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.

Background. Epidemiological studies aggregating data on newly diagnosed multiple myeloma (MM) from real-world clinical practice allow to analyze various clinical and laboratory parameters characterizing general trends in MM diagnosis, treatment outcomes, and life expectancy. Aim. To determine overall survival rates in patients with newly diagnosed MM and to examine factors influencing survival in a population-based study. Materials and methods. A total of 3,184 patients with a newly diagnosed MM from 40 regions of Russia were enrolled in a multicenter prospective study from January 2, 2015, to October 4, 2018. Complete registration of newly diagnosed cases was conducted at each of the 44 participating centers. Information on clinical and laboratory parameters, antitumor therapy regimens administered to patients, including autologous transplantation, was analyzed. Results. The median age of MM patients in Russia was 63 years. It was shown that 39 % of the patients belonged to the older age group 66–90 years, and the percentage of patients under 55 years was 22 %. Only 9 % of patients underwent bone marrow cytogenetic testing. The proportion of patients with kidney damage was 26 %, while 5 % of patients were dialysis dependent. It was shown that 78 % of patients were diagnosed with stage 3 according to Durie–Salmon system, and in 52 % of cases, stage III according to International Staging System was diagnosed. Induction therapy included the use of bortezomib in 94 % of cases, the VCD regimen (bortezomib + dexamethasone + cyclophosphamide) was mainly used, lenalidomide was prescribed extremely rarely – in 1 % of cases. The proportion of patients under 65 years of age who received at least one autologous hematopoietic stem cell transplant was only 18 %. With autologous hematopoietic stem cell transplantation, the median overall survival was 107 months, and in the case of maintenance therapy with lenalidomide, it increased to 129 months. Five-year overall survival of the entire group of patients was 40 %. In a multivariate analysis, independent predictors of worse overall survival were Durie–Salmon stage III, substage B, profound anemia (hemoglobin ≤80 g/L), albumin ≤30 g/L, and hypercalcemia (≥3 mmol/L). Conclusion. This multicenter prospective study of the epidemiological and clinical parameters of newly diagnosed multiple myeloma is unique in Russia in terms of coverage and follow-up duration (over 10 years). Analysis of treatment data for newly diagnosed multiple myeloma identified key issues in diagnosis and treatment.

BACKGROUND. The etiology of plasmacytomas in multiple myeloma (MM) remains unclear, whereas the pathogenetic mechanisms of extramedullary lesions are of particular importance because of extremely poor prognosis. The incidence of bone and extramedullary plasmacytomas complicating the course of MM as well as the efficacy of therapy for this disease are still underresearched. AIM. To assess the incidence of bone and extramedullary plasmacytomas in patients with newly diagnosed MM (ndMM) and analyze the overall survival (OS) rates depending on whether soft-tissue components were detected. MATERIALS & METHODS. This multi-center prospective study enrolled 3184 ndMM patients (1339 men and 1845 women) from 40 regions of Russia, aged 24–90 years and treated during the period from January 2015 to October 2018. The electronic patient records included demographic, clinical, laboratory, and instrumental documentation data accessible on MM diagnosis date. Bortezomib-based regimens were administered in the first-line therapy to 94 % of patients. RESULTS. Bone and extramedullary plasmacytomas were diagnosed in 763 (24 %) out of 3184 ndMM patients. At disease onset, the median hemoglobin concentration was significantly higher in patients with bone (105 g/L) and extramedullary (102 g/L) plasmacytomas than in patients without them (95 g/L; p < 0.05). The median percentage of plasma cells in the bone marrow was significantly lower in cases of bone plasmacytoma accounting for 25 % vs. 30 % in cases of extramedullary components, and it was 31 % in patients without plasmacytomas at MM onset (p < 0.05). Most commonly, plasmacytomas were detected in the thoracic spine (24.3 %; n = 132) and ribs (13.6 %; n = 74). The median OS of patients without plasmacytomas was 46 months (95% confidence interval [95% CI] 43–49 months) vs. 38 months (95% CI 42–46 months) in patients with them (p = 0.1245). The median OS was 17 months (95% CI 10–35 months) in patients with extramedullary plasmacytomas and 13 months (95% CI 1–69 months) if soft-tissue components were detected in the bones of the lower extremities. CONCLUSION. The course of ndMM is complicated by bone plasmacytomas in 23 % of cases and extramedullary plasmacytomas in 1 % of cases. The antitumor response rate as well as the OS rates are similar in patients with and without bone plasmacytomas. Extramedullary lesions in MM are associated with extremely low rate of OS with the median not exceeding 17 months.

AIM. To assess the changes in quality of life and symptom profile in polycythemia vera (PV) patients during ruxolitinib treatment as well as to study the efficacy and safety of this drug in a real-world setting. MATERIALS & METHODS. This multi-center prospective observational study enrolled 50 PV patients from 9 medical centers of the Russian Federation (median age 58 years, male/female ratio 1:1). All patients had indications for ruxolitinib therapy. The median disease duration was 48.3 months. Splenomegaly was observed in 86 % of cases; stratification of patients yielded an intermediate/high-risk group with 76 % and low-risk group with 24 % of them. Patients completed SF-36 and MPN10 questionnaires prior to treatment and then 1, 3, 6, 9, and 12 months after therapy onset along with a patient satisfaction checklist. Clinic response was assessed in accordance with the National Clinical Guidelines. The total MPN10 score decrease by ≥ 50 % during the treatment was regarded as a symptom-related response. The statistical analysis of the parameters changing over time was performed using generalized estimating equations (GEE). RESULTS. Ruxolitinib therapy for 12 months resulted in pronounced positive changes in quality of life and symptom reduction in patients with both intermediate/high and low risk of thrombotic complications (p < 0.001). Therapy response with total MPN10 score decrease ≥ 50 % was observed in 59 % of patients after 1 month and in 70 % of patients in subsequent treatment periods. Nine months after therapy onset, a complete clinical and hematologic response was achieved in 57.8 % and a partial one in 37.8 % of patients; after 12 months it was reported in 59.2 % and 36.7 % of patients, respectively. Hematocrit improvement was achieved in more than 85 % of cases in different treatment periods. Adverse events (AEs), commonly grade 1/2, were identified in 11 (22 %) patients, and serious AEs (ischemic stroke and second tumor) were detected in 2 patients. During the follow-up period, the vast majority of PV patients reported improved well-being and satisfaction with ruxolitinib treatment outcomes. CONCLUSION. In a real-world setting, ruxolitinib therapy showed good clinical efficacy, favorable safety profile as well as pronounced positive changes in quality of life and disease symptoms throughout 12 months after therapy onset.

Introduction . Before the introduction of targeted agents, multiple myeloma (MM) was characterized by low overall survival (OS) rates. Survival during the first year of life is a clear indicator of the effectiveness of not only antitumor therapy but also accompanying therapy. Analysis of the cause of death in the MM patient population will allow for refined approaches to diagnosis, antitumor treatment, and accompanying therapy. Aim : to determine the one-year OS rates in patients with newly diagnosed MM, to study the cause of death, and to estimate the probability of death within the first year after diagnosis. Materials and Methods . This multicenter prospective study, conducted from January 2015 to October 2018, included 3,184 patients (1,339 men and 1,845 women) with newly diagnosed MM from 40 regions of Russia, aged 24–90 years. One-year OS, one-year mortality, and causes of death were analyzed. Results . During the follow-up period, 2,307 deaths were recorded, 261 patients were lost to follow-up, and 616 patients continue to receive antitumor therapy or remain untreated. The median OS of patients with MM was 44 months, with a 5-year OS probability of 40 % and a 10-year OS probability of 17 %. Among 2,307 patients with documented fatal outcomes, causes of death were available in 1,350 cases. In 55.7 % (n = 752) of cases, death was determined to be due to MM progression, in 19 % (n = 257) to infectious complications, in 8.2 % (n = 111) to acute cardiovascular failure, and in 4 % (n = 54) to acute kidney injury. Pulmonary embolism was documented in 3.6 % of patients (n = 49), and mesenteric vascular thrombosis was diagnosed in 0.3 % (n = 4) of cases. Acute cerebrovascular accident, both ischemic and hemorrhagic, was diagnosed in 3.7 % (n = 50) of cases resulting in death. A second malignancy was the primary cause of death in 1.6 % (n = 22) of patients with MM. The one-year OS rate for patients newly diagnosed with myeloma from 2015 to 2018 was 82 %. The 5-year probability of death from myeloma progression was 31 %, while the 1-year probability was 9 %. Meanwhile, the 5-year probability of death from causes unrelated to myeloma progression was 45 %, while the 1-year probability was 11 %. Conclusion . The probability of death from causes unrelated to refractory myeloma was comparable across all age cohorts. This highlights the need for knowledge regarding concomitant therapy and the correction of comorbid factors. Differences in the one-year OS of patients with myeloma were demonstrated depending on the registration center, due to differences in access to second- and subsequent-line anticancer drugs. It is important to note the need to develop a separate treatment strategy for the cohort of patients with myeloma diagnosed in old age.