The effect multiple sclerosis (MS) has on the social functioning and integration of patients has been recently considered as an important factor of the disease. To assess social participation of MS patients with regard to demographic and disease-related variables. The study comprised 201 MS patients: 140 women, 61 men, aged 24-69 years. The World Health Organization Disability Assessment Schedule (WHODAS 2.0) was applied to assess the aspects of social functioning and the Beck Depression Inventory (BDI) was applied to evaluate the level of depression. Disease duration, degree of disability in Expanded Disability Status Scale (EDSS), most disabling symptoms and type of treatment were determined. WHODAS 2.0 scores (total and within particular domains) and their relationships with age, gender, disease-related variables and level of depression were analyzed. The results of WHODAS 2.0 for 27.4% of patients exceeded the 90th percentile compared to the population norms (with the highest scores for "getting around" and "participation in society" domains). The results of BDI and WHODAS 2.0 were strongly correlated (p < 0.001; β = 0.73) and mobility impairment was related to both of them (p < 0.001; β = -0.12 and 0.25, respectively). Other disabling symptoms were associated with scores in domains "understanding and communicating", "getting around" and "participation in society". Social participation of the MS patients is affected by the impact of disease and associated with depression. Particular symptoms of neurological deficit (motor and visual impairment, fatigue) influence social functioning more than general disease-related variables.

Background. Kidney diseases are the main causative factors of secondary hypertension (HTN) in children. Although primary HTN is less common in the pediatric population, its increasing prevalence, especially among teenagers, makes early diagnosis an emerging issue. Objectives. To analyze the potential differences between primary HTN and HTN secondary to renal diseases, in order to tailor diagnostic procedures to pediatric patients with suspicion of HTN. Material and methods. A retrospective evaluation was performed of medical records of 168 children (aged from 1 month to 18 years) diagnosed with arterial HTN in the Pediatric Nephrology Department of Wroclaw Medical University (Poland). The comparative analysis concerned demographics, causes of HTN, clinical picture, laboratory tests, and parameters of ambulatory blood pressure monitoring (ABPM). Results. Out of 168 children, 47% were diagnosed with primary HTN and 53% with secondary renal HTN. The patients with primary HTN were significantly older than those with HTN secondary to renal disease. Among the children with primary HTN, 26% were overweight and 42% were obese; among those with renal HTN, the proportions were 16% and 19%, respectively. The patients with primary HTN had significantly higher body mass index (BMI) percentiles and z-scores, and tended toward higher pulse pressure (PP) values. In the group with secondary HTN, ABPM parameters of diastolic blood pressure (DBP) and total cholesterol were significantly elevated. The BMI z-scores correlated positively with PP in the whole group. Conclusions. As expected, HTN secondary to renal disease prevails in younger children, but primary HTN has become an emerging issue in teenagers. The diagnostics of HTN secondary to kidney disease have revealed risk factors worsening the prognosis, including higher values of cholesterol or of parameters connected with DBP. Primary HTN risk factors include obesity and a tendency towards higher PP values.

The optimal bridging therapy before CAR-T cell infusion in pediatric relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r BCP-ALL) still remains an open question. The administration of blinatumomab prior to CAR-T therapy is controversial since a potential loss of CD19+ target cells may negatively impact the activation, persistence, and, as a consequence, the efficacy of subsequently used CAR-T cells. Here, we report a single-center experience in seven children with chemorefractory BCP-ALL treated with blinatumomab before CAR-T cell therapy either to reduce disease burden before apheresis (six patients) or as a bridging therapy (two patients). All patients responded to blinatumomab except one. At the time of CAR-T cell infusion, all patients were in cytological complete remission (CR). Four patients had low positive PCR-MRD, and the remaining three were MRD-negative. All patients remained in CR at day +28 after CAR-T infusion, and six out of seven patients were MRD-negative. With a median follow-up of 497 days, four patients remain in CR and MRD-negative. Three children relapsed with CD19 negative disease: two of them died, and one, who previously did not respond to blinatumomab, was successfully rescued by stem cell transplant. To conclude, blinatumomab can effectively lower disease burden with fewer side effects than standard chemotherapeutics. Therefore, it may be a valid option for patients with high-disease burden prior to CAR-T cell therapy without clear evidence of compromising efficacy; however, further investigations are necessary.

Introduction: Posaconazole is recommended for prophylaxis in pediatric immunocompromised patients. Due to its variability in bioavailability and drug-to-drug interactions, EBMT recommends regimens based on therapeutic drug monitoring (TDM). Materials and methods: In total, 171 oncohematological pediatric patients on posaconazole prophylaxis were included. Full pharmacokinetic posaconazole profiles were assessed in 51 children. The efficacy and safety of posaconazole was evaluated by measuring the drug concentration, with dose modification attempted in patients with suboptimal results. The influence of modifying factors on the posaconazole plasma concentration (PPC) was investigated. Results: An insufficient PPC was the main issue, but no significant increase in prophylaxis failure was reported. The modification of the dosage resulted in the optimization of PPC in 50% of patients. No significant correlation between age, gender, diagnosis or the posaconazole dosage and the PPC was found. HCT, total parenteral nutrition and diarrhea were associated with a lower PPC. Hypoalbuminemia was related to both higher and lower PPC. The concomitant administration of specified drugs significantly impacted the PPC. Conclusions: TDM allows the identification of patients receiving non-optimal treatment and offers an opportunity to improve the efficacy and safety of the therapy. However, further research involving larger patient groups and longer observation periods are needed to determine the optimal dosing and target PPC in pediatric patients.

CD19-targeted CAR-T cell therapy has revolutionized the treatment of relapsed/refractory (r/r) pre-B acute lymphoblastic leukemia (ALL). However, it can be associated with acute toxicities related to immune activation, particularly cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). Cytokines released from activated immune cells play a key role in their pathophysiology. This study was a prospective analysis of proinflammatory proteins and cytokines in children treated with tisagenlecleucel. Serial measurements of C-reactive protein, fibrinogen, ferritin, IL-6, IL-8, IL-10, IFNγ, and TNFα were taken before treatment and on consecutive days after infusion. The incidence of CRS was 77.8%, and the incidence of ICANS was 11.1%. No CRS of grade ≥ 3 was observed. All complications occurred within 14 days following infusion. Higher biomarker concentrations were found in children with CRS grade ≥ 2. Their levels were correlated with disease burden and CAR-T cell dose. While cytokine release syndrome was common, most cases were mild, primarily due to low disease burden before lymphodepleting chemotherapy (LDC). ICANS occurred less frequently but exhibited various clinical courses. None of the toxicities were fatal. All of the analyzed biomarkers rose within 14 days after CAR-T infusion, with most reaching their maximum around the third day following the procedure.

Background and aim of the study: Developmental prosopagnosia (DP) is defi ned asimpaired face recognition in the absence of brain injury or intellectual defi cit. Becauseof the complexity of validated face memory and perception tests, its online distributiongreatly improves the effectiveness and convenience of conducting research. Assessmentof DP occurrence in the Polish population concerning study sample collection andcharacteristics.Material and methods: An online questionnaire consisting of the 20-item ProsopagnosiaIndex (PI20) was administered to assess self-reported problems with face recognition.Cambridge Face Memory Test (CFMT) and Glasgow Face Matching Test (GFMT)were applied to assess respondents’ face memory and perception. Additional data onrespondents’ gender, age and handedness were also obtained. All of the components werearranged together using Google Forms tool. Respondents were recruited via social mediaannouncements. The study was conducted from 1st January to 31st March 2019. Each ofthe respondents was provided with personalised results analysis, which was sent with ane-mail. They were also able to stay in contact with research authors, asking additional questionsand giving remarks.Results: During the study, a total of 1349 questionnaires were collected, out of which1276 met inclusion criteria and were enrolled in the study. 66.1% of the respondent groupwere females and 33.9% males. The mean age was 28.3 ± 9.5 years (range: 14-75). Themean PI20 score was 49.6 ± 18 (range: 20-99). In 11.8% of respondents PI20 result indicatedself-reported mild, in 8.3% moderate and in 3.9% severe DP. The mean CFMT totalscore was 58.1 (median: 59, range: 26-72) and mean GFMT score was 33.9 (median: 34,range: 18-40). According to the cutoff values from original papers, 81 (6.3%) of respondentsscored below threshold in CFMT, 27 (2.4%) in GFMT. Out of 50 respondents whoself-reported symptoms indicating severe DP, only 15 (30%) scored below the cutoff inCFMT and 6 (12%) in GFMT. On the contrary, 30 (3.1%) and 16 (1.4%) out of 970 respondentsreporting no problems with face recognition had total scores below the cutoff inCFMT and GFMT, respectively.Conclusions: The estimated prevalence of developmental prosopagnosia in Polishpopulation is higher than in normative data. These discrepancies may result from specifi cdemographics of participant group more than an online-character of the study. It is important to focus on experiment design and methodology in order to maintain high quality ofcollected data. Development of country-specifi c norms and stimuli for face-recognitiontests should also be considered .