Gulf J Oncolog. 2017 Jan;1(23):15-20.
Chronic myeloid leukemia (CML) is relatively rare malignancy in childhood. There are limited studies of use of Imatinib Mesylate (IM) alone in management of CML in this age group.
We retrospectively analyzed the outcome of 30 consecutive children with CML chronic phase treated with IM alone.
The median age at the time of diagnosis was 11 years with male preponderance. Asthenia and abdominal discomfort due to splenomegaly were the most common presenting features and splenomegaly a dominant sign. At time of starting IM, 19 children were in early CP while 11 were in late CP. Complete hematological remission was achieved in 90% with a median time to achieve CHR was 60 days. Among evaluable children, 83.3% achieved cytogenetic response (CyGR). Those who achieved complete CyGR were in major molecular remission at the time of last follow up. At 3 years, progression-free survival was 81.5% and overall survival was 100%. At 10 years, 12 (40%) children failed on IM therapy of which 3(10%) children developed primary IM resistance while 9 (30%) developed secondary IM resistance. IM was well tolerated and severe (grade III-IV) events were infrequent. Non-haematological toxicities were uncommon except hypopigmentation of skin which was seen in 60% of the cohort.
Presenting features of CML-CP in children is comparable to other Indian and international studies. IM is very effective and safe drug for the first line treatment of CML-CP in children. It is very effective in inducing CHR. Adherence to treatment is very important for achieving CyGR and long term survival. This data will be useful for financially deprived children in developing countries where allogeneic stem cell transplant (SCT) or second line tyrosine kinase inhibitors (TKIs) is not an affordable option.
慢性髓性白血病（CML）在儿童期是相对罕见的恶性肿瘤。关于在该年龄组中单独使用甲磺酸伊马替尼（IM）治疗CML的研究有限。
我们回顾性分析了30例连续接受单独IM治疗的CML慢性期儿童的治疗结果。
诊断时的中位年龄为11岁，男性居多。乏力和因脾肿大引起的腹部不适是最常见的临床表现，脾肿大是主要体征。开始使用IM时，19例儿童处于慢性期早期，11例处于慢性期晚期。90%的患儿实现了完全血液学缓解，达到完全血液学缓解的中位时间为60天。在可评估的儿童中，83.3%实现了细胞遗传学反应（CyGR）。那些实现完全细胞遗传学反应的患儿在最后一次随访时处于主要分子缓解状态。3年时，无进展生存率为81.5%，总生存率为100%。10年时，12例（40%）儿童IM治疗失败，其中3例（10%）儿童出现原发性IM耐药，9例（30%）出现继发性IM耐药。IM耐受性良好，严重（III-IV级）事件很少见。非血液学毒性不常见，除了60%的队列患儿出现皮肤色素减退。
儿童CML慢性期的临床表现与其他印度及国际研究结果相当。IM是儿童CML慢性期一线治疗非常有效且安全的药物。它在诱导完全血液学缓解方面非常有效。坚持治疗对于实现细胞遗传学反应和长期生存非常重要。这些数据对于发展中国家经济贫困的儿童将是有用的，在这些国家，异基因干细胞移植（SCT）或二线酪氨酸激酶抑制剂（TKIs）不是可负担得起的选择。
