双口服铁化 (DOIC) 在患有输血依赖β血症的儿童:现实世界的有效性数据
Amitoj Singh Chadha1, Abaan Ul Haq Khazi Mohammad1, Naveen Kanth Dhãmi Nadakuditi1
1Department of Pediatric Hematology Oncology, St. Johns Medical College Hospital, Bangalore, India.
概括
使用deferiprone和deferasirox进行双口服铁化,有效降低了在患有输血依赖性血症的儿童的铁过载. 这种组合疗法耐受性良好,并显著降低费里水平,特别是在初始值较高的患者中.
科学领域:
- 血液学 血液学 血液学
- 儿科医学 儿科医学
- 药理学 药理学是指药理学的学科.
背景情况:
- 铁过载是患有输血依赖性thalassemia (TDT) 的儿童的常见并发症.
- 化疗对于管理TDT患者的铁过载至关重要.
- 与deferiprone (DFP) 和deferasirox (DFX) 结合化提供了一个治疗选择.
研究的目的:
- 评估双口服铁化 (DOIC) 在TDT儿童的疗效和安全性.
- 评估DOIC对随着时间的推移对血清费里水平的影响.
- 通过DOIC识别影响费里丁减少的因素.
主要方法:
- 对2015年至2022年期间在DOIC上启动的TDT儿童的回顾性图表审查.
- 在DOIC启动之前和之后分析血清费里水平.
- 评估治疗持续时间和不良影响.
主要成果:
- 45名TDT儿童接受了DOIC.
- 30个月后,平均血清费里丁从2929 ng/dl下降到1962.10 ng/dl.
- 82.22%的儿童达到费里丁<2000 ng/dl,60%的儿童达到<1000 ng/dl.
- 较高的初始费里水平 (>2500 ng/dl) 在12-24个月内迅速降低.
- DOIC被很好地容忍,没有严重的不良反应.
结论:
- 双口服铁化 (DOIC) 是一种有效和安全的治疗方法,用于减少TDT儿童的铁过载.
- DOIC导致血清费里水平显著降低.
- 该疗法在具有高基线费里水平的患者中特别有效,并且耐受性良好.
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