1,721,064 research outputs found
Thrombotic complications in children with haematologic malignancies
No full textThrombosis is rare in children but they are more common and severe in children with hematological malignances. Thrombotic events in children with acute leukemia, lymphoma, acute lymphoblastic leukemia and chronic myeloproliferative neoplasms are explored and evaluate
Philadelphia-negative chronic myeloproliferative disorders in children
No full textPhiladelphia-negative chronic myeloproliferative disorders (Ph-MPDs) are clonal haematopoietic disorders typically found in medianadvanced
age. The recently discovered acquired JAK2V617F mutation and other somatic mutations in JAK2, TPO, MPL and EPO-R in
the myeloid cells of these patients are now considered specific biological markers, and their presence is considered a main diagnostic
criterion of these diseases. Ph-MPD are extremely rare in children and need to be distinguished from other more common causes
of thrombocytosis and erythrocytosis. Essential thrombocythaemia (ET) is the less rare Ph-MPD, while polycythaemia vera (PV)
and primary myelofibrosis (PMF) are occasional findings in paediatrics. In this group of patients the incidence of haemorrhagic and
thrombotic complications, as well as transformation into MF and acute leukaemia, seem to be rarer than in adults. Moreover, the
biological markers found in adults with Ph-MPDs are not as common in paediatric cases, and familial forms are relatively frequent.
Therefore, the tests used in adults are not exhaustive for most children. Because gene mutations are detectable in a minority of
paediatric cases, new markers are needed to better understand such cases. Therapy is devoted to reducing symptoms, not improving
collateral drug effects. New drugs targeting the JAK2 molecule are under development and open new possibilities in the treatment of
these rare diseases
Pediatric myeloproliferative neoplasms
No full textThe increasing knowledge accumulated over recent years in adults with Philadelphia-negative
myeloproliferative disorders (Ph-MPD) has prompted better evaluation of the rare pediatric essential
thrombocythemia (ET), polycythemia vera (PV) and primary idiopathic myelofibrosis (PMF). A few cases
of PV, ET and PMF in children were published in the English literature and an up-date of these works
is here given. The experience recently collected by the Italian Pediatric Hemato-Oncology Association
is also reported. Overall, the findings suggest that pediatric Ph-negative MPD are heterogeneous diseases
and before the diagnosis is made, hereditary disorders have to be excluded. Because JAK2 and
other genes mutations are only detectable in a minority of children with sporadic forms, complementary
markers, such as clonality of hematopoieis, spontaneous erythroid colony growth, and so on
should be performed for the diagnosis and new tests have to be identified. Diagnostic criteria that fit
pediatric ET, PV and PMF have to be considered. Prognosis in children is still unknown and treatment
guidelines need to be established; therefore, prospective observations and clinical trials are needed
Essential Thrombocythemia in Children and Adolescents
Full text linkThis paper reviews the features of pediatric essential thrombocythemia (ET). ET is a rare disease in children, challenging pediatric and adult hematologists alike. The current WHO classification acknowledges classical Philadelphia-negative MPNs and defines diagnostic criteria, mainly encompassing adult cases. The presence of one of three driver mutations (JAK2V617F, CALR, and MPL mutations) represent the proof of clonality typical of ET. Pediatric ET cases are thus usually confronted by adult approaches. These can fit only some patients, because only 25-40% of cases present one of the driver mutations. The diagnosis of hereditary, familial thrombocytosis and the exclusion of reactive/secondary thrombocytosis must be part of the diagnostic process in children and can clarify most of the negative cases. Still, many children present a clinical, histological picture of ET, with a molecular triple wild-type status. Moreover, prognosis seems more benign, at least within the first few decades of follow-up. Thrombotic events are rare, and only minor hemorrhages are ordinarily observed. As per the management, the need to control symptoms must be balanced with the collateral effects of lifelong drug therapy. We conclude that these differences concert a compelling case for a very careful therapeutic approach and advocate for the importance of further cooperative studies
Antithrombin plasma levels and fibtem determination in children with acute lymphoblastic leukemia undergoing asparaginase treatment
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Polycythemia vera and high serum erythropoietin in young patients: occurrence of VHL mutation
No full textHETEROGENEITY OF TDT+, HLA-DR+ ACUTE-LEUKEMIA - IMMUNOLOGICAL, IMMUNOCYTOCHEMICAL AND CLINICAL-EVIDENCE OF LYMPHOID AND MYELOID ORIGIN
No full text: IMATINIB MESYLATE INDUCES HIGH COMPLETE CYTOGENETIC AND MAJOR MOLECULAR RESPONSE RATES IN CHILDREN AND ADOLESCENTS WITH PHILADELPHIA CHROMOSOME-POSITIVE CHRONIC MYELOGENOUS LEUKAEMIA IN CHRONIC PHASE
No full textEarly cerebral sinovenous thrombosis in a child with acute lymphoblastic leukemia carrying the prothrombin G20210A variant: a case report and review of the literature.
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