Supporting the assignment of NMR spectra with variable-temperature experiments.

Nuclear magnetic resonance (NMR) spectroscopy is one of the most powerful tools in analytical chemistry. An important step in the analysis of NMR data is the assignment of resonance frequencies to the corresponding atoms in the molecule being investigated. The traditional approach considers the spectrum's characteristic parameters: chemical shift values, internuclear couplings, and peak intensities. In this paper, we show how to support the process of assigning a series of spectra of similar organic compounds by using temperature coefficients, that is, the rates of change in chemical shift values associated with given changes in temperature.

Performance of portable emergency suction devices in pre-hospital conditions: a pilot study in the fire brigade.

A i m: Assessment of the effectiveness and efficiency of three mobile (portable) rescue aspirators models in the opinion of state fire service officers. Comparison with the use of the medical simulation element. MATERIAL AND METHODS: The study was conducted in organizational units of the State Fire Service (24-hour officers). The research consisted in carrying out the task with the use of three models of mobile rescue aspirators (manual, hand-foot, battery). Each participating firefighter had the task of sucking up an equal amount of fluid (100 ml, respectively) with each model of an aspirator. The test fluid was water at room temperature in a homogeneous 1:1 mixture with sugar (increased viscosity and density, simulated real conditions). Immediately after three suction attempts (with measured suction time), each officer completed a questionnaire on the three models used. Descriptive statistics were used to characterize the variables. The following measures were calculated for the variables: mean (M) and standard deviation (SD), minimum, maximum. The following measures were calculated for categorical variables: number (n) and frequency (%). RESULTS: 184 officers (182 M and 2 F) took part in the study, including commanders 18.43%, rescuers 65.22%, drivers 16.30%. In the study area 1,609 officers serve in the combat division as at the end of 2021. The studied group accounts for 11.43%. Age of respondents M 34.04 SD 8.24 Min 21 Max 52, length of service M 8.48, SD 7.20 Min 1, Max 25. The longest mean time of completing the task was recorded for model 2 (hand-foot) and it was 6.77 sec. CONCLUSIONS: SFS officers highly appreciated the usefulness and effectiveness of the battery-operated automatic aspirator. This assessment may contribute to the widespread introduction of such a model to rescue sets in the SFS. Time of performing the task by mode 1 was significantly longer by elderly people. People with experience with the model 1 during rescue and firefighting operations had a significantly shorter time of performing the task with the use of the model 2. According to the subjective assessment of firefighters, the most effective is model 3, which is confirmed by the suction time obtained at the work station.

Silver route to cuprate analogs.

The parent compound of high-[Formula: see text] superconducting cuprates is a unique Mott insulator consisting of layers of spin-[Formula: see text] ions forming a square lattice and with a record high in-plane antiferromagnetic coupling. Compounds with similar characteristics have long been searched for without success. Here, we use a combination of experimental and theoretical tools to show that commercial [Formula: see text] is an excellent cuprate analog with remarkably similar electronic parameters to [Formula: see text] but larger buckling of planes. Two-magnon Raman scattering and inelastic neutron scattering reveal a superexchange constant reaching 70% of that of a typical cuprate. We argue that structures that reduce or eliminate the buckling of the [Formula: see text] planes could have an antiferromagnetic coupling that matches or surpasses the cuprates.

Different blood-brain-barrier disruption profiles in multiple sclerosis, neuromyelitis optica spectrum disorders, and neuropsychiatric systemic lupus erythematosus.

AIM OF THE STUDY: To assess differences in BBB damage profiles by measuring serum levels of soluble vascular cell adhesion molecule-1 (sVCAM-1), soluble platelet endothelial cell adhesion molecule-1 (sPECAM-1), soluble intercellular adhesion molecule-1 (sICAM-1), and S100 calcium-binding protein B (S100B) in relapsing-remitting multiple sclerosis (RRMS), neuromyelitis optica spectrum disorders (NMOsd), and neuropsychiatric systemic lupus erythematosus (NPSLE) patients. CLINICAL RATIONALE FOR THE STUDY: Blood-brain-barrier (BBB) disruption is one of the key pathological processes involved in various demyelinating diseases of the central nervous system (CNS) and is associated with shedding of cell adhesion molecules and S100B into the serum compartment. Therefore, making an assessment of serum levels of the above-mentioned molecules could provide information about disease pathogenesis, severity of BBB disruption, and disease activity. MATERIAL AND METHODS: We recruited 42 RRMS, 19 NMOsd and 35 NPSLE patients. Subjects were treated with beta-interferons or glatiramer acetate (RRMS), oral steroids and/or azathioprine (NMOsd, NPSLE), other immunosuppressants (NPSLE), or antimalarials (NPSLE). The clinical condition of the patients was assessed using the Kurtzke Expanded Disability Status Scale for MS and NMOsd, and the Systemic Lupus Erythematosus Disease Activity Index for NPSLE. Serum levels of sVCAM-1, sPECAM-1, sICAM-1 and S100B were determined using enzyme-linked immunosorbent assay (ELISA). RESULTS: We found the lowest levels of sPECAM-1, sICAM-1 and S100B in sera from NMOsd patients. The highest levels of sPECAM-1 and sICAM-1 were observed in NPSLE, and in NPSLE and MS, respectively. There were no statistically significant differences in sVCAM-1 levels between the examined groups. In MS and NMOsd, there was a negative correlation between the EDSS score and the following molecules: sPECAM-1, sICAM-1 and S100B. CONCLUSIONS AND CLINICAL IMPLICATIONS: We conclude that there is a different profile of blood-brain-barrier disruption reflected by cell adhesion molecules shedding in the spectrum of autoimmune CNS disorders with disseminated white matter lesions. These molecules could become new biomarkers to be used in CNS demyelinating diseases differential diagnoses and monitoring disease activity, but further studies on larger groups of patients are necessary.

2022 Systemic lupus erythematosus remission in clinical practice. Message for Polish rheumatologists.

Systemic lupus erythematosus (SLE) is a complicated multiorgan disease and can lead to organ damage and increased risk of morbidity and mortality. The strategy of management while avoiding complications, especially caused by chronic glucocorticoid therapy, improves outcomes. Different definitions of the treatment goal in different configurations of lupus activity indexes have appeared over the years. In 2021 the definition of remission and recommendations for its achievement were published and it become a way to implement a treat-to-target strategy. The main goal of treatment has become DORIS (definition of remission in SLE) remission and the alternative LLDAS (low lupus disease activity state). Prolonging remission with clinical and immunological lupus activity restrictions and minimizing or stopping steroid doses reduced flares and damage accrual. The analysis and neutralization of poor prognosis predictive factors in lupus could be the most beneficial for less morbidity and mortality and better quality of life.

Phase 2, randomized, placebo-controlled trial of dapirolizumab pegol in patients with moderate-to-severe active systemic lupus erythematosus.

OBJECTIVE: To evaluate the dose-response, efficacy and safety of dapirolizumab pegol (DZP) in patients with SLE. METHODS: Adults with moderately to severely active SLE (SLEDAI-2K score ≥6 and ≥1 BILAG A or ≥2 BILAG B domain scores), receiving stable CS (≤40 mg/day prednisone-equivalent), antimalarial or immunosuppressant drugs were included. Patients with stable LN (proteinuria ≤2 g/day) not receiving high-dose CS or CYC were permitted entry. Randomized patients received placebo or i.v. DZP (6/24/45 mg/kg) and standard-of-care (SOC) treatment every 4 weeks to week 24, after which patients received only SOC to week 48. The primary objective was to establish a dose-response relationship based on week 24 BILAG-Based Composite Lupus Assessment (BICLA) responder rates. RESULTS: All DZP groups exhibited improvements in clinical and immunological outcomes vs placebo at week 24; however, BICLA responder rates did not fit pre-specified dose-response models [best-fitting model (Emax): P = 0.07]. Incidences of serious treatment-emergent adverse events across DZP groups were low and similar to placebo. Following DZP withdrawal, SLEDAI-2K, physician's global assessment (PGA), BILAG, and Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) scores stabilized; BICLA and SLE Responder Index (SRI-4) responder rates declined (likely due to interventions with disallowed escape medications), BILAG flares increased, and immunologic parameters returned towards baseline. CONCLUSIONS: Although the primary objective was not met, DZP appeared to be well tolerated, and patients exhibited improvements across multiple clinical and immunological measures of disease activity after 24 weeks relative to placebo. The potential clinical benefit of DZP warrants further investigation.

Improving the quality of training paramedics by means of cadavers - a pilot study.

BACKGROUND: Paramedics are authorised to perform emergency procedures, including trauma assessment according to global standards. The aim of the study was to answer the question whether the use of cadavers in teaching practical competencies to medical rescue students, in the field of trauma assessment, is necessary as a supplement to learning in simulated conditions with the use of mannequins. METHODS: Research included several stages. The first stage was conduction of classes for 27 students in the field of rapid trauma assessment, in accordance with the guidelines of the International Trauma Life Support. In the second stage, a plan of a test in which students had to perform an analogous procedure of rapid trauma assessment, but with the use of cadavers, human unfixed specimens, was prepared. The Delphi method was used to develop and approve checklists, as well as a scale to assess the global correctness of identification of head, torso and limb injuries by medical rescue students. RESULTS: The identification rate was 76.54% in the head area, 67.90% in the torso area, while in the limb area it equalled 44.45%. A significant difference in scores, compared to the examination performed on a mannequin, was observed (Wilcoxon = 4.541; p = 0.000). The most difficult to make a correct diagnosis were injuries related to a fracture of the proximal end of the femur and a dislocated wrist (only 18.52% of correct answers). The students highly rated the usefulness of the examination, by awarding it an average of 4.76 points (SD ± 0.56) on the Likert scale (0-5). CONCLUSIONS: The study shows that the use of cadavers to teach practical competencies in the field of trauma assessment to medical rescue students can be an effective supplement to simulated learning. Students could feel the difference between the human body and the mannequin. More research is needed to assess whether realistic simulation translates into objective endpoints, such as the effectiveness of diagnosis in the examination of trauma patients. However, it should be remembered that the introduction of this teaching method is expensive and requires adequate base, as well as the compliance with a number of formal requirements.

The NACA score as a predictor of ventricular cardiac arrhythmias - A retrospective six-year study.

BACKGROUND: Helicopter Emergency Medical Service (HEMS) conducts the evaluation of a patient's condition using NACA score before transporting the patient to hospital. The conditions inside the rescue helicopter limit or even make it impossible to conduct some medical procedures. An appropriate classification of the patient may lead to a lower possibility of occurrence of adverse events during the flight. The aim of the research was to evaluate the correlation of NACA score with the cardiac arrhythmia that may be life threatening. METHODS: A retrospective observational study included a group of 47,131 patients, who were transported by HEMS services between 2012 and 2017. The research was conducted using the analysis of variance ANOVA running a post hoc test. In order to calculate the correlation of variables, Kruskal-Wallis and r-Pearson tests were carried out, interpreting the results according to J. Gilville's scale. The significance level was set at α = 0,05. RESULTS: The average number of points using NACA score for the studied group was 4,06 (SD ±â€¯1,38). Twelve heart rhythms were selected while evaluating correlations using NACA score. There was a significant relation between the ECG variable and NACA score (p = 0,003). There was a very strong correlation between NACA score and the following: VF/pVT (r-Pearson = 0,856; p = 0,006), PEA (r-Pearson = 0,810; p = 0,015) and Asystole (r-Pearson = 0,728; p = 0,026). CONCLUSIONS: NACA score allows to predict the risk of occurrence of ventricular arrhythmia of the myocardium as well as cardiac arrest. The possibility of occurrence of a life-threatening rhythm is significantly higher in patients classified as NACA IV or higher.

The use of analgesics in pediatric patients with body injuries in pre-hospital conditions.

BACKGROUND: Despite the positive dynamic changes in medicine and science, injuries continue to be the dominant cause of death among people under 45 years of age. Children's injuries are particularly dangerous as they can lead to hypovolemic shock, disability or even death. EMS uses opioid and nonopioid analgesics in order to provide relief to patients suffering from pain. The aim of this work is to analyze the types of pharmacological substances used in children in pre-hospital conditions. METHODS: The research tools included medical documentation of the emergency medical services in Poland. The analysis included 68,441 medical emergency treatment forms from 2016-2017, from which 464 interventions for children after trauma were selected. The general patient profile, characteristics of the event site, type of injuries suffered, type of intervening EMS, pharmacotherapy being used, as well as vital signs (GCS, RTS, HR, NiBP) were subject to assessment. The χ2 independence tests were performed, considering the results as statistically significant for p<0.05. RESULTS: There were 354 instances of BEMS (Basic Emergency Medical Services - without a doctor) and 110 instances of SEMS (Specialist Emergency Medical Services - with a doctor) sent to the emergency site. The mean age was 10.8 (SD ± 4.84) years. Boys were subject to intervention more often than girls (n=283 vs. n=181). Analgesics were administered in case of 49 (10.56%) patients only. NSAIDs (Nonsteroidal Anti-Inflummatory Drugs) were the analgesics of choice, which were used in 25 patients (50%), while opioid analgesics were used in 10 patients (20.40%). Analgesia was mostly used in patients with lower limb (34.69%) and upper limb (30.61%) injuries. The lowest percentage of analgesic use was observed in patients with head and neck injuries (3.06%). There was a significant relationship demonstrated between the type of EMS and the use of analgesics (χ2=6.330; p=0.012). CONCLUSIONS: Analgesia with EMS in children with injuries is critically insufficient. NSAIDs are the most commonly administered drugs in pre-hospital conditions. Head and torso injuries reduce the percentage of drugs administered by EMS. The EMS teams with no doctors (BEMS) are significantly more likely to use anesthetics, compared to SEMS.

Kikuchi-Fujimoto disease associated with primary Sjögren's syndrome - literature review based on a case report.

Primary Sjögren's syndrome (pSS) is a chronic, autoimmune disease predominantly involving exocrine glands. Lymphadenopathy is one of the possible symptoms of pSS. It may also suggest development of non-Hodgkin lymphoma (NHL), the most severe complication of pSS, or be a symptom of less common diseases, such as Kikuchi-Fujimoto disease (KFD), presented in this paper. Kikuchi-Fujimoto disease is an extremely rare, benign and self-limiting disorder, characterized by regional lymphadenopathy. This paper presents a case of previously unreported association of pSS, KFD and renal cancer in a patient with recurrent cervical lymphadenopathy, as well as a discussion on the coexistence of these diseases based on available literature searching for PubMed, Scopus and Google Scholar databases, particularly in this subject. These three clinical entities may manifest lymphadenopathy each, causing a diagnostic dilemma. The treatment is also challenging under such circumstances. In this particular situation, it was a combination of immunosuppressive therapy and surgery.

Work instability and associated factors among patients with rheumatoid arthritis in Greater Poland.

OBJECTIVES: Rheumatoid arthritis (RA) affects patients' capacity to work. The Rheumatoid Arthritis Work Instability Scale (RA-WIS) is a reliable method to measure work instability (WI) (1-3). We lack data on the relationship between RA and work instability among Polish patients. Our study aimed to assess WI and associated factors among patients with RA. MATERIAL AND METHODS: The authors conducted a multi-centre cross-sectional observational study. 315 patients from three rheumatology centres were enrolled and filled in questionnaires, including demographic and self-reported clinical data, RA-WIS, and the Health Assessment Questionnaire (HAQ). Swollen and tender joint counts (SJC, TJC) were assessed by the attending physician, and current erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP) were collected. We excluded 41 patients due to an incorrectly filled in form and analysed questionnaires of 274 patients. DAS28 (Disease Activity Score in 28 joints) and DAS28-CRP were calculated. We performed statistical analysis with Statistica v. 13.3 using the Mann-Whitney U test, χ2 test, and Spearman's correlation. RESULTS: 140 (51%) patients were currently employed and their characteristics were analysed. In univariable analysis we identified the following risk factors for high risk WI: moderate-to-high disease activity (DAS28 ≥ 3.2 - OR 2.29, 95% CI 1.06-4.96, p = 0.033; DAS28-CRP ≥ 3.2 - OR 2.34, 95% CI 1.04-5.27, p = 0.038), ESR ≥ 30 mm/h in women and ≥ 20 mm/h in men (OR 2.65, 95% CI 1.20-5.89, p = 0.010), CRP ≥ 1 mg/dl (OR 4.02, 95% CI 1.78-9.10, p < 0.001), HAQ-DI > 1.0 (OR 2.23, 95% CI 1.04-4.81, p = 0.037) and at least moderate pain on the visual analogue scale (VAS p ≥ 4.5 cm - OR 5.31, 95% CI 2.36-11.96, p < 0.001).Correlations were moderate between RA-WIS and VASp (RS = 0.59, p < 0.001) and HAQ-DI (RS = 0.52, p < 0.001) but weak with disease activity indices (DAS28 [RS = 0.31, p < 0.001]; DAS28-CRP [RS = 0.28, p < 0.001]). CONCLUSIONS: Pain and disability are the main factors strongly associated with work instability among patients with RA.

Nonstationary Two-Dimensional Nuclear Magnetic Resonance: A Method for Studying Reaction Mechanisms in Situ.

Nuclear magnetic resonance spectroscopy (NMR) is a versatile tool of chemical analysis allowing one to determine structures of molecules with atomic resolution. Particularly informative are two-dimensional (2D) experiments that directly identify atoms coupled by chemical bonds or a through-space interaction. Thus, NMR could potentially be powerful tool to study reactions in situ and explain their mechanisms. Unfortunately, 2D NMR is very time-consuming and thus often cannot serve as a "snapshot" technique for in situ reaction monitoring. Particularly difficult is the case of spectra, in which resonance frequencies vary in the course of reaction. This leads to resolution and sensitivity loss, often hindering the detection of transient products. In this paper we introduce a novel approach to correct such nonstationary 2D NMR signals and raise the detection limits over 10 times. We demonstrate success of its application for studying the mechanism of the reaction of AgSO4-induced synthesis of diphenylmethane-type compounds. Several reactions occur in the studied mixture of benzene and toluene, all with rather low yield and leading to compounds with similar chemical shifts. Nevertheless, with the use of a proposed 2D NMR approach we were able to describe complex mechanisms of diphenylmethane formation involving AgSO4-induced toluene deprotonation and formation of benzyl carbocation, followed by nucleophilic attacks.

Corynebacterium coyleae as potential urinary tract pathogen.

Corynebacterium coyleae is part of the commensal microflora of the skin, urethra, mucous membranes, and genital tract. Isolates from patients with urinary tract infection (UTI) were reported, but the pathogenic potential of this species has not been defined yet. The aim of the study is to determine whether C. coyleae could be the etiological agent of UTI and to analyze its antibiotic susceptibility. Urine samples were cultured quantitatively according to accepted laboratory procedures. The identification of bacterial isolates was carried out using the Vitek MS (bioMérieux) and antibiotic susceptibility was tested using disc diffusion according to EUCAST guidelines. Between 1 January 2017 and 30 October 2018, a total of 39 C. coyleae strains were isolated. This represented 0.32% of all urine samples cultured in the laboratory during the collection period. The strains were isolated from samples obtained from 35 women and 3 men (age median for all-64 years). One female patient presented with C. coyleae in her urine twice at an interval of 21 months. In six cases of UTI, C. coyleae was isolated in monoculture. The isolates had the same resistance pattern. A total of 11 strains were obtained from cases with a clinical diagnosis of UTI. In 13 cases, the strain was cultured in a monoculture and in 28 cases with accompanying species. All strains were susceptible to vancomycin. However, resistance to ciprofloxacin was observed for 58.4% of the strains. Urine isolates of C. coyleae must be considered as contamination or normal flora in most cases (28/39, 72%). In the remaining cases, it can be considered as potential etiologic agents, mostly in women and especially in the 6 UTI cases where C. coyleae was found as the single culture-positive species. Several of these isolates demonstrate resistance to antibiotics commonly used in empiric treatment of urinary tract infections.

Factors associated with quality of life in systemic sclerosis: a cross-sectional study.

INTRODUCTION: Systemic sclerosis (SSc) is a connective tissue disease characterized by progressive fibrosis of the skin and internal organs, leading to their failure and disturbances in the morphology and function of blood vessels. The disease affects people in different ways, and identifying how the difficulties and limitations are related to quality of life may contribute to designing helpful interventions. The aim of this study was to identify factors associated with quality of life in people with SSc. METHODS: This was a cross-sectional study conducted in 11 rheumatic centres in Poland. Patients diagnosed with SSc were included. Quality of life was measured using the SSc Quality of Life Questionnaire (SScQoL). The following candidate factors were entered in preliminary multivariable analysis: age, place of residence, marital status, occupational status, disease type, disease duration, pain, fatigue, intestinal problems, breathing problems, Raynaud's symptoms, finger ulcerations, disease severity, functional disability, anxiety and depression. Factors that achieved statistical significance at the 10% level were then entered into a final multivariable model. Factors achieving statistical significance at the 5% level in the final model were considered to be associated with quality of life in SSc. RESULTS: In total, 231 participants were included. Mean age (SD) was 55.82 (12.55) years, disease duration 8.39 (8.18) years and 198 (85.7%) were women. Factors associated with quality of life in SSc were functional disability (ß = 2.854, p < 0.001) and anxiety (ß = 0.404, p < 0.001). This model with two factors (functional disability and anxiety) explained 56.7% of the variance in patients with diffuse SSc and 73.2% in those with localized SSc. CONCLUSIONS: Functional disability and anxiety are significantly associated with quality of life in SSc. Interventions aimed at improving either of these factors may contribute towards improving the quality of life of people with SSc.

Polish Helicopter Emergency Medical Service (HEMS) Response to Stroke: A Five-Year Retrospective Study.

BACKGROUND A stroke is a serious life-threatening emergency that requires immediate intervention in an appropriate therapeutic center. The aim of this study was to analyze the time of medical procedures at the scene and changes in the state of stroke patients during transport by HEMS in Poland. The presented research is the first nationwide study covering such a large group of stroke patients, for whom aerial support was used in the therapeutic process. MATERIAL AND METHODS A retrospective cross-sectional study of 48553 missions performed by Polish Medical Air Rescue (PMAR) during the 5-year study period resulted in 3906 stroke patients who, after medical rescue operations by HEMS crew, were transported by helicopters to hospitals. RESULTS Helicopters in 3475 (88.97%) cases were utilized as a support for Ground Emergency Medical Service (GEMS). The maximum duration of HEMS operation from activation to patient transfer to the hospital did not exceed 108 min and the median was 60 min. Over 87% of patients with HEMS reported stroke symptoms and arrived at the medical center with the possibility of implementing thrombolytic therapy. The factor that affected the deterioration of patients' condition was the drawing out of the extent of time spent by the crew at the scene. CONCLUSIONS The use of HEMS in Poland in the case of patients with stroke symptoms ensures fast and professional assistance at the site of the medical emergency as well as safe transport to specialized centers, shortening the time of proper treatment implementation.

Maladaptive perfectionism and authentic leadership skills in emergency medicine students

Introduction: Leadership skills can be critical in emergency medicine. However, there are no works that analyze this issue in much more details. The aim: To analyze the level of leadership skills in emergency medicine students, and also checking if despondency perfectionism is a variable that reduce the correlation between self-efficacy and leadership skills. Material and methods: The analyzed group consisted of 75.76% of all emergency medicine students taking up education at the Medical University of Warsaw in 2018 (n = 150, W = 74, M = 71). The average age was 23 years (SD = 1.7). All students were divided into two groups: Group 0 - without maladaptive perfectionism (n = 64), and group 1- with maladaptive perfectionism (n = 79). In the cross-sectional study, three standardized research tools were used: Authentic Leadership Questionnaire, Almost Perfect Scale-Revised (APS-R), and General Self-Efficacy Scale (GSES). ANCOVA analysis was used. Results: The linear regression coefficients for both comparison groups were significantly different (interaction of variables: "maladaptive perfectionism * self-efficacy": F = 4.841, p = .029). Comparing adjusted mean values for both groups (0 vs 1), it can be stated that students from group 0 had a significantly higher level of authentic leadership skills compared to group 1 (F = 4.432, p = .037). Conclusions: Studies to determine the mechanisms of a positive relationship between the self-efficacy and leadership skills in emergency medicine students with high maladaptive perfectionism are required. This will allow the development of effective programs to strengthen the leadership skills of these students.

Autologous hematopoietic stem cell transplant for progressive diffuse systemic sclerosis: procedural success and clinical outcome in 5-year follow-up.

Systemic sclerosis is an autoimmune connective tissue disease affecting both skin and internal organs. Progressive disease with multiple organ involvement is considered to have a poor prognosis. Treatment possibilities are limited, but certain patients may benefit from autologous hematopoietic stem cell transplantation (auto-HSCT). We report a case of a 30-year-old woman with progressive diffuse systemic sclerosis treated with parenteral cyclophosphamide with unsatisfactory results. Due to progression of the disease and lack of alternative therapies auto-HSCT was performed. After instituting treatment with autologous hematopoietic stem cell transplantation no immunosuppressive therapy has been required during 5-year follow-up. Improvement in exertion tolerance, partial regression of skin lesions and stabilization of pulmonary and cardiovascular changes were observed. Currently therapeutic options in patients with progressive systemic sclerosis are limited. Hematopoietic stem cell transplantation might become an alternative therapeutic solution not only in the early phase of the disease but also among selected patients with progressive systemic sclerosis resistant to standard therapy.

Dual inhibition of tumour necrosis factor and interleukin-17A with ABT-122: open-label long-term extension studies in rheumatoid arthritis or psoriatic arthritis.

Objectives: To evaluate the safety and maintenance of efficacy with ABT-122, a bi-specific monoclonal antibody targeting TNF and IL-17A, in patients with RA or PsA in open-label, 24-week extensions [open-label extensions (OLEs)] of 12-week, randomized, double-blind studies. Methods: All patients received ABT-122 (RA, 120 mg; PsA, 240 mg) subcutaneously every other week on background MTX. Safety assessments included adverse events (AEs) and laboratory parameters. Efficacy was evaluated with ACR responses, 28-joint DAS using high-sensitivity CRP [DAS28 (hsCRP)], and Psoriasis Area and Severity Index (PsA study). Results: The RA OLE study enrolled 158 patients; the PsA OLE study enrolled 168 patients. In the RA OLE study, the incidence of treatment emergent AEs (TEAEs; 41%) appeared similar to the double-blind study (36-43%). In the PsA OLE study, 57% of patients reported ⩾1 TEAE (double-blind study, 42-53%). Most TEAEs were mild or moderate in severity. There were no neutrophil abnormalities greater than grade 2. Grade 3 and/or 4 laboratory abnormalities were reported for lymphocytes, alanine aminotransferase, aspartate aminotransferase, bilirubin and haemoglobin; the number of these severe laboratory values was low (0.6-3.0%), except grade 3 lymphocyte count decreased (11.5%) in the RA study. In both OLE studies, efficacy assessed by ACR responses and other disease activity scores was maintained over the 24 weeks. Conclusion: ABT-122 demonstrated acceptable tolerability and maintenance of efficacy for up to 36 weeks in patients with RA or PsA receiving background MTX. Trial registration: ClinicalTrials.gov, http://clinicaltrials.gov, NCT02433340 and NCT02429895.

Tofacitinib in the treatment of patients with rheumatoid arthritis: position statement of experts of the Polish Society for Rheumatology.

Tofacitinib is a newly approved small-molecule targeted synthetic disease-modifying antirheumatic drug. The drug was designed as a selective and specific inhibitor of pro-inflammatory receptor signalling. Tofacitinib inhibits the process of intracellular signalling from the receptor to the cellular nucleus and inhibits the inflammation process via a new pathway (inhibition of the Janus kinases), which is unavailable to biological medicines. Tofacitinib has been approved for use in the treatment of patients with moderate to severe active RA. The drug may be used in combination with methotrexate or another conventional synthetic disease-modifying antirheumatic drug or in monotherapy. The efficacy of tofacitinib has been confirmed in several clinical trials. The drug inhibits radiographic progression of the disease. The innovative mechanism of action of tofacitinib is a noteworthy feature because it offers hope of effective treatment for patients who fail to respond to other drugs. The presented article discusses the mechanism of action and the clinical application of tofacitinib. Tofacitinib represents a new group of disease-modifying antirheumatic drugs that can be placed on an equal footing with biological drugs already available.

Satisfaction and discontent of Polish patients with biological therapy of rheumatic diseases: results of a multi-center questionnaire study.

OBJECTIVES: Biologics are medications widely applied in the management of inflammatory rheumatic diseases. The drugs were found to be effective but their application is associated with some disadvantages. Medication with biologics is relatively expensive, and in Poland, it is carried out in specialized centers. The study was designed to evaluate various aspects of satisfaction and dissatisfaction of Polish patients treated with biologics. MATERIAL AND METHODS: An anonymous questionnaire was distributed in 23 Polish rheumatological centers involved in the treatment; 1212 returned questionnaires were used for analysis. Responses were received from 606 patients with rheumatoid arthritis, 427 with ankylosing spondylitis, 117 psoriatic arthritis, and 62 adult patients with juvenile idiopathic arthritis (in whom administration of the drugs had been introduced before they were 18 years old). The investigated group constituted about one-fifth of all rheumatic patients on biologics in Poland. RESULTS: A beneficial or very beneficial influence of the medication on the state of physical health was found mostly in patients with rheumatoid arthritis (51.3 and 30.5%) and ankylosing spondylitis (51.0 and 36.8%). Family life was improved by the treatment especially in patients with ankylosing spondylitis (40.7 and 35.6% beneficial and very beneficial, respectively), sleep quality and sexual life mostly in those with ankylosing spondylitis (beneficial/very beneficial influence 41.5/38.4, and 38.7/23.9, respectively). There was a rather small influence of biological treatment on the financial situation of the patients. In general, satisfaction with the treatment was evaluated as positive or very positive in 88% of all investigated patients.In a significant part of the patients, transportation to the medical center was considered as a disadvantage of the treatment. About one-third of the patients considered laboratory and imaging tests to be done before initiation of the medication as a difficulty, and for about 40% waiting time for qualification for the medication was a significant disadvantage. The route of drug administration was without importance for 4/5 of the patients. CONCLUSIONS: Summing up, the results were similar in the patients suffering from various diseases although those with psoriatic arthritis felt the highest satisfaction (possibly due to the positive aesthetic effect), and those with ankylosing spondylitis had significant improvement in sexual life (probably due to younger age). Relatively low satisfaction was found in patients with juvenile idiopathic arthritis. There was a small influence of medication on financial status of the patients. Application of biologics has few disadvantages and most of them are associated with the organization of health services (waiting time for the tests, transportation to the medical centers).