Mitura-Lesiuk Małgorzata, Filiks-Litwin Barbara, Małek Urszula, Kowalczyk Jerzy R
Klinika Hematologii i Onkologii Dzieciecej, Dzieciecy Szpital Kliniczny, Chodźki 2, 20-093 Lublin, Poland.
Med Wieku Rozwoj. 2004 Jul-Sep;8(3 Pt 2):834-8.
Essential thrombocythemia is a myeloproliferative disease resulting from the clonal proliferation of a multipotent myeloid stem cell with an increased platelet count in peripheral blood. In 2% of cases the cause remains unknown. It is then called idiopathic essential thrombocythemia.
The objective of this study was to present the diagnostic and therapeutic management of essential thrombocythemia in children.
The authors analysed complete medical records of 5 patients treated for essential thrombocythemia in the Department of Children Haematology and Oncology, Medical University Lublin during a 6 years (1996-2002) period. The investigated group consisted of 5 children: 3 boys and 2 girls with mean age of 7 years and 8 months (varying from 5 years and 3 months to 11 years and 8 months).
The peripheral platelet count was above 1200 x 10(9)/L. Three patients developed functional symptoms of microvascular blood flow alteration (headaches, sensory disorders. paresthesiae). In 2 patients thrombocythemia was asymptomatic and the diagnosis was made incidentally. Diagnosis of essential thrombocythemia was made according to the criteria of the Polycythemia Vera Study Group and treatment with anagrelide hydrochloride was started. One patient due to bad drug tolerance was treated with hydroxyurea. Treatment of essential thrombocythemia in all patients resulted in: (1) decreased (500-600 x 10(9)/L) platelet count in peripheral blood; (2) resolving the symptoms of small vessels obturation (headaches, sensory disorders, paresthesiae); (3) low platelet separating megakaryocytes count in control myelograms (4) no transformation into acute neoplastic disease.
In the treatment of children with essential thrombocythemia anagrelid was effective in four of five patients.
原发性血小板增多症是一种骨髓增殖性疾病，由多能髓系干细胞的克隆性增殖引起，外周血血小板计数增加。2%的病例病因不明，称为特发性原发性血小板增多症。
本研究的目的是介绍儿童原发性血小板增多症的诊断和治疗管理。
作者分析了卢布林医科大学儿童血液学和肿瘤学系在6年（1996 - 2002年）期间治疗的5例原发性血小板增多症患者的完整病历。研究组包括5名儿童：3名男孩和2名女孩，平均年龄为7岁8个月（范围从5岁3个月至11岁8个月）。
外周血小板计数高于1200×10⁹/L。3例患者出现微血管血流改变的功能性症状（头痛、感觉障碍、感觉异常）。2例患者血小板增多症无症状，诊断为偶然发现。根据真性红细胞增多症研究组的标准诊断为原发性血小板增多症，并开始用盐酸阿那格雷治疗。1例患者因药物耐受性差用羟基脲治疗。所有患者原发性血小板增多症的治疗结果为：（1）外周血血小板计数降低（500 - 600×10⁹/L）；（2）小血管阻塞症状（头痛、感觉障碍感觉异常）消失；（3）对照骨髓检查中血小板分离巨核细胞计数降低；（4）未转化为急性肿瘤性疾病。
在儿童原发性血小板增多症的治疗中，阿那格雷对5例患者中的4例有效。
