Itonaga Hidehiro, Ota Shuichi, Ikeda Takashi, Taji Hirohumi, Amano Itsuto, Hasegawa Yuichi, Ichinohe Tatsuo, Fukuda Takahiro, Atsuta Yoshiko, Tanizawa Akihiko, Kondo Takeshi, Miyazaki Yasushi
Department of Hematology, Nagasaki University Hospital, Nagasaki, Japan.
Department of Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan.
Leuk Res. 2018 Dec;75:50-57. doi: 10.1016/j.leukres.2018.11.003. Epub 2018 Nov 13.
Atypical chronic myeloid leukemia (aCML) and chronic neutrophilic leukemia (CNL) are rare BCR-ABL1 fusion gene-negative myeloid neoplasms with a predominance of neutrophils. Since no standard therapeutic strategy currently exists for these diseases, we retrospectively evaluated the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for aCML and CNL. Data from 14 aCML and 5 CNL patients as their diagnoses were collected using a nationwide survey. Allo-HSCT was performed between 2003 and 2014. Preconditioning regimens included myeloablative (n = 15), reduced-intensity (n = 3), and non-myeloablative (n = 1) regimens. Transplanted stem cells were obtained from HLA-matched related donors (n = 5) and alternative donors (n = 14). Neutrophil engraftment was successfully achieved in 17 patients. One-year overall survival rates (OS) were 54.4% (95% confidence interval [CI], 24.8 to 76.7%) and 40.0% (95% CI, 5.2 to 75.3%) in patients with aCML and CNL, respectively. Among aCML patients, 1-year OS were 76.2% (95% CI, 33.2 to 93.5%) and 20.0% (95% CI, 0.8 to 58.2%) in patients with <5% myeloblasts (n = 9) and ≥5% myeloblasts (n = 5) in peripheral blood before allo-HSCT, respectively. These results suggest that allo-HSCT achieves long-term survival in patients with aCML and CNL. Better pre-transplant management is required to improve the outcomes of aCML patients with ≥5% blasts in peripheral blood.
非典型慢性髓性白血病（aCML）和慢性中性粒细胞白血病（CNL）是罕见的BCR-ABL1融合基因阴性的髓系肿瘤，以中性粒细胞增多为主。由于目前这些疾病尚无标准治疗策略，我们回顾性评估了异基因造血干细胞移植（allo-HSCT）治疗aCML和CNL的疗效。通过全国性调查收集了14例aCML和5例CNL患者确诊时的数据。allo-HSCT于2003年至2014年间进行。预处理方案包括清髓性方案（n = 15）、减低强度方案（n = 3）和非清髓性方案（n = 1）。移植的干细胞来自HLA配型相合的相关供者（n = 5）和替代供者（n = 14）。17例患者成功实现中性粒细胞植入。aCML和CNL患者的1年总生存率（OS）分别为54.4%（95%置信区间[CI]，24.8%至76.7%）和40.0%（95%CI，5.2%至75.3%）。在aCML患者中，allo-HSCT前外周血原始粒细胞<5%（n = 9）和≥5%（n = 5）的患者1年OS分别为76.2%（95%CI，33.2%至93.5%）和20.0%（95%CI，0.8%至58.2%）。这些结果表明，allo-HSCT可使aCML和CNL患者获得长期生存。需要更好的移植前管理以改善外周血原始粒细胞≥5%的aCML患者的疗效。
