Publications by authors named "R A P Raymakers"
Article Synopsis
- The immune response plays a crucial role in diseases like cancer and autoimmune disorders, affecting disease progression and treatment outcomes.
- A new technology called STAP-STP allows for simultaneous measurement of activity in different signaling pathways of immune cells by analyzing their mRNA.
- This technology was used to create distinct activity profiles for various immune cell types and was applied in a clinical study on rheumatoid arthritis, highlighting its potential for diagnosing and developing treatments for immune-related diseases.
Article Synopsis
- - Dyskeratosis congenita (DC) is a rare inherited condition that leads to bone marrow failure and is largely linked to mutations affecting telomere biology, with about 35% of cases having unidentified genetic causes.
- - Research on a wide range of DC and 'DC-like' cases uncovered new pathogenic variants, including findings in the novel X-linked gene POLA1 and in known genes POT1 and ZCCHC8, enhancing the understanding of the genetic basis of these disorders.
- - Functional studies indicated that the new variants in POLA1 and POT1 disrupt crucial protein interactions that are essential for telomere maintenance, while ZCCHC8 variants lead to inflammation in patients, thereby contributing to the understanding of
The classical BCR::ABL1-negative myeloproliferative neoplasms (MPN) form a group of bone marrow (BM) diseases with the potential to progress to acute myeloid leukemia or develop marrow fibrosis and subsequent BM failure. The mechanism by which BM fibrosis develops and the factors that drive stromal activation and fibrosis are not well understood. Cellular Communication Network 2 (CCN2), also known as CTGF (Connective Tissue Growth Factor), is a profibrotic matricellular protein functioning as an important driver and biomarker of fibrosis in a wide range of diseases outside the marrow.
View Article and Find Full Text PDFArticle Synopsis
- A phase 3 clinical trial evaluated the effectiveness and safety of adding eltrombopag to standard immunosuppressive therapy (horse ATG plus cyclosporine) in treating patients with severe aplastic anemia.
- Results showed a higher complete response rate at 3 months (22% with eltrombopag vs. 10% without) and improved overall response rates at 6 months (68% vs. 41%).
- The addition of eltrombopag enhanced the treatment's efficacy without increasing severe side effects, suggesting it could be a beneficial option for newly diagnosed patients.