Epidemiology and prevention strategies of SARS-CoV-2 infection in pediatric hematology and oncology centers in Poland

IntroductionEpidemiological analysis of severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infections in pediatric hematology and oncology (PHO) and hematopoietic cell transplant (HCT) centers in a Polish nationwide study, as well as analysis of the preventive strategies in these centers.MethodsAll of the 18 PHO/HCT centers participated in eight surveys and questionnaires conducted over the first 5 months of the SARS-CoV-2/coronavirus disease 2019 (COVID-19) pandemic in Poland. Epidemiological data were collected at eight regular time points, and the strategy of preventive management was done once after 4 months of the pandemic.ResultsDuring this analyzed period, eight patients were positive for SARS-CoV-2. The estimated incidence of SARS-CoV-2 positivity in Polish PHO/HCT centers was 0.5%. After exclusion of HCT patients (with one patient being infected), the estimated incidence of SARSCoV-2 positivity was between 0.5 and 0.6%. In all but one case, the course of COVID-19 was asymptomatic or mild, and it was moderate in one case. None of them developed SARS or respiratory insufficiency, none of them required treatment in the intensive care unit (ICU), and no patient died due to SARS-CoV-2 infection. As of July 1, parents staying in the hospital together with their children were regularly tested for the virus in 13 centers. Asymptomatic healthcare personnel were regularly tested for the virus in seven centers.ConclusionsThe estimated incidence of SARS-CoV-2 infection among PHO/HCT patients is lower than in Western Europe; however, these patients cannot be regarded as a low-risk group. The low COVID-19 incidence should be interpreted as a result of strictly and continuously implemented detailed preventive measures in the PHO/HCT wards and in hospitals.

Eltrombopag use in chronic immune thrombocytopenia of childhood: results from nationwide therapeutic program

BackgroundThrombopoietin receptor agonists have been repeatedly confirmed to be safe, efficient, and well tolerated in pediatric patients with chronic immune thrombocytopenia (cITP).Material and methodsIn this report, we present data summarizing the Polish experience of the use of eltrombopag in cITP patients, refractory to standard first-line care. Our analysis was based on clinical and epidemiological data from the Nationwide Therapeutic Program 2018–2020. Quality of the response to the eltrombopag treatment was defined according to the International Consensus Guidelines as follows: complete response (CR) defined as platelet count (PLT) ≥100 × 109/L and absence of bleeding; response (R) defined as PLT ≥30 × 109/L and at least two-fold increase in the baseline count and absence of bleeding.ResultsWe evaluated 60 patients (33 boys and 27 girls) with chronic and refractory ITP. Median age at beginning of treatment was 9.5 years. Median PLT at the first eltrombopag administration was 30 × 109/L. The median follow-up was 7 months (range, 3–22 months). After 1 week of treatment, response (R) was noted in 53.3% (95% confidence interval [CI]: 40.7%–66.0%) patients, and complete response (CR) was seen in 21.6% (95% CI: 11.2%–32.1%). We evaluated the long-term duration of the response and found that it was obtained in 84.4% (95% CI: 71.8%–97.0%) and 88.9% (95% CI: 77.0%–100%) of patients after 6 and 12 months, respectively, of eltrombopag therapy, while CR was reached, respectively, in 46.9% (95% CI: 29.6%–64.2%) and 29.6% (95% CI: 12.4%–46.9%) patients. No serious adverse events were reported.ConclusionOur data support the safety and efficacy of eltrombopag use in cITP pediatric patients.

Medical dilemmas – erythrocytosis

Erythrocytosis is defined not only by an increase in the erythrocyte count, hemoglobin concentration, and hematocrit value, but also by the occurrence of specific symptoms, the intensity and frequency of which depend on the character of the initial genetic lesion. Ischemic episodes and thrombotic complications caused by increased blood viscosity are frequently the first clinical manifestation of the disease. This paper represents the current level of knowledge about the pathogenesis of erythrocytosis and the diagnostic algorithms used to precisely define the type of the disease.

Iron deficiency anemia – new possibilities of iron supplementation in various clinical conditions

Iron deficiency anemia (IDA) treatment is done to eliminate the causes of iron deficiency, iron supplementation, and rarely red blood cell transfusion. Divalent iron salts are the first line of oral treatment, but their use lead to frequent gastrointestinal adverse reactions. Iron is administered intravenously in the event of contraindications, intolerance, or inefficiency of oral therapy, but the parenteral route of drug delivery is not easily accepted by the patients. Intravenous preparations for single administration of a large dose of iron have a good therapy safety profile, but are more expensive than oral and are usually administered in a hospital setting. The availability of new iron compounds: sucrosomial iron, ferric citrate complexes, and ferric maltol widen the possibilities of IDA therapy and enable the better selection of iron preparations in various clinical situations. The innovative structure of sucrosomial iron leads to absorption in different ways (through endocytosis, the paracellular pathway, M cells of Peyer's patches), ensures high bioavailability, and good tolerance of therapy. Ferric citrate, in addition to iron supplementation, reduces phosphate levels, and is beneficial to chronic kidney disease. Ferric maltol is currently being studied for IDA treatment with various comorbidities. Some studies indicate that new iron formulas may be used where intravenous intake has been recommended so far. So, we can expect treatment with iron nanoparticles and drugs that affect the intestinal microflora in the future. The paper presents current knowledge about new iron preparations that are already available in everyday practice, but also those that are at various stages of pre-clinical and clinical studies.

Monoclonal gammopathies of undetermined significance and smoldering myeloma

Monoclonal gammopathy of undetermined significance (MGUS) is a clonal plasma cell disorder implicated as a precursor of multiple myeloma (MM), while smoldering multiple myeloma (SMM) is a malignant plasma cell disorder without evidence of a myeloma-defining event(s) (MDE). This is a review article of both disorders outlining their current definition and management according to the current standard of care. We focus on the pathogenesis of MM and the role of MGUS and SMM in the development of active MM. MGUS is a benign disorder and, subsequently, is followed by observation. In contrast, for SMM, although the current standard of care is “watch and wait”, this paper will explore the circumstances in which treatment should be considered to prevent MDE.

The beneficial role of allogenic hematopoietic cell transplantation in blastic plasmacytoid dendritic cell neoplasm

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a very rare aggressive hematopoietic malignancy. The median survival is approximately 12 months, and for patients >65 years the survival rate is 7 months, when only chemotherapy is administered. Clinically, it is characterized by skin involvement and most often bone marrow lesions accompanied by lymphadenopathy and in some cases hepato- and/or splenomegaly. The diagnosis is based on histopathological examination of the skin or bone marrow lesions and tumor cell immunophenotyping. The etiopathogenesis of the disease is not fully understood. Therapeutic decisions are based only on the results of a few retrospective analyses and case reports. This article presents the important role of allogeneic hematopoietic cell transplantation in the treatment of BPDCN.

Myeloid-derived suppressor cells level and MUC1 expression in de novo acute myeloid leukemia

BackgroundAcute myeloid leukemia (AML) is the most common acute leukemia occurring in adults. It is an aggressive myeloid neoplasm with maturation arrest of myelopoiesis, leading to an accumulation of myeloblasts in the bone marrow and peripheral blood.ObjectiveTo evaluate alterations in myeloid-derived suppressor cells level and MUC1 gene expression in patients with de novo acute myeloid leukemia concerning disease characteristics and response to induction chemotherapy.Patients and methodsThe study was performed on 50 AML patients and 50 healthy controls. Detection of myeloid-derived suppressor cells (MDSCs) in peripheral blood was performed by mononuclear separation and flow cytometry. MUC1 gene expression was performed by RNA extraction, reverse transcription, and real-time PCR at Hematology Department Medical Research Institute, Alexandria University.ResultsWe have demonstrated that AML patients had both increased presence of MDSCs in peripheral blood as well as MUC1 overexpression in comparison to normal controls. MDSCs showed a significant correlation regarding response to induction chemotherapy on day 28. While MDSCs and not MUC1 are associated with inferior response to induction chemotherapy on day 28.ConclusionThe current data suggested that AML patients exhibit an increased presence of MDSCs as well as MUC1 gene overexpression in comparison with normal controls. While MDSCs showed a significant correlation regarding response to induction chemotherapy on day 28, MDSCs and not MUC1 are associated with inferior response to induction chemotherapy on the same day.

Clinical characteristics of essential thrombocythemia patients depend on the mutation status

The impact of the mutation status on the clinical course and the outcome of essential thrombocythemia (ET) patients has not yet been completely established. A total of 171 patients with diagnosed ET were tested and subsequently grouped, according to their mutation status – Janus Kinase 2 (JAK2) – 112 patients, calreticulin (CALR) – 36 patients, and thrombopoietin receptor (MPL) – 5 patients. Moreover, 18 individuals were triple-negative (with non-mutated JAK2, CALR, and MPL). CALR-mutated patients preferentially were male, with higher platelets (PLT) counts (mean PLT = 1 002.3) and lower hemoglobin and hematocrit levels at the diagnosis, compared to the JAK2 (mean PLT = 933.6), MPL (mean PLT = 940.8) and triple-negative patients (mean PLT = 822.6) (p = 0.0035). The patients with CALR mutated, and the triple-negative ones had a lower risk of arterial and venous thrombosis (3% and 5.6% cases at the time of diagnosis, respectively) than the patients with JAK2 mutation (7.2%) (p = 0.9210). The overall survival rate did not differ statistically between the groups.