Ramiz Sarah, Elhaj Osama, Siddiqui Khawar, Khan Saadiya, AlSaedi Hawazen, AlAnazi Awatif, Al-Ahmari Ali, Al-Jefri Abdullah, Sahvan Oudai, Ayas Mouhab, Ghemlas Ibrahim
Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital and Research Center, Riyadh 11211, Saudi Arabia.
College of Medicine, Alfaisal University, Riyadh 11533, Saudi Arabia.
J Hematol. 2023 Feb;12(1):16-26. doi: 10.14740/jh1065. Epub 2023 Feb 25.
Despite pronounced improvement in overall survival (OS) in pediatric leukemia, a proportion of patients continue to suffer from lack of response or relapse, and the management of such patients is exceedingly difficult. Immunotherapy and engineered chimeric antigen receptor (CAR) T-cell therapy have shown promising results in the course of relapsed or refractory acute lymphoblastic leukemia (ALL). However, conventional chemotherapy continues to be utilized for re-induction purposes whether independently or in combination with immunotherapy.
Forty-three pediatric leukemia patients (age < 14 years at diagnosis) consecutively diagnosed at our institution and got treated with clofarabine based regimen at a single tertiary care hospital between January 2005 and December 2019 were enrolled in this study. ALL comprised of 30 (69.8%) patients of the cohort while the remaining 13 (30.2%) were with acute myeloid leukemia (AML).
Post-clofarabine bone marrow (BM) was negative in 18 (45.0%) cases. Overall clofarabine failure rate was 58.1% (n = 25) with 60.0% (n = 18) in ALL and 53.8% (n = 7) in AML (P = 0.747). Eighteen (41.9%) patients eventually underwent hematopoietic stem cell transplantation (HSCT); 11 (61.1%) were from ALL group and remaining seven (38.9%) were AML (P = 0.332). Three- and 5-year OS of our patients was 37.7±7.6% and 32.7±7.3%. There was a trend of better OS for ALL patients compared to AML (40.9±9.3% vs. 15.4±10.0%, P = 0.492). Cumulative probability of 5-year OS was significantly better in transplanted patients (48.1±12.1% vs. 21.4±8.4%, P = 0.024).
Though almost 90% of our patients proceeded to HSCT with complete response post-clofarabine treatment, yet clofarabine-based regimens are associated with the significant burden of infectious complications and sepsis-related deaths.
尽管小儿白血病的总生存期（OS）有了显著改善，但仍有一部分患者持续存在反应不佳或复发的情况，对此类患者的管理极为困难。免疫疗法和工程化嵌合抗原受体（CAR）T细胞疗法在复发或难治性急性淋巴细胞白血病（ALL）的治疗过程中已显示出有前景的结果。然而，无论是单独使用还是与免疫疗法联合使用，传统化疗仍被用于再诱导治疗。
本研究纳入了2005年1月至2019年12月期间在我们机构连续诊断并在一家三级护理医院接受基于氯法拉滨方案治疗的43例小儿白血病患者（诊断时年龄<14岁）。该队列中，ALL患者有30例（69.8%），其余13例（30.2%）为急性髓系白血病（AML）。
氯法拉滨治疗后，18例（45.0%）患者的骨髓（BM）呈阴性。氯法拉滨总体失败率为58.1%（n = 25），其中ALL为60.0%（n = 18），AML为53.8%（n = 7）（P = 0.747）。18例（41.9%）患者最终接受了造血干细胞移植（HSCT）；11例（61.1%）来自ALL组，其余7例（38.9%）为AML（P = 0.332）。我们患者的3年和5年总生存率分别为37.7±7.6%和32.7±7.3%。ALL患者的总生存率有高于AML的趋势（40.9±9.3%对15.4±10.0%，P = 0.492）。移植患者的5年总生存累积概率明显更好（48.1±12.1%对21.4±
