儿科状细胞疾病中的脏并发症:回顾性队列审查
Alex George1, Shannon E Conneely2, Ross Mangum3
1Department of Pediatrics, Section of Hematology/Oncology, Wake Forest University School of Medicine and Brenner Children's Hospital, Winston-Salem, North Carolina, USA.
Pediatric blood & cancer
|July 26, 2024
概括
患有状细胞疾病 (SCD) 的儿童根据基因型经历了各种脏并发症. 基尿素治疗,特别是在两岁之前,与更严重的脊髓封存危机 (ASSC) 有关.
科学领域:
- 血液学 血液学 血液学
- 儿科医学 儿科医学
- 遗传学 遗传学 是一个
背景情况:
- 状细胞疾病 (SCD) 是一种具有重大并发症的遗传性血液疾病.
- 脏功能障碍在SCD中很常见,但其他脏并发症需要进一步研究.
- 了解功能损失之外的脏问题的自然史对于儿科护理至关重要.
研究的目的:
- 调查SCD儿童脏并发症的自然史,不包括脏功能丧失.
- 分析特定脏问题的患病率和发病率,如大,急性脏封存危机 (ASSC),超,切除术.
- 检查基尿素治疗对ASSC的发生和严重性的影响.
主要方法:
- 德克萨斯州儿童医院SCD的儿科患者的回顾性图表审查.
- 确定脊髓并发症的诊断日期,ASSC事件,以及氧尿素治疗.
- 统计分析以评估氧尿素和ASSC发病/严重程度之间的关联.
主要成果:
- 累计患病率:24.7%的脊髓巨变,24.2%的ASSC,9.6%的超,5.6%的脊髓切除术.
- 血红蛋白Sβ的发病率最高 (69.2%),其次是SS (33.3%) 和SC (9.0%).
- 基尿素的使用,特别是在两岁之前,与ASSC的更高发病率相关.
结论:
- 在SCD中脏并发症的患病率和严重程度是基因型依赖的,血红蛋白Sβ0显示最高负担.
- 基尿素治疗与ASSC的发病率增加有关,尤其是在生命早期开始时.
- 这些发现凸显了基因型特异性管理的必要性,并仔细考虑了儿科SCD患者中基氨酸尿素启动时间.
相关概念视频
Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption
Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...
Pharmacokinetics in Pediatric Patients: Drug Distribution
Drug distribution in the pediatric population exhibits unique challenges and considerations due to the physiological differences between children, particularly neonates and infants, and adults. A crucial aspect of pediatric pharmacology is understanding how these differences impact the pharmacokinetics of various drugs, necessitating age-specific dosing strategies to ensure efficacy and safety.Neonates and infants have a higher total body water content, ~75%–90% of their body weight, compared...
Pharmacokinetics in Pediatric Patients: Drug Metabolism
In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses a challenge in...
Pharmacokinetics in Pediatric Patients: Drug Excretion
In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...


