Lymphoblastic predominance of blastic phase in children with chronic myeloid leukaemia treated with imatinib: A report from the I-CML-Ped Study.

Background: Chronic myeloid leukaemia (CML) is a rare disease in children. The frequency and outcome of children evolving to accelerated phase (AP) or blastic phase (BP) under treatment with imatinib is unknown. The aim of the current study is to assess the incidence of progression from CML in chronic phase with imatinib frontline in a paediatric setting and describe the management and outcome of these patients.

Hypovitaminosis D Influences the Clinical Presentation of Immune Thrombocytopenia in Children with Newly Diagnosed Disease.

Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia defined as platelet count in peripheral blood <100 × 10/L. Hypovitaminosis D is very common in children with autoimmune diseases. To analyze whether hypovitaminosis D is associated with the clinical presentation of ITP in children, medical records of 45 pediatric patients with newly diagnosed immune thrombocytopenia in the coastal region of Croatia were evaluated.

Prognostic discrimination based on the EUTOS long-term survival score within the International Registry for Chronic Myeloid Leukemia in children and adolescents.

The EUTOS Long-Term Survival score was tested in 350 children with chronic myeloid leukemia in first chronic phase treated with imatinib and registered in the International Registry for Childhood Chronic Myeloid Leukemia. With a median follow up of 3 years (range, 1 month to 6 years) progression and/or death (whichever came first) occurred in 23 patients. For the entire cohort of patients the 5-year progression-free survival rate was 92% (95% CI: 87%-94%) and the 5-year survival accounting for chronic myeloid leukemia deaths was 97% (95% CI: 94%-99%).

Additional cytogenetic abnormalities and variant t(9;22) at the diagnosis of childhood chronic myeloid leukemia: The experience of the International Registry for Chronic Myeloid Leukemia in Children and Adolescents.

Background: In the adult population with newly diagnosed chronic myeloid leukemia (CML), variant translocations are usually not considered to be impairing the prognosis, whereas some additional cytogenetic abnormalities (ACAs) are associated with a negative impact on survival. Because of the rarity of CML in the pediatric population, such abnormalities have not been investigated in a large group of children with CML.

Methods: The prognostic relevance of variant t(9;22) and ACAs at diagnosis was assessed in 301 children with CML in the chronic phase who were enrolled in the International Registry for Chronic Myeloid Leukemia in Children and Adolescents.

Rare localization of extrarenal nephroblastoma in 1-month-old female infant.

Extrarenal occurrence of Wilms' tumor is exceptional and the diagnosis is almost always made after surgery. The exact mechanism whereby a Wilms' tumor occurs in extrarenal tissue is unknown. The tumor is most commonly located in the retroperitoneum or inguinal region.

Relationship between tumor vascularity and vascular endothelial growth factor as prognostic factors for patients with neuroblastoma.

Although the role of angiogenesis in tumor progression and response to treatment is generally well-characterized, for neuroblastomas clinical data regarding the contribution of angiogenesis and its predictive capacity remain unclear. The aim of this study was to evaluate whether tumor vascularity in the combination with expression of vascular endothelial growth factor (VEGF) represent prognostic factors for patients with neuroblastoma. Immunohistochemistry using anti-CD34 and anti-VEGF antibodies was used to analyze paraffin-embedded primary tumor tissues from 56 patients diagnosed with neuroblastoma.

Parental type of personality, negative affectivity and family stressful events in children with cancer.

Objective: Psychological interactions between parents,children and social environment are very important for childhood health. The type of personality and stressful events are probably also cancer risk factors. We investigated personality types A/B and D (negative affectivity and social inhibition) in parents of children with cancer (PCC), as well as social environmental factors, and family / children's stressful events before the appearance of cancer.

Common acute lymphoblastic leukemia Ph+ following Langerhans cell histiocytosis in a multi-malformed child with INV (9) (p12;q13) (mat): case report.

The occurrence of Langerhans cell histiocytosis (LCH) and another malignancy in the same patient is infrequent but has been recognized. The genetic changes that could be responsible for LCH and/or concomitant leukemia development are obscure. To the best of our knowledge, this is the first description of constitutional maternally derived inv (9) (p12;q13) in an LCH patient, and also of the development of common ALL Ph after LCH diagnosis and therapy.

Familial hemophagocytic lymphohistiocytosis in a 6-week-old male infant.

Familial hemophagocytic lymphohistiocytosis (FLH) is an autosomal recessively inherited multisystem disease. This defect in cellular cytotoxicity is a life threatening condition characterized by fever, rash, splenomegaly, cytopenias and neurologic manifestations. PRF1, UNC13D and STX11 gene defects underlie in about 40-50% of primary cases.

Expression and prognostic value of the Ki-67 in Wilms' tumor: experience with 48 cases.

Purpose: Ki-67, tumor proliferation marker, is an important prognostic factor in a variety of cancers. In the present study, we investigated the expression and the prognostic value of Ki-67 in nephroblastoma.

Methods: Ki-67 expressions were investigated by immunohistochemistry on paraffin-embedded material in 48 children operated on because of nephroblastoma.

Vascular endothelial growth factor in children with neuroblastoma: a retrospective analysis.

Background: Despite aggressive therapy, advanced stage neuroblastoma patients have poor survival rates. Although angiogenesis correlates with advanced tumour stage and plays an important role in determining the tumour response to treatment in general, clinical data are still insufficient, and more clinical evaluations are needed to draw conclusions. The aim of this study was to evaluate vascular endothelial growth factor (VEGF) expression in patients with neuroblastoma, determine whether it correlates with other prognostic factors and/or therapeutic response, and to assess should VEGF be considered in a routine diagnostic workup.

Double-blind, placebo-controlled, multiple-ascending-dose study on the effects of ABIO-08/01, a novel anxiolytic drug, on perception and cognition, utilizing event-related potential mapping and low-resolution brain electromagnetic tomography.

Early pharmacological studies in animals demonstrated that ABIO-08/01, a new isoxazoline, exerted anxiolytic and anticonvulsant, but also cognition-enhancing properties. Thus, the aim of the present double-blind, placebo-controlled multiple-ascending-dose study was to investigate the effect of the new compound on event-related potentials (ERPs). In a randomized ascending-dose design for phase-1 studies, 16 young healthy male subjects aged 30.

Use of recombinant factor VIIa for emergency reversal of anticoagulation.

Context: There is limited data regarding the use of activated recombinant factor VII (rFVIIa) in anticoagulated patients requiring reversal.

Aims: To identify and describe characteristics of subjects who received rFVIIa as part of emergency treatment aimed at improving hemostasis.

Settings And Design: Data was obtained from an international peer-reviewed registry haemostasis.

Anti-CD20 monoclonal antibody (rituximab) for therapy of CD20-positive nodular lymphocyte-predominant Hodgkin lymphoma in an 10-year-old girl.

Biologic treatments including antibody-based therapies are still in early-phase development in Hodgkin lymphoma. The authors present the case of a 10-year-old girl with massive, solid, unilateral cervical, nodular lymphocyte-predominant Hodgkin lymphoma. Chemotherapy (doxorubicin, bleomycin, vinblastine, and dacarbazine [ABVD]) and radiotherapy were given, according to the Italian Association of Pediatric Hematology and Oncology (AIEOP) MH-96 study protocol, but the patient failed to enter complete remission.

Correlations among age, cytokines, lymphocyte subtypes, and platelet counts in autoimmune thrombocytopenic purpura.

While autoimmune thrombocytopenic purpura is mediated by autoantibodies, accumulating evidence suggests that T helper cells and the cytokines they produce also play a key role. We determined correlations among age, serum cytokine concentrations, circulating lymphocyte, and platelet counts in adult (n=19) and children (n=29) with autoimmune thrombocytopenic purpura. Correlations between age and cytokine levels were also assessed in healthy controls (n=50).

Successful management of bleeding with recombinant factor VIIa (NovoSeven) in a patient with Burkitt lymphoma and thrombosis of the left femoral and left common iliac veins.

We present the case of an 18-year-old female with Burkitt lymphoma involving the intra-abdominal and inguinal lymph nodes. The tumor had invaded the left femoral and common iliac veins causing secondary thrombosis and vessel occlusion. Chemotherapy and anticoagulant treatment resulted in mild thrombocytopenia and a prolonged prothrombin time, respectively, which exacerbated postoperative bleeding following surgical removal of a deep inguinal necrosis.

Control of bleeding caused by thrombocytopenia associated with hematologic malignancy: an audit of the clinical use of recombinant activated factor VII.

This paper presents an analysis of 24 cases in which recombinant factor VIIa (rFVIIa) was used in the management of hemorrhage in patients with thrombocytopenia associated with hematologic malignancies. This is the largest case aggregation to date and focuses on preliminary experience in the off-label use of this hemostatic agent. Data were extracted from the international, Internet-based registry, www.

Treatment of post-traumatic trabecular mashwork thrombosis and secondary glaucoma with intracameral tissue plasminogen activator in previously unrecognized sickle cell anemia.

Intracameral tissue plasminogen activator (t-PA) application in a child with previously unrecognized sickle cell anemia, post-traumatic hyphema, thrombosis in trabecular mashwork and consecutive acute glaucoma showed positive results. Thirteen year-old boy, son of African father and Caucasian mother, was admitted to hospital, with symptoms of acute glaucoma and partial hyphema after right eye trauma. Visual acuity of affected eye was 0.

Cold injury syndrome and neurodevelopmental changes in survivors.

Background: The aim of this study was to extend and develop knowledge of the clinical parameters of cold injury syndrome (CIS) in hypothermic infants and newborns, so that morbidity, mortality, and neurodevelopmental disturbances associated with the condition can be reduced.

Methods: This retrospective, 10-year cohort study investigated 103 hypothermic infant and newborn patients (aged 0-60 days) admitted to the Pediatric Department between January 1, 1976 and December 31, 1985. Medical records of 67 survivors and 36 children who died were analyzed.

Disseminated BCG infection resembling langerhans cell histiocytosis in an infant with severe combined immunodeficiency: a case report.

We present a very rare congenital immunologic disease, severe combined immunodeficiency syndrome (SCID) in 6-months-old-boy with prolonged mucocutaneous candidiasis, severe anaemia, skin rash similar to the infiltrative eczema of Langerhans cell histiocytosis (LCH) and subcutaneous nodules with histiocytic infiltration. Laboratory findings show profound absence of humoral and cell-mediated immunity. Pathology specimens analysis of subcutaneous nodule revealed numerous S-100 protein and Cd1a negative histiocytes, occupied by BCG intracellular growth.