Hereditary angioedema (HAE), arising from a deficiency of C1 inhibitor (C1-INH), clinically manifests as swelling of the subcutaneous tissue and mucous membranes. The standard prophylactic therapy consists of intravenous (i.v.) substitution of C1-INH. We present a case study of successful long-term subcutaneous (s.c.) prophylaxis in a patient who had to discontinue i.v. prophylaxis.

Over the past decades, hemophilia treatment has made significant progress—from factor therapy to prophylactic regimens and non-factor products, and even to gene therapy. Its development has greatly improved the quality of life for hemophiliacs and reduced the incidence of bleeding episodes. However, they cannot be completely eliminated, which is why further options for monitoring bleeding and joint status are still being explored. In addition to classical methods, biomarkers are attracting attention, but their practical use is still the subject of studies. A recently published work by a team of European and American experts, who assessed collagen turnover as a biomarker of treatment efficacy with rurioctocog alfa pegol, came with interesting results.

We present a case study of a patient with non-small cell lung cancer (NSCLC) with a proven activating mutation of the epidermal growth factor receptor (EGFR) gene, who developed resistance to the first-generation EGFR tyrosine kinase inhibitor (TKI) gefitinib during treatment. After the secondary mutation EGFR T790M was confirmed, osimertinib, a third-generation EGFR TKI, was introduced, resulting in disease regression, which is ongoing. This case study illustrates the significance of the role of osimertinib in the treatment of patients with NSCLC with an activating EGFR mutation, whose disease progresses during treatment with first and second-generation EGFR TKIs due to the emergence of the secondary EGFR T790M mutation.

Alpha-1 antitrypsin deficiency (AATD) plays a significant role in the pathogenesis of pulmonary emphysema, chronic obstructive pulmonary disease (COPD), and bronchiectasis. However, its deficiency may also play a role in other lung diseases.

Hereditary angioedema (HAE) can be encountered in emergency rooms and specialist offices. Patients with this rare disorder are often misdiagnosed with a more common condition, and they may learn their correct diagnosis only after many years. This is because HAE manifests with a combination of symptoms that are typical of various other diseases. What should pediatricians, otorhinolaryngologists, and gastroenterologists be aware of? Providing better understanding of HAE symptoms from different specializations' perspectives and emphasizing the importance of interdisciplinary collaboration for accurate diagnosis and patient care was the focus of a professional webinar we have summarized for you.

The number of patients with type 2 diabetes mellitus (DM2) and heart failure is increasing worldwide, and the combination of both diseases significantly worsens the prognosis of patients. One of the main causes of increased mortality in these patients is macrovascular complications of DM2. The burden on the cardiovascular system is even greater with the concurrent occurrence of chronic heart failure.

At the psychiatric symposium titled 'Chance for a Better Quality of Life with a Schizophrenia Diagnosis', which took place on October 18, 2021, in Prague, Professor of Psychiatry and Molecular Biology Christoph U. Correll from the Donald and Barbara Zucker School of Medicine at Hofstra/Northwell spoke. His lecture included insights on current approaches to schizophrenia therapy and key information on one of the newest antipsychotics -⁠ lurasidone.

A beneficial impact on social functioning is an important outcome of schizophrenia treatment. A recently published Japanese study is the first to demonstrate the efficacy of lurasidone compared to placebo on the social functioning of patients with schizophrenia. The results are comparable to other atypical antipsychotics.

Osteopenia or osteoporosis are known comorbidities of hemophilia and concern many of these patients. What all contributes to their development?

The effect of two commonly used antihypertensives was evaluated in a randomized clinical trial in patients with primary hypertension and COVID-19.

In the pivotal phase III RECOURSE randomized placebo-controlled study, therapy with trifluridine/tipiracil (FTD/TPI) was associated with improved overall survival and progression-free survival in pre-treated metastatic colorectal cancer (mCRC) patients. The below-presented TACTIC study provides prospective data from German real-world practice.

Standard prophylaxis for bleeding in hemophilia A, based on the administration of coagulation factor VIII (FVIII), depends on the patient's weight, the severity of the coagulation factor deficiency, and the nature of bleeding episodes. The current trend, however, is an individualized approach in prophylaxis, which can improve clinical outcomes and optimize bleeding disorder therapy. A clinical study evaluated the efficacy and safety of two prophylactic regimens with different target trough levels of FVIII.

In Phase III clinical trials, teriflunomide demonstrated its ability to slow down brain tissue atrophy in patients with relapsing-remitting multiple sclerosis (RRMS). What impact did the treatment have on the cognitive state of these patients?

Rurioctocog alfa pegol represents a modern modality for the treatment of hemophilia from the group of factors with an extended half-life. The authors of a systematic review and meta-analysis published last year assessed its efficacy and safety in patients previously treated for hemophilia A.

The primary analysis of results from the APHINITY clinical trial supported the combined anti-HER2 treatment with trastuzumab and pertuzumab in combination with chemotherapy in patients with early HER2-positive breast cancer. At the end of last year, efficacy and safety results of this treatment after more than 6 years of follow-up were presented.