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Pediatric myelodysplastic syndrome (MDS) are a heterogeneous group of clonal disorders often occur in the context of inherited bone marrow failure syndromes, acquired aplastic anemia or gene predisposition. Germ line syndromes predisposing individuals to develop familial MDS or acute myeloid leukemia have recently been identified – mutations in RUNX1, ANKRD, GATA2, ETV6, SRP72, DDX41. Juvenile myelomonocytic leukemia (JMML) occurs in context of inherited and somatic mutations PTPN11, KRAS, NRAS, CBL, NF1. In pathogenesis of these disorders there are a several factors – hypermethylation, clonal hematopoiesis/cytopenia of undetermined significance, disturbances of bone marrow microenvironment, telomeres, immune mechanisms. Allogeneic hematopoietic stem cell transplantation is the main method of MDS and JMML treatment but it is necessary to take into account special indications for refractory cytopenia (infections, dependence on blood transfusions) and be careful for JMML with CBL mutation.

Allogeneic hematopoietic stem cell transplantation (allo-SCT) is used widely in the management of children with hematological, oncological and inherited diseases. Study to compare efficiency of steroid-refractory acute graft versus host disease treatment (extracorporeal photopheresis (ECP) vs anticytokine therapy) was conducted in Raisa Gorbacheva Memorial Institute of Children Oncology, Hematology and Transplantation, First Pavlov State Medical University of Saint Petersburg. Sixty four children were included in the analysis. Patients were divided into two groups: first “group with ECP” (n = 31; 50,5 %) ("ECP group") and second - "group without ECP" (n = 33; 49,5 %). Treatment in the second group consisted of anticytokine therapy (etanercept (n = 12), infliximab (n = 9), daclizumab (n = 8)) and alemtuzumab (n = 4). Ten-year overall survival (OS) of children with steroid-refractory acute graft versus host disease was 39 % without difference in OS between ECP and anticytokine therapy (5-year OS 40 % vs 35 %, p = 0,34). Response rate to the therapy was also the same in both groups (68 % after ECP and 70 % after anticytokine therapy, p = 0,77). Difference in cumulative incidence of relapse in "ECP group" and "group without ECP" was not statistically significant (18 % and 7 %, respectively, p = 0,2). In conclusion, ECP and anticytokine therapy are equally effective in the treatment of children with steroid-refractory acute graft versus host disease and are associated with the same cumulative incidence of relapse.

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