Sharif Mohamed I, Alotaibi Ahmad S, Alyamany Ruah, Alahmari Ali, Alkhaldi Hanan, Saad Ayman, Alfayez Mansour
Department of Hematology, Stem Cell Transplant and Cellular Therapy, Oncology Centre, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.
Leuk Res Rep. 2025 Mar 18;23:100507. doi: 10.1016/j.lrr.2025.100507. eCollection 2025.
Acute myeloid leukemia (AML) is a heterogeneous disease with diverse molecular cytogenetic characteristics. Philadelphia-positive acute myeloid leukemia, a rare subtype of AML, is traditionally considered a high-risk, with the standard recommendation being an allogeneic hematopoietic cell transplant (HCT) in first remission. More recently, with better characterization and understanding of AML biology, novel therapies have been introduced. Drawing parallels from the advances seen in Philadelphia-positive acute lymphoblastic leukemia (ALL), the question arises whether potent tyrosine kinase inhibitors (TKI), such as ponatinib, in combination with AML-directed therapies, could be used in Philadelphia-positive AML, potentially eliminating the need for HCT in the first remission. In this report, we review the literature on Philadelphia-positive AML, study a case where HCT was omitted, and explore potential signals that could support successful HCT omission.
急性髓系白血病（AML）是一种具有多种分子细胞遗传学特征的异质性疾病。费城染色体阳性急性髓系白血病是AML的一种罕见亚型，传统上被认为是高危型，标准治疗建议是在首次缓解期进行异基因造血细胞移植（HCT）。最近，随着对AML生物学特征的更好描述和理解，新型疗法不断涌现。借鉴费城染色体阳性急性淋巴细胞白血病（ALL）取得的进展，人们提出疑问，强效酪氨酸激酶抑制剂（TKI），如波纳替尼，联合针对AML的治疗方法，是否可用于费城染色体阳性AML，从而有可能在首次缓解期无需进行HCT。在本报告中，我们回顾了关于费城染色体阳性AML的文献，研究了一例未进行HCT的病例，并探讨了可能支持成功省略HCT的潜在信号。
