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.
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.
Thrombopoietin receptor agonists have been repeatedly confirmed to be safe, efficient, and well tolerated in pediatric patients with chronic immune thrombocytopenia (cITP).
Material and methods
In 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.
We 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.
Our data support the safety and efficacy of eltrombopag use in cITP pediatric patients.
Epidemiological 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.
All 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.
During 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.
The 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.
In this article, we report familial cases of type 2 congenital erythrocytosis (ECYT2) in two siblings, a 2-year-old boy and his younger sister. Both patients were diagnosed based on laboratory findings including erythrocytosis, elevated hemoglobin levels, and hematocrit. Acquired erythrocytosis was excluded based on the clinical features and genetic analysis of JAK2/CALR/MPL genes. Next-generation sequencing was employed for older brother revealing NM_000551.4: c.598C>T, p.Arg200Trp homozygous variant in the VHL gene, the similar variant was detected in the younger sibling. Sequencing analysis confirmed the VHL c.598C>T heterozygous variant in both parents. To the best of our knowledge, these are the first confirmed cases of familial erythrocytosis type 2, also known as Chuvash type, in Poland.
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.