Ruxolitinib combined with venetoclax and azacitidine in the treatment of refractory T-ALL patients with JAK1, JAK3, and STAT5B gene mutations: a case report and literature review
10.3760/cma.j.cn121090-20240412-00138
- VernacularTitle:芦可替尼联合维奈克拉及阿扎胞苷治疗伴JAK1、JAK3、STAT5B基因突变的难治性T-ALL患者1例报告并文献复习
- Author:
Peipei XU
1
;
Tong ZHOU
;
Yueyi XU
;
Miaoxin PENG
;
Ying DU
;
Ting XIE
;
Yonggong YANG
;
Jian OUYANG
;
Bing CHEN
Author Information
1. 南京大学附属鼓楼医院血液内科,南京 210008
- From:
Chinese Journal of Hematology
2024;45(9):872-875
- CountryChina
- Language:Chinese
-
Abstract:
Refractory acute T-lymphoblastic leukemia (T-ALL), which is characterized by a low sensitivity to conventional induction therapy and poor prognosis, poses significant challenges during treatment. This study reported a case of refractory T-ALL patient with mutations in the JAK1, JAK3, and STAT5B genes from Nanjing University’s Gulou Hospital. Following an unsuccessful course of standard VDLP regimen chemotherapy, the treatment was modified to include ruxolitinib in combination with venetoclax and azacitidine. Subsequent to this therapy, the patient achieved bone marrow minimal residual disease (MRD) negativity. Notably, pleural effusion and mediastinal mass significantly improved the post-chest cavity infusion of dexamethasone combined with etoposide at the same stage. The patient also underwent allogeneic hematopoietic stem cell transplantation upon achieving bone marrow remission and was followed up until January 2024. Ruxolitinib combined with venetoclax and azacytidine has shown promising efficacy and safety in treating refractory T-ALL harboring the JAK1, JAK3, and STAT5B mutations, providing a novel therapeutic approach for such patients.