The Need for Treatment of Neovascular Age-Related Macular Degeneration: A Study Based on the Polish National Registry.

Introduction: The Polish National AMD Therapeutic Program offered us a unique opportunity to determine the need for treatment of neovascular age-related macular degeneration (nAMD).

Methods: A search, extraction, and analysis of data from the monitoring system of the Therapeutic Program of the National Health Fund was performed. Demographic data from the Central Statistical Office were also obtained and analyzed.

Hearing Loss as the Main Clinical Presentation in -Associated Autoinflammatory Disease.

The gene mutations are the cause of autosomal dominant autoinflammatory disorders (NLRP3-AID). Recently, hearing loss (HL) has been found to be the sole or major manifestation of NLRP3-AID. Here, we tested 110 autosomal dominant HL families with a custom panel of 237 HL genes and found one family carrying the c.

Early or late tracheotomy in patients after multiple organ trauma.

The analysis of the study group of 124 patients revealed a statistically significant shortening of mechanical ventilation requirement period in patients in whom tracheotomy had been performed before hospitalization day 10 (G1). The average length of mechanical ventilation was shorter by 20.3 days in G1 as compared to G2.

Three-Year Outcomes of Wet Age-Related Macular Degeneration Treatment in Polish Therapeutic Programs.

: Wet age-related macular degeneration (wAMD) is a chronic, progressive disease of the central part of the retina. Standard treatment for wAMD consists of multiple intravitreal injections of anti-vascular endothelial growth factor drugs. The study goal was to evaluate the three-year effectiveness of wAMD treatment with aflibercept and ranibizumab as part of the therapeutic program in routine clinical practice.

Anakinra and canakinumab for patients with R92Q-associated autoinflammatory syndrome: a multicenter observational study from the AIDA Network.

Background: This study aims at describing the therapeutic outcome of patients carrying the R92Q variant in the gene treated with anakinra (ANA) or canakinumab (CAN) and identifying any factors predictive of complete response to IL-1 inhibition.

Methods: Clinical data of patients treated with ANA or CAN for recurrent inflammatory attacks due to the presence of the R92Q variant were retrospectively collected and analysed.

Results: Data about 20 treatment courses with IL-1 inhibitors (16 with ANA and 4 with CAN) from 19 patients were collected.

Biologicals in childhood severe asthma: the European PERMEABLE survey on the .

Introduction: Severe asthma is a rare disease in children, for which three biologicals, anti-immunoglobulin E, anti-interleukin-5 and anti-IL4RA antibodies, are available in European countries. While global guidelines exist on who should receive biologicals, knowledge is lacking on how those guidelines are implemented in real life and which unmet needs exist in the field. In this survey, we aimed to investigate the and identify open questions in biological therapy of childhood asthma across Europe.

Tumor Necrosis Factor Receptor-Associated Periodic Syndrome (TRAPS) with a New Pathogenic Variant in Gene in a Family of the Adult Male with Renal AA Amyloidosis-Diagnostic and Therapeutic Challenge for Clinicians.

Tumor necrosis factor receptor-associated periodic syndrome (TRAPS) belongs to systemic autoinflammatory diseases (AIDs). Many of these syndromes are genetically conditioned and can be inherited. Diagnosis relies on clinical symptoms and should be confirmed by genetic testing.

Secondary immunodeficiencies with predominant antibody deficiency: multidisciplinary perspectives of Polish experts.

At present, secondary immune deficiencies have become a clinical problem, recognized in different specialties. The aim of this paper was to increase awareness and support the need for screening at-risk populations. Secondary immune deficiencies result in variety of conditions, but not all of them require immunoglobulin replacement therapy, as specific antibody response might be preserved.

Effects of vitamin D on macrophages and myeloid-derived suppressor cells (MDSCs) hyperinflammatory response in the lungs of COVID-19 patients.

Vitamin D regulates homeostasis, anti-microbial response, and inflammation. The vitamin D receptors are expressed in the macrophages and other immune cells, regulating the transcription of many different genes, including those coding the anti-microbial peptides. One of the most severe complications of the SARS-CoV-2 infection is the acute respiratory distress syndrome (ARDS) caused by the hyperinflammatory response (commonly called cytokine storm) of the lung macrophages.

Hematopoietic Stem Cell Transplantation Positively Affects the Natural History of Cancer in Nijmegen Breakage Syndrome.

Purpose: Nijmegen breakage syndrome (NBS) is a DNA repair disorder with a high predisposition to hematologic malignancies.

Experimental Design: We describe the natural history of NBS, including cancer incidence, risk of death, and the potential effectiveness of hematopoietic stem cell transplantation (HSCT) in preventing both pathologies: malignancy and immunodeficiency.

Results: Among 241 patients with NBS enrolled in the study from 11 countries, 151 (63.

Schnitzler Syndrome in a 27-Year-Old Man: Diagnostic and Therapeutic Dilemma in Adult Auto-Inflammatory Syndromes A Case Report and Literature Review.

A 32-year-old-man, with a history of chronic urticaria from the age of 27, diagnosed with an adult-onset Still's disease and received a low dose of glucocorticoids, methotrexate and tocilizumab. Despite the long-term combined treatments, he suffered from chronic urticaria, low-grade fever and bone pain. He was found to have high inflammatory markers, hypogammaglobulinemia, monoclonal IgM - kappa light chain in serum and increased radiotracer uptake in the whole bone scintigraphy.

Tonsillectomy in own material.

<b>Introduction:</b> Tonsillectomy belongs to the most frequently performed surgical treatments; however, the necessity of its performance is questioned. Therefore, there are many attempts to unify and define the indications for the procedure. <br><b>Aim:</b> The main objective of the current dissertation was an analysis of the clinical symptoms occurring in patients qualified for tonsillectomy, as well as a comparison of those with a histopathological image of the removed tonsils in a repeatedly carried out, unified pathomorphological examination.

From infections to autoimmunity: Diagnostic challenges in common variable immunodeficiency.

Common variable immunodeficiency (CVID) is the most common clinically significant primary antibody deficiency diagnosed in adults. The early symptoms are not specific. They include common infections, mainly of the respiratory tract, caused by typical microorganisms, so cases can be missed in primary care.

Facilitated Subcutaneous Immunoglobulin Replacement Therapy in Clinical Practice: A Two Center, Long-Term Retrospective Observation in Adults With Primary Immunodeficiencies.

Facilitated subcutaneous immunoglobulin (fSCIG) replacement therapy is the latest method of IgG administration; however, real-life data are limited. We retrospectively analyzed the everyday experience of fSCIG administration, particularly, the method used to switch from intravenous immunoglobulin (IVIG) or subcutaneous immunoglobulin (SCIG) to fSCIG and the dosing modifications required. Of the 39 adult patients with primary immunodeficiency (PID) who received fSCIG, 34 remained on the therapy at the end of the study.

Shorter Diagnostic Delay in Polish Adult Patients With Common Variable Immunodeficiency and Symptom Onset After 1999.

Common variable immunodeficiency (CVID) is the most clinically significant primary antibody immunodeficiency recognized in adulthood. Previously published data have shown an average diagnostic delay of 10 years for Polish adult patients with CVID. In the current study, we aimed to analyze the current diagnostic delay of adult patients with CVID in Poland.

One-year outcomes of the Polish treatment program for the wet form of age-related macular degeneration using intravitreal therapy.

Purpose: To report 12-month outcomes of a Polish National Treatment Program using aflibercept and ranibizumab in eyes with wet, age-related macular degeneration in routine clinical practice.

Material And Methods: This was a non-randomized, retrospective, observational multicenter study. Anonymous data contained in the electronic Therapeutic Program Monitoring System were utilized in this study.

The identification and management of interstitial lung disease in systemic sclerosis: evidence-based European consensus statements.

Background: Systemic sclerosis-associated interstitial lung disease (ILD) carries a high mortality risk; expert guidance is required to aid early recognition and treatment. We aimed to develop the first expert consensus and define an algorithm for the identification and management of the condition through application of well established methods.

Methods: Evidence-based consensus statements for systemic sclerosis-associated ILD management were established for six domains (ie, risk factors, screening, diagnosis and severity assessment, treatment initiation and options, disease progression, and treatment escalation) using a modified Delphi process based on a systematic literature analysis.

Periodic fever, aphthous stomatitis, pharyngitis and cervical adenitis syndrome persisting to adulthood - an example of a diagnostic and therapeutic challenge.

Periodic fever, aphthous stomatitis, pharyngitis and cervical adenitis (PFAPA) syndrome is the most common cause of periodic fever in childhood. Reports of adult patients are sparse. In adults the clinical picture is more heterogeneous than in children, so PFAPA can be a real diagnostic challenge.

Determination of antibodies in everyday rheumatological practice.

Antibody determination is routinely used in everyday rheumatological practice. Its result repeatedly determines the diagnosis or exclusion of a particular disease. Antibodies are immunoglobulins, i.

A case report of pregnancy in a patient with common variable immunodeficiency emphasizing the need for personalized immunoglobulin replacement.

Rationale: Subcutaneous immunoglobulin administration facilitated by recombinant human hyaluronidase is a new mode of immunoglobulin replacement. It has been approved for treatment in primary and secondary antibody immunodeficiency. To date, it has not been reported in the literature as therapy of choice during pregnancy.

Effect of Phacoemulsification on Visual Acuity and Macular Morphology in Patients with Wet Age-Related Macular Degeneration.

BACKGROUND This article discusses the effectiveness of phacoemulsification cataract surgery with intraocular lens implantation in patients with wet age-related macular degeneration in the inactive phase of the disease. MATERIAL AND METHODS Forty-nine patients (50 eyes) aged 78.94±5.

The Cross-Talk Between Sphingolipids and Insulin-Like Growth Factor Signaling: Significance for Aging and Neurodegeneration.

Bioactive sphingolipids: sphingosine, sphingosine-1-phosphate (S1P), ceramide, and ceramide-1-phosphate (C1P) are increasingly implicated in cell survival, proliferation, differentiation, and in multiple aspects of stress response in the nervous system. The opposite roles of closely related sphingolipid species in cell survival/death signaling is reflected in the concept of tightly controlled sphingolipid rheostat. Aging has a complex influence on sphingolipid metabolism, disturbing signaling pathways and the properties of lipid membranes.

Drug Policy in Poland.

We presented a general overview of the health care system as well as the pricing and reimbursement environment in Poland. Poland aims to ensure proper access to safe and effective medicines while reducing patients' share in treatment costs. Nevertheless, the co-payment for pharmacotherapy is still high (more than 60%).