Jabbar Naeem, Khayyam Naema, Arshad Uzma, Maqsood Sidra, Hamid Syed Ahmer, Mansoor Neelum
Pediatric Hematology-Oncology Section of Pediatric Department, The Indus Hospital, Plot C-76, Sector 31/5, Opposite Darussalam Society, Korangi Crossing, Karachi, 75190 Pakistan.
Jinnah Medical College Hospital, Karachi, Pakistan.
Indian J Hematol Blood Transfus. 2021 Oct;37(4):569-575. doi: 10.1007/s12288-021-01404-1. Epub 2021 Jan 30.
The overall survival of Acute Promyelocytic Leukemia (APL), reported in recent studies, is approaching to 90% wherein, arsenic trioxide (ATO) and all-trans retinoic acid (ATRA) are used as the mainstay of treatment with either limited or no use of anthracycline and cytarabine. This study is aimed to ascertain the outcome of children with APL using similar approach. A total of 30 patients with APL, registered from January 2015 to December 2018, were reviewed. Diagnosis was established on bone marrow aspirate and confirmed by the presence of PML-RARA translocation. Treatment protocol was based on Australian APML 4 study performed by Australian Leukemia Lymphoma Group (ALLG). Lumbar puncture was not performed as it was not part of the protocol due to the risk of bleeding. The mean age in current cohort was 9 years with 53% males. Seven (23.3%) patients died and three (10%) abandoned treatment during induction. Twenty patients completed the intensive phase of chemotherapy and all (100%) of them attained molecular remission (MR). One patient dropped out after MR whereas, 19 remain on follow up with no evidence of disease, reflecting disease free survival (DFS) of 95%. With a median follow up of 2.5 years (range 2.1-4.8 years) the 5 years Kaplan-Meier estimate of OS was 63% and 73%, with and without abandonment, respectively. Analysis of outcome according to risk groups revealed inferior outcome of high risk (HR) group (38% and 50% with and without abandonment, respectively) in contrast to standard risk (SR) group which showed better outcome (82% and 88% with and without abandonment, respectively). The attainment of 100% molecular remission and absence of relapse supports the effectiveness of this regimen. Moreover, it is found to be less toxic and therefore, can be conveniently managed in day-care settings.
近期研究报道,急性早幼粒细胞白血病(APL)的总生存率接近90%,其中三氧化二砷(ATO)和全反式维甲酸(ATRA)被用作主要治疗药物,蒽环类药物和阿糖胞苷的使用有限或未使用。本研究旨在确定采用类似方法治疗的APL患儿的治疗结果。回顾了2015年1月至2018年12月登记的30例APL患者。通过骨髓穿刺确诊,并经PML-RARA易位证实。治疗方案基于澳大利亚白血病淋巴瘤组(ALLG)进行的澳大利亚APML 4研究。由于存在出血风险,腰椎穿刺未作为方案的一部分进行。当前队列的平均年龄为9岁,男性占53%。7例(23.3%)患者在诱导期死亡,3例(10%)放弃治疗。20例患者完成了强化化疗阶段,所有患者(100%)均达到分子缓解(MR)。1例患者在达到MR后退出,19例仍在随访中,无疾病证据,无病生存率(DFS)为95%。中位随访2.5年(范围2.1 - 4.8年),5年Kaplan-Meier估计的总生存率(OS)分别为63%(未放弃治疗)和73%(放弃治疗)。根据风险组分析结果显示,高危(HR)组的结果较差(放弃治疗和未放弃治疗的分别为38%和50%),而标准风险(SR)组的结果较好(放弃治疗和未放弃治疗的分别为82%和88%)。100%的分子缓解率和无复发支持了该方案的有效性。此外,发现该方案毒性较小,因此可以在日间护理环境中方便地进行管理。