Wang Lei, Yan Xiaojing, He Juan
Department of Hematology, The First Affiliated Hospital of China Medical University, Shenyang, Liaoning Province, China.
Medicine (Baltimore). 2020 Oct 23;99(43):e22923. doi: 10.1097/MD.0000000000022923.
Most acute promyelocytic leukemia (APL) patients respond to all-trans-retinoic acid (ATRA)and have a good prognosis. However, variants APL who carry PLZF/RARа, STAT5B/RARа, and STAT3/RARа are insensitive to ATRA and have poor prognoses. The standard treatment for variants APL is still unclear due to the small sample size.
Here we reported a Chinese male who was admitted to our hospital with the complaint of rib pain, dyspnea, and fever (37.5°C). Blood tests showed leukopenia (1.83 × 10/L), anemia (hemoglobin 73 g/L), and thrombocytopenia (54 × 10/L). Prothrombin time and activated partial thromboplastin time were normal.
The patient was diagnosed as STAT5b-RARa-positive APL based on the clinical and laboratory findings.
ATRA was used immediately for induction treatment, then he was treated with ATRA + arsenic trioxide and got the severe cardiac insufficiency. Subsequently, consolidation chemotherapy was added with ATRA + Huangdai tablets + idarubicin and decitabine, cytarabine, aclamycin (DCAG).
The patient relapsed soon after his first molecular complete remission (CRm), fortunately, he got a second CRm with DCAG. He has survived for more than 9 months and remains CRm, now he is looking for a suitable donor to prepare for hematopoietic stem cell transplantation (HSCT).
APL patients with STAT5B-RARa is not only resistant to ATRA, but also to conventional combination chemotherapy such as daunorubicin and cytarabine/idarubicin and cytarabine or other regimens. Relapse and extramedullary infiltration is common, HSCT is a effective treatment, and the best time for HSCT is after the first CR. It should be noted that this patient got CRm with DCAG after relapse, so the role of decitabine in APL with STAT5B-RARa needs to be considered.
大多数急性早幼粒细胞白血病（APL）患者对全反式维甲酸（ATRA）有反应，预后良好。然而，携带PLZF/RARα、STAT5B/RARα和STAT3/RARα的变异型APL对ATRA不敏感，预后较差。由于样本量小，变异型APL的标准治疗方法仍不明确。
我们报告了一名中国男性，因肋骨疼痛、呼吸困难和发热（37.5°C）入院。血液检查显示白细胞减少（1.83×10/L）、贫血（血红蛋白73g/L）和血小板减少（54×10/L）。凝血酶原时间和活化部分凝血活酶时间正常。
根据临床和实验室检查结果，该患者被诊断为STAT5b-RARa阳性APL。
立即使用ATRA进行诱导治疗，随后接受ATRA+三氧化二砷治疗，但出现严重心脏功能不全。随后，巩固化疗采用ATRA+黄黛片+去甲氧柔红霉素以及地西他滨、阿糖胞苷、阿克拉霉素（DCAG）。
患者在首次分子完全缓解（CRm）后不久复发，幸运的是，通过DCAG治疗再次获得CRm。他已存活超过9个月，仍处于CRm状态，目前正在寻找合适的供体准备造血干细胞移植（HSCT）。
STAT5B-RARa阳性的APL患者不仅对ATRA耐药，而且对柔红霉素和阿糖胞苷/去甲氧柔红霉素和阿糖胞苷等传统联合化疗或其他方案也耐药。复发和髓外浸润很常见，HSCT是一种有效的治疗方法，HSCT的最佳时机是在首次CR后。需要注意的是，该患者复发后通过DCAG获得CRm，因此需要考虑地西他滨在STAT5B-RARa阳性APL中的作用。
