Bone Marrow Transplant. 2025 Sep 20. doi: 10.1038/s41409-025-02710-2. Online ahead of print.
ABSTRACT
Bone marrow failure (BMF) syndromes (BMFSs) are a group of disorders characterized by the bone marrow’s inability to produce sufficient blood cells. Over the years, diagnostic and therapeutic approaches have significantly evolved, now progressing towards personalized treatment strategies. Traditionally, these disorders include both acquired forms and inherited conditions. The inherited conditions, in particular, are becoming increasingly complex, with new genetically-defined BMFSs being identified beyond the well-known Fanconi Anemia (FA), Dyskeratosis Congenita (DKC), Diamond-Blackfan Anemia (DBA) and Shwachman-Diamond syndrome (SDS). A dedicated conference, supported by leading international scientific networks, such as the European School of Hematology (ESH), European Bone Marrow Transplantation (EBMT), European Hematology Association (EHA), and the International PNH Interest Group (IPIG), was recently organized to discuss and to emphasize how our knowledge in BMFs field has improved, eventually becoming a topic of interest for general hematologists and those involved in hematopoietic cell transplantation (HCT). This manuscript highlights some key findings from this 3rd ESH-EBMT-EHA-IPIG Translational Research Conference, focusing on the pathogenic mechanisms of BMFSs and their secondary malignancies, as well as advances in pathophysiology-driven treatments for both acquired and inherited BMFSs.
PMID:40975757 | DOI:10.1038/s41409-025-02710-2
