Ambulatory Anaesthesia in a Patient with Niemann-Pick Disease Type C

Araújo, A. M.

doi:10.4172/2155-6148.1000509

Cited by 3 publications

(5 citation statements)

References 11 publications

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“…Information about gender differences in the dose of anesthetic management in patients with Niemann-Pick disease has not yet been reported. Only possible complications of anesthetic management and the necessary measures in NPC patients have been shown [ 122 , 123 , 124 , 125 , 126 , 127 , 128 ] and explained by the disease-related interference with pathologies of liver, spleen, lung, and heart [ 129 ]. Due to both the liver involvement and the probability of an alteration in drug metabolism, anesthetic drugs should be used with caution in NPC patients [ 122 ].…”

Section: Discussionmentioning

confidence: 99%

Gender-Specific Effects of Two Treatment Strategies in a Mouse Model of Niemann-Pick Disease Type C1

Holzmann

Witt

Rolfs

et al. 2021

IJMS

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In a mouse model of Niemann-Pick disease type C1 (NPC1), a combination therapy (COMBI) of miglustat (MIGLU), the neurosteroid allopregnanolone (ALLO) and the cyclic oligosaccharide 2-hydroxypropyl-β-cyclodextrin (HPßCD) has previously resulted in, among other things, significantly improved motor function. The present study was designed to compare the therapeutic effects of the COMBI therapy with that of MIGLU or HPßCD alone on body and brain weight and the behavior of NPC1−/− mice in a larger cohort, with special reference to gender differences. A total of 117 NPC1−/− and 123 NPC1+/+ mice underwent either COMBI, MIGLU only, HPßCD only, or vehicle treatment (Sham), or received no treatment at all (None). In male and female NPC1−/− mice, all treatments led to decreased loss of body weight and, partly, brain weight. Concerning motor coordination, as revealed by the accelerod test, male NPC1−/− mice benefited from COMBI treatment, whereas female mice benefited from COMBI, MIGLU, and HPßCD treatment. As seen in the open field test, the reduced locomotor activity of male and female NPC1−/− mice was not significantly ameliorated in either treatment group. Our results suggest that in NPC1−/− mice, each drug treatment scheme had a beneficial effect on at least some of the parameters evaluated compared with Sham-treated mice. Only in COMBI-treated male and female NPC+/+ mice were drug effects seen in reduced body and brain weights. Upon COMBI treatment, the increased dosage of drugs necessary for anesthesia in Sham-treated male and female NPC1−/− mice was almost completely reduced only in the female groups.

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Section: Discussionmentioning

confidence: 99%

Gender-Specific Effects of Two Treatment Strategies in a Mouse Model of Niemann-Pick Disease Type C1

Holzmann

Witt

Rolfs

et al. 2021

IJMS

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“…Additionally, its rapid washing out after discontinuation helped us to achieve rapid recovery with minimal postoperative sequel on respiration and ventilation. Similar to the approach of Araujo et al ( 5 ), we used remifentanil as the analgesic because of its short duration of action, which provided us with a rapid and predictable recovery. Moreover, its metabolism, which is independent of liver function, rendered it a good analgesic choice for our patient.…”

Section: Discussionmentioning

confidence: 99%

“…In Niemann-Pick type C, there is a broad spectrum of clinical manifestations, including liver and spleen involvement and neurological problems such as seizure, progressive dementia, and dystonia. Respiratory problems have also been noted ( 1 , 5 ).…”

Section: Introductionmentioning

confidence: 99%

“…Due to the complex clinical progression and multi-organ involvement, the anesthetic management of Niemann-Pick patients is challenging. However, only a few articles are available concerning the anesthetic implications of such patients ( 1 , 5 , 6 ). Hence, in this case report we describe the anesthetic management of a Niemann-Pick patient.…”

Section: Introductionmentioning

confidence: 99%

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Anesthetic Management in a Child With Niemann-Pick Disease

et al. 2016

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Niemann-Pick is a lipid storage disease that results from a lysosomal enzyme deficiency (sphingomyelinase). It has different presentations, and it may affect various organs such as the central nervous system, kidney, liver, and spleen. Due to the complexity of the disease, careful perianesthetic management is necessary in order to reduce the risks and sequels. As there is little evidence available in the literature regarding the anesthetic implications of such patients, in this case report we describe the anesthetic management of a two-year-old female with Niemann-Pick disease.

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“…The typical disease course is gradual neurological decline over years, resulting in increasing morbidity and eventual death from aspiration pneumonia, respiratory failure, or intractable epilepsy. Patient longevity is correlated to age of onset of neurological symptoms with an earlier manifestation indicating a less favorable prognosis 1,2 …”

Section: Introductionmentioning

confidence: 99%

Anesthetic management of pediatric patients with Niemann‐Pick disease type C for intrathecal 2‐hydroxypropyl‐β‐cyclodextrin injection

Ulloa

Froyshteter

Kret

et al. 2020

Pediatric Anesthesia

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Background Niemann‐Pick disease type C is an autosomal‐recessive, lysosomal storage disorder with variable age of onset and a heterogeneous clinical presentation that includes neurological, psychiatric, and visceral findings. Serial intrathecal injections of 2‐hydroxypropyl‐beta‐cyclodextrin are being evaluated as a treatment modality for Niemann‐Pick disease type C with a subset of patients requiring anesthesia for this procedure. Aims The aim of this study was to evaluate the safety of anesthesia provided for patients undergoing intrathecal injection of 2‐hydroxypropyl‐beta‐cyclodextrin. Methods A retrospective review of pediatric patients who received serial intrathecal injections of 2‐hydroxypropyl‐beta‐cyclodextrin with anesthesia at two tertiary care centers was conducted from December 2015 through April 2019. Data were extracted for analysis included preoperative comorbidities, demographics, vital signs, intraoperative anesthesia course, airway management technique, venous access, postoperative course, and perioperative complications. In total, 19 patients were identified and a total of 394 anesthetic encounters were included in this study. Results All 394 2‐hydroxypropyl‐beta‐cyclodextrin administration procedures were successfully performed, and there were no changes made in the anesthetic plan during the anesthesia encounters. Three hundred forty‐nine anesthetics were performed utilizing inhalation induction and mask maintenance, and 45 anesthetics were performed with placement of a supraglottic airway device due to patient body habitus and provider preference. The incidence of a major adverse event (aspirations, arterial desaturation) was 5/394 (1.3%, 95% CI 0.05%‐3.1%). Minor adverse events (emesis, delirium, hypotension, seizure, and airway obstruction) were observed in 19/394 encounters (4.8%, 95% CI 3.0%‐7.5%). Conclusions Our findings suggest that general anesthesia induced via inhalation induction and maintained with volatile anesthetic via mask or supraglottic airway is a safe and effective option for pediatric patients with Niemann‐Pick disease type C undergoing serial intrathecal injections of 2‐hydroxypropyl‐beta‐cyclodextrin, supporting this technique as a viable option for anesthetic care in these patients.

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Ambulatory Anaesthesia in a Patient with Niemann-Pick Disease Type C

Cited by 3 publications

References 11 publications

Gender-Specific Effects of Two Treatment Strategies in a Mouse Model of Niemann-Pick Disease Type C1

Gender-Specific Effects of Two Treatment Strategies in a Mouse Model of Niemann-Pick Disease Type C1

Anesthetic Management in a Child With Niemann-Pick Disease

Anesthetic management of pediatric patients with Niemann‐Pick disease type C for intrathecal 2‐hydroxypropyl‐β‐cyclodextrin injection

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