Successful Bone Marrow Transplantation in a Patient with Acute Myeloid Leukemia Developed from Severe Congenital Neutropenia Using Modified Chemotherapy and Conditioning Regimen for Leukemia.

IF 16.4 1区化学 Q1 CHEMISTRY, MULTIDISCIPLINARY Accounts of Chemical Research Pub Date : 2024-02-26 DOI:10.3390/hematolrep16010010

Risa Matsumura, Shinji Mochizuki, Yusuke Morishita, Hiroko Hayakawa, Shuhei Karakawa, Hiroshi Kawaguchi, Satoshi Okada, Nobuyuki Hyakuna, Masao Kobayashi

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Abstract

Severe congenital neutropenia (SCN) is characterized by chronic neutropenia with recurrent infections from early infancy and a predisposition to myelodysplastic syndrome/acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for patients with SCN who develop myelodysplastic syndrome/AML. We report an 8-year-old girl with SCN carrying an ELANE mutation that had been refractory to granulocyte colony-stimulating factor. The patient experienced recurrent infections and then developed AML. The counts of leukemic blasts that harbored both CSF3R and RUNX1 mutations spontaneously decreased with antimicrobial therapy, leading to partial remission. After AML recurrence, HSCT was successfully performed using modified chemotherapy and a conditioning regimen. Serial donor lymphocyte infusions against mixed chimerism induced complete donor chimerism over 4 years without any infections or AML relapse. This case suggests the importance of carefully managing neutropenia-related infections, leukemia progression, and HSCT in patients with SCN developing AML.

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使用改良的白血病化疗和治疗方案，成功为一名因严重先天性中性粒细胞减少症而患上急性髓性白血病的患者进行骨髓移植。

重度先天性中性粒细胞减少症（SCN）的特点是慢性中性粒细胞减少，从婴儿期就开始反复感染，并易患骨髓增生异常综合症/急性髓性白血病（AML）。异基因造血干细胞移植（HSCT）是治疗患骨髓增生异常综合征/急性髓细胞白血病的 SCN 患者的唯一治愈方法。我们报告了一名携带ELANE基因突变的8岁SCN女孩，她对粒细胞集落刺激因子治疗无效。患者经历了反复感染，随后发展为急性髓细胞白血病。经抗菌治疗后，携带CSF3R和RUNX1突变的白血病血细胞数量自发减少，病情得到部分缓解。急性髓细胞性白血病复发后，患者采用改良化疗和调理方案成功进行了造血干细胞移植。针对混合嵌合体的连续供体淋巴细胞输注诱导了4年的完全供体嵌合，且未发生任何感染或急性髓细胞性白血病复发。该病例表明，对于罹患急性髓细胞性白血病的 SCN 患者，仔细处理中性粒细胞减少症相关感染、白血病进展和造血干细胞移植非常重要。

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来源期刊

Accounts of Chemical Research 化学-化学综合

CiteScore

31.40

自引率

1.10%

发文量

312

审稿时长

2 months

期刊介绍： Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.