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两例异基因造血干细胞移植后复发并接受新型免疫治疗与帕纳替尼治疗的费城染色体阳性急性淋巴细胞白血病患者的成功治疗

英文原题:Successful treatment of two cases with Philadelphia-chromosome positive acute lymphoblastic leukemia who relapsed after allogeneic stem cell transplantation and the treatments with novel immunotherapies and ponatinib.

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Successful treatment of two cases with Philadelphia-chromosome positive acute lymphoblastic leukemia who relapsed after allogeneic stem cell transplantation and the treatments with novel immunotherapies and ponatinib.

PubMed 2024/06/03(内容时间) Hematology Q3 · IF 2(JCR 2025)

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中文摘要

对新型药物(如酪氨酸激酶抑制剂、inotuzumab ozogamicin(InO)和 blinatumomab)耐药的复发性费城染色体阳性急性淋巴细胞白血病(Ph+ALL)患者预后极差。

我们治疗了两例对此类药物耐药的 Ph+ALL 病例,通过嵌合抗原受体(CAR)T 细胞疗法或采用序贯预处理方案进行第二次异基因造血干细胞移植(HCT),患者均获得长期生存。病例 1:一名 15 岁男孩确诊 Ph+ALL。接受 ponatinib 和 blinatumomab 治疗后进行第二次 HCT,但仍发生血液学复发。InO 治疗无效,患者转至 CAR-T 中心。CAR-T 细胞治疗后达到可测量残留病灶(MRD)阴性,并在未接受维持治疗的情况下持续 38 个月。病例 2:一名 21 岁男性确诊 Ph+ALL。首次 HCT 后出现血液学复发。尽管接受 InO、ponatinib 和 blinatumomab 治疗,仍未达到血液学缓解。随后采用克拉屈滨序贯预处理方案进行第二次 HCT。此后患者达到 MRD 阴性,并在未接受维持治疗的情况下持续 42 个月。这些策略对治疗多种免疫疗法耐药的 Ph+ALL 具有参考价值。

展开英文摘要原文

The outcomes of relapsed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL) resistant to new drugs such as tyrosine kinase inhibitors, inotuzumab ozogamicin (InO) and blinatumomab are dismal.

We treated two cases of Ph+ALL resistant to these drugs that achieved long-term survival after treatment with chimeric antigen receptor (CAR)-T cell therapy or a second allogeneic hematopoietic stem cell transplantation (HCT) with a sequential conditioning regimen. Case 1: A 15-year-old boy was diagnosed with Ph+ALL. Despite the second HCT after the treatment of ponatinib and blinatumomab, hematological relapse occurred. InO was ineffective and he was transferred to a CAR-T center. After the CAR-T cell therapy, negative measurable residual disease (MRD) was achieved and maintained for 38 months without maintenance therapy.

Case 2: A 21-year-old man was diagnosed with Ph+ALL. Hematological relapse occurred after the first HCT. Despite of the treatment with InO, ponatinib, and blinatumomab, hematological remission was not achieved. The second HCT was performed using a sequential conditioning regimen with clofarabine. Negative MRD was subsequently achieved and maintained for 42 months without maintenance therapy. These strategies are suggestive and helpful to treat Ph+ALL resistant to multiple immunotherapies.

论文信息

作者
Tachibana T、Tanaka M、Noguchi Y、Najima Y、Sadato D、Harada Y、Tamai Y、Doki N
第一作者单位
Department of Hematology, Kanagawa Cancer Center, Yokohama, Japan.Japan
通讯作者单位
Department of Hematology and Clinical Immunology, Yokohama City University School of Medicine, Yokohama, Japan.Japan
文献类型
病例报告
期刊
Hematology (Amsterdam, Netherlands)2024 Dec
原文标识
PubMed 38828928 · DOI 10.1080/16078454.2024.2360843