Choroideremia (CHM) is a rare genetically conditioned retinal disease caused by mutations in the CHM gene, leading to a lack of production of the protein REP1 (ras-associated binding escort protein 1). Currently, there is no approved treatment for CHM. The aim of the study (Tübingen Choroideremia Gene Therapy) published last year in the journal JAMA Ophthalmology was to evaluate the safety and efficacy of subretinally applied gene therapy using an adeno-associated virus vector (AAV2), designed to deliver a functional copy of the CHM gene to patients with choroideremia.

MUDr. Věra Geierová from the Institute of Hematology and Blood Transfusion in Prague prepared a summary of what was discussed about emicizumab during the ASH 2020 congress for this year's Prague Hematology Days. In this context, she highlighted the comprehensive analysis of phase 3 HAVEN 1–4 studies, which assessed the safety and efficacy of emicizumab in individuals with hemophilia A with or without FVIII inhibitors.

Thanks to the expansion of modern therapies for chronic lymphocytic leukemia, many patients now have regimens based on venetoclax. Do you want to help them better understand the practical aspects and principles of this therapy? For this purpose, the Czech Group for Chronic Lymphocytic Leukemia (ČSCLL) in collaboration with PharmDr. Petra Rozsívalová from the Department of Clinical Pharmacy of the Faculty of Medicine, Charles University, and the University Hospital Hradec Králové has compiled a new brochure. It clearly and simply informs how this treatment works, the available dosages, how to use it correctly, and the most common side effects. Of course, it includes many practical tips, such as what to do if a patient forgets to take a dose, which foods interact with the treatment, and whether it affects the efficacy of vaccinations.

Malnutrition represents a potentially serious complication for cancer patients. If it is not possible to meet the nutritional demands of the body through the enteral route, parenteral nutrition must be initiated. A doctor will recommend it in situations where the patient is at risk from the consequences of malnutrition. Up to 45% of these patients struggle with at least a 10% loss of their body weight.

Ursodeoxycholic acid (UDCA) is the cornerstone treatment for PBC according to the guidelines of the European Association for the Study of the Liver (EASL). However, many patients (25–50%) do not achieve an adequate response to it, and some patients develop intolerance leading to the necessity of discontinuation (in the phase III POISE study, this concerned 7% of participants). Then, obeticholic acid (OCA) is indicated, which is recommended as the only registered second-line treatment option for PBC by current professional guidelines both European (EASL 2017) and Czech (2018). The latest data on OCA from clinical studies as well as real-world practice was also presented at this year's XLVIII May Hepatology Days.

Overall survival of patients with advanced-stage follicular lymphoma (FL) who experience early disease progression after chemotherapy remains low. Currently used clinical prognostic models do not provide optimal sensitivity and specificity for identifying this patient subgroup. The presented study by an international team of authors introduced a new assessment index that achieves better outcomes.

Several studies and meta-analyses in the past have pointed to a possible positive effect of low-molecular-weight heparins (LMWH) in the treatment of cancer. The RASTEN study therefore focused on the potential effect of enoxaparin in patients with small-cell lung cancer (SCLC).

The Journal of Wound Care published the results of an observational study on the benefits of Resposorb Super superabsorbent dressings in the treatment of acute and chronic wounds with higher levels of exudate.

Diabetes mellitus (DM) is a risk factor for the development of nephrolithiasis. Recent studies associated with nephrolithiasis suggest a possible beneficial effect of sodium-glucose cotransporter 2 inhibitors (SGLT2i, also known as gliflozins), a significant group of modern antidiabetics. Below we summarize findings from a study that explored this potential in empagliflozin using conducted phase I–IV studies, including EMPA-REG OUTCOME.

Combined treatment with liposomal pegylated irinotecan, 5-fluorouracil (5-FU), and leucovorin (LV) significantly improves overall survival in patients with metastatic ductal carcinoma of the pancreas. Analysis of the NAPOLI clinical trial revealed prognostic markers for long-term survival in patients treated this way.

In December of last year, the Czech multicenter research database on COPD was completed. This was an extensive 5-year project in which researchers included 784 patients with significant chronic obstructive pulmonary disease (FEV₁ ≤ 60%) from 14 centers. This represents approximately 1% of the total number of patients with this diagnosis in the Czech Republic, making the study sample fairly representative for obtaining data on treatment in Czech real practice. Three-year data are currently available. What have they shown so far? This was summarized during this year's Hradec Pulmonology Days by Dr. Jaromír Zatloukal, Ph.D., from the Clinic of Pulmonary Diseases and TB at the Faculty of Medicine, Palacký University and University Hospital Olomouc.

Up to 2% of the population suffers from leg ulcers. The standard treatment for ulcers of venous etiology is compression. The following French study, whose results were recently published in the International Journal of Angiology, examined its effectiveness and safety in patients with mixed (arteriovenous) etiology ulcers.

Compression therapy represents an important part of the treatment of venous leg ulcers; it also plays a role in preventing possible recurrences. The 4-year British ESCHAR study evaluated the impact of combining compression therapy with surgical correction of reflux in the superficial venous system on the healing and potential recurrence of venous leg ulcers compared to compression therapy alone.

The expert consensus published in February 2024 offers guidance on the use of bimekizumab in the treatment of patients with moderate to severe plaque psoriasis and psoriatic arthritis. It is based on a literature review and an assessment of data from 19 relevant studies. A panel of 9 American dermatologists specializing in psoriasis treatment created and adopted 14 consensus statements and recommendations, 12 of which have a strength of “A”.