通过鼻胃管给药稀释口服冻干去氨加压素制剂治疗残疾儿童中心性尿崩症:回顾性病例系列。

IF 3.4 3区 医学 Q1 PEDIATRICS Pediatric Drugs Pub Date : 2023-09-01 DOI:10.1007/s40272-023-00578-7
Hüseyin Anıl Korkmaz, Ved Bhushan Arya, Ahmet Gönüllü, Fulya Coşkunol, Behzat Ozkan
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引用次数: 0

摘要

背景:鼻胃给药DDAVP (desamino- d -精氨酸-8-加压素)冻干液(ODL)治疗伴有吞咽协调困难的中心性尿囊症(CDI)残疾儿童的经验有限。目的:我们旨在评估鼻胃应用ODL治疗CDI残疾儿童的安全性和有效性。与正常智力和CDI患儿舌下DDAVP治疗后血清钠恢复正常所需时间进行比较。方法:对2012年至2022年在土耳其Behcet Uz儿童医院经鼻胃管行ODL治疗的12例CDI残疾儿童的临床、实验室和神经影像学特征进行评估。结果:男6例,女6例,平均(±SD)年龄43(±40)个月。这些儿童(平均[±SD]体重标准差评分[SDS] - 1.2±1.7;平均[±SD]身高SDS - 1.3±1.4)表现为发育不全、烦躁、发热延长、多尿、高钠血症(平均血清钠162[±3.6]mEq/L)。诊断时,平均血清和尿液渗透压分别为321(±14)mOsm/kg和105(±7.8)mOsm/kg。精氨酸加压素(AVP)水平未检测到(结论:在这个小型回顾性研究中,鼻胃给药口服DDAVP冻干制剂治疗残疾儿童CDI是安全有效的。
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Management of Central Diabetes Insipidus in Disabled Children with Diluted Oral Desmopressin Lyophilisate Formulation Administered Through Nasogastric Tube: A Retrospective Case Series.

Background: Experience with nasogastric administration of oral DDAVP [desamino-D-arginine-8-vasopressin] lyophilisate (ODL) for central diabetes insipidus (CDI) in disabled children with swallowing coordination difficulties is limited.

Objective: We aimed to assess the safety and efficacy of nasogastric use of ODL in disabled children with CDI. Time to serum sodium normalisation was compared with that of children with normal intellect and CDI treated with sublingual DDAVP.

Methods: Clinical, laboratory and neuroimaging characteristics were evaluated for 12 disabled children with CDI treated with ODL through nasogastric tube at Dr Behcet Uz Children's Hospital, Turkey, between 2012 and 2022.

Results: Six boys and six girls with a mean (±SD) age of 43 (± 40) months were evaluated. These children (mean [±SD] weight standard deviation score [SDS] - 1.2 ± 1.7; mean [±SD] height SDS - 1.3 ± 1.4) presented with failure to thrive, irritability, prolonged fever, polyuria and hypernatraemia (mean serum sodium 162 [±3.6] mEq/L). At diagnosis, mean serum and urine osmolality were 321 (± 14) mOsm/kg and 105 (± 7.8) mOsm/kg, respectively. Arginine vasopressin (AVP) levels were undetectable (< 0.5 pmol/L) at diagnosis in all patients. Nasogastric tube administration of DDAVP lyophilisate (120 µg/tablet) dissolved in water (10 mL) was commenced at a dose of 1-5 µg/kg/day in two divided doses together with controlled water intake to avoid hyponatraemia. The frequency and dose of DDAVP were titrated based on urine output and serum sodium concentration. Serum sodium declined at a rate of 0.11 ± 0.03 mEq/L/h and reached normal range in a mean duration of 174 ± 46.5 h. Serum sodium declined faster in children with normal intellect and CDI treated with sublingual DDAVP (1.28 ± 0.39 mEq/L/h; p = 0.0003). Three disabled children needed rehospitalisation because of hypernatraemia due to unintentional DDAVP omission by caregivers. No episode of hyponatraemia was observed. Weight gain and growth were normal during the median (± interquartile range) follow-up duration of 32 ± 67 months.

Conclusions: Nasogastric administration of oral DDAVP lyophilised formulation was safe and effective in the treatment of CDI in disabled children in this small retrospective series.

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来源期刊
Pediatric Drugs
Pediatric Drugs PEDIATRICS-PHARMACOLOGY & PHARMACY
CiteScore
7.20
自引率
0.00%
发文量
54
审稿时长
>12 weeks
期刊介绍: Pediatric Drugs promotes the optimization and advancement of all aspects of pharmacotherapy for healthcare professionals interested in pediatric drug therapy (including vaccines). The program of review and original research articles provides healthcare decision makers with clinically applicable knowledge on issues relevant to drug therapy in all areas of neonatology and the care of children and adolescents. The Journal includes: -overviews of contentious or emerging issues. -comprehensive narrative reviews of topics relating to the effective and safe management of drug therapy through all stages of pediatric development. -practical reviews covering optimum drug management of specific clinical situations. -systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. -Adis Drug Reviews of the properties and place in therapy of both newer and established drugs in the pediatric population. -original research articles reporting the results of well-designed studies with a strong link to clinical practice, such as clinical pharmacodynamic and pharmacokinetic studies, clinical trials, meta-analyses, outcomes research, and pharmacoeconomic and pharmacoepidemiological studies. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pediatric Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.
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