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We present the case of an 18.5-year-old boy with hemophilia A and FVIII inhibitor detected at the age of 8 months. No persistent inhibitor eradication was achieved, despite three attempts of immune tolerance induction (ITI) with temporary inhibitor resolution and subsequent administration of 11 doses of rituximab. In spite of hemostatic treatment, rehabilitation, and radiosynovectomy, the patient suffered from recurrent bleeding episodes into almost all major joints, which resulted in the development of progressive hemophilic arthropathy. Currently, due to high frequency of bleeding episodes, the patient is being treated prophylactically and receives prophylaxis with activated prothrombin complex concentrate (aPCC).

Disseminated intravascular coagulation (DIC) is usually observed in acute promielocytic leukemia (AML-M3). DIC is diagnosed in other types of acute myeloid leukemia less frequently, but among them, the largest percentage applies to patients with acute monoblastic leukemia (AML-M5), with the release of procoagulation substances and cytokines from blasts. Laboratory and clinical symptoms of DIC appeared at the time of diagnosis of leukemia or just after initiation of induction treatment.

According to WHO classification, classic myeloproliferative neoplasms (MPN) without Philadelphia chromosome include polycythemia vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). The discoveries in recent years revealed molecular causes of these diseases. These include mutations in JAK2(V617F, exon 12), MPLand CALRgenes. MPN are rarely diagnosed in children, and the molecular basis of these disorders is not well understood. In the first part of this review, we describe diagnostic criteria and therapy of PV and ET. Most cases of erythrocytosis and thrombocytosis in children are reactive and warrant a thorough differential diagnosis.

Posterior reversible encephalopathy syndrome (PRES) embraces serious neurological symptoms, including seizures, headache, visual disturbances, mental disturbances and aphasia, usually accompanied by increased blood pressure. Diagnosis is based on clinical symptoms and characteristic central nervous system lesions, localized in the white and grey matter of the parieto-occipital part of the brain, revealed on magnetic resonance imaging (MRI). In the majority of cases, PRES symptoms subside spontaneously, both clinically and radiologically, without permanent neurological consequences. Risk factors of PRES development described in literature include chemotherapy and immunosuppression. In this report, we present a 13-year-old boy with acute lymphoblastic leukaemia who developed acute neurological symptoms in the form of two episodes of generalized seizures. PRES was diagnosed based on clinical signs and characteristic lesions visible on MRI. Prompt diagnosis and administration of anti-hypertensive drugs resulted in a regression of the observed symptoms.

Patients with hemato-oncologic diseases are particularly vulnerable to severe infections. Adult patients with blood cancers infected with SARS-CoV-2 had poorer treatment outcomes and higher mortality than patients with COVID-19 without burden. However, in pediatric patients with hemato-oncologic diseases the course of COVID-19 is milder than in adults in the same group of patients. In this report, we describe the case of our patient with acute lymphoblastic leukemia infected with SARS-CoV-2 and treated with remdesivir. We also review the existing literature of pediatric patients who have been diagnosed with both hemato-oncologic diseases and COVID-19.

Autoimmune hemolytic anemia (AIHA) is a rare disorder in which the immune system produces pathologic antibodies directed against its own red blood cells (autoantibodies). AIHA is most commonly diagnosed by a positive result of the direct antiglobulin test (DAT, Coombs test). Depending on the temperature at which autoantibodies react with red blood cells in vitroAIHA is classified as warm-type AIHA with incomplete IgG autoantibodies and cold-type AIHA with cold IgM agglutinins (CAS – cold agglutinin syndrome) or with biphasic hemolysins (PCH – paroxysmal cold hemoglobinuria). In mixed-type AIHA there are simultaneously warm autoantibodies and cold agglutinins.

To analyze the incidence and outcome of infections with Staphylococcusspp. in children with malignancies treated in pediatric hematology and oncology (PHO) centers or undergoing hematopoietic stem cell transplantation (HSCT) over a period of 24 months.

This article presents the current recommendations from the Polish Pediatric Group for Hematopoietic Stem Cell Transplantation concerning the indications for hematopoietic stem cell transplantation (HSCT) in children and adolescents suffering from hematological malignancies, solid tumours, and congenital or acquired non-malignant disorders. Indications for HSCT are established in context of the recent results of conventional treatment, i.e. obtained with non-HSCT strategies; it means transplantation is justifiable exclusively, when it significantly increases individual patient's chances to be cured, despite of the risk of HSCT-related mortality. Hence, due to the advances of non-transplant treatment strategies as well as progress in the field of HSCT the indications for HSCT require to be regularly up-dated. The recommendations presented in this article are based on the current guidelines from the European Group for Blood and Bone Marrow Transplantation (EBMT), including those from the EBMT Pediatric Diseases Working Party and the EBMT Inborn Errors Working Party, and from the international treatment protocols currently applied in the centers of the Polish Pediatric Leukemia/Lymphoma Study Group and Polish Pediatric Solid Tumours Study Group. The recommendations are addressed not only to the Polish pediatric transplant centers, but first of all to the Polish pediatric centers involved in diagnostics and treatment of the malignancies and non-malignant disorders in children and adolescents with non-transplant strategies, because it is their responsibility to identify as soon as possible indications for HSCT and refer patient at the appropriate time to pediatric transplant center.

In the article case of thrombotic thrombocythopenic purpura in 3-year-old boy is presented, therapeutic and diagnostic problems are described. TTP is not common disease, but the diagnosis of TTP must be taken into consideration in patients with microangiopathic hemolytic anemia, thrombocythopenia, neurologic or renal abnormalities, lung and liver dysfunction. In presented case inhibitor ADAMTS13 and severe deficiency of ADAMTS 13 activity was detected and acquired TTP recognized. Patient was treated with plasma exchange, coticosteroids and azatiopryne and remission was established.

Sferocytoza wrodzona należy do najczęściej występujących wrodzonych niedokrwistości hemolitycznych.

Talasemie to grupa genetycznie uwarunkowanych niedokrwistości hemolitycznych, spowodowanych ilościowym zaburzeniem syntezy łańcuchów globiny, zaliczana do hemoglobinopatii ilościowych. Talasemie należą do najczęstszych chorób jednogenowych.

Napadowa zimna hemoglobinuria (NZH) jest rzadko występującą postacią niedokrwistości hemolitycznej. Cechuje się wystąpieniem masywnej hemolizy z hemoglobinurią po ekspozycji na zimno. Jej przyczyną są hemolizyny Donatha-Landsteinera. Przedstawiono historie chorób sześciorga dzieci, u których na podstawie wyników obserwacji klinicznej i badań serologicznych rozpoznano napadową zimną hemoglobinurię. Po zastosowaniu przetoczeń koncentratu krwinek czerwonych i steroidów uzyskano dobry efekt terapeutyczny. W artykule zwrócono uwagę na konieczność unikania wyziębiania pacjentów z niedokrwistością autoimmunologiczną do czasu wykluczenia zimnych autoprzeciwciał.

W pracy przedstawiono przypadek rodziny, w której u obojga rodziców wykryto heterozygotyczną postać talasemii β, a u jednego z synów homozygotyczną postać talasemii. Drugi z synów jest zdrowy.

Nadpłytkowość samoistna zaliczana jest do chorób mieloproliferacyjnych. Występuje u osób dorosłych i bardzo rzadko u dzieci. Chorobę rozpoznaje się na podstawie liczby płytek wyższej od 1000 × 109/l, po wykluczeniu chorób prowadzących wtórnie do nadpłytkowości. Przedstawiono przypadek 12-letniej dziewczynki z nadpłytkowością samoistną leczonej przez 52 miesiące anagrelidem.

Wirusowe zapalenie wątroby typu A (WZW A) to jedna z najczęstszych na świecie chorób zakaźnych. W Polsce aż 93% dzieci do 15 roku życia nie ma przeciwciał chroniących przed nią. Konsekwencjami przebycia WZW A mogą być aplazja szpiku, żółtaczka cholestatyczna, ostra niewydolność nerek, a nawet ostra niewydolność wątroby prowadząca do zgonu. WZW A jest przyczyną około 0,3–8% nabytych anemii aplastycznych (NAA) u dzieci, których przebieg na ogół jest bardzo ciężki i kończy się śmiercią u ok. 50% chorych. W latach 1990–2006 w obu klinikach u 81dzieci rozpoznano NAA, w tym u 6 z nich po przebytym WZW A. 4 pacjentów otrzymało leczenie farmakologiczne, a 2 dzieci miało wykonany przeszczep komórek hematopoetycznych od zgodnego dawcy rodzinnego. Do dziś 3 dzieci żyje w remisji, w tym jedno po przeszczepie. Zmarło 3 pacjentów.

Przedstawiono przypadek chłopca, u którego rozpoznano zespół limfohistiocytozy hemofagocytarnej. Opisano trudności diagnostyczne i terapeutyczne związane z tą bardzo ciężką, często śmiertelną chorobą układu krwiotwórczego.

W pracy przedstawiono trudności diagnostyczne w rozpoznawaniu niedokrwistości hemolitycznej na przykładzie 16-letniego chłopca z talasemią beta.

Immunoterapia jest jednym z najnowszych kierunków w leczeniu nowotworów. W ostrej białaczce limfoblastycznej (ALL) u dzieci możliwe jest przekształcenie komórek nowotworowych w komórki dendrytyczne prezentujące antygeny białaczkowe limfocytom T gospodarza. W ten sposób, być może, będzie można uzyskać eliminację klonu białaczkowego, tym samym wyleczenie pacjenta.

The therapeutic management in patients with stage IV Hodgkin disease is still controversial. Among 783 children with Hodgkin disease treated from 1971 to 1996, 56 patients (7.3%) were diagnosed with stage IV. The treatment consisted of MVPP or MVPP/B-DOPA chemotherapy combined with involved-field radiotherapy in 50 children. The results of treatment of stage IV patients were compared in the three sequential time periods, during which the therapy was modified. In these periods, the first complete remission was obtained in 67%, 86%, and 90% of children, respectively, and the 10-year event-free survival was 42%, 64%, and 85%, respectively. Alternate multidrug chemotherapy combined with low-dose involved-field radiotherapy is at present a satisfactory therapeutic method in children with stage IV Hodgkin disease. Med. Pediatr. Oncol. 33:382–387, 1999. © 1999 Wiley-Liss, Inc.

Peripheral blood samples were collected from 13 children with confirmed SAA. Patients (9 girls and 4 boys) aged 7–19 yrs have all received rabbit ATG in a dose of 3.75 mg/kg/day for 5 days and CSA in a dose of 5mg/kg/day for 12–14 months). Remission was assessed on 180th and 360th day of treatment. Control peripheral blood samples were obtained from 12 healthy children (3 girls and 9 boys, aged 3–17 yrs), 12 healthy adultand from 4 SAA children parents. Informed consent was obtained from all adult donors and from parents of participating children.DNA extraction and qPCR Genomic DNA was extracted from whole blood using AxyPrep Blood Genomic DNA Miniprep Kit (Axygene). Telomere length was determined using the quantitive PCR (qPCR) method described by Elisa Pavesi et al (1). Briefly, two qPCR reactions were run for each DNA sample: amplification of telomere product and amplification of a single copy gene (SEP15). Each qPCR reaction contained: Power SYBR1 Green PCR Master Mix (Applied Biosystems), template DNA and primers (270nM Tel1 and 900nM Tel2 or 500nM of each SEP15 primers). Telomere/single copy gene (T/S) ratios for samples were calculated using Human reference DNA (Applied Biosystems) standard curve. Reactions were preformed in Real Time PCR CFX96 system (Biorad). Statistica software version 9.1 (StatSoft) was used for statistical analysisAmong 13 SAA patients telomere shortening was found in 9 children while 4 patients had significantly elongated telomeres. All of the latter ones responded poorly to IST treatment: 3 of them did not respond (NR) to IST (neither on day 180 nor on 360) and one patient had a partial remission (PR). 2 patients in the NR group were randomized to unrelated bone marrow transplantation and one had another course of IST (no unrelated donor), after which he reached a PR. All patients with elongated telomeres had normal serum adriostendion level. Telomere elongation was also found in parents compared to the control group of adults (n=12, age range 31–57). Among 9 patients with shortened telomeres, 4 achieved complete remission (CR), 1 - PR and 4 did not respond (NR) to treatment on check times points.Our observations indicate that SAA patients with shortened telomeres respond to immunosuppressive therapy better then those with elongated telomeres. Telomere length may be considered therefore as a predicting factor in this patients. Studies on a larger group of patients should be performed to confirm our observations. Short telomere definition –samples, which T/S value is less than the first Quartile of controls. Long telomere - samples, which T/S value is in III quartile of controls. Very long telomere - samples, which T/S value is higher than the IV Quartile of controls.No relevant conflicts of interest to declare.

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