案例报告:药剂师主导的精密治疗框架,用于管理CSF1R相关白细胞脑病变后的侵入性真菌感染,Allo-HSCT
Jin Lu1,2, Mengqi Jia3, Xinghua Luan2,4,5,6
1Department of Pharmacy, Shanghai Sixth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Frontiers in pharmacology
|December 15, 2025
概括
血造干细胞移植 (HSCT) 对于带有球形 (HDLS) 的遗传性扩散性白细胞大脑病 (HDLS) 具有侵入性真菌病 (IFD) 的风险. 由多学科团队指导的精密抗真菌疗法改善了CSF1R相关疾病患者的结果.
科学领域:
- 神经科学是一个神经科学.
- 免疫学 免疫学 免疫学
- 药理学 药理学是指药理学的学科.
背景情况:
- 带有球体的遗传性扩散性白细胞性脑病变 (HDLS) 是一种罕见的,由CSF1R突变引起的自体主导性白细胞性脑病变,导致神经系统的快速衰退.
- 造血干细胞移植 (HSCT) 是对HDLS的潜在治疗方法,但侵入性真菌病 (IFD) 是移植后的一个重要并发症.
- 在CSF1R相关疾病 (CRD) 中,内在的微质功能障碍可能会损害免疫防御,增加HSCT后对感染的易感性.
研究的目的:
- 描述在接受全源性HSCT的HDLS患者中侵入性真菌病 (IFD) 的管理.
- 突出抗真菌治疗在HSCT后CRD患者的挑战和策略.
- 强调多学科团队和精确诊断在管理复杂感染中的作用.
主要方法:
- 一个中国男性的病例报告,该男性患有非热点CSF1R突变 (c.2443-1G>C),他接受了全基性HSCT.
- 由多学科团队实施个性化的抗真菌管理策略,考虑免疫状态,感染风险,药理动力学和下一代病原体诊断.
- 根据临床进展和病原体 (Aspergillus flavus) 鉴定,从预防性沃里康纳和勒沃撒到美罗,提科普拉宁和卡斯波辛的治疗升级.
主要成果:
- 尽管采取了抗真菌预防措施,但患者发病了发烧性中性质衰竭和中耳炎.
- 肺部感染Aspergillus flavus被诊断通过针对性测序的支气管支气管洗.
- 强化沃里康纳治疗导致菌感染在HSCT后+70天的临床解决.
结论:
- 由于系统性免疫抑制和内在的微质功能障碍,在患有CRD的HSCT接受者中管理IFD是复杂的.
- 在多学科专业知识和药理监测的指导下,精确的抗真菌治疗对于改善这种高风险人群的结果至关重要.
- 这一案例强调了在罕见的遗传神经系统疾病中,需要针对感染管理采取量身定制的方法.
相关概念视频
Cystic Fibrosis: Management
Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic sinusitis...
Sinus disease and chronic sinusitis...
Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions
PK–PD modeling has significantly influenced FDA regulatory decisions, particularly drug approval, dosage optimization, and labeling. These models integrate pharmacokinetics (PK) and pharmacodynamics (PD) to predict drug behavior and effects, aiding in optimizing dosing regimens and enhancing the probability of clinical trial success.One notable example is Nesiritide (Natrecor®), a recombinant human brain natriuretic peptide for treating acute decompensated congestive heart failure (CHF).
Cryptococcal Meningitis
Cryptococcal meningitis is a life-threatening opportunistic infection predominantly associated with HIV/AIDS, accounting for over 100,000 deaths annually worldwide. However, it also affects individuals with other forms of immunosuppression, including those undergoing immunosuppressive therapy, organ transplant recipients, patients with innate immunodeficiencies, and individuals with hematological disorders. The infection is caused mainly by Cryptococcus neoformans and Cryptococcus gattii,...


