Bad Liebenwerda

Immune thrombocytopenia (ITP) is a rare blood disorder where the immune system causes a shortage of platelets, leading to increased bleeding risk. New treatment options have emerged recently, but clinical studies often focus on specific patient groups. This research collects real-world data from a broad range of ITP patients to better understand the diagnosis, treatment, and outcomes in everyday care. The study also aims to improve personalized therapy and patient results by gathering detailed clinical and biospecimen information. The study involves creating a national registry where clinical data and biospecimens are collected from patients diagnosed with primary or secondary ITP. Data are gathered prospectively at defined points during the disease course, and patients can also be included retrospectively within 12 months of diagnosis if ongoing documentation is available. This includes information about disease factors, treatment types, complications, quality of life, fatigue, and survival over 5 years. Participants will provide written consent and undergo clinical assessments at enrollment and follow-up visits. Researchers will collect epidemiological data such as disease incidence, age and sex distribution, causes, treatment types, and remission status over 5 years. The registry also includes biospecimen collection to support high-quality, standardized research. This ongoing monitoring will help improve knowledge of ITP and support better patient care.

Cholangiocarcinoma is a rare and aggressive bile duct cancer with limited treatment options and a poor outlook. This study investigates the use of ivosidenib, a targeted IDH1 inhibitor, which was approved in May 2023 by the European Medicines Agency for adults with locally advanced or metastatic cholangiocarcinoma carrying an IDH1 R132 mutation, who have already received at least one prior systemic therapy. The study aims to collect real-world data on the effectiveness, safety, and impact on quality of life of ivosidenib treatment. Ivosidenib will be given to patients according to the current prescribing information (SmPC). This is a prospective, multicenter, observational study conducted in Germany, enrolling a broad patient population receiving ivosidenib as part of routine care. Treatment is not assigned by the study but is observed in real-world settings, allowing for the collection of comprehensive data on this therapy. Participants will be monitored for up to 38 months to measure progression-free survival, among other outcomes. Data on quality of life and safety will also be collected. Patients will provide informed consent, and those opting into the patient-reported outcomes module will complete assessments in German. The study will provide valuable insights into how ivosidenib performs outside of clinical trials.

Researchers are evaluating treatment options for unstable odontoid fractures in adults aged 70 years and older. This study compares surgery and rigid collar bracing to determine which leads to better recovery in daily activities, pain control, disability, and quality of life. It aims to provide clear evidence about which treatment supports improved functional outcomes and healing for older adults with these fractures. Participants are randomly assigned to either surgical stabilization using a posterior C1-C2 screw-rod system or conservative treatment with a rigid cervical collar worn for 12 weeks. Surgery typically lasts about 100 minutes and requires a hospital stay of around 6 days without additional external bracing. The conservative group will record collar use in diaries to monitor compliance. Patients are followed for 12 weeks and 6 months, with an extra visit about 2 weeks after surgery for those who undergo the procedure. Those in the collar group may switch to surgery within 12 weeks if needed. During the study, participants will attend visits at 12 weeks and 6 months to complete questionnaires on daily functioning, pain, and quality of life. CT scans and medical assessments will be performed to monitor fracture healing and complications. Researchers will track treatment adherence, any crossover to surgery, and adverse events. The main outcome measured is change in the Barthel Index from baseline to 12 weeks, which reflects recovery in daily living activities.

Researchers are studying the use of venetoclax in people with chronic lymphocytic leukemia (CLL) to understand how effective it is and to assess the costs and patient-reported outcomes when used in real-life medical settings. The study looks at venetoclax given alone or combined with rituximab, obinutuzumab, ibrutinib, or acalabrutinib, based on doctors' decisions and local treatment guidelines. Participants receive venetoclax therapy as prescribed by their physician, which may include the drug alone or along with one of the mentioned combination treatments. The study follows patients in Austria, Germany, and Switzerland, observing how the treatments are used in everyday practice without altering the prescribed therapy. During the study, participants are monitored for up to 12 months to measure the percentage who achieve the best overall response rate (ORR) to treatment. Researchers also collect data on health economics and patient experiences to better understand the impact of these therapies in routine care.