Fanconi anemia (AF) is a hereditary genetic disease characterized by developmental abnormalities, progressive bone marrow failure, hypersensitivity to alkylating agents, and a tendency to hematological and solid tumors throughout life. The only curative option in the treatment of bone marrow failure in patients with AF is allogeneic hematopoietic stem cell transplantation (allo-HSCT). There are no detailed descriptions of allo-HSCT in patients with AF in the Russian-language literature. On the example of a clinical case with AF at the onset of myelodysplastic syndrome, a choose of method for treating bone marrow failure is presented.
There is no doubt that allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the most effective treatments for many serious diseases. However, despite significant progress, allo-HSCT is still associated with a high rate of complications and mortality in the posttransplant period due to the toxicity of conditioning regimens, infectious and immune conditions. Acute complications such as endothelial injury, acute and chronic graft-versus-host disease (GVHD) remain the main causes of mortality after allo-HSCT. In our clinical case, we demonstrated an example of the development of such life-threatening complications as transplant-associated thrombotic microangiopathy and GVHD in a patient after repeated allo-HSCT, as well as the successful relief of these complications by modern therapeutic methods, including the introduction of closely related donor mesenchymal stem cells and the complement blocker eculizumab.
Despite improved understanding of the biology of the disease and the use of multicomponent chemotherapy, the prognosis for children with relapsed or refractory B-line acute lymphoblastic leukemia (B-ALL) remains poor. Currently, the only definitive treatment for these patients is allogeneic hematopoietic stem cell transplantation (allo-HSCT), which can be performed after achieving immunohematological remission. Conducting highintensity polychemotherapy (PCT) blocks to achieve negative values of minimal residual disease (MRD) is often limited due to high toxicity. The developed monoclonal antibodies targeting cell surface antigens, such as CD19 and CD20, are actively used in children with relapsed/refractory B-ALL as part of “bridge therapy”, which allows achieving MRD-negative status without the use of intensive chemotherapy. However, new strategies are needed to improve the prognosis of these patients. The drug Inotuzumab ozogamicin has demonstrated efficacy in relapses of B-ALL and is actively used to achieve a negative MRD status before the allo-HSCT stage in children. In the presented article, in addition to a brief review of the literature, clinical experience with the use of this drug is demonstrated.
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