Okada Yosuke, Tachi Noriaki, Shimazu Yutaka, Murata Makoto, Nishiwaki Satoshi, Onishi Yasushi, Jinguji Atsushi, Uchida Naoyuki, Tanaka Masatsugu, Hasegawa Yuta, Ito Ayumu, Kako Shinichi, Nishida Tetsuya, Onodera Koichi, Sawa Masashi, Nakamae Hirohisa, Toyosaki Masako, Kanda Yoshinobu, Onizuka Makoto, Fukuda Takahiro, Ohbiki Marie, Atsuta Yoshiko, Arai Yasuyuki, Tachibana Takayoshi
Division of Hematology, Jichi Medical University Saitama Medical Center, Saitama, Japan.
Division of Hematology, National Defense Medical College Hospital, Saitama, Japan.
Cancer. 2025 Jan 1;131(1):e35627. doi: 10.1002/cncr.35627. Epub 2024 Nov 4.
De novo chronic myeloid leukemia in blastic phase (CML-BP) showing lymphoid immunophenotype mimics Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph-positive ALL). Although upfront allogeneic hematopoietic cell transplantation (HCT) is considered in both diseases, it is not yet clear whether the transplant outcomes are also similar.
Using a registry database, the transplant outcomes between de novo CML-BP and Ph-positive ALL in negative-minimal residual disease (MRD), positive MRD, and nonremission cohorts were compared, respectively. All of the included patients had received tyrosine kinase inhibitor therapy before HCT and underwent HCT between 2002 and 2021. Regarding Ph-positive ALL, patients with p210 transcripts were excluded because there was concern that this group might include patients with de novo CML-BP.
Although most of the outcomes were comparable, in patients with positive MRD at HCT, de novo CML-BP was significantly associated with superior disease-free survival (DFS) (hazard ratio [HR] 0.6, p = .0032), overall survival (HR 0.66, p = .027), and a lower risk of relapse (HR 0.48, p = .0051). In subgroup analyses, BCR::ABL1 mutation status had a significant interaction with the disease (p for interaction = .0027). De novo CML-BP seemed to be associated with superior disease-free survival in a BCR::ABL1 mutation-positive cohort, whereas this association was not observed in a mutation-negative cohort.
Considering previous reports that showed inferior outcomes for de novo CML-BP compared to Ph-positive ALL, the data suggested that allogeneic HCT could overcome the poor prognosis of de novo CML-BP. These findings highlight the importance of distinguishing de novo CML-BP from Ph-positive ALL.
呈现淋巴样免疫表型的初发性急变期慢性髓性白血病（CML-BP）酷似费城染色体阳性急性淋巴细胞白血病（Ph阳性ALL）。尽管这两种疾病都考虑进行 upfront 异基因造血细胞移植（HCT），但移植结果是否相似尚不清楚。
利用登记数据库，分别比较了初发性CML-BP与Ph阳性ALL在微小残留病（MRD）阴性、MRD阳性和未缓解队列中的移植结果。所有纳入患者在HCT前均接受了酪氨酸激酶抑制剂治疗，并在2002年至2021年间接受了HCT。对于Ph阳性ALL，排除了具有p210转录本的患者，因为担心该组可能包括初发性CML-BP患者。
尽管大多数结果具有可比性，但在HCT时MRD阳性的患者中，初发性CML-BP与无病生存率（DFS）显著相关（风险比[HR]0.6，p = 0.0032）、总生存率（HR 0.66，p = 0.027）以及较低的复发风险（HR 0.48，p = 0.0051）。在亚组分析中，BCR::ABL1突变状态与疾病有显著的相互作用（相互作用p = 0.0027）。初发性CML-BP似乎在BCR::ABL1突变阳性队列中与无病生存率较高相关，而在突变阴性队列中未观察到这种关联。
考虑到先前的报告显示初发性CML-BP的结果比Ph阳性ALL差，数据表明异基因HCT可以克服初发性CML-BP的不良预后。这些发现凸显了区分初发性CML-BP与Ph阳性ALL的重要性。
