form which provided information on social, educational and vocational outcomes. Results Compared with the AO control group, CO and YAO patients were between 45 and 80 times more likely to live with their parents in adulthood; CO and YAO patients were also less likely to live in partnership and to have children. The impact on educational and vocational outcomes was less marked than on social outcomes with no significant differences compared with the AO control group. Educational and vocational outcomes showed the lowest level in male and female CO and YAO patients who had been previously diagnosed with a brain tumour. ConclusionsSocial outcomes were more affected than educational and vocational outcomes. Although CO patients are more adversely affected, YAO patients were also failing to achieve social milestones. This has consequences for the delivery of endocrine care in both paediatric and adult services.
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| 10. |
- Ragnarsson, Oskar, 1971, et al.
(författare)
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The relationship between glucocorticoid replacement and quality of life in 2737 hypopituitary patients.
- 2014
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Ingår i: European journal of endocrinology / European Federation of Endocrine Societies. - 1479-683X .- 0804-4643. ; 171:5, s. 571-9
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Tidskriftsartikel (refereegranskat)abstract
- Quality of life (QoL) is impaired in hypopituitary patients and patients with primary adrenal insufficiency. The aim of this study was to analyse the impact of glucocorticoid (GC) replacement on QoL. The main hypothesis was that ACTH-insufficient patients experience a dose-dependent deterioration in QoL.
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