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- Title
Pediatric Bone Marrow Failure: A Broad Landscape in Need of Personalized Management.
- Authors
Vissers, Lotte T. W.; van der Burg, Mirjam; Lankester, Arjan C.; Smiers, Frans J. W.; Bartels, Marije; Mohseny, Alexander B.
- Abstract
Irreversible severe bone marrow failure (BMF) is a life-threatening condition in pediatric patients. Most important causes are inherited bone marrow failure syndromes (IBMFSs) and (pre)malignant diseases, such as myelodysplastic syndrome (MDS) and (idiopathic) aplastic anemia (AA). Timely treatment is essential to prevent infections and bleeding complications and increase overall survival (OS). Allogeneic hematopoietic stem cell transplantation (HSCT) provides a cure for most types of BMF but cannot restore non-hematological defects. When using a matched sibling donor (MSD) or a matched unrelated donor (MUD), the OS after HSCT ranges between 60 and 90%. Due to the introduction of post-transplantation cyclophosphamide (PT-Cy) to prevent graft versus host disease (GVHD), alternative donor HSCT can reach similar survival rates. Although HSCT can restore ineffective hematopoiesis, it is not always used as a first-line therapy due to the severe risks associated with HSCT. Therefore, depending on the underlying cause, other treatment options might be preferred. Finally, for IBMFSs with an identified genetic etiology, gene therapy might provide a novel treatment strategy as it could bypass certain limitations of HSCT. However, gene therapy for most IBMFSs is still in its infancy. This review summarizes current clinical practices for pediatric BMF, including HSCT as well as other disease-specific treatment options.
- Subjects
BONE marrow; HEMATOPOIETIC stem cell transplantation; GRAFT versus host disease; APLASTIC anemia; PAROXYSMAL hemoglobinuria; GENE therapy
- Publication
Journal of Clinical Medicine, 2023, Vol 12, Issue 22, p7185
- ISSN
2077-0383
- Publication type
Article
- DOI
10.3390/jcm12227185