Background. In recent years, due to the progress of approaches for allogeneic hematopoietic stem cell transplantation (allo-HSCT), as well as the active development of bone marrow registries, the number of transplants has been steadily growing. Clinical outcomes after allo-HSCT depends on many factors, including ethnic compatibility in a patient-donor pair. There is evidence that allo-HSC performed from national donors have a better outcome compared with transplantation from donor’s research in international registers. There are suggestions that this may be due to the fact that not only individual HLA allele disparities, but also non-HLA polymorphisms in the major histocompatibility complex have an impact on clinical outcomes.

Aim. We undertook a study to define results of HSCTs using Russian donor registry compared to international ones.

Materials and methods. This retrospective study included 585 patients transplanted from HLA-matched unrelated donors at the RM Gorbacheva Research Institute. Allo-HSCT was performed from international donors in 444 of patients and from Russian donor in 141. All patients had post-transplant cyclophosphamide in GVHD prophylaxis. A paired difference test was also performed, and the groups were matched by their pretransplant characteristics: age, sex, diagnosis, conditioning regimen, graft source, the proportion of salvage transplantation and year of transplantation.

Results. With the median follow-up of 17 months (1-74) we obtained comparable results of allo-HSCT from Russian and international donors: overall survival was 64% and 62% respectively (р=0,19), event-free survival- 57% and 43% respectively (р=0,14), cumulative incidence of non-relapse mortality - 17% and 18% respectively (р=0,62), cumulative incidence of relapse- 12% and 20% (p = 0,08). In paired difference test, we received similar comparable results in both groups. There were no significant differences in the incidence of acute and chronic GVHD.

Conclusion. In this study we demonstrate comparable long-term results of allo-HSCT from Russian and international donors. In this regard, further development of the Federal Registry of bone marrow donors will make allo-HSCT more accessible to the citizens of the Russian Federation, which is especially relevant in the context of difficulties with logistics, financial and time costs when applying to international donors.

Aim. To study sociomedical profile and quality of life in patients with paroxysmal nocturnal hemoglobinuria (PNH) with a suboptimal response to C5i therapy as well as to test usefulness of FACIT-Fatigue to evaluate fatigue and its impact on everyday life in this patients’ population.

Materials and methods. PNH patients with suboptimal response to current C5i treatment participated in multicenter cross-sectional study. All the patients filled out EQ-5D and FACIT-Fatigue as well as checklist with questions about common PNH symptoms, demographics, disease, treatment and work status. Methods of descriptive statistics, Mann-Whitney test, χ2 criterion and Spearman correlations were used for analysis.

Results. In total, 45 patients with PNH from 12 medical centers of RF were included in the analysis (classic PNH – 64.4%). Median age was 41 years, 62% – female. The mean duration of suboptimal response was 3.2 years. The majority of patients reported problems in mobility (55.6%), anxiety/depression (62.2%), and usual activities (68.9%). The majority of patients reported that PNH affected moderately or significantly their everyday activities and social/family life. Overall, 61% of employed patients mentioned that PNH moderately or significantly affected their productivity at work. The majority of patients experienced fatigue (96%), headaches (86%), shortness of breath (83%), difficulty focusing, sleeping difficulties (82%), rapid heart (77%), yellowing of the skin (76%), back pain (71%). Fatigue was <40 scores by FACIT-Fatigue in 70% patients. Statistically significant rela­tionships were found between FACIT-Fatigue score and EQ-5D VAS as well as severity of dyspnea, rapid heart, loss of concentration.

Conclusions. Sociomedical profile and quality of life impairment in PNH patients with suboptimal response to C5i have been demonstrated. FACIT-Fatigue is an informative questionnaire to assess fatigue and its impact on everyday life in this patients’ population.

Myelofibrosis (MF) is a chronic myeloproliferative neoplasm characterized by bone marrow fibrosis, anemia, extramedullary hematopoiesis, and splenomegaly. The overall survival of patients with myelofibrosis is significantly lower than that of the general population, partly due to constitutional symptoms that considerably impair the quality of life of patients, in particular, life-threatening thrombosis in the portal vein system and Budd-Chiari syndrome. In phase III clinical trials, ruxolitinib provided rapid and sustained reduction in splenomegaly and improvement in constitutional symptoms. Despite this, the role of ruxolitinib therapy in reducing the risk of thrombosis in this patient category remains unknown. The aim of this study is to evaluate the effectiveness and safety of ruxolitinib therapy in patients with primary myelofibrosis and Budd-Chiari syndrome, portal vein thrombosis. The article describes our own successful experience in using this drug in patients with thrombosis in the portal vein system, hepatic veins, and those with transplanted livers. Monitoring of side effects when using ruxolitinib and immunosuppressive drugs, dose modification leads to rapid stabilization of patients' somatic conditions, reduction in spleen size, alleviation of constitutional symptoms, and control of graft-versus-host disease (GVHD).