Alkhatib Mohammed Awad
Department of Pediatrics, University of Khartoum, Khartoum, Sudan.
Pediatr Hematol Oncol. 2011 Mar;28(2):100-5. doi: 10.3109/08880018.2010.512949. Epub 2010 Nov 17.
Chronic myeloid leukemia (CML) is infrequent in children. The best-known treatment is stem cell transplant. In a country with limited resources like Sudan, such expensive therapy is not available. Alternative approaches are needed to help these children. The tyrosine kinase inhibitor-imatinib-might be an answer to this problem. The objective of this study is to determine the pattern of children with CML, their hematological response to imatinib, and tolerance and side effects to this drug. All patients with confirmed BCR-ABL by polymerase chain reaction (PCR) were included in this study. The relevant data were collected and the patients were started on imatinib. Response to treatment was assessed clinically and hematologically only. Cytogenetics and molecular studies are not available. The average age of the 31 patient evaluated was 8.7 years, 2 patients were less than 1 year, and 5 patients, ie, 16%, were 2 years old or less. Chloroma was observed in 6 (19%) patients. The average of the white blood cell (WBC) count was 206.6 x 10(9)/L and the platelet count average was 523 x 10(9)/L. Two (6.5%) of the 31 patients presented as acute myeloid leukemia (AML). All patients had hematological remission within 2 months. Twenty-three (74%) had a sustained remission over an average follow-up period of 26 months (2-67 months). Six (19%) patients died with AML or sepsis. Side effects to imatinib were infrequent, observed in 4 out of 29 (13.7%) patients, and mild. One patient only needed dose modification. No resistance was observed during this period. CML patients present at an earlier age than in other parts of the world. Imatinib is safe and effective in treating pediatric CML where stem cell transplant in not available. Further cytogenetics are important to monitor response and proper management.
慢性髓性白血病(CML)在儿童中并不常见。最著名的治疗方法是干细胞移植。在像苏丹这样资源有限的国家,这种昂贵的治疗方法无法获得。需要其他方法来帮助这些儿童。酪氨酸激酶抑制剂伊马替尼可能是解决这个问题的答案。本研究的目的是确定CML患儿的模式、他们对伊马替尼的血液学反应以及对该药物的耐受性和副作用。所有通过聚合酶链反应(PCR)确诊为BCR-ABL的患者均纳入本研究。收集相关数据,患者开始使用伊马替尼治疗。仅通过临床和血液学评估治疗反应。无法进行细胞遗传学和分子研究。接受评估的31例患者的平均年龄为8.7岁,2例患者年龄小于1岁,5例患者(即16%)年龄为2岁或更小。6例(19%)患者出现绿色瘤。白细胞(WBC)计数平均为206.6×10⁹/L,血小板计数平均为523×10⁹/L。31例患者中有2例(6.5%)表现为急性髓性白血病(AML)。所有患者在2个月内均实现血液学缓解。23例(74%)患者在平均26个月(2 - 67个月)的随访期内持续缓解。6例(19%)患者死于AML或败血症。伊马替尼的副作用并不常见,29例患者中有4例(13.7%)出现,且症状轻微。仅1例患者需要调整剂量。在此期间未观察到耐药情况。CML患者的发病年龄比世界其他地区更早。在无法进行干细胞移植的情况下,伊马替尼治疗儿童CML安全有效。进一步的细胞遗传学检查对于监测反应和合理管理很重要。