Observational Study on Treatment, Quality of Life, and Disease Progression in Myelofibrosis with Anemia

In myelofibrosis, excessive scar tissue (fibrosis composed of reticulin and collagen fibers) forms in the bone marrow. This displaces the blood-forming tissue and disrupts normal blood formation in the bone marrow, often leading to anemia—a deficiency of red blood cells. A distinction is made between a primary form, which occurs on its own, and secondary forms, which can develop from other chronic blood disorders, particularly polycythemia vera or essential thrombocythemia. In the treatment of myelofibrosis, stem cell transplantation from donors is the only potentially curative option and is primarily used in younger patients who are in good general health, as the treatment is associated with significant side effects. For patients for whom a cure is not possible, so-called JAK inhibitors are available. These are medications that inhibit Janus kinases (JAK), a signaling pathway that is persistently overactive in myelofibrosis. This study includes both patients who have never been treated with a JAK inhibitor and those who have already received one.

The goal of this observational study is to systematically document, over several years, how patients with myelofibrosis and anemia are treated in everyday clinical practice, how their disease progresses, and what effects the disease and/or treatment have on their quality of life. The study does not interfere with treatment but rather records, across multiple study centers, how patients are actually treated in everyday clinical practice. At the outset, information on the disease, comorbidities, disease characteristics, and prior treatments is collected. As the study progresses, all systemic (whole-body) treatments and their outcomes will be documented. A particular focus is on health-related quality of life, which is reported directly by the patients themselves and assessed using a standardized questionnaire. In total, patients are followed for up to three years.

Eligible participants are patients aged 18 and older with primary or secondary myelofibrosis who are experiencing anemia at the time of enrollment and who are starting their first or subsequent systemic treatment for myelofibrosis. Additional requirements include a willingness to participate in the quality-of-life surveys and consent to be included in an accompanying registry (GSG-MPN Bioregistry). The signed consent form for RHODOLITE must be submitted no later than six weeks after the start of the respective systemic treatment.