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Background There is currently limited data to guide treatment selection, dosing, combination strategies and sequencing in the management of myasthenia gravis (MG). Additionally, MG symptoms are heterogenous between people and with time, requiring an individualized treatment approach. As such, successful patient-physician communication is key. This Delphi project was undertaken by MG specialists and patient representatives to explore their opinion on improving MG management via effective physician-patient communication and collaboration approach. Methods This mixed-methods study was conducted in two phases. In Phase I, seven MG specialists and two patient representatives contributed to idea generation. Relevant insights informed the development of the Phase II Delphi survey. A panel of 16 MG specialists and seven patient representatives participated in a two-round Delphi survey. Consensus was defined as ≥ 70% agreement or disagreement on a 6-point Likert scale. Participants responded to the survey from what they considered an ideal scenario, regardless of their real-world experience. Results Consensus was achieved on 89% of all statements. Key areas of alignment were – importance of incorporating patient preferences on quality of life (QoL), sustained symptom control, route of administration, and side effects profile. Both groups agreed that current clinical practices insufficiently integrate mental health considerations and patient engagement remains suboptimal. Two key areas of misalignment were – patient representative and physicians had different perspective on how well physicians understood patient preferences and both groups showed different interpretations of side effects (adverse reaction versus issues of tolerability). Conclusion This Delphi consensus found that while physician and patient representatives shared similar perspectives these were not always reflected in current clinical practice due to differences in understanding of patient preferences. Prioritizing structured conversations on QoL, treatment expectations, and side effect were found important for physicians while improving access to information, openly communicating and actively participating in treatment decisions were key outcomes for patients. This study lays a valuable foundation for deepening conversations and alignment on key topics in MG management among the medical, patient and caregiving communities.

Background Hereditary gelsolin (AGel) amyloidosis is an autosomal dominantly inherited systemic amyloidosis that manifests with the characteristic triad of progressive ophthalmological, neurological and dermatological signs and symptoms. The National Finnish Gelsolin Amyloidosis Registry (FIN-GAR) was founded in 2013 to collect clinical data on patients with AGel amyloidosis, including altogether approximately one third of the Finnish patients. We aim to deepen knowledge on the disease burden and life span of the patients using data from the updated FIN-GAR registry. We sent an updated questionnaire concerning the symptoms and signs, symptomatic treatments and subjective perception on disease progression to 240 members of the Finnish Amyloidosis Association (SAMY). We analyzed the lifespan of 478 patients using the relative survival (RS) framework. Results The updated FIN-GAR registry includes 261 patients. Symptoms and signs corresponding to the classical triad of ophthalmological (dry eyes in 93%; corneal lattice amyloidosis in 89%), neurological (numbness, tingling and other paresthesias in 75%; facial paresis in 67%), and dermatological (drooping eyelids in 86%; cutis laxa in 84%) manifestations were highly prevalent. Cardiac arrhythmias were reported by 15% of the patients and 5% had a cardiac pacemaker installed. Proteinuria was reported by 13% and renal failure by 5% of the patients. A total of 65% of the patients had undergone a skin or soft tissue surgery, 26% carpal tunnel surgery and 24% at least unilateral cataract surgery. As regards life span, relative survival estimates exceeded 1 for males and females until the age group of 70–74 years, for which it was 0.96. Conclusions AGel amyloidosis causes a wide variety of ophthalmological, neurological, cutaneous, and oral symptoms that together with repeated surgeries cause a clinically significant disease burden. Severe renal and cardiac manifestations are rare as compared to other systemic amyloidoses, explaining in part the finding that AGel amyloidosis does not shorten the life span of the patients at least for the first 75 years.

Background Impact of changing diagnostic criteria for the population-based incidence of multiple sclerosis (MS) has not been investigated. Objective To assess the effect of changing diagnostic criteria on national MS incidence and prevalence in Finland from 1974 to 2021. Methods We identified patients with MS (pwMS) through the National MS registry and the national Care Register for Healthcare and divided them into four groups based on the year of MS diagnosis: 1) Schumacher criteria (1974–1982), 2) Poser criteria (1983–2000), 3) Earlier McDonald criteria (2001–2016), and 4) Current McDonald criteria (2017–2021). Age-adjusted incidence and prevalence were calculated. Results Age-adjusted incidence per 105 person years increased from 3.7 (95% CI 3.5–3.8) during the Schumacher criteria period to 9.2 (95% CI 9.0–9.4) during the earlier McDonald criteria. During the Current McDonald criteria incidence stabilized to 8.6 (95% CI 8.3–9.0). Prevalence increased from 24.3 (95% CI 22.8–25.8) to 241.5 (95% CI 237.3–245.6) per 105 person years. Conclusion Both incidence and prevalence of MS increased significantly. Incidence showed a sharp increase when entering the twenty-first century, after which it stabilized. Increasing incidence was likely related to incorporation of MRI in the diagnostic criteria. Current diagnostic criteria did not further increase the incidence.

Background Gelsolin amyloidosis (AGel amyloidosis) is a hereditary form of systemic amyloidosis featuring ophthalmological, neurological and cutaneous symptoms. Previous studies based mainly on patients’ self-reporting have indicated that hearing impairment might also be related to the disease, considering the progressive cranial neuropathy characteristic for AGel amyloidosis. In order to deepen the knowledge of possible AGel amyloidosis-related hearing problems, a clinical study consisting of the Speech, Spatial and Qualities of Hearing Scale (SSQ) questionnaire, clinical examination, automated pure-tone audiometry and a speech-in-noise test was designed. Results Of the total 46 patients included in the study, eighteen (39%) had self-reported hearing loss. The mean scores in the SSQ were 8.2, 8.3 and 8.6 for the Speech, Spatial and Qualities subscales, respectively. In audiometry, the mean pure tone average (PTA) was 17.1 (SD 12.2) and 17.1 (SD 12.3) dB HL for the right and left ears, respectively, with no difference to gender- and age-matched, otologically normal reference values. The average speech reception threshold in noise (SRT) was − 8.2 (SD 1.5) and − 8.0 (SD 1.7) dB SNR for the right and left ears, respectively, which did not differ from a control group with a comparable range in PTA thresholds. Conclusion Although a significant proportion of AGel amyloidosis patients experience subjective difficulties in hearing there seems to be no peripheral or central hearing impairment at least in patients up to the age of 60 years.

The treatment landscape for myasthenia gravis (MG) has evolved with the introduction of novel therapies. An international consensus on patient selection criteria and optimal time to initiate these therapies could improve clinical outcomes and reduce delays for likely beneficiaries. This Delphi consensus was undertaken by MG specialists from selected European countries to explore gaps in the application of national guidelines and elicit expert opinion in practice. A mixed-method approach was used; qualitative and quantitative study phases were combined to explore key concepts and reach consensus. The qualitative first phase involved seven healthcare professionals (HCPs) and two patient advocacy group representatives who participated in idea generation. Findings from this phase supported the development of a Delphi survey, which was completed by 16 HCPs in two rounds. This constituted the quantitative second phase of the study. Consensus was defined as ⩾70% agreement or disagreement on a 6-point Likert scale. In total, 65% of statements achieved consensus. Key findings include-HCPs highly regard international guidelines but find critical discrepancies between the \"ideal\" scenario and current clinical practices. Consensus was achieved on the importance of incorporating patient-related quality of life in decision-making, despite limited current methods. Consensus was obtained on steroid tapering and treatment-switch criteria based on steroid dose and duration. Consensus was also achieved on suitable patient profiles, including those with persistent symptoms, severe side effects, or needing rapid control. This study recognized that guidelines offer valuable direction but do not replace individualized treatment decisions. This study identified the areas of alignment and opportunities to refine patient selection criteria and treatment-switch categories, particularly to integrate novel therapy use in MG management, highlighting a path to a more patient-centric approach.

The objective was to investigate adherence measured by an electronic auto-injector device, and self-reported adherence and treatment convenience in subjects with relapsing remitting multiple sclerosis (RRMS), using an electronic auto-injector Rebismart® to self-inject interferon β-1a. Thirty one patients with RRMS using the electronic auto-injector Rebismart® for selfinjecting interferon β-1a subcutaneously three times weekly were included in a reallife clinical multicenter study for 24 weeks in Finland. Mean adherence reported by the device and mean self-assessment of adherence were studied. Reasons for missing injections and treatment convenience were assessed. Association between adherence and gender and age were studied. The mean adherence calculated from the device data was 93.5%. The mean self-assessment of adherence was 96.6%. The most common reason for missing an injection was forgetfulness. Adherence (measured by the device) was not changed over time. In the high adherence group there were more females and young patients (<30 years of age). The auto-injector was found to substantially ease the treatment by 90% of the patients. The electronic auto-injector was associated with high adherence to treatment. The device was found to ease the patient’s treatment and it was perceived as easy to use. It is a convenient tool to assess patient’s adherence to treatment.

Background: Treating hyperglycemia in acute ischemic stroke may be beneficial, but knowledge on its prognostic value and optimal target glucose levels is scarce. We investigated the dynamics of glucose levels and the association of hyperglycemia with outcomes on admission and within 48 h after thrombolysis. Methods: We included 851 consecutive patients with acute ischemic stroke treated with intravenous thrombolysis in the Helsinki University Central Hospital during 1998–2008. Outcome measures were unfavorable 3- month outcome (3–6 on the modified Rankin Scale), death, and symptomatic intracerebral hemorrhage (sICH) according to NINDS criteria. Hyperglycemia was defined as a blood glucose level of ≧8.0 mmol/l. Four groups were identified based on (a) admission and (b) peak glucose levels 48 h after thrombolysis: (1) persistent normoglycemia (baseline plus 48-hour normoglycemia), (2) baseline hyperglycemia (48-hour normoglycemia), (3) 48-hour hyperglycemia (baseline normoglycemia), and (4) persistent hyperglycemia (baseline plus 48-hour hyperglycemia). Results: 480 (56.4%) of our patients (median age 70 years; onset-to-needle time 199 min; National Institutes of Health Stroke Scale score 9), had persistent normoglycemia, 59 (6.9%) had baseline hyperglycemia, 175 (20.6%) had 48-hour hyperglycemia, while persistent hyperglycemia appeared in 137 (16.1%) patients. Persistent and 48-hour hyperglycemia independently predicted unfavorable outcome [odds ratio (OR) = 2.33, 95% confidence interval (CI) = 1.41–3.86, and OR = 2.17, 95% CI = 1.30–3.38, respectively], death (OR = 6.63, 95% CI = 3.25–13.54, and OR = 3.13, 95% CI = 1.56–6.27, respectively), and sICH (OR = 3.02, 95% CI = 1.68–5.43, and OR = 1.89, 95% CI = 1.04–3.43, respectively), whereas baseline hyperglycemia did not. Conclusions: Hyperglycemia (≧8.0 mmol/l) during 48 h after intravenous thrombolysis of ischemic stroke is strongly associated with unfavorable outcome, sICH, and death.

Background and purpose Exacerbation of myasthenia gravis (MG) with respiratory failure requires intensive care. We aimed to study the risk factors for intensive care unit admission for MG exacerbation and myasthenic crisis (MC) and the prognosis of people with MG (pwMG) thereafter. Methods This retrospective study investigated patients in the Helsinki and Uusimaa hospital district during the years 2008–2021. PwMG (International Classification of Diseases, 10th revision code G70.0) were identified through a data repository search, followed by a chart review of patient records. Risk factors for intensive care due to MG exacerbation were evaluated as compared with the patients only treated in the outpatient clinic and those treated in the neurological ward for MG exacerbation. The outcomes of patients in intensive care for any reason were also compared with those of patients in intensive care for exacerbation of bronchial asthma. Results Of 577 pwMG, 35 (6.1%) needed intensive care for MG within a median of 5.3 months from diagnosis. The mean (±SD) age at MG diagnosis was higher in the intensive care group (60.5 [±16.1] years) compared to the outpatient (48.3 [±20.9] years; p < 0.001) and neurological ward groups (53.4 [±20.8] years; p = 0.044). Thymoma (odds ratio [OR] 4.8, 95% confidence interval [CI] 1.19–19.43; p = 0.028) and female sex (OR 2.1, 95% CI 1.02–4.48; p = 0.045) were independent risk factors for intensive care. In‐hospital mortality was 4% for MC patients. Six‐month mortality after intensive care for MG exacerbation (14.3%) was twice that for asthma exacerbation (7.7%). Conclusion Our study shows an increased risk of intensive care treatment for patients with late‐onset MG, female sex or thymoma, occurring usually within 6 months from diagnosis, which emphasises the importance of early treatment choices.

BackgroundIncidence and prevalence rates of myasthenia gravis (MG) vary considerably across studies, and mortality risk is rarely addressed. We examined the prevalence and incidence rates, mortality and factors associated with mortality with MG.MethodThis was a registry linkage study based on nationwide health and administrative registries of Denmark, Finland and Sweden (populations of 5.9, 5.6 and 10.5 million, respectively). Patients with MG were identified based on International Classification of Diseases codes from inpatient and outpatient specialised care registries. Yearly prevalence, incidence and mortality rates in relation to the total background population were calculated from 2000 to 2020 (study period). The causes of death and factors associated with mortality were addressed separately.ResultsThe overall incidence of MG was 1.34 (95% CI 1.27 to 1.41), 1.68 (95% CI 1.60 to 1.75) and 1.62 (95% CI 1.56 to 1.68) per 100 000, and the overall prevalence per 100 000 was 18.56 (95% CI 18.31 to 18.81), 20.89 (95% CI 20.62 to 21.16) and 23.42 (95% CI 23.21 to 23.64) in Denmark, Finland and Sweden, respectively. The overall standardised mortality ratio (SMR) was 1.32 (95% CI 1.23 to 1.42) among patients with MG in Denmark, 1.23 (95% CI 1.15 to 1.33) in Finland, and 1.20 (95% CI 1.14 to 1.26) in Sweden, with higher SMR observed in women than men. Annual incidence and prevalence increased over time, whereas the SMR remained stable. The most common causes of death were MG, chronic ischaemic heart disease and acute myocardial infarction.ConclusionsThis population-based study from three Nordic countries highlights the need for improved care of patients with MG, especially young women.

Introduction Population-based longitudinal data on mortality and causes of death (COD) for people with Multiple Sclerosis (pwMS) is scarce. We studied all-cause and cause-specific mortality in Finnish pwMS in a nationwide registry study. Methods PwMS from 1st January 1971 until end of 2019 were identified from the Finnish MS registry and national health care register. Standardized mortality ratios (SMRs), excess death rates (EDRs), life expectancies, and causes of death (COD) were determined by linkage to national registries. Results For 16,602 pwMS, 3936 deaths occurred between 1980 and 2020. During 1980–1999, SMR for pwMS was 3.07 (95% CI 2.91–3.25) and EDR 14.05 (95% CI 13.72–14.37), and during 2000–2020 2.18 (95% CI 2.10–2.26) and 7.48 (95% CI 7.2–7.75), respectively. SMRs were higher for female pwMS and for patients diagnosed under age 30. EDRs were higher for males. Risk of death was lower for pwMS diagnosed 1996–2005 versus 1980–1995 (HR 0.49; 95% CI 0.43–0.55; p  < 0.001). MS was the underlying cause in 51.2%, and a mentioned cause in 73.1% of deaths during 2000–2020. Mortality by underlying cause was higher than expected for gastrointestinal diseases (SMR 2.15, 95% CI 1.53–2.77), respiratory infections (SMR 1.99, 95% CI 1.22–2.75), and vascular diseases (SMR 1.38, 95% CI 1.25–1.51). Median lifetime expectancy was shortened by 7 years. Conclusion Excess mortality in Finnish pwMS has decreased during the last 40 years. Life expectancy is shortened by 7 years and MS itself is the most frequent underlying COD. Risk of death is lower for pwMS diagnosed during the therapeutic era.