Unit. Patients were aged 24-85 years (median 68), and were diagnosed as having essential thrombocythemia (ET) (n = 149, 45%), polycythaemia vera (PV)(n = 94, 29%), primary myelofibrosis (PMF)(n = 69, 21%) and unclassifiable MPN (n = 17, 5%). Diagnosis were made according to the 2008 WHO Classification. JAK-2 mutation status was either established or revised in our center, but was unknown in 55 (17%) patients. Data about treatment and cardiovascular and thromboembolic incidents was retrieved from medical records. Results: Forty patients (12%) developed thrombosis after median of 36 (range 1-240) months from the time of diagnosis. Fifteen (5%) patients developed venous thrombosis, twenty-four (7%) arterial, while one (0,3%) patient both type of thrombosis. Regarding the disease type, thrombosis was described in 16 patients with ET (11%), 18 with PV (19%) and 6 with PMF (8%). The risk of thrombosis was the highest in patients with previous thrombotic events, but did not correlate with the type of therapy (cytoreductive and/or antiagreggation) nor the JAK-2 status. Summary/Conclusion: The risk of both arterial and venous thrombosis was found to be increased in patients with MPN even after introducing appropriate treatment. The highest risk of post treatment thrombosis was found in patients with previously diagnosed thrombotic events.
Background. Due to the significant increase in life expectancy and the quality of life in patients with chronic myeloid leukemia (CML) as well as the growing need for expensive tyrosine kinase inhibitors (TKI), the analysis of cost-effectiveness and lifelong monitoring of patients is especially important. Aim. We present the results of a multicenter observational study “The Russian Registry of Chronic Myeloid Leukemia in routine clinical practice (2011-2016)”. Materials & Methods. The study included Russian patients with CML, confirmed by the detection of a Ph-chromosome or a BCR-ABL transcript. The statistical analysis (July 1, 2016) included 7609 patients from 80 regions of the Russian Federation (covering 95 % of the population). The annual increase in the number of patients with newly diagnosed CML was 600-650 patients. At the time of the statistical analysis, 6995 (92 %) patients remained under observation, 473 (6 %) died and 141 (2 %) were withdrawn. The registry included 44 % of men and 56 % of women, the median age was 49 years (range 2-94 years). The peak incidence (46.3 %) occurred at the age of 40-60 years. The median disease duration by the time of the analysis was 6 years (range 0.1-30 years). Results. The disease was diagnosed in the chronic phase (CP), acceleration phase, and blast crisis in 6560 (93.8 %), 380 (5.5 %) and 47 (0.7 %) patients, respectively. The proportion of risk groups according to Sokal for low, intermediate and high risk in CP was 49 %, 30 %, and 21 %, respectively. TKI were administered to 6473 (92.5 %) patients. Imatinib and the second generation TKI (TKI2) were administered to 5570 (86 %) and 903 (14 %) patients, respectively. The total of 30.4 % of patients received the increased imatinib dose of 600-800 mg. In the TKI2 group, 558 (61.7 %) patients received nilotinib and 345 (38.2 %) patients received dasatinib. The proportion of patients with completed molecular genetic studies (MGS) in 2014, 2015 and the first 6 months of 2016 amounted to 61 %, 58 % and 23 %, respectively. The proportion of patients with cytogenetic studies (CS) for the same period was 28 %, 26 % and 7 %, respectively. No CS or MGS data were presented for 34 %, 35 % and 63 % of patients during this period. Optimal molecular response and major molecular response (MMR) for TKI therapy were observed in 23 % and 58 % of patients treated < 12 months and > 12 months, respectively. When nilotinib was used in the second line, MMR was obtained in 42 % of patients, and a deep molecular response was obtained in 25 % of patients (BCR-ABL < 0.01 %). Conclusion. The high efficacy of TKI therapy was observed in the majority of patients with the possibility of achieving a minimal residual disease. The problems concerning untimely monitoring and suboptimal administration of second line treatment were identified. In general, the CML patient registry allowed the data integration of data and information management of population with CML in Russia.
Aim. To assess the efficacy of targeted therapy with ruxolitinib in patients with myelofibrosis in real clinical practice in Russia. To determine the prognostic value of spleen reduction in the early stages of ruxolitinib treatment and its effect on overall survival. Materials & Methods. The present retrospective study was based on the data of 10 centers of Russia. It included 56 myelofibrosis (primary or post-polycythemic and post-throm-bocythemic) patients who received ruxolitinib. The median age of patients was 56 years (range 26-76 years). Most of them (59 %) were considered intermediate-1 risk according Results. By the start of data collection most of patients (79 %) had been treated with ruxolitinib. In no case therapy was withdrawn for the reason of drug toxicity. On ruxolitinib constitutional symptoms were reversed in 70 %, 87 %, and 98 % of patients by months 1, 3 and 6, respectively. In 36 % and 46 % of patients by months 3 and 6, respectively, > 50 % decrease in spleen size was observed. Overall, in 31 % and 27 % of cases the size of the spleen decreased by less than 25 % by months 3 and 6, respectively. The factors affecting the changes in spleen size have not been identified. The probability of overall survival by years 2 and 5 of follow-up was 97 % and almost 70 %, respectively. This parameter was significantly affected by the extent of spleen size reduction by month 3 of follow-up as well as by its initial size. Conclusion. Ruxolitinib shows high efficacy for both decrease of general myelofibrosis symptoms and reduction in spleen size. The extent of spleen reduction is an important prognostic factor. It seems, that in patients with insufficient spleen reduction an increase in drug dose is advisable. If it is not possible, alternative methods of treatment should be sought.
Background. Much attention has been paid to molecule-genetic diagnostics of chronic myeloid leukemia (CML) and its treatment using new highly effective methods of therapy. The baseline characteristics of patients at primary CML diagnosis are hardly discussed in literature. Aim. To provide clinical, hematological, molecular genetic and demographic characteristics of patients obtained at primary diagnosis of CML. Patients & Methods. Characteristics of CML patients are based on data gathered by the Russian Investigational Group for CML within the international project European Treatment and Outcome Study of CML in Europe (EUTOS, the European Treatment and Outcomes Study). The study included 197 patients with newly diagnosed CML in 6 regions of the Russian Federation (Mordovia, Kirov, Perm (2 sites), Bryansk, Irkutsk, and Chita) over the period from 2009 till 2012. Results. The study demonstrated that 94 % of CML cases were diagnosed in the chronic phase (CP) and 6 % of cases in the acceleration phase (AP) and the blast crisis phase (BC). In 40 % of patients there were no clinical symptoms, and CML was suspected only due to changes in the CBC test. Fatigue was the main subjective complaint presented by 77 % of patients in the CP and 100 % of patients with the AP and BC. Peripheral blood leukocytosis, left shift to immature myeloid cells and increased granulocytic lineage in bone marrow were typical for the patients. In all patients, the CML diagnosis was confirmed by cytogenetic or molecular tests. The social and demographic characteristics of CML patients and comorbidities at diagnosis were analyzed. Conclusion. Based on the results of the study, a modern «portrait of a CML patient» was obtained. The study demonstrated that cytogenetic and molecular methods allow to diagnose CML in most patients at early stages of the disease in the absence of clinical signs of progression. The data on comorbidities require a special attention while choosing a therapy considering its duration. Demographic and social characteristics of CML patients demonstrate that they are socially active, particularly interested in retaining the working capacity and quality of life.
Introduction. Given the possibility of preserving molecular remission in 40–60 % of patients with chronic myeloid leukemia (CML) with a stable deep molecular response (MR) after discontinuation of tyrosine kinase inhibitors (TKI), it is important to determine the number of candidates for observation in a treatment-free remission (TFR) and terms of treatment cancellation.Aim — to evaluate the probability of stable deep MR and the rate of patients who meet the criteria for TFR observation in the Russian part of the international multicenter prospective population study EUTOS PBS (European Treatment and Outcome Study — Population-Based Study).Materials and methods. Registration of all CML cases in the EUTOS PBS was conducted in 6 regions of Russia from September 2009 to December 2012. The main inclusion criterion was the diagnosis of CML confirmed by cytogenetic or molecular study in patients aged over 18 years. In total, 197 CML patients were included: 181 (92 %) with chronic phase (CP) CML, 14 (7 %) with accelerated phase (AP) and 2 (1 %) with blast crisis (BC) at diagnosis. Data on therapy and results was updated annually.Results. Deep MR (at least MR4 or BCR::ABL1 level less than 0.01 % IS) was achieved in 104 (54 %) of 192 patients receiving TKI therapy, with a median observation period of 7 years (range from 3 months to 10 years). The probability of a deep MR after 5 years of treatment was 48 % (95 % confidence interval (95% CI): 40–55 %) in patients with CP. The cumulative incidence of a stable deep MR with duration of more than 2 years in CML CP patients was 16 % (95% CI: 11–22 %) after 5 years of therapy, 29 % (95% CI: 22–37 %) after 7 years of therapy and 50 % (95% CI: 38–60 %) after 9 years of therapy. The cumulative incidence of a stable deep MR was significantly higher in those patients who had achieved a deep MR at 36 months of therapy compared to patients with only MMR: 40 % (95% CI: 28–53 %) vs. 3 % (95% CI: 0–13 %) at 5 year of therapy; 66 % (95% CI: 52–77 %) vs. 15 % (95% CI: 5–30 %) at 7 year and 89 % (95% CI: 64–97 %) vs. 48 % (95% CI: 25–67 %) at 9 year (p < 0.0001) in patients without MMR by 36 months. No patients without MMR at 36 months of therapy subsequently gained a stable deep MR. Fifty four patients met the TKI discontinuation criteria for transition into TFR phase: CP CML with a typical BCR::ABL1 p210 transcript, TKI therapy for more than 3 years and a stable deep MR for over 2 years. The rate of possible candidates for cancellation of therapy was 28 % of all 192 patients who received TKI in the study or 31 % in terms of patients with CP CML. Predominantly, patients with low-risk by Sokal or ELTS score were among the potential TFR candidates 26 (48 %) and 33 (61 %), respectively. No patients with long-term resistance to therapy were the TFR candidates.Conclusion. In the Russian portion of the prospective observational multicenter study EUTOS PBS, it was found that with a median duration of TKI therapy of 7 years, about a third of patients with CP CML may be candidates for the controlled therapy discontinuation. If half of these patients remain in molecular remission, up to 15 % of the initial number of patients will be able to continue observation in the TFR. Achievement of MMR and deep MR at 36 months of therapy is associated with a significantly greater likelihood of meeting the criteria for follow-up in the TFR phase in the future.
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