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Essential versus reactive thrombocythemia in children: Retrospective analyses of 12 cases
Author(s) -
ElMoneim Abeer Abd,
Kratz Christian P.,
Böll Silke,
Rister Manfred,
Pahl Heike L.,
Niemeyer Charlotte M.
Publication year - 2007
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.21128
Subject(s) - medicine , thrombocytosis , anagrelide , essential thrombocythemia , asymptomatic , myelofibrosis , organomegaly , retrospective cohort study , population , pediatrics , surgery , thrombosis , polycythemia vera , leukocytosis , platelet , disease , bone marrow , environmental health
Background Essential thrombocythemia (ET) rarely occurs in the pediatric population and little is known about the clinical course and the molecular characteristics. Procedure In this retrospective multi‐institutional study we examine the clinical, hematological, and molecular features of 12 children aged 5–16 years with thrombocytosis and a suspected diagnosis of ET. Results Median follow‐up was 59 months (range 10–72). Seven patients presented with clinical symptoms potentially related to thrombocytosis. The remaining five patients were diagnosed incidentally. Median platelet count at diagnosis was 1,325 × 10 9 /L (range 600–3,050). In 11 out of 12 cases bone marrow morphology was consistent with ET, the remaining patient had chronic idiopathic myelofibrosis. Cytogenetic analyses were normal in all studied cases and only one out of nine analyzed cases harbored a JAK V617F allele. Within 6 months after initial presentation one patient who was initially asymptomatic developed thrombosis and another patient had mild bleeding. Eight patients were treated with acetylsalicylic acid, one patient received hydroxyurea, and two patients received anagrelide. At last follow‐up, all patients were alive and none had developed leukemia. Five patients experienced hematological remission. Two children had not received any therapy. During the course of their disease, nine patients developed symptoms possibly attributable to an elevated platelet count. Conclusions In JAK2 mutation negative cases, long‐term follow‐up is helpful to distinguish between primary and secondary thrombocytosis. Secondary cases are not associated with organomegaly but may present with unspecific symptoms. Indications for treatment in children remain unclear. Pediatr Blood Cancer 2007;49:52–55. © 2006 Wiley‐Liss, Inc.