在高度活跃的儿科多发性硬化症中,用大量延长间隔的Rituximab/ocrelizumab剂量控制疾病活性
Melany Venet1,2, Anne Lepine3, Adil Maarouf1
1Department of Neurology, Aix Marseille Univ, APHM, Hôpital de la Timone, CNRS, CRMBM, Marseille, France.
概括
拉长时间间隔的Rituximab/ocrelizumab (RTX/OCR) 剂量对于非常活跃的儿科发病多发性硬化症 (PoMS) 是安全有效的. 在这个儿科队列中,平均18个月的间隔维持了治疗有效性.
科学领域:
- 神经学 神经学
- 免疫学 免疫学 免疫学
- 儿科 儿科 儿科
背景情况:
- 成人研究表明,超过12个月的Rituximab/ocrelizumab (RTX/OCR) 延长剂量间隔是安全的,并可能提高安全性.
- 儿科发病多发性硬化症 (PoMS) 是这种疾病的一种罕见而激烈的形式.
研究的目的:
- 评估RTX/OCR在非常活跃的PoMS中延长间隔剂量的安全性和有效性.
- 为了在这个人群中比较标准的6个月间隔剂量与早期延长间隔剂量.
主要方法:
- 对患有非常活跃的PoMS的儿科患者进行观察性队列研究.
- 患者接受了标准6个月间隔RTX/OCR (n=9) 或早期延长间隔RTX/OCR (n=12,中位数为18个月).
- 随访时间中位数为31个月,以评估复发,残疾恶化和新的MRI病变.
主要成果:
- 一名接受标准间隔剂量的患者出现了复发.
- 没有患者显示残疾恶化或新的T2加权病变.
- RTX/OCR的有效性保持在18个月的中位延长间隔.
结论:
- 对于非常活跃的PoMS,RTX/OCR的延长间隔剂量 (中位数为18个月) 似乎是安全有效的.
- 这种剂量策略可以保持治疗疗效,同时有可能提高儿科患者的安全性.
- 需要进一步的研究,以在更大的队列中证实这些发现.
相关概念视频
Treatment Resistant Cancers
Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
Drug Dosing: Infants and Children
Pediatric patient dosages diverge from adults due to disparities in body surface area, total body water, and extracellular fluid per kilogram of body weight. The dosing regimen considers the variations in pharmacokinetics and pharmacology across distinct age groups, encompassing preterm newborns, infants, young children, older children, and adolescents. Calculation of pediatric patient doses is predicated on determining body surface area, which exhibits a superior correlation with the child's...
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 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...
Respiratory Syncytial Virus Disease
Human respiratory syncytial virus (RSV) is a widespread pathogen that primarily targets infants and young children but also poses a serious health risk to elderly and immunocompromised individuals. Belonging to the Pneumoviridae family, RSV is a negative-sense, single-stranded RNA virus within the Pneumovirus genus. Its global health burden is significant, with millions of cases annually resulting in hospitalizations and mortality, particularly in resource-limited settings. Although most...


