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Results: Serum OPG concentrations increased throughout the study period (p = 7.26x10 -5 ) while RANKL concentrations did not change (p = 0.19). The OPG:RANKL ratio exhibited a 6.2-fold increase between Days 1 and 5. CTX-1 concentrations were lower (p = 0.006) 30 minutes after the 50 U/kg FVIII infusion. CTX-1 response for each participant was assessed using linear regression throughout the time course. Spine L1-L4 Z-score (SZ) and Hip Total Z-score (HTZ) correlate with CTX-1 response (p = 3.4x10 -4 and 0.014, respectively). The mean age of participants was 25.2 ± 2.1 at time of first study visit. Participants had a mean SZ of −0.74 ± 0.34, HTZ of −0.17 ± 0.32, Hip Neck Z-score (HNZ) of −0.16 ± 0.35, and HJHS of 20 ± 5. HTZ and HNZ decreased significantly with patient age (p = 0.027 and 0.032) while SZ and HJHS did not (p = 0.18 and 0.16). Consequently, age correction was applied to BMD comparisons. Statistical comparisons between HAL, EQ-5D-3L, and BHQ answers, BMD data, and HJHS are shown in Table 1. Summary/Conclusion: This prospective study demonstrates a relationship between FVIII deficiency and bone disease in PwH. OPG acts as a decoy ligand to RANKL, inhibiting osteoclastic bone resorption. The observed increase in OPG:RANKL ratio suggests FVIII has a direct impact on this pathway. Decreased CTX-1 concentrations following factor infusion and correlations between CTX-1 response and BMD further suggests that FVIII plays a role in moderating bone remodeling. Hemophilia-associated bone and joint pathology is associated with decreased quality of life. Correlations between BMD and questionnaire responses demonstrate that poor bone and joint health is physically limiting, causes discomfort, and negatively impacts perceptions of personal well-being. Furthermore, correlations between BMD and HJHS and ISTH-BAT responses (Table 1) illustrate a relationship between bleeding management and skeletal health. PwH with lower BMD report more nose and gum bleeds, suggesting a link between FVIII replacement and skeletal health. Bleeds in the extremities were not correlated with decreased BMD or increased HJHS, but abdominal (stomach, iliopsoas) and other bleeds were. Analysis of these data is ongoing.

Introduction. Primary immune thrombocytopenia (idiopathic thrombocytopenic purpura, ITP) is a rare autoimmune disorder, whose main clinical manifestation consists in the hemorrhagic syndrome of varying severity. The therapy for ITP is aimed at eliminating hemorrhagic manifestations and maintaining patients’ quality of life. There is no information on the incidence of ITP, as well as on the clinical course and response to the treatment of ITP patients, in the Russian Federation.Aim. To estimate the epidemiological and clinical characteristics of ITP in the adult population of the Russian Federation.Materials and methods. We studied clinical and laboratory data derived from electronic medical records of ITP patients having participated in a multi-centre, prospective, observational, cohort study “Epidemiological and Clinical Characteristics of ITP in Adults in Russia” carried out from 2014 to 2017. The data was processed employing methods of descriptive statistics, as well as frequency analysis, event analysis and analysis of variance. The calculations were performed using the SAS V9.4 statistical package.Results. The incidence of ITP in the adult population in three selected regions of the Russian Federation averaged 2.09 per 100 thousand people per year. The highest incidence rate in gender-age groups was observed in women under 40 years of age mounting to 2.7 per 100 thousand. A gradual increase in the incidence rate from 1.09 to 2.5 per 100 thousand was noted in the men over 60 years of age. A significant correlation was found between the risk of developing the hemorrhagic syndrome, its severity and the platelet count at the onset of the disease (p<0.0001). In 92.2 % of cases, glucocorticosteroid hormones were prescribed as the first line of therapy. The analysis of methods used as the second line of therapy revealed that, although splenectomy is performed relatively frequently, its likelihood has decreased from 26 to 17 % over the past three years. In addition, the proportion of patients being treated with thrombopoietin receptor agonists has increased from 5.9 to 45.7 %.Conclusion: The incidence of ITP in three selected regions of the Russian Federation is comparable to that in other European countries.Conflict of interest: the authors declare no conflict of interest.