Optimal stem cell mobilization regimen for poor mobilizers do not exist. The aim of this trial was to compare efficacy and toxicity of stem cell mobilization using cytarabine + G-CSF with cyclophosphamide + G-CSF and plerixafor + G-CSF.

Methods. In this retrospective study mobilization results of 87 ‘predicted poor mobilizer’ were evaluated. Thirty-three of them received cytarabine at the dose 400 mg/m2/12 h + G-CSF 10 µg/kg starting from day 5, 18 patients received cyclophosphamide at the dose 2-4 g/m2 + G-CSF 10 µg/kg starting from day 5 and 33 patients received G-CSF 10 µg/kg followed by plerixafor 0,24 mg/kg on day 5.

Results. Median of CD34+ cells yield was 8,1 x 106/kg in the cytarabine group compared to 6,5 x 106/kg in the cyclophosphamide group and 2,8 x 106/kg in the plerixafor group (р<0,0001). The most frequent complications were thrombocytopenia gr 4 (44% of patients in the cytarabine group and 6% of patients in the cyclophosphamide group, p=0,004) and neutropenia gr 4 (42% of patients in the cytarabine group and 22% of patients in the cyclophosphamide group, p=0,23).

Conclusion: stem cell mobilization with intermediate dose cytarabine is effective and safe regimen for predicted poor mobilizers.

Background. Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal disease of the blood system, one of the manifestations of which is hemolytic anemia. Due to the predominantly intravascular hemolysis, iron metabolism in PNH patients has several features. Pathogenetic therapy with inhibitors of C5-component of complement modifies the course of hemolysis and, as a consequence, introduces changes in iron metabolism.

Aim. To characterize serum parameters of iron metabolism in patients with PNH and results of liver and kidney MRI in T2-R2* method.

Materials and methods. A total of 99 MRI studies of the liver and kidneys and 97 studies of serum parameters of iron metabolism in 82 patients were performed.

Results. Among patients not receiving C5 inhibitors, iron overload of liver tissue was detected in 10/38 (26.3%) patients. Among patients receiving a C5-component inhibitor, liver tissue iron overload was significantly more frequent in 28/40 (70%). Liver iron overload was more pronounced in patients with suboptimal hematological response. Among patients not receiving C5 inhibitors, renal tissue iron overload was found in all (34/34) patients. Among patients receiving a C5-component inhibitor, iron overload of kidneys was significantly less frequent in 21/37 (57%) patients.

Conclusion. Renal cortical overload occurs in all patients with PNH not receiving complement C5 inhibitors. Liver iron overload is associated with hemotransfusions and suboptimal response to pathogenetic therapy. Serum indices of iron metabolism in PNH patients have lower sensitivity in detecting hepatic and renal iron overload compared to T2-R2* mode MRI. Achievement of optimal response to pathogenetic therapy is characterized by normalization of iron content in renal parenchyma and absence of hepatic hemosiderosis.

The use of tyrosine kinase inhibitors (TKIs) for chronic myeloid leukemia (CML) patients treatment significantly improved a prognosis for the majority of patients in the chronic phase (CP), however, the problem of TKI therapy failure is still an issue. Currently, the genetic profile of tumor cells in CML patients and the role of somatic mutations of non-BCR::ABL1 genes in the development of resistance to therapy is being actively studied. New data on the frequency of somatic mutations at the time of diagnosis of CML in CP and advanced phases, on clonal changes during treatment, including disease progression, are emerging. Of particular interest is the study of somatic mutations’ role in disease transformation, the temporal relationship between the occurrence of somatic mutations and CML progression. The purpose of this review is to present the data of actual studies of molecular-genetic profile in CML patients at different stages of the disease. In their works, the authors seek to identify associations between the presence of somatic mutations and response to therapy, to assess the prognostic value of mutations detected at diagnosis and during the therapy. In the future, this knowledge could be used to optimize treatment: the choice of the most effective TKI, the prescription of targeted therapy aimed at alternative genetic abnormalities or early allogeneic bone marrow transplantation. Thus, a detailed study of the role of the most common somatic mutations in CML patients could help to determine the management of patients in different clinical situations.