Results of rhabdomyosarcoma treatment in a developing country.

Fifty-one children (median age: 4.5 years; 4 months-16 years) diagnosed with rhabdomyosarcoma were treated in our center between 1980-1999. The primary sites were head and neck in 31.4%, the genito-urinary system in 21.6%, and extremities in 9.8% of the patients. The histopathologic subtypes were embryonal in 80.4%, alveolar in 9.8%, and undifferentiated in 9.8%. The majority of the patients were considered group III (47%) and group IV (25.5%) according the criteria of the Intergroup Rhabdomyosarcoma Study (IRS). Primary total tumour resection was performed in only 27.5% of the patients. The patients were treated with assigned regimens of IRS II and IRS III protocols. Radiotherapy was applied to 92.1% of the patients. Thirty-four patients (66.7%) were lost to follow up, and of the remaining 17 patients, 7 patients (41.2%) died, relapse occurred in 9 patients (52.9%) and 10 patients (58.8%) are alive. The percentage of cases lost to follow up during the first 10 years and the following 9 years of the study were 77.4% and 50%, respectively. In compliance with cancer treatment remains a major problem in developing countries.</p

Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency

Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34+ cell gene therapy (GT) following busulfan reduced-intensity conditioning is a promising therapeutic approach for ADA-SCID, but long-term data are warranted. Here we report an analysis on long-term safety and efficacy data of 43 patients with ADA-SCID who received retroviral ex vivo bone marrow-derived hematopoietic stem cell GT. Twenty-two individuals (median follow-up 15.4 years) were treated in the context of clinical development or named patient program. Nineteen patients were treated post-marketing authorization (median follow-up 3.2 years), and two additional patients received mobilized peripheral blood CD34+ cell GT. At data cutoff, all 43 patients were alive, with a median follow-up of 5.0 years (interquartile range 2.4-15.4) and 2 years intervention-free survival (no need for long-term enzyme replacement therapy or allogeneic hematopoietic stem cell transplantation) of 88% (95% confidence interval 78.7-98.4%). Most adverse events/reactions were related to disease background, busulfan conditioning or immune reconstitution; the safety profile of the real world experience was in line with premarketing cohort. One patient from the named patient program developed a T cell leukemia related to treatment 4.7 years after GT and is currently in remission. Long-term persistence of multilineage gene-corrected cells, metabolic detoxification, immune reconstitution and decreased infection rates were observed. Estimated mixed-effects models showed that higher dose of CD34+ cells infused and younger age at GT affected positively the plateau of CD3+ transduced cells, lymphocytes and CD4+ CD45RA+ naive T cells, whereas the cell dose positively influenced the final plateau of CD15+ transduced cells. These long-term data suggest that the risk-benefit of GT in ADA remains favorable and warrant for continuing long-term safety monitoring. Clinical trial registration: NCT00598481 , NCT034786

The effects of heat treatment on the synthesis of nickel ferrite (NiFe2O4) nanoparticles using the microwave assisted combustion method

NiFe2O4 nanoparticles were synthesized using the microwave assisted combustion method based on metal nitrate salts and urea. To remain of organic matters and to stabilize the particles, samples were thermally treated at various temperatures from 300-800 degrees C. The prepared samples were characterized by X-ray diffraction (XRD), transmission electron microscopy (TEM), scanning electron microscopy (SEM), energy dispersive X-ray spectroscopy (EDX) thermogravimetric analysis (TGA) and vibrating sample magnetometry (VSM). The heat treated samples show the reflection planes of (111), (220), (222), (311), (400), (422), (511), and (440) which perfectly confirm to a cubic spinel phase of NiFe2O4 and no secondary phases were detected in the XRD patterns of the samples. The crystallite sizes calculated using the Debye-Scherrer formula were found to increase with the heat treatment temperature, from about 4 nm at 300 degrees C-85 nm at 800 degrees C. EDX results verify that the compositional mass rations were relevant, as expected from the synthesis. The micrographs of SEM and TEM showed that all of the samples have nano-crystalline behavior and particles indication cubic shape. Magnetization measurements were obtained at room temperature by using a VSM, which demonstrated that the all of the samples synthesized with heat treatment exhibited ferromagnetic behaviors. (C) 2014 Elsevier B.V. All rights reserved

PROTEIN-C AND ANTITHROMBIN-III IN CHILDREN WITH ACUTE-LEUKEMIA

In this study Protein C (PC) and antithrombin III (AT III) levels in childhood acute leukemia were investigated. The mean PC activity levels in 19 newly diagnosed cases of acute leukemia were significantly lower as compared with the normal controls (p < 0.05). A significant increase was found (p < 0.01) in the patients in remission. Prior to treatment 78.8 percent of patients had decreased PC activity levels, but all patients had normal PC activity during remission