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阿西米尼联合帕纳替尼作为桥接 brexucabtagene autoleucel 及维持治疗用于一例复发难治性费城阳性 B 细胞急性淋巴细胞白血病患者

英文原题:Combination Therapy With Asciminib and Ponatinib as a Bridge to Brexucabtagene Autoleucel and Maintenance in a Patient With Relapsed Refractory Philadelphia Positive B-Cell Acute Lymphoblastic Leukemia.

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Combination Therapy With Asciminib and Ponatinib as a Bridge to Brexucabtagene Autoleucel and Maintenance in a Patient With Relapsed Refractory Philadelphia Positive B-Cell Acute Lymphoblastic Leukemia.

PubMed 2024/09/20(内容时间) J Med Cases Q3 · IF 0.5(JCR 2025)

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中文摘要

酪氨酸激酶抑制剂(TKIs)已改变了费城染色体阳性B细胞急性淋巴细胞白血病(ALL)的预后;然而,多种TKIs治疗后的复发/难治性疾病仍然是临床挑战。Brexucabtagene autoleucel(brexu-cel)是FDA新近批准用于复发/难治性ALL的疗法。鉴于制备时间较长,brexu-cel前通常采用桥接治疗。本文报道一例75岁西班牙裔男性复发/难治性费城染色体阳性B细胞ALL患者,伴有髓外病变,累及腹部淋巴结和皮肤。他最初接受化疗联合imatinib治疗,随后接受dasatinib,之后接受blinatumomab和nilotinib。随着患者疾病进展,他接受了ponatinib联合低剂量挽救性化疗,未显示激酶域突变。作为最后努力,一种ponatinib联合asciminib的新型组合被用作brexu-cel前的桥接治疗,之后作为brexu-cel后的维持治疗。这一新型组合能够在brexu-cel前控制疾病达2个月,并维持缓解至少10个月。本报告表明,ponatinib联合asciminib的新型组合作为brexu-cel后的桥接和维持治疗是可耐受且有效的。

展开英文摘要原文

Tyrosine kinase inhibitors (TKIs) have changed the prognosis of Philadelphia-positive B-cell acute lymphoblastic leukemia (ALL); however, relapsed and refractory disease after multiple TKIs continues to be a clinical challenge. Brexucabtagene autoleucel (brexu-cel) is a novel FDA-approved therapy for relapsed and refractory ALL. Given the lengthy manufacturing time, bridging therapy is commonly employed prior to brexu-cel.

Here we describe a case of a 75-year-old Hispanic male patient with relapsed/refractory Philadelphia-positive B-cell ALL with extramedullary disease involving abdominal lymph nodes and skin. He was initially treated with chemotherapy in combination with imatinib, and later received dasatinib and subsequently blinatumomab and nilotinib. As the patient progressed, he received ponatinib with low-dose salvage chemotherapy and did not show kinase domain mutation.

In a final effort, a novel combination of ponatinib with asciminib was used as a bridge therapy before brexu-cel and later as maintenance therapy after brexu-cel. This novel combination was able to control disease prior to brexu-cel for 2 months and maintained remission for at least 10 months. This report shows that the novel combination of ponatinib and asciminib is tolerable and effective as a bridge and maintenance therapy after brexu-cel.

论文信息

作者
Amin Muhammad A、Mir Ghazi E、Ali A、Tam E、Woan K、Chaudhary P、Yaghmour G、Ladha A
第一作者单位
Department of Medicine, Liaquat National Hospital and Medical College, Karachi, Pakistan.Pakistan
通讯作者单位
Jane Anne Nohl Division of Hematology, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.United States
文献类型
病例报告
期刊
Journal of medical cases2024 Oct
原文标识
PubMed 39328803 · DOI 10.14740/jmc4287