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Last update 08 May 2025

Hydrocephalus, Endocardial Fibroelastosis, and Cataracts

Last update 08 May 2025

Basic Info

Synonyms

Communicating hydrocephalus, endocardial fibroelastosis (EFE) and congenital cataracts, HEC (hydrocephalus, endocardial fibroelastosis, cataract) syndrome, HEC SYNDROME

+ [7]

Introduction

Syndrome that is characterised by communicating hydrocephalus, endocardial fibroelastosis and congenital cataracts. It has been described in two children, both of whom died a few months after birth (the first as a result of a respiratory infection and the second due to cardiac complications). The aetiology of the syndrome is unknown but a viral or genetic origin has been proposed.

Drugs associated with Hydrocephalus, Endocardial Fibroelastosis, and Cataracts

Fosaprepitant Dimeglumine

Target

NK1R

Mechanism

NK1R antagonists

Active Org.

Shandong Luoxin Pharmaceutical Group Stock Co., Ltd. [+7]

Originator Org.

Merck & Co., Inc.

Active Indication

Nausea [+2]

Inactive Indication

Advanced Lung Non-Small Cell Carcinoma [+8]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

European Union [+3]

First Approval Date11 Jan 2008

Clinical Trials associated with Hydrocephalus, Endocardial Fibroelastosis, and Cataracts

CTR20171385

/ CompletedPhase 3

在HEC患者中进行福沙匹坦与福沙匹坦（意美）预防CINV的有效性及安全性的随机、拟双盲、阳性对照研究

[Translation] A randomized, quasi-double-blind, positive-controlled study of the efficacy and safety of fosaprepitant versus fosaprepitant (Imetabolic) in preventing CINV in patients with HEC

在高致吐化疗患者中比较注射用福沙匹坦双葡甲胺与注射用福沙匹坦双葡甲胺（意美®）预防化疗所致的恶心、呕吐（CINV）的有效性及安全性。

[Translation]

To compare the efficacy and safety of fosaprepitant dimeglumine for injection and fosaprepitant dimeglumine for injection (Imperidone®) in preventing chemotherapy-induced nausea and vomiting (CINV) in patients receiving highly emetogenic chemotherapy.

Start Date30 Mar 2018

Sponsor / Collaborator

Hangzhou Jiuyuan Genetic Biopharmaceutical Co., Ltd.

CTR20150598

/ CompletedPhase 3

对比福沙匹坦双葡甲胺与阿瑞匹坦在预防HEC所致恶心、呕吐中的有效性及安全性的随机、双盲、阳性对照研究

[Translation] A randomized, double-blind, positive-controlled study comparing the efficacy and safety of fosaprepitant dimeglumine and aprepitant in preventing nausea and vomiting caused by HEC

评价注射用福沙匹坦双葡甲胺预防高致吐性化疗引起的恶心和呕吐（CINV）的有效性和安全性。

[Translation]

To evaluate the efficacy and safety of fosaprepitant dimeglumine for injection in preventing nausea and vomiting (CINV) induced by highly emetogenic chemotherapy.

Start Date06 Feb 2015

Sponsor / Collaborator

Jiangsu Haosen Pharmaceutical Research Institute Co., Ltd.

[+1]

100 Clinical Results associated with Hydrocephalus, Endocardial Fibroelastosis, and Cataracts

100 Translational Medicine associated with Hydrocephalus, Endocardial Fibroelastosis, and Cataracts

0 Patents (Medical) associated with Hydrocephalus, Endocardial Fibroelastosis, and Cataracts

Literatures (Medical) associated with Hydrocephalus, Endocardial Fibroelastosis, and Cataracts

18 Feb 2025·Science Signaling

Sustained chromosomal passenger complex activity preserves the pluripotency of human embryonic carcinoma cells

Article

Author: Guardavaccaro, Daniele ; Arakaki, Rieko ; Mouri, Yasuhiro ; Ishimaru, Naozumi ; Murano, Kensaku ; Kudo, Yasusei ; Shao, Wenhua ; Ruppert, Jan G. ; Tsunematsu, Takaaki ; Pagano, Michele

Human embryonic carcinoma (hEC) cells are derived from teratocarcinomas, exhibit robust proliferation, have a high differentiation potential, are the malignant counterparts of human embryonic stem cells (hESCs), and are considered hESC-like. The chromosomal passenger complex (CPC), made up of the microtuble binding protein Borealin, the kinase Aurora-B, the CPC-stabilizing inner centromere protein (INCENP), and the inhibitor of apoptosis family member Survivin, regulates cell division and is active exclusively during mitosis in somatic cells. The anaphase-promoting complex/cyclosome and its cofactor Cdh1 (APC/C Cdh1 ) is a ubiquitylating complex that catalyzes the degradation of Aurora-B and Borealin in somatic cells but has low activity during interphase in hESCs. Here, we found that Borealin and Aurora-B exhibited sustained stability throughout the cell cycle of hEC cells due to low APC/C Cdh1 activity. In contrast with somatic cells, CPC activity persisted across the cell cycle of hEC cells because of diminished APC/C Cdh1 activity. Disrupting the CPC complex by depleting its constituents triggered spontaneous differentiation in hEC cells. As hEC cells differentiated, APC/C Cdh1 activation curtailed CPC activity. Inactivating the CPC by pharmacologically inhibiting Aurora-B induced hEC cell differentiation by activating the epithelial-to-mesenchymal transition (EMT) program. Hence, APC/C Cdh1 -mediated termination of CPC activity triggered hEC cell differentiation. Collectively, these findings demonstrate a role for the CPC in governing hESC cell fate.

01 Oct 2024·Journal of NeuroInterventional Surgery

Safety and efficacy of tailored antiplatelet therapy using prasugrel or ticagrelor based on clopidogrel responsiveness in endovascular treatment for intracranial aneurysms: a meta-analysis

Article

Author: Cho, Young Dae ; Jang, Kyoung Min ; Choi, Hyunho ; Jang, Ju Sung

BackgroundClopidogrel (CPG)-based dual antiplatelet therapy (DAPT) in combination with aspirin has been widely used before endovascular procedures for intracranial aneurysms to prevent procedural thromboembolic complication (TEC). However, the main drawback of CPG is the high proportion of hyporesponders. This study sought to investigate the usefulness of tailored DAPT using novel P2Y12 inhibitors (prasugrel or ticagrelor, (PSG/TCG)) guided by a platelet reactivity test (PRT), compared with CPG-based conventional DAPT.MethodData were extracted from PubMed, Embase, and Cochrane Central Register of Controlled Trials by two independent reviewers. A random effects model was used to investigate the procedural TEC and hemorrhagic complications (HEC) of the tailored DAPT and conventional therapy by risk ratios (RR) and 95% confidence intervals (95% CI). Additionally, we performed subgroup analyses to directly compare prasugrel/ticagrelor with CPG.ResultsSix studies comprising 2557 patients were included in the analysis. Compared with conventional non-tailored therapy, PRT-guided tailored DAPT with PSG/TCG was associated with a lower risk of TEC (RR 0.40, 95% CI 0.22 to 0.74, P=0.004) without increasing HEC rates. The subgroup analysis showed that the switch to PSG/TCG in CPG hyporesponders was related to a lower incidence of TEC (RR 0.46, 95% CI 0.23 to 0.95, P=0.03) without a difference in HEC, compared with maintenance of CPG in CPG responders.ConclusionEvidence from this analysis supports PRT-guided tailored DAPT (using PSG/TCG) as a better choice for preparation towards endovascular procedures to treat aneurysms. Furthermore, it suggests that PSG/TCG is not limited to the role of a substitute for CPG but may be a first-line agent for DAPT.

02 Jun 2024·Expert Opinion on Drug Metabolism & Toxicology

Pharmacokinetics, pharmacodynamics, safety, and tolerability of dopamine-receptor antagonists for the prevention of chemotherapy-induced nausea and vomiting

Review

Author: Nguyen, Carolyn ; Orhan, Adile ; Herrstedt, Jørn ; Chan, Alexandre

INTRODUCTIONDopamine (D)_2,3-receptor antagonists (RAs) were the first antiemetics used in the prophylaxis of chemotherapy-induced nausea and vomiting (CINV).AREAS COVEREDEight D_2,3-RAs, amisulpride, domperidone, droperidol, haloperidol, metoclopramide, metopimazine, olanzapine and prochlorperazine are reviewed focusing on pharmacokinetics, pharmacodynamics, antiemetic effect and side effects.EXPERT OPINIONSince the introduction of D_2,3-RAs, antiemetics such as corticosteroids, 5-hydroxytryptamine (5-HT)₃-RAs and neurokinin (NK)₁-RAs have been developed. The classical D_2,3-RAs are recommended in the prophylaxis of CINV from low emetic risk chemotherapy, but not as a fixed component of an antiemetic regimen for moderately or highly (HEC) emetic risk chemotherapy. D_2,3-RAs are also used in patients with breakthrough nausea and vomiting. It should be emphasized, that most of these drugs are not selective for dopamine receptors.The multi-receptor targeting agent, olanzapine, is recommended in the prophylaxis of HEC-induced CINV as part of a four-drug antiemetic regimen, including a 5-HT₃-RA, dexamethasone and a NK₁-RA. Olanzapine is the most effective agent to prevent chemotherapy-induced nausea.Side effects differ among various D_2,3-RAs. Metopimazine and domperidone possess a low risk of extrapyramidal side effects. Domperidone and metoclopramide are prokinetics, whereas metopimazine delays gastric emptying and haloperidol does not influence gastric motility. Many D_2,3-RAs increase the risk of prolonged QTc interval; other side effects include sedation and orthostatic hypotension.

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Hydrocephalus, Endocardial Fibroelastosis, and Cataracts

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