External quality monitoring facilitates improvement in already well-performing stroke units: insights from RES-Q Poland.

INTRODUCTION: The Registry of Stroke Care Quality (RES-Q) is used in Poland for quality monitoring by numerous hospitals participating in the Angels Initiative. Our aim was to assess the degree of improvement in highly stroke-oriented centres that report cases to the RES-Q each year. MATERIAL AND METHODS: This retrospective analysis included Polish stroke units that from January 2017 to December 2020 contributed to the RES-Q at least 25 patients annually. RESULTS: Seventeen out of 180 Polish stroke units reported patients each year (2017, n = 1,691; 2018, n = 2,986; 2019, n = 3,750; 2020, n = 3,975). The percentage of ischaemic stroke patients treated with alteplase remained stable (26%, 29%, 30% and 28%, respectively). The door-to-needle time progressively decreased, from a median 49 minutes to 32 minutes. The percentage of patients treated ≤ 60 minutes and ≤ 45 minutes significantly increased (from 68% to 86% and from 43% to 70%, respectively), with no change observed between 2019 and 2020. Despite a general improvement in dysphagia screening (81%, 91%, 98% and 99%), screening performed within the first 24h from admission became less frequent (78%, 76%, 69% and 65%). In-hospital mortality significantly increased (11%, 11%, 13% and 15%), while the proportion of patients discharged home remained stable. CONCLUSIONS: Quality-oriented projects facilitate the improvement of stroke care, even in centres demonstrating good baseline performance. Polish stroke units that consistently reported cases to the RES-Q demonstrated improvement in terms of door-to- -needle time and dysphagia screening. However, there is still a need to shorten the time to dysphagia screening, and carefully monitor stroke unit mortality following the COVID-19 pandemic.

Seven decades of clinical experience with Wilson's disease: Report from the national reference centre in Poland.

BACKGROUND AND PURPOSE: Wilson's disease (WD) is a rare autosomal recessive disorder causing excessive copper deposition and a spectrum of manifestations, particularly neurological and hepatic symptoms. We analysed the clinical characteristics of patients with WD admitted to the country's only reference centre, which provided long-term care to most adult patients in Poland over seven decades (pre-1959 to 2019). METHODS: Electronic prospective data collection began in the 2000s and, for prior years, medical records were analysed retrospectively. Demographic and clinical characteristics, treatment and outcomes were analysed by decade of diagnosis. Life-years lost were estimated in patients with WD compared with the general population. Kaplan-Meier curves were used for a time-to-death analysis using 2000-2009 as a reference. RESULTS: In total, 929 patients were analysed. The number of patients increased from 21 before 1959 to 315 for 2000 to 2009 period. Mostly males were diagnosed before the 1990s, but the numbers of female patients diagnosed increased thereafter. Initially, most patients presented with neurological manifestations; however, the incidence of hepatic manifestations and asymptomatic presentations increased over time as patients were diagnosed early and consequently were more independent at diagnosis. Fewer Kayser-Fleischer rings were detected recently. Prior to 1970, patients were treated with D-penicillamine (DP); however, since the introduction of zinc, both therapies have been used as often. Since the 1990s, switches between DP and zinc were recorded in 6%-7% of patients. Consistent improvement in survival has been observed over the years. CONCLUSIONS: This is the largest cohort of patients with WD reported in Poland, with the longest follow-up. Earlier diagnosis and prognosis have improved over seven decades.

EQ-5D-Derived Health State Utility Values in Hematologic Malignancies: A Catalog of 796 Utilities Based on a Systematic Review.

OBJECTIVES: We performed a systematic review of health state utility values (HSUVs) obtained using the EQ-5D questionnaire for patients with hematologic malignancies. METHODS: The following databases were searched up to September 2018: MEDLINE, EMBASE, The Cochrane Library, and the EQ-5D publications database on the EuroQol website. Additional references were extracted from reviewed articles. Only studies presenting EQ-Index results were incorporated. In view of the heterogeneity across the included publications, we limited ourselves to a narrative synthesis of original HSUVs found. RESULTS: Fifty-nine studies (described in 63 articles) met the inclusion criteria. Data from 21 635 respondents provided 796 HSUV estimates for hematologic malignancy patients. EQ-Index scores ranged from -0.025 to 0.980. The most represented area was multiple myeloma (4 studies, 11 112 patients, and 249 HSUVs). In clinical areas such as chronic myeloid leukemia, acute myeloid leukemia, chronic lymphocytic leukemia, non-Hodgkin lymphoma, and mantle cell lymphoma, we described over 50 health utilities in each. In contrast, we identified only 13 HSUVs (based on 4 studies and the data of 166 patients) for Hodgkin lymphoma. Areas without EQ-5D-based health utilities comprised: polycythemia vera, primary myelofibrosis, essential thrombocythemia, mastocytosis, myeloid sarcoma, chronic myelomonocytic, eosinophilic leukemia, and neutrophilic leukemia. CONCLUSIONS: There is a wide range of HSUVs available for hematologic cancer patients with different indications. The review provides a catalog of utility values for use in cost-effectiveness models for hematologic malignancies.

Gastropathy in patients with Wilson disease.

Background: Gastrointestinal symptoms are common in patients with Wilson disease (WD) and may be related to the disease itself or to adverse drug reactions (ADRs).Aim: To investigate gastroscopy findings in patients with WD and to analyze the risk of gastropathy in the context of different manifestations and treatments of WD as well as Helicobacter pylori infection status.Methods: This cross-sectional study included patients diagnosed or monitored for WD between 2007 and 2017. All enrolled patients were examined with gastroscopy and checked for infection with a urease test. Based on predominant manifestations, WD was classified as pre-symptomatic, hepatic (only liver symptoms) or neurological. Patients were divided into three treatment groups: untreated, treated with d-penicillamine (DPA) or zinc sulfate therapy.Results: Of 115 patients, 58 were male and the median age was 30 years. Gastropathy was observed in 65.2% of all patients. Factors that increased the risk of gastropathy were zinc sulfate (odds ratio [OR] = 3.01; 95% confidence interval [CI]: 1.12-8.09, p = .03), H. pylori infection (OR = 2.96; 95%CI: 1.34-6.56, p = .01) and neurological manifestations (OR = 2.55; 95%CI: 1.16-5.60, p = .02). In total, 9.6% of patients had gastric or duodenal ulcers and 29.6% had esophageal varices but no difference was seen by treatment status. In multivariate analysis, zinc sulfate remained associated with higher risk of gastropathy compared with no treatment (OR = 4.57; 95%CI: 1.21-17.19; p = .03) and DPA (OR = 6.28; 95%CI: 1.43-27.56; p = .01).Conclusions: Our results show that gastropathy in WD may be influenced by the treatment used.KeypointsIn a retrospective study of 115 patients with Wilson's disease, gastric injury was frequent.Patients receiving zinc sulfate had increased gastropathy risk compared with those receiving no treatment or d-penicillamine.

The cost-effectiveness of food consistency modification with xanthan gum-based Nutilis Clear® in patients with post-stroke dysphagia in Poland.

BACKGROUND: Dysphagia is a well-known stroke complication characterised by difficulty in swallowing. It may affect the majority of stroke patients and increases mortality and morbidity, due to aspiration pneumonia and malnutrition. Food thickening may help patients to feed themselves, and its effectiveness was demonstrated. However, the cost-effectiveness studies are lacking. We evaluate the cost-utility of xanthan gum-based consistency modification therapy (Nutilis Clear®) in adult post-stroke patients from the public payer perspective in Poland. METHODS: Routine clinical practice was used as a comparator, as no alternative specific treatment for dysphagia is available. To verify the robustness of the results against the modelling approach, we built two models: a static (a fixed simple-equations model, 8-week time horizon of dysphagia) and a dynamic one (Markov model, with a possible dysphagia resolution over a 1-year horizon). In both models, the treatment costs, health state utilities, and clinical events (i.e. aspiration, aspiration pneumonia, death) were included. Parameters were estimated jointly for both models, except for the duration of dysphagia and the risk of aspiration pneumonia (specific to the time horizon). We only assumed Nutilis Clear® to prevent aspirations, without affecting dysphagia duration. RESULTS: The average cost of one quality-adjusted life year (i.e. the incremental cost-utility ratios, ICURs) amounted to 21,387 PLN (€1 ≈ 4.5 PLN), and 20,977 PLN in static and dynamic model, respectively; far below the cost-effectiveness threshold in Poland (147,024 PLN). The one-way, scenario, and probabilistic sensitivity analysis confirmed these findings. CONCLUSIONS: Nutilis Clear® is highly cost-effective in Poland from the public payer perspective. Our approach can be used in other countries to study the cost-effectiveness of food thickening in stroke patients.

Fluoxetine for stroke recovery improvement - the doubleblind, randomised placebo-controlled FOCUS-Poland trial.

AIM OF STUDY: The Fluoxetine Or Control Under Supervision (FOCUS)-Poland trial tested in a Polish cohort the hypothesis that fluoxetine improves recovery after stroke. CLINICAL RATIONALE FOR STUDY: Some studies have suggested that fluoxetine may improve functional outcomes after stroke, but these results needed confirmation. Between 2012 and 2014, large clinical trials were initiated by the FOCUS Trial Collaboration. Recently, results from the UK, Sweden, Australia, New Zealand and Vietnam have been published. We here present the results of the FOCUS trial conducted in Poland. MATERIAL AND METHODS: This was a randomised, double-blind, placebo-controlled study based on the FOCUS trial protocol. Patients who had a persisting neurological deficit were randomly assigned 2-15 days after stroke onset to receive for six months either fluoxetine 20 mg/day or a placebo. The primary outcome was functional status measured using the modified Rankin Scale (mRS) at six months after randomisation. Functional status at 12 months was also assessed, as was neurological deficit at six and 12 months. Data was also collected on adverse events. RESULTS: Between 19 December 2014 and 13 March 2018, 30 patients were given fluoxetine and 31 were given a placebo. For the primary outcome, the distribution across mRS categories was similar for the fluoxetine and placebo groups at six months (common odds ratio 0.88; 95% confidence interval 0.31-2.50; p = 0.81), and there was no difference at 12 months (p = 0.864). There were no differences between groups in stroke recovery or in motor function recovery of the affected hand. There were no significant differences in any other secondary outcomes at six or 12 months. Patients given fluoxetine were less likely than those given the placebo to receive new antidepressant medication within six months (2 [6.67%] vs. 4 [12.90%]). CONCLUSIONS AND CLINICAL IMPLICATIONS: Consistent with other trials based on the FOCUS protocol, fluoxetine did not improve motor recovery or general stroke outcome at six and 12 months in the Polish cohort studied. However, patients receiving fluoxetine required therapy with additional antidepressant medication less frequently.

Differences in acute ischaemic stroke care in Poland: analysis of claims database of National Health Fund in 2017.

Selected and basic indicators of acute ischaemic stroke care in Poland are reported cross-regionally based on the analysis of claims data of the National Health Fund (NFZ) in 2017, the most reliable source of healthcare funding in the country, being a single public payer. For research purposes, a selection algorithm based on the diagnosis coded as I63 according to the International Classification of Diseases (ICD-10) was used to identify all ischaemic stroke patients in the claims database provided by the NFZ. Stroke units and other centres providing treatment for acute ischaemic stroke patients were examined. The analysis showed marked differences between provinces in terms of stroke unit treatment availability. The crude and standardised rates of acute ischaemic stroke admissions to stroke units varied between provinces. Moreover, substantial differences were observed for the thrombolysis implementation rate, access to rehabilitation, hospital stay and early prognosis. As the leading cause of disability and the second leading cause of death in developed countries, stroke requires a well-organised, evidence-based healthcare system provided for both acute treatment and rehabilitation. Continuous monitoring of healthcare is crucial to identify weaknesses and areas for improvement.

Persistence with treatment for Wilson disease: a retrospective study.

BACKGROUND: Wilson disease (WD) is genetically induced failure of copper metabolism which can be successfully treated with pharmacological agents. The prognosis for survival in most WD patients is favorable if diagnosis and anti-copper treatment are provided early. Many observations imply that persistence with drug treatment is generally low in patients with chronic diseases, which impact the treatment effectiveness, but such results are very limited in WD. The aim of our study was to assess persistence with treatment among WD patients, to analyze its effect on patient outcome and to identify factors that might be related to persistence. METHODS: 170 newly diagnosed, symptomatic patients with WD who started treatment between 1995 and 2005 were analyzed retrospectively to assess treatment non-persistence, which was defined as at least one reported break of more than 3 months or minimum two breaks lasting longer than 2 months. Results were further analyzed according to selected clinical variables. RESULTS: Only 74.1% of patients were persistent with treatment during the mean 11.7 years of follow up. Treatment persistence closely impacted positive clinical outcomes. In patients classified as persistent, improvement and lack of WD progression were observed more often compared to those classified as non-persistent (29.4 and 68.3% vs. 2.3 and 45.5%; p < 0.001, respectively). In contrast, non-persistent patients presented more often with worsening WD than persistent patients (52.3% vs. 2.4%). Type of WD treatment, gender, phenotypic presentation, adverse events and duration of treatment were not related to treatment persistence. Higher or upper/post-secondary education and a supportive family attitude towards treatment were the most important factors related to persistence. CONCLUSIONS: One quarter of WD patients were not taking anti-copper treatment regularly and this had an important negative effect on clinical outcome. Family support played an important role in treatment persistence.

Measurement of Nutritional Status Using Body Mass Index, Waist-to-Hip Ratio, and Waist Circumference to Predict Treatment Outcome in Females and Males with Acute First-Ever Ischemic Stroke.

BACKGROUND: We aimed to investigate whether increased waist-to-hip ratio (WHR), waist circumference (WC), or improper body mass index (BMI) may differently predict short-term outcomes in females and males with first-ever acute ischemic stroke. METHODS: This was a retrospective study of consecutive patients (1109 females and 939 males) admitted for first-ever ischemic stroke between 2003 and 2015. Data were collected in a detailed hospital stroke registry. BMI of 18.5-24.9 kg/m2 and gender-specific normal values of WHC and WC were used as references for comparisons. Logistic regression was used to calculate the odds of in-hospital death or being dead or dependent at discharge, adjusted for patients' age and prestroke disability. RESULTS: In both sexes a high WHR increased the odds of death or dependency at discharge (odds ratio [OR], 1.8; 95% confidence interval [CI], 1.05-3.08 for females and 1.43; 95% CI, 1.00-2.04 for males), but not in-hospital death alone. Increased WC was significantly associated with lower odds of either death or death and dependency at discharge in females only (OR, .36; 95% CI, .22-.58 and .69; 95% CI, .48-.97, respectively). BMI did not show any clear predictive value in either sex. CONCLUSIONS: Our findings suggest that being overweight measured with WC is a strong predictor of good outcome in women but not in men. The WHR less consistently predicts stroke outcome, as it is not associated with death at discharge alone; however, the WHR seems to be of similar clinical relevance in both genders. BMI seems to have the least clinical value in predicting stroke outcome in both genders.

Activation of blood coagulation and thrombin generation in acute ischemic stroke treated with rtPA.

The impact of thrombolysis with recombinant tissue plasminogen activator (rtPA) on blood coagulation in acute ischemic stroke (AIS) patients is not completely understood. We studied the effect of thrombolysis on the thrombin generation (TG) profile as well as coagulant activity of activated factors IX (FIXa), XI (FXIa) and tissue factor (TF) in AIS patients. In a case-control study, TG parameters as well as FIXa, FXIa and TF levels were assessed in 95 AIS patients, including individuals receiving rtPA treatment within 4.5 h since AIS onset (n = 71, 74.7%) and those ineligible for thrombolysis (n = 24, 25.3%). Blood samples were collected at baseline and after 24 h since admission. The two groups were similar with regard to demographics and clinical factors. In thrombolysed patients, all TG parameters measured after 24 h were markedly decreased, with strongest impact on lag time (LT), when compared with the baseline values (81.3% longer LT, p < 0.0001), as well as when compared to the non-thrombolysed group (86% longer LT, p = 0.002). In non-thrombolysed AIS patients the TG remained unaltered. Logistic regression adjusted for potential confounders showed that high baseline ETP value (the top quartile) was solely predicted by the presence of circulating FIXa, whereas after 24 h FXIa predicted high ETP in the subgroup of thrombolysed and in all AIS patients. Thrombolysis in AIS patients markedly attenuates the TG. Elevated FXIa contributes to thrombin formation capacity after 24 h, highlighting a role of this factor in the regulation of blood coagulation in AIS.

Status of etoricoxib in the treatment of rheumatic diseases. Expert panel opinion.

Pain is one of the most disabling symptoms of rheumatoid diseases. Patients with pain secondary to osteoarthritis (OA), rheumatoid arthritis (RA), ankylosing spondylitis (AS) or gout require effective analgesic treatment, and the physician's task is to select a drug that is best suited for an individual patient. The choice of pharmacotherapy should be based both on drug potency and clinical efficacy, and its safety profile, particularly in the elderly population, as the number of comorbidities (and hence the risk of treatment complications and drug interactions) rises with age. In cases involving a high risk of gastrointestinal complications or concerns about hepatotoxicity, with a low cardiovascular risk, the first-line nonsteroidal anti-inflammatory drugs to consider should be coxibs including etoricoxib.

Lifestyle modification for stroke prevention: facts and fiction.

PURPOSE OF REVIEW: The purpose is to summarize recent evidence on lifestyle modifications and first or recurrent stroke risk. RECENT FINDINGS: Weight reduction, low-risk diet, regular physical activity, smoking cessation, and low-to-moderate alcohol consumption may reduce stroke risk up to 50% or more, but level one evidence is still lacking for several interventions. Appropriate food ingredients can significantly decrease stroke risk as recently confirmed for Mediterranean diet. The optimal intensity and amount of physical exercise is still not well established before and after stroke, although modest levels of activity already show benefits. Passive smoking represents an important health hazard. The impact of tobacco withdrawal using e-cigarette is currently uncertain. Alcohol and stroke risk relation is probably J-shaped for ischaemic stroke and linear for intracranial haemorrhage. Coffee consumption is J-shaped for overall stroke. Several interventions have failed to show significant effects, including regular intake of 'healthy' forms of fatty acids, various vitamin supplements, and other antioxidants. Both individualized and public educational programmes are likely needed on a repetitive basis to induce and maintain a healthy lifestyle before or after a stroke.

The impact of a belief in life after death on health-state preferences: True difference or artifact?

PURPOSE: In most religions, the preservation of one's own, God-given, life is considered obligatory, while the time trade-off method (TTO) forces one to voluntarily forego life years. We sought to verify how this conflict impacts TTO-results among the religious. METHODS: We used the data from the only EQ-5D valuation in Poland (2008, three-level, 321 respondents, 23 states each)-a very religious, mostly Catholic country. We measured the religiosity with the belief in afterlife question on two levels: strong (definitely yes) and some (also rather yes), both about a third of the sample. RESULTS: The religious more often are non-traders, unwilling to give up any time in exchange for quality of life: odds ratio (OR) equal to 1.97 (strong religiosity), OR 1.55 (some religiosity); and less often consider a state worse than death: OR 0.67 (strong), OR 0.81 (some). These associations are statistically significant ([Formula: see text]) and hold when controlling for possible demographic confounders. Strong religiosity abates the utility loss: in the additive approach by 0.14, in the multiplicative approach by the factor of 2.1 (both [Formula: see text]), especially among the older. Removing the effect of religiosity from the value set reduces the utility by 0.05 on average. CONCLUSION: The results may stem from a true difference in preferences or be a TTO-artifact and would vanish for other elicitation methods. Juxtaposing our findings with comments from respondents in other studies suggests the latter. Therefore, this Weltanschauung effect should be removed in cost-utility analysis.

Variations in acute hospital stroke care and factors influencing adherence to quality indicators in 6 European audits.

BACKGROUND AND PURPOSE: We compared compliance with standards of acute stroke care between 6 European audits and identified factors associated with delivery of appropriate care. METHODS: Data were derived from stroke audits in Germany, Poland, Scotland, Catalonia, Sweden, and England/Wales/Northern-Ireland participating within the European Implementation Score (EIS) collaboration. Associations between demographic and clinical characteristics with adherence to predefined quality indicators were investigated by hierarchical logistic regression analyses. RESULTS: In 2007/2008 data from 329 122 patients with stroke were documented. Substantial variations in adherence to quality indicators were found; older age was associated with a lower probability of receiving thrombolytic therapy, anticoagulant therapy, or stroke unit treatment and a higher probability of being tested for dysphagia. Women were less likely to receive anticoagulant or antiplatelet therapy or stroke unit treatment. No major weekend effect was found. CONCLUSIONS: Detected variations in performance of acute stroke services were found. Differences in adherence to quality indicators might indicate population subgroups with specific needs for improving care delivery.

Drug-related risk of severe hypoglycaemia in observational studies: a systematic review and meta-analysis.

BACKGROUND: Diabetes mellitus (DM) leads to multiple complications, including severe hypoglycaemia events (SHEs). SHEs can impact a patient's quality of life and compliance and may directly result in additional costs to the health care system. The aim of this review was to evaluate the risk of severe hypoglycaemia in patients with type 1 (T1) and 2 (T2) DM as observed in everyday clinical practice for various drug regimens. METHODS: We conducted a systematic review of observational (retrospective or prospective) studies in the MEDLINE, Embase, and Cochrane Library databases that covered at least 100 children or adults with T1/T2 DM. In T1 DM, basal-bolus/pre-mix insulin (human or analogue) and insulin pump were reviewed, and in T2 DM, basal-bolus/pre-mix insulin (human or analogue), oral antidiabetic drugs supported with basal insulin (human or analogue), sulfonylureas in monotherapy, and combined oral treatment were reviewed. In order to estimate SHE rates, we extracted data on the time horizon of the study, number of patients, number of SHEs, and number of patients experiencing at least one SHE. We used a random effects model to estimate the annual SHE rate. We considered the risk for other antidiabetic medications in T2 DM to be negligible and the results of our main review yielded no observational data for premixes in T1 DM so they were assessed based on relative rates taken from additional systematic reviews. The study, being a desk research, did not involve any human subjects (including human material or human data) and no ethical committee approval was asked for. For the same reason there was no need to collect informed consent for participation in the study. RESULTS: We identified 76 observational studies encompassing 707,722.30 patient-years. The estimated annual SHE rate varied from 0.168 (95 % CI 0.123-0.237) for insulin pump up to 1.628 for biphasic human insulin in T1 DM patients, and from 0.0035 for oral antidiabetic drugs up to 0.554 (95 % CI 0.157-7.534) for basal-bolus with human insulin in T2 DM patients. CONCLUSIONS: Our review indicates that SHE rates differ between patients depending on treatment regimen. However, SHEs are also driven by other factors. Proper modelling techniques are needed to use various types of information in published studies.

Validity of EQ-5D-5L in stroke.

PURPOSE: To assess EQ-5D-5L (5L) validity in patients with acute stroke, in comparison with EQ-5D-3L (3L). METHODS: Cross-sectional study of 408 patients during index hospitalization. We compared 5L and 3L in terms of feasibility, frequency of unique health states, ceiling effect and discriminatory power (informativity). We assessed construct validity in terms of known-groups validity and convergent validity of 5L dimensions with other stroke outcome measures. RESULTS: The overall proportion of patients with acute stroke reporting 'no problems' with 3L-6.1 % was further reduced to 5.6 % with 5L (relative reduction of 8.2 %). The highest improvement in relative discriminatory power, when moving from 3L to 5L, was noticed in pain/discomfort and anxiety/depression dimensions (Shannon Evenness Index 0.91 for both 5L dimensions; relative increase 34.4 and 29.1 %, respectively). Known-groups validity tests confirmed prior hypotheses: Health state utilities were lower in following subpopulations-females, patients with high modified Rankin Scale (mRS) score, low Barthel Index (BI) or VAS score, patients with subarachnoid hemorrhage or intracerebral hemorrhage, and when proxy respondent was used. Convergence of EQ-5D-5L dimensions with mRS, BI and EQ VAS was improved or at least the same as for 3L dimensions. CONCLUSIONS: Results support the validity of the EQ-5D-5L descriptive system as a generic health outcome measure in patients with acute stroke, demonstrating some psychometric advantages in comparison with EQ-5D-3L.

Comparing responsiveness of the EQ-5D-5L, EQ-5D-3L and EQ VAS in stroke patients.

AIMS: To date, evidence to support the construct validity of the EQ-5D-5L has primarily focused on cross-sectional data. The aims of this study were to examine the responsiveness of EQ-5D-5L in patients with stroke and to compare it with responsiveness of EQ-5D-3L and visual analogue scale (EQ VAS). METHODS: We performed an observational longitudinal cohort study of patients with stroke. At 1 week and 4 months post-stroke, patients were assessed with modified Rankin Scale (mRS) and Barthel Index (BI) and were administered the EQ-5D-5L and EQ-5D-3L, including the EQ VAS. The EQ-5D-5L index scores were derived using the crosswalk methodology developed by the EuroQol Group. We classified patients according to two external criteria, based on mRS or BI, into 3 categories: 'improvement,' 'stable' or 'deterioration'. We assessed the responsiveness of each measure in each patient subgroup using: effect size (ES), standardized response mean (SRM), F-statistic, relative efficiency and area under the receiver operating characteristic curve. RESULTS: A total of 112 patients (52% females; mean age 70.6 years; 93% ischemic stroke) completed all the instruments at both occasions. In subjects with clinical improvement, EQ-5D-5L was consistently responsive, showing moderate ES (0.51-0.71) and moderate to large SRM (0.69-0.86). In general, EQ-5D-3L index appeared to be more responsive (ES 0.63-0.82; SRM 0.77-1.06) and EQ VAS less responsive (ES 0.51-0.65; SRM 0.59-0.69) than EQ-5D-5L index. CONCLUSIONS: The EQ-5D-5L index, based on the crosswalk value set, seems to be appropriately responsive in patients with stroke, 4 months after disease onset. As far as EQ-5D-5L index is scored according to crosswalk approach, the EQ-5D-3L index appears to be more responsive in stroke population.

Pheochromocytoma presenting as takotsubo-like cardiomyopathy following delivery.

OBJECTIVE: Diagnosis of pheochromocytoma during pregnancy can be difficult, and the tumor carries an unfavorable prognosis if not diagnosed and treated in a timely manner. METHODS: To present a case of Takotsubo-like cardiomyopathy characterized by transient left ventricular apical ballooning due to pheochromocytoma following delivery. RESULTS: A few hours after Caesarean section, a 32-year-old Caucasian female presented with pulmonary edema followed by cardiac arrest with echocardiographic and ventriculographic evidence of reversible acute myocardial failure characteristic of Takotsubo-like cardiomyopathy. A previously unrecognized adrenal pheochromocytoma was found during her clinical work-up. Left ventricle (LV) function normalized after surgical removal of the tumor, which was carried out after implementing an alpha-adrenoreceptor blockade. Hemorrhagic necrosis of the pheochromocytoma was seen on histopathologic analysis; this may have triggered the sequence of events leading to the development of Takotsubo-like cardiomyopathy and hemodynamic collapse. CONCLUSION: To the best of our knowledge, this is the first reported case of Takotsubo-like cardiomyopathy related to pheochromocytoma following delivery. This emphasizes the increased cardiovascular risk if pheochromocytoma is not diagnosed and treated in a timely manner, especially during pregnancy.

AOTMiT reimbursement recommendations compared to other HTA agencies.

Our objective was to compare AOTMiT (Polish: Agencja Oceny Technologii Medycznych i Taryfikacji) recommendations to other HTA (Health Technology Assessment) agencies for newly registered drugs and new registration indications issued by the European Medicines Agency between 2014 and 2019. The study aims to assess the consistency and justifications of AOTMiT recommendations compared to that of other HTA agencies in 11 countries. A total of 2494 reimbursement recommendations published by 12 HTA agencies for 464 medicinal products and 525 indications were analyzed. Our analysis confirmed that the Polish AOTMiT agency seems to bear the closest resemblance to the corresponding HTA agencies from Canada (CADTH) and New Zealand (PHARMAC), when it comes to the outcome of HTA recommendations (positive or negative). Poland had a general scheme for justifying recommendations, similar to that of Ireland-four aspects (i.e., clinical efficacy, safety profile, cost-effectiveness, and impact on the payer's budget) are important for Poland when formulating the final decision. Compared to other countries, Poland shows a noticeably different pattern of justifying reimbursement recommendations, as revealed primarily in terms of budget impact and somewhat less so for cost-effectiveness rationales.

Importance of aligning the implementation of new payment models for innovative pharmaceuticals in European countries.

INTRODUCTION: The uptake of complex technologies and platforms has resulted in several challenges in the pricing and reimbursement of innovative pharmaceuticals. To address these challenges, plenty of concepts have already been described in the scientific literature about innovative value judgment or payment models, which are either (1) remaining theoretical; or (2) applied only in pilots with limited impact on patient access; or (3) applied so heterogeneously in many different countries that it prevents the health care industry from meeting expectations of HTA bodies and health care payers in the evidence requirements or offerings in different jurisdictions. AREAS COVERED: This paper provides perspectives on how to reduce the heterogeneity of pharmaceutical payment models across European countries in five areas, including 1) extended evaluation frameworks, 2) performance-based risk-sharing agreements, 3) pooled procurement for low volume or urgent technologies, 4) alternative access schemes, and 5) delayed payment models for technologies with high upfront costs. EXPERT OPINION: Whilst pricing and reimbursement decisions will remain a competence of EU member states, there is a need for alignment of European pharmaceutical payment model components in critical areas with the ultimate objective of improving the equitable access of European patients to increasingly complex pharmaceutical technologies.