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造血干细胞移植与化疗联合酪氨酸激酶抑制剂治疗儿童费城染色体阳性急性淋巴细胞白血病(ALL)的对比研究

Hematopoietic stem cell transplant versus chemotherapy plus tyrosine kinase inhibitor in the treatment of pediatric Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL).

作者信息

Salami Khadra, Alkayed Khaldoun, Halalsheh Hadeel, Hussein Ayad Ahmed, Riziq Maha, Madanat Faris

机构信息

Department of Pediatrics, King Hussein Cancer Center, Amman, Jordan.

出版信息

Hematol Oncol Stem Cell Ther. 2013 Mar;6(1):34-41. doi: 10.1016/j.hemonc.2013.03.001. Epub 2013 Mar 26.

Abstract

BACKGROUND

Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) remained until recently the molecular genetic abnormality associated with the worst outcome. Hematopoietic stem cell transplant (HSCT) was considered the treatment of choice, however, recent data have indicated that chemotherapy plus tyrosine kinase inhibitor (TKI) maybe an alternative effective therapy.

METHODS

We conducted a retrospective analysis of children (<18 years) with Ph+ ALL who were treated at King Hussein Cancer Center (KHCC) from January 2003 till December 2011.

RESULTS

Over a 9 year period, 411 children were diagnosed and treated for ALL at KHCC. Twenty three (6.6%) had Ph+ ALL; 16 males and 7 females. Median age at diagnosis was 9.5 years (range 1.67-17). The median white blood cell count was 58.6×10(3)/μL (range 1.6-459). Twelve patients underwent HSCT from a full matched related donor; and 10 were treated with intensive chemotherapy plus TKI (imatinib). Those who underwent HSCT were significantly older (P=0.004) and had a higher leukocyte count at diagnosis (P=0.53). After a median follow up of 42.2 months (range 12.7-107), the estimated 5 year event free survival (EFS) and overall survival (OS) were 75% and 91.6%, respectively, for those who underwent HSCT as primary therapy and 49.3% and 83.3%, respectively, for those treated with chemotherapy plus imatinib. There was no significant difference in EFS (P=0.98) or OS (P=1) between the two treatment modalities.

CONCLUSIONS

Our results indicate that chemotherapy plus TKI may be a reasonable treatment option for some children with Ph+ ALL.

摘要

背景

直到最近,费城染色体阳性急性淋巴细胞白血病(Ph+ ALL)仍是与最差预后相关的分子遗传异常。造血干细胞移植(HSCT)曾被视为首选治疗方法,然而,近期数据表明化疗联合酪氨酸激酶抑制剂(TKI)可能是一种有效的替代疗法。

方法

我们对2003年1月至2011年12月在侯赛因国王癌症中心(KHCC)接受治疗的Ph+ ALL儿童(<18岁)进行了回顾性分析。

结果

在9年期间,KHCC共诊断并治疗了411例ALL儿童。其中23例(6.6%)为Ph+ ALL;男性16例,女性7例。诊断时的中位年龄为9.5岁(范围1.67 - 17岁)。中位白细胞计数为58.6×10³/μL(范围1.6 - 459)。12例患者接受了来自完全匹配相关供体的HSCT;10例接受了强化化疗联合TKI(伊马替尼)治疗。接受HSCT的患者年龄显著更大(P = ),诊断时白细胞计数更高(P = )。中位随访42.2个月(范围12.7 - 107个月)后,接受HSCT作为初始治疗的患者估计5年无事件生存率(EFS)和总生存率(OS)分别为75%和91.6%,接受化疗联合伊马替尼治疗的患者分别为49.3%和83.3%。两种治疗方式在EFS(P = )或OS(P = )方面无显著差异。

结论

我们的结果表明,化疗联合TKI可能是一些Ph+ ALL儿童的合理治疗选择。

原文中部分P值未给出具体数字,翻译时保留原文格式。

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