Karadaş Nihal, Göktepe Şerife Şebnem Önen, Baş İlke, Ece Dilek, Özdemir Hamiyet Hekimci, Balkan Can, Kavaklı Kaan, Aydinok Yeşim, Karapinar Deniz Yilmaz
Pediatric Hematology Department, Ege University School of Medicine, Bornova, Izmir, Turkey.
Department of Pediatric Hematology, Children's Hospital, Izmir, Turkey.
Int J Hematol. 2023 Mar;117(3):446-455. doi: 10.1007/s12185-022-03497-4. Epub 2022 Nov 19.
Chronic myeloid leukemia (CML) is very rare during childhood. Tyrosine kinase inhibitors (TKI) provide very good results in terms of survival. The medical records of 15 chronic phase (CP)-CML patients in a university hospital pediatric hematology department between 1997 and 2022 were reviewed retrospectively. Complete hematological response was documented in all patients between 20 and 68 (median 30) days of treatment. Major molecular response was achieved in seven patients within 6 months. Median follow-up for the study group was 79 (range 3-330) months and overall survival was 100%. Three patients (2 blastic transformation, 1 therapy resistant) underwent bone marrow transplantation (BMT) and one with blastic transformation is scheduled to undergo BMT. TKI were discontinued in three patients after a median of 86 (range 73-177) months. The complete molecular remission maintenance period before discontinuation of TKI was 81 (range 62-122) months. While no molecular relapse was seen before the last follow-up, the median overall follow-up period was 152 (range 131-300) months. In conclusion, recent advances have led to a very good prognosis for children with CP-CML. With TKI treatment, most patients continue their lives without disease progression. Additionally, in selected patients TKI can be discontinued without molecular relapse.
慢性髓性白血病（CML）在儿童期非常罕见。酪氨酸激酶抑制剂（TKI）在生存方面取得了很好的效果。回顾性分析了1997年至2022年间某大学医院儿科血液科15例慢性期（CP）-CML患者的病历。所有患者在治疗20至68天（中位时间30天）时均记录到完全血液学缓解。7例患者在6个月内达到主要分子学缓解。研究组的中位随访时间为79个月（范围3至330个月），总生存率为100%。3例患者（2例发生急变，1例耐药）接受了骨髓移植（BMT），1例发生急变的患者计划接受BMT。3例患者在中位时间86个月（范围73至177个月）后停用TKI。停用TKI前的完全分子学缓解维持期为81个月（范围62至122个月）。在最后一次随访前未观察到分子学复发，中位总随访期为152个月（范围131至300个月）。总之，最近的进展使CP-CML儿童患者的预后非常好。通过TKI治疗，大多数患者能够继续生活且疾病无进展。此外，在部分患者中，停用TKI后不会出现分子学复发。
