Childhood ITP: can we venture below 20 000?
نویسنده
چکیده
Contrary to the exploits of Captain Nemo and his crew in Jules Verne’s epic novel, there have been few hearty souls willing to venture below the surface of conventional childhood acute immune thrombocytopenic purpura (ITP) management and ask whether symptoms, platelet count, and intervention at diagnosis could predict bleeding severity and incidence in the next month. Accordingly, controversy still exists regarding management of ITP in these children. Specifically, should treatment be administered at all, for whom, and with which drugs? Drug treatment of ITP is often used to prevent serious bleeding, and the initial platelet count is assumed to be a surrogate for bleeding potential, especially for those who present with minimal bleeding. By most accounts, life-threatening hemorrhage, especially intracranial or central nervous system (CNS) bleeding, is quite rare (approximately 1 in 800 cases; 0.125%).1 But clinical anxiety often creeps in when there is mild mucosal bleeding, and the platelet count is firmly below sea level (less than or equal to 20 000 per mm3). This study by Neunert et al is the first to prospectively chronicle the severity of hemorrhage at diagnosis and in the next 4 weeks, as well as the relationship of clinical symptoms to the platelet count and treatment intervention. Eight hundred sixty-three of 1106 enrolled patients were fully evaluable, and the vast majority had no or mild bleeding at diagnosis and a platelet count of less than or equal to 20 000 per mm3. Therapy aimed at raising the platelet count was left to provider discretion. The table illustrates the distribution of various treatments for those presenting with no to mild bleeding symptoms and the number of patients (n 3) with serious bleeding within the first month after diganosis. Severe bleeding was defined as mucosal or CNS bleeding, requiring hospitalization and/or blood transfusions. All 3 patients had nonCNS bleeding. As expected, there was a significant difference between the platelet count and bleeding symptoms at diagnosis: mild bleeding equals 17 000/mm3, moderate equals 10 000/mm3, and severe equals 9000/mm3. A model of disease progression. Multiple myeloma is characterized by the clonal expansion of malignant plasma cells driven by initiating genetic events, such as oncogene activating translocations, for example, t(4;14), hyperdiploidy, deletion of chromosome 13, and deletion of 17p13. During subclinical growth, an inherent genomic instability characteristic of these terminally differentiated cells leads to secondary genetic events, such as gains of chromosome 1q and deletion of 1p as well as gains of 8q24, that provide a growth and/or survival advantage to a subpopulation of cells. A gene expression–based signature, reflective of a minimum proportion of cells with these secondary lesions, can define high-risk disease. Following therapy, such as stem-cell supported high-dose melphalan, a sizeable portion of patients achieve complete remission that can be monitored by MFC. Toward relapse, both the percentage of tumor cells with gains of chromosome 1q and molecular risk score invariably increase. These data suggest that a subpopulation of high-risk cells survives therapy and eventually contributes to progressively resistant relapses. The use of MFC to measure MRD, as reported by Paiva et al, appears well suited for the identification of cell-surface marker(s)discriminating tumorsubpopulations,exhibiting differential cytoreduction and regrowth kinetics, with significant impact on the clinical management of the disease.
منابع مشابه
Rituximab treatment of refractory fludarabine-associated immune thrombocytopenia in chronic lymphocytic leukemia.
Fludarabine can exacerbate idiopathic thrombocytopenia (ITP) in chronic lymphocytic leukemia (CLL). We report 3 CLL patients with refractory fludarabine-associated ITP who responded to rituximab. The patients had Rai stages III, III, and IV disease. Before fludarabine treatment, the platelet counts were 141 000/microL, 118 000/microL, and 70 000/microL. ITP developed within week 1 of cycle 3 in...
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OBJECTIVE To find the pattern of idiopathic thrombocytopenic purpura (ITP) (acute/chronic) and to describe presenting features and clinical characteristics of the disease in children below 14 years of age in a newly developed Arabian society. METHODS This retrospective, descriptive study was carried out at the Pediatric Department of the Hamad General Hospital, Hamad Medical Corporation, Qata...
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Controversy exists regarding management of children newly diagnosed with immune thrombocytopenic purpura (ITP). Drug treatment is usually administered to prevent severe hemorrhage, although the definition and frequency of severe bleeding are poorly characterized. Accordingly, the Intercontinental Childhood ITP Study Group (ICIS) conducted a prospective registry defining severe hemorrhage at dia...
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عنوان ژورنال:
- Blood
دوره 112 10 شماره
صفحات -
تاریخ انتشار 2008