Shifting gears to differentiation agents in acute promyelocytic leukemia with resource constraints—a cohort study

Treatment of acute promyelocytic leukaemia has emerged as a major success in hemato-oncology. While literature from the developed world boasts of outstanding outcomes, there is a paucity of data from the developing world. This study aimed to assess complications and outcomes of acute promyelocytic leukaemia in a resource-constrained setting.
We retrospectively collected data from patients diagnosed with APL from January 2016 to December 2020.
Sixty-four patients were treated-32 in both the Sanz high and low-risk groups. In the Sanz low-risk group, 12.5% of patients received ATRA with daunorubicin and 81.25% received ATRA with ATO. In the Sanz high-risk group, 18.8% of patients received ATRA with daunorubicin, 34.3% received ATRA with daunorubicin and ATO while 40.6% received ATRA with ATO. 56.25% of patients developed differentiation syndrome. The incidence was higher in Sanz high-risk group as compared to Sanz low-risk group. 57.4% of patients had an infection at the time of presentation. 62.5% of patients developed neutropenic fever during treatment. 17.2% of patients developed pseudotumor cerebri. The 4-year EFS and OS were 71.25 and 73.13%, respectively. Sanz low-risk group had a better 4-year EFS and OS as compared to the Sanz high-risk group. Haemoglobin at presentation and Sanz high-risk group were associated with poorer outcomes with a hazard ratio of 0.8 and 3.1, respectively. Outcomes in high-risk patients were better with the use of ATRA + ATO + daunorubicin.
In the Indian population, APL patients have a high incidence of differentiation syndrome, pseudotumor cerebri, and infections during induction. CR, EFS, and OS compared to the developed world can be achieved with optimal therapy. Low haemoglobin at presentation and Sanz high-risk group were associated with poorer outcomes. ATRA, ATO, and daunorubicin combination is the preferred protocol for treating high-risk patients.

急性早幼粒细胞白血病（acute promyelocytic leukaemia, APL）的治疗已成为血液肿瘤学领域的重大成功典范。尽管发达国家的相关文献报道了优异的治疗结局，但发展中国家的同类研究数据仍较为匮乏。本研究旨在评估资源受限医疗环境下急性早幼粒细胞白血病患者的并发症发生情况与治疗结局。
本研究回顾性收集了2016年1月至2020年12月期间确诊为APL的患者临床数据。
本研究共纳入64例接受治疗的APL患者，其中Sanz高危组与低危组各32例。在Sanz低危组中，12.5%的患者接受全反式维甲酸（all-trans retinoic acid, ATRA）联合柔红霉素（daunorubicin）治疗，81.25%的患者接受ATRA联合三氧化二砷（arsenic trioxide, ATO）治疗。在Sanz高危组中，18.8%的患者接受ATRA联合柔红霉素治疗，34.3%的患者接受ATRA联合柔红霉素与ATO治疗，40.6%的患者接受ATRA联合ATO治疗。56.25%的患者出现分化综合征，且Sanz高危组的分化综合征发生率显著高于低危组。57.4%的患者在初诊时合并感染，62.5%的患者在治疗期间出现中性粒细胞减少性发热，17.2%的患者发生假性脑瘤。本研究中患者的4年无事件生存期（Event-Free Survival, EFS）与总生存期（Overall Survival, OS）分别为71.25%与73.13%。Sanz低危组患者的4年EFS与OS均显著优于高危组。初诊时血红蛋白水平与Sanz高危分层均与不良结局相关，对应的风险比（hazard ratio, HR）分别为0.8与3.1。采用ATRA+ATO+柔红霉素联合方案治疗的高危患者结局更佳。
在印度人群中，APL患者在诱导治疗期间的分化综合征、假性脑瘤与感染发生率较高。通过优化治疗方案，可达到与发达国家相当的完全缓解（Complete Remission, CR）、EFS与OS水平。初诊时血红蛋白水平偏低与Sanz高危分层均与不良预后相关。ATRA、ATO与柔红霉素联合方案为高危APL患者的首选治疗方案。