Molecular response with asciminib 20 mg QD in a patient intolerant to multiple TKIs.

IF 3 3区医学 Q2 HEMATOLOGY

Annals of Hematology Pub Date : 2025-04-07 DOI:10.1007/s00277-025-06339-5

Ruth Stuckey, Laura Navarrete Bullón, Asunción Borrero Borrego, Violeta Vidal Ballester, Carlos Rodríguez-Medina, Alejandro Morales Curbelo, Leslie González Pinedo, Melissa Torres Ochando, Alvaro Veiga Vaz, María Del Mar Perera, Cristina Bilbao-Sieyro, Juan Francisco López Rodríguez, Adrián Segura-Díaz, María Teresa Gómez-Casares

引用次数: 0

Abstract

This case highlights a young CML patient who achieved a major molecular response (MMR) after just four months with a subtherapeutic dose of asciminib. The patient could not tolerate full-dose asciminib, or the full dose of two previous tyrosine kinase inhibitors (TKI) due to myelotoxicity, but blood counts recovered rapidly upon TKI suspension and reduced dosing enabled sustained treatment. This is the second reported case of the use of 20 mg QD asciminib, four times below the recommended dose, and the first to demonstrate efficacy at this dose. The study emphasizes asciminib's potential for overcoming treatment challenges associated with multi-resistant/intolerant CML patients.

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本病例重点介绍了一位年轻的 CML 患者，她在服用亚治疗剂量的阿西米尼仅四个月后就获得了主要分子反应（MMR）。由于骨髓毒性，该患者不能耐受全剂量阿西替尼，也不能耐受之前两种酪氨酸激酶抑制剂（TKI）的全剂量，但在停用 TKI 后血细胞计数迅速恢复，减少剂量使治疗得以持续。这是第二例报告使用 20 毫克 QD asciminib（低于推荐剂量四倍）的病例，也是第一例证明该剂量疗效的病例。这项研究强调了阿西米尼克服与多耐药/不耐药 CML 患者相关的治疗难题的潜力。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Annals of Hematology 医学-血液学

CiteScore

5.60

自引率

2.90%

发文量

304

审稿时长

2 months

期刊介绍： Annals of Hematology covers the whole spectrum of clinical and experimental hematology, hemostaseology, blood transfusion, and related aspects of medical oncology, including diagnosis and treatment of leukemias, lymphatic neoplasias and solid tumors, and transplantation of hematopoietic stem cells. Coverage includes general aspects of oncology, molecular biology and immunology as pertinent to problems of human blood disease. The journal is associated with the German Society for Hematology and Medical Oncology, and the Austrian Society for Hematology and Oncology.