1,721,548 research outputs found
Targeted Therapies for Pediatric AML: Gaps and Perspective
Full text linkAcute myeloid leukemia (AML) is a hematopoietic disorder characterized by numerous cytogenetic and molecular aberrations that accounts for ~25% of childhood leukemia diagnoses. The outcome of children with AML has increased remarkably over the past 30 years, with current survival rates up to 70%, mainly due to intensification of standard chemotherapy and improvements in risk classification, supportive care, and minimal residual disease monitoring. However, childhood AML prognosis remains unfavorable and relapse rates are still around 30%. Therefore, novel therapeutic approaches are needed to increase the cure rate. In AML, the presence of gene mutations and rearrangements prompted the identification of effective targeted molecular strategies, including kinase inhibitors, cell pathway inhibitors, and epigenetic modulators. This review will discuss several new drugs that recently received US Food and Drug Administration approval for AML treatment and promising strategies to treat childhood AML, including FLT3 inhibitors, epigenetic modulators, and Hedgehog pathway inhibitors
Evaluation of IgE Sensitization Profiles in a Pediatric Population with Wheat Allergy.
Full text linkIntroduction: IgE-mediated reactions to wheat can occur after ingestion, inhalation, contact or exercise. Among wheat allergens, Tri a14 and Tri a19 not only cause food allergy, but also baker's asthma (Tri a4) and exercise-induced anaphylaxis (Tri a19). Despite the prevalence of adverse reactions, few studies have been conducted in children.
Aim: To evaluate the pattern of sensitization to wheat allergenic components in a group of pediatric patients with sIgE to wheat referring to the Pediatric Allergy Unit of University of Bologna.
Materials and Methods: Patients were assessed by skin prick-test and serum specific IgE against pollens, wheat, gluten and the molecular allergens rTri a19 and rTri a14. The diagnosis was confirmed with open food challenges.
Results: The diagnosis of wheat allergy was confirmed in 7 patients (64%), of whom 2 (29%) suffered also from grass pollen allergy. The levels of specific IgE (geometric mean) to wheat and gluten were 5 times higher in allergic patients compared to tolerant ones. The comparison between the patterns of sensitization showed a higher prevalence of sensitization against gluten (100% vs. 75% in tolerant patients) and the molecular components rTri a14 (71% vs. 25%) and rTri a19 (71% vs. 0%) in the wheat-allergic group. Positive predictive value for rTria a19 was higher than rTri a14 (100% vs. 83%).
Conclusion: Patients with wheat allergy have different profiles of sensitization than the tolerant ones; in particular rTri a19 showed a higher positive predictive value than rTri a14. These findings need to be confirmed in a larger population
Targeting Hedgehog pathway in pediatric acute myeloid leukemia: challenges and opportunities
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Diagnostic accuracy of component resolved diagnosis in children with wheat allergy.
No full textBackground: Wheat allergy (WA) affects about 0.4-1% of the worldwide population and can occur after ingestion (food allergy), inhalation (baker’s asthma), contact (contact urticaria) or physical exercise after eating wheat-based foods. Among the 21 allergenic components of wheat, gliadins are markers of genuine wheat sensitization and ω-5 gliadin (Tri a 19) is a significant allergen in young children with immediate reactions to ingested wheat. Moreover, the non-specific lipid transfer protein (nsLTP) Tri a 14 is a relevant food allergen in wheat allergic patients from the Mediterranean area and is also associated with baker’s asthma. The aim of this study is to investigate the molecular patterns of wheat sensitization in a pediatric population and to assess the diagnostic accuracy of Tri a 14 and Tri a 19 in predicting IgE-mediated adverse reactions to wheat.
Method: For this cross-sectional study, we consecutively enrolled 19 patients (13 males, mean age 8 yrs - range 0,6-17) with sensitization to wheat grain referring to the Pediatric Allergy Outpatient Unit of S. Orsola- Malpighi Hospital of Bologna (Italy) from October 2013 to December 2014. Patients were assessed by SPT and serum sIgE against pollens, wheat, gluten and the molecular allergens rTri a 19 and rTri a 14. The diagnosis of WA was confirmed with open food challenges (OFC).
Results: The diagnosis of WA was confirmed in 6/19 patients (32%), of whom 2 (33%) suffered also from grass pollen allergy. The levels of sIgE (geometric mean) to wheat were almost 4 times higher in allergic patients compared to the tolerant ones (35,2 vs.8,2 kU/L). The comparison between the different patterns of sensitization showed among the wheat-allergic group a higher prevalence of sIgE sensitization against gluten (100% vs. 75%), rTri a 14 (50% vs. 46%) and rTri a 19 (83% vs. 0%). The molecular component rTria a19 showed a good sensitivity (83,3%) and specificity (100%) in diagnosing WA, and a PPV and a NPV higher than rTri a 14 (respectively 100% vs. 33,3% and 92,9% vs. 70%).
Conclusion: Patients with OFC confirmed WA have different profiles of sensitization than the tolerant ones; in particular the diagnostic accuracy of Tri a 19 is better than rTri a14 in the diagnosis of WA. However these findings need to be confirmed in a larger study population.
1. Palosuo K, Varjonen E, Kekki OM, et al. Wheat omega-5 gliadin is a major allergen in children with immediate allergy to ingested wheat. JACI 2001;108:634-38.
2. Mäkelä MJ, Eriksson C, Kotaniemi-Syrjänen A, et al. Wheat allergy in children - new tools for diagnostics. CEA 2014;44:1420-30
CBFA2T3-GLIS2-positive acute myeloid leukaemia. A peculiar paediatric entity
No full textThe scenario of paediatric acute myeloid leukaemia (AML), particularly non-Down syndrome acute megakaryoblastic leukaemia (non-DS-AMKL), has been recently revolutionized by the advent of large-scale, genomic sequencing technologies. In this changing landscape, a significantly relevant discovery has been represented by the identification of the CBFA2T3-GLIS2 fusion gene, which is the result of a cryptic inversion of chromosome 16. It is the most frequent chimeric oncogene identified to date in non-DS-AMKL, although it seems not to be exclusively restricted to the French-American-British M7 subgroup. The CBFA2T3-GLIS2 fusion gene characterizes a subtype of leukaemia that is specific to paediatrics, having never been identified in adults. It characterizes an extremely aggressive leukaemia, as the presence of this fusion is associated with a grim outcome in almost all of the case series reported, with overall survival rates ranging between 15% and 30%. Although the molecular basis that underlies this leukaemia subtype is still far from being completely elucidated, unique functional properties induced by CBFA2T3-GLIS2 in the leukaemogenesis driving process have been recently identified. We here review the peculiarities of CBFA2T3-GLIS2-positive AML, describing its intriguing clinical and biological behaviour and providing some challenging targeting opportunities
72th Congress of the Italian Society of Pediatrics. Joint National Meeting SIP, SINP, Study Group on Accreditation and Improvement of the Quality, SIP National Work Group on Migrant Child.
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Going Beyond Counting First Authors in Author Co-citation Analysis
Full text linkThe present study examines one of the fundamental aspects of author co-citation analysis (ACA) - the way co-citation
counts are defined. Co-citation counting provides the data on which all subsequent statistical analyses and mappings
are based, and we compare ACA results based on two different types of co-citation counting - the traditional type that
only counts the first one among a cited work's authors on the one hand and a non-traditional type that takes into
account the first 5 authors of a cited work on the other hand. Results indicate that the picture produced through this non-traditional author co-citation counting contains more coherent author groups and is therefore considerably clearer. However, this picture represents fewer specialties in the research field being studied than that produced through the traditional first-author co-citation counting when the same number of top-ranked authors is selected and analyzed. Reasons for these effects are discussed
Perinatal Stem Cell Therapy to Treat Type 1 Diabetes Mellitus: A Never-Say-Die Story of Differentiation and Immunomodulation
Full text linkHuman term placenta and other postpartum-derived biological tissues are promising sources of perinatal cells with unique stem cell properties. Among the massive current research on stem cells, one medical focus on easily available stem cells is to exploit them in the design of immunotherapy protocols, in particular for the treatment of chronic non-curable human diseases. Type 1 diabetes is characterized by autoimmune destruction of pancreatic beta cells and perinatal cells can be harnessed both to generate insulin-producing cells for beta cell replenishment and to regulate autoimmune mechanisms via immunomodulation capacity. In this study, the strong points of cells derived from amniotic epithelial cells and from umbilical cord matrix are outlined and their potential for supporting cell therapy development. From a basic research and expert stem cell point of view, the aim of this review is to summarize information regarding the regenerative medicine field, as well as describe the state of the art on possible cell therapy approaches for diabetes
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