A case of recurrent Miller-Fisher syndrome with multiple anti-ganglioside antibodies / 中国综合临床

Guillain-Barré syndrome (GBS) defines a kind of Immune-mediated acute inflammatory peripheral neuropathy. Miller-Fisher Syndrome (MFS) is a special variant of GBS, with mostly one-way course and rare clinical recurrence. Only a few recurrent cases have been reported in China. Here we report a case of a young male patient with double vision and progressive aggravation of limb numbness, acute onset, with symptoms of upper respiratory tract infection before onset, accompanied by pupil abnormalities and autonomic nervous dysfunction, who was was admitted to our hospital for similar symptoms 3 years ago and was improved by immunotherapy. The patient had a triad of “ataxia, areflexia and ophthalmoplegia”. Cerebrospinal fluid showed protein-cell separation. Serum anti-Sulfatides antibody IgM, anti-GT1a antibody IgG, anti-GQ1b antibody IgG and anti-GM3 IgM were positive. Recurrent MFS was diagnosed and the symptoms improved after immunotherapy. This case suggests that MFS is clinically heterogeneous, a few patients can present with relapse and generally have a better prognosis with immunotherapy. Pre-existing infection and anti-GQ1b antibody production may be predisposing factors for MFS recurrence.

Efficacy，safety and cost-effectiveness of edaravone dexborneol and compound porcine cerebroside ganglioside in the treatment of acute ischemic stroke / 中国药房

OBJECTIVE To evaluate efficacy， safety and cost-effectiveness of edaravone dexborneol and compound porcine cerebroside ganglioside in the treatment of acute ischemic stroke， and to provide decision-making reference for clinical treatment selection. METHODS The medical records of 488 patients with acute ischemic stroke hospitalized from Jan. 2021 to Dec. 2021 were collected and divided into two groups according to the treatment plan， i.e. 268 patients in edaravone dexborneol group， and 220 patients in compound porcine cerebroside ganglioside group. After baseline levels of the two groups were balanced using propensity score matching method， curative effect was evaluated according to the changes of NIHSS scores before and after treatment； the occurrence of adverse drug reactions in patients were collected from the hospital adverse reaction reporting system； from the perspective of China’s health system， the cost-effectiveness of the two options were analyzed， and one-way sensitivity analysis was conducted. RESULTS After the propensity score matching， 125 patients were included in the edaravone dexborneol group and compound porcine cerebroside ganglioside group， respectively. The response rates were 81.6% and 74.4%， respectively， with no significant difference. The average costs were 13 560.30 yuan and 14 958.68 yuan， respectively； the cost of edaravone dexborneol group was lower than that of compound porcine cerebroside ganglioside group. No adverse reaction reporting information was retrieved in both groups. Results of one-way sensitivity analysis showed that other drug costs in compound porcine cerebroside ganglioside group was relatively sensitive parameters. CONCLUSIONS Short-term efficacy and safety of edaravone dexborneol are equivalent to those of compound porcine cerebroside ganglioside in treating acute ischemic stroke. But edaravone dexborneol regimen had lower cost and is a more economical scheme.

Evaluation of the effect of intrathecal GM1 in 24, 48, and 72 hours after acute spinal cord injury in rats

Abstract Objective The aim of this study was to evaluate the best timing and feasibility of intrathecal application of sodium monosialoganglioside (GM1) after spinal cord contusion in Wistar rats as an experimental model. Methods Forty Wistar rats were submitted to contusion spinal cord injury after laminectomy. The animals were randomized and divided into four groups: Group 1 - Intrathecal application of GM1 24 hours after contusion; Group 2 - Intrathecal application of GM1 48 hours after contusion; Group 3 - intrathecal application of GM1 72 hours after contusion; Group 4 - Sham, with laminectomy and intrathecal application of 0.5 mL of 0.9% saline solution, without contusion. The recovery of locomotor function was evaluated at seven different moments by the Basso, Beattie, and Bresnahan (BBB) test. They were also assessed by the horizontal ladder, with sensory-motor behavioral assessment criteria, pre-and postoperatively. Results This experimental study showed better functional scores in the group submitted to the application of GM1, with statistically significant results, showing a mean increase when evaluated on known motor tests like the horizontal ladder and BBB, at all times of evaluation (p < 0.05), especially in group 2 (48 hours after spinal cord injury). Also, fewer mistakes and slips over the horizontal ladder were observed, and many points were achieved at the BBB scale analysis. Conclusion The study demonstrated that the intrathecal application of GM1 after spinal cord contusion in Wistar rats is feasible. The application 48 hours after the injury presented the best functional results.

Guillain-Barré syndrome / 中华神经科杂志

Guillain-Barré syndrome (GBS) is a rare, but potentially fatal, immune-mediated disease of the peripheral nerves and nerve roots, which is usually triggered by infections. It is characterized by rapidly progressive, symmetrical weakness of the extremities. Some patients develop respiratory insufficiency and many show signs of autonomic dysfunction. Diagnosis can usually be made on clinical grounds, but lumbar puncture and electrophysiological studies can help to substantiate the diagnosis and to differentiate demyelinating from axonal subtypes of GBS. Molecular mimicry of pathogen-borne antigens, leading to generation of crossreactive antibodies that also target gangliosides, is generally accepted pathogenesis of GBS. The treatment of GBS is intravenous immunoglobulin or plasma exchange with general clinical treatment. Most patients have a good prognosis and basically recover within weeks to months. A few patients have persistent neurological dysfunction or even death.

Guillain-Barré Syndrome in Mexico: An Updated Review Amid the Coronavirus Disease 2019 Era

ABSTRACT Guillain-Barré syndrome (GBS) is the most frequent cause of acute flaccid paralysis and if not diagnosed and treated timely, a significant cause of long-term disability. Incidence in Latin America ranges from 0.71 to 7.63 cases/100,000 person-years. Historically, GBS has been linked to infections (mainly gastrointestinal by Campylobacter jejuni) and vaccines (including those against severe acute respiratory syndrome coronavirus 2 [SARS-CoV-2]); however, a trigger cannot be detected in most cases. Regarding SARS-CoV-2, epidemiological studies have found no association with its development. Acute motor axonal neuropathy is the most common electrophysiological variant in Mexico and Asian countries. Intravenous immunoglobulin or plasma exchanges are still the treatment cornerstones. Mortality in Mexico can be as high as 12%. Advances in understanding the drivers of nerve injury in GBS that may provide the basis for developing targeted therapies have been made during the past decade; despite them, accurate criteria for selecting patients requiring acute treatment, prognostic biomarkers, and novel therapies are still needed. The newly-developed vaccines against SARS-CoV-2 have raised concerns regarding the potential risk for developing GBS. In the midst of coronavirus disease 2019 and vaccination campaigns against SARS-CoV-2, this review discusses the epidemiology, clinical presentation, management, and outcomes of GBS in Mexico.

Effects of ganoderma triterpenoids combined with exogenous GM1 on cognitive function and hippocampal synaptic structure in rats with epilepsy / 中华行为医学与脑科学杂志

Objective:To study the intervention effect of ganoderma triterpenoids combined with exogenous monosialotetrahexosyl ganglioside(GM1) on cognitive dysfunction and synaptic ultrastructure of hippocampal neurons in rats with epilepsy caused by pentylenetetrazol(PTZ).Methods:A total of 40 Sprague-Dawley rats were divided into blank control group, epileptic model group, ganoderma triterpenoids group, GM1 group and GM1 combined with ganoderma triterpenoids group according to the random number table method( n=8 in each group). The rats were intraperitoneally injected with PTZ subconvulsant dose (35 mg·kg -1·d -1) once a day for 28 days to replicate the models of chronic epilepsy. And the rats in different medication groups were given corresponding administration based on daily intraperitoneal injection of PTZ(GM1: intraperitoneal injection of 30 mg·kg -1·d -1, ganoderma triterpenoids: gavage 1 000 mg·kg -1·d -1). Morris water maze was used to test the spatial exploration and learning and memory ability of epileptic rats.Transmission electron microscopy was used to observe the ultrastructure of hippocampal neurons in epileptic rats.Immunofluorescence staining was used to observe expression levels of cofilin and SYN protein in hippocampus CA1 of rats. In addition, Western blot was used to detect the expression levels of cofilin, p-cofilin and synaptophysin(SYN) protein in hippocampus of rats. SPSS 17.0 software was used for statistical analysis. Repeated one-way ANOVA was used for comparing among groups, LSD test was used for pairwise comparisons. Results:Morris water maze results showed that there were statistically significant differences in escape latency, times of crossing the platform and time spent in the target quadrant among the groups( F=5.259, 8.240, 5.961, all P<0.05). Compared with the epilepsy model group, the escape latencies((20.31±7.39) s, (21.81±6.05) s, (17.66±4.76) s) of the ganoderma triterpenoids group, GM1 group and GM1 combined with ganoderma triterpenoids group were shorter (all P<0.05), the numbers of crossing the platform ((4.63±1.41) times, (4.50±1.93) times, (5.50±1.77) times) were more (all P<0.05), the residence time in target quadrant ((31.91±5.00) s, (30.49±5.72) s, (35.70±5.34) s) were longer (all P<0.05). And the most obvious change was found in the GM1 combined with ganoderma triterpenoids group ( P<0.01). The results of transmission electron microscope showed that there were significant differences in the numbers of hippocampal neurons synapses, the synaptic gap, the density of postsynaptic membrane and length of active area of postsynaptic membrane among the groups( F=3.693, 7.201, 5.012, 4.033, all P<0.05). Compared with the epilepsy model group, the numbers of synapses ((8.00±1.79), (7.83±1.84), (8.50±1.87)) in the ganoderma triterpenoids group, GM1 group and GM1 combined with ganoderma triterpenoids group were all more (all P<0.05), synaptic gap ((33.83±3.81)nm, (32.43±4.14)nm, (30.23±3.08)nm)were narrower, and the postsynaptic dense substances ((57.50±6.03)nm, (58.10±2.40)nm, (60.73±3.81)nm) were all thicker (all P<0.05). The length of active region of postsynaptic membrane ((271.66±11.80) nm, (279.06±13.58) nm) in ganoderma triterpenoid group and GM1 combined with ganoderma triterpenoids group were longer than that in epilepsy model group (both P<0.05). Immunofluorescence results showed that the average fluorescence intensity of cofilin in the epilepsy model group was higher than that in the blank control group, and the average fluorescence intensity of SYN was lower than that in the blank control group (both P<0.05). The average fluorescence intensity of cofilin in GM1 group and GM1 combined with ganoderma triterpenoids group were lower than that in epilepsy model group (both P<0.05), and the average fluorescence intensity of SYN in ganoderma lucidum triterpenoids combined with GM1 group was higher than that in epilepsy model group ( P<0.05). Western blot showed that the expression levels of cofilin protein in the epilepsy model group was higher than that in the blank control group ((1.454±0.080), (1.092±0.099), P<0.05), and the expression of p-cofilin and SYN were lower than those in the blank control group ((1.103±0.120) vs (1.420±0.934), (1.650±0.062) vs (1.958±0.062), both P<0.05). The expression of cofilin protein ((1.227±0.071), (1.262±0.078), (1.162±0.129), P<0.05) in ganoderma triterpenoids group, GM1 group and GM1 combined with ganoderma triterpenoids group were lower than that in epilepsy model group, and the expression levels of p-cofilin(1.357±0.199) and SYN protein(1.873±0.010) in ganoderma triterpenoids combined with GM1 group were higher than that in epilepsy model group (both P<0.05). Compared with ganoderma lucidum triterpenoids group and GM1 group, there was no significant difference in each index of GM1 combined with ganoderma triterpenoids group (all P>0.05). Conclusion:GM1 combined with ganoderma triterpenoids may promote the synaptic plasticity of neurons, improve the learning and memory ability of epileptic rats.Combination medication is better than single medication in some observed indicators.

Effects of ganglioside GM1 and erythropoietin on spinal cord injury in mice: Functional and immunohistochemical assessments

ABSTRACT Objectives: To evaluate the functional and immunohistochemical effects of ganglioside GM1 and erythropoietin following experimental spinal cord injury. Methods: Thirty-two male BALB/c mice were subjected to experimental spinal cord injury using the NYU Impactor device and were randomly divided into the following groups: GM1 group, receiving standard ganglioside GM1 (30 mg/kg); erythropoietin group, receiving erythropoietin (1000 IU/kg); combination group, receiving both drugs; and control group, receiving saline (0.9%). Animals were evaluated according to the Basso Mouse Scale (BMS) and Hindlimb Mouse Function Score (MFS). After euthanasia, the immunohistochemistry of the medullary tissue of mice was analyzed. All animals received intraperitoneal treatment. Results: The GM1 group had higher BMS and MFS scores at the end of the experiment when compared to all other groups. The combination group had higher BMS and MFS scores than the erythropoietin and control groups. The erythropoietin group had higher BMS and MFS scores than the control group. Immunohistochemical tissue analysis showed a significant difference among groups. There was a significant increase in myelinated axons and in the myelinated axon length in the erythropoietin group when compared to the other intervention groups (p < 0.01). Conclusion: Erythropoietin and GM1 have therapeutic effects on axonal regeneration in mice subjected to experimental spinal cord injury, and administration of GM1 alone had the highest scores on the BMS and MFS scales.

Clinical effect of monosialotetrahexosyl ganglioside in the treatment of neonatal intracranial hemorrhage / 中国基层医药

Objective To investigate the clinical effect of monosialotetrahexosyl ganglioside in the treatment of neonatal intracranial hemorrhage.Methods From January 2016 to December 2018,142 neonates with intracranial hemorrhage admitted to the Maternal and Child Health Hospital of Zhoushan were randomly divided into observation group (71 cases) and control group (71 cases) according to the digital table.The control group was treated with routine treatment,while the observation group was treated with ganglioside needle on the basis of the control group.Both two groups were treated for 14 days.The therapeutic effects,muscle tone recovery time,reflex recovery time and consciousness recovery time were compared.The changes of neurobehavioral assessment score (NABA score),TNF-αt,IL-1β,MMP-2,T IMP-1 and NSE levels before and after treatment were compared.Results The total effective rate of the observation group (92.96％) was higher than that of the control group (77.47％) (x2 =6.762,P ＜ 0.05).The recovery time of muscle tension,reflex and consciousness in the observation group [(7.68 ± 1.29) d,(6.83 ± 1.20) d and (8.34 ± 1.54) d] were shorter than those in the control group [(10.25 ± 2.31) d,(9.17 ±1.86) d and (10.53 ± 1.08) d] (t =8.185,8.908,9.811,all P ＜ 0.05).After treatment,the NABA score of the observation group [(40.37 ± 0.65) points] was higher than that of the control group [(37.16 ± 0.93) points] (t =23.838,P ＜ 0.05).The serum levels of TNF-α [(26.37 ± 4.25) pg/L],IL-1β [(16.74 ± 3.24) ng/L],MMP-2 [(78.39 ± 16.57)g/L],TIMP-1 [(179.32 ± 17.65) ng/mL] and NSE [(13.52 ± 2.19) g/L] in the observation group were lower than those in the control group [(53.21 ± 7.39) pg/L,(28.93 ± 5.64) ng/L,(97.42 ±12.63) g/L,(238.63 ± 28) ng/mL and (21.43 ± 2.89) μg/L] (t =26.529,15.792,7.696,14.938,1 8.381,all P ＜ 0.05).Conclusion Ganglioside has good therapeutic effect on neonatal intracranial hemorrhage.It can reduce the serum levels of TNF-α,IL-1β,MMP-2,TIMP-1 and NSE,and improve the neurobehavioral function of neonates.

Pattern of Extraocular Muscle Involvements in Miller Fisher Syndrome

BACKGROUND AND PURPOSE: The most-common initial manifestation of Miller Fisher syndrome (MFS) is diplopia due to acute ophthalmoplegia. However, few studies have focused on ocular motility findings in MFS. This study aimed to determine the pattern of extraocular muscle (EOM) paresis in MFS patients. METHODS: We consecutively recruited MFS patients who presented with ophthalmoplegia between 2010 and 2015. The involved EOMs and the strabismus pattern in the primary position were analyzed. Antecedent infections, other involved cranial nerves, and laboratory findings were also reviewed. We compared the characteristics of the patients according to the severity of ophthalmoplegia between complete ophthalmoplegia (CO) and incomplete ophthalmoplegia (IO). RESULTS: Twenty-five patients (15 males and 10 females) with bilateral ophthalmoplegia were included in the study. The most-involved and last-to-recover EOM was the lateral rectus muscle. CO and IO were observed in 11 and 14 patients, respectively. The patients were aged 59.0±18.4 years (mean±SD) in the CO group and 24.9±7.4 years in the IO group (p<0.01), and comprised 63.6% and 21.4% females, respectively (p=0.049). Elevated cerebrospinal fluid protein was identified in 60.0% of patients with CO and 7.7% of patients with IO (p=0.019) for a mean follow-up time from the initial symptom onset of 3.7 days. CONCLUSIONS: The lateral rectus muscle is the most-involved and last-to-recover EOM in ophthalmoplegia. The CO patients were much older and were more likely to be female and have an elevation of cerebrospinal fluid protein than the IO patients.

Analysis of gene mutation in a pedigree with autosomal dominant Charcot-Marie-Tooth disease / 南方医科大学学报

OBJECTIVE@#To investigate the molecular genetic mechanism of Charcot- Marie-Tooth (CMT) disease in a pedigree.@*METHODS@#Genomic DNA was extracted from the peripheral blood of the family members of a pedigree with autosomal dominant CMT disease, and 65 candidate genes of the proband were screened using target exon capture and the next generation sequencing, and the suspicious genes were verified using Sanger sequencing. PolyPhen-2, PROVEAN and SIFT software were used to predict the function of the mutant genes, and PyMOL-1 software was used to simulate the mutant protein structure.@*RESULTS@#A heterozygous missense mutation [c.371A>G (p.Y124C)] was detected in exon 3 of gene of the proband. This heterozygous mutation was also detected in both the proband's mother and her brother, but not in her father. Multiple sequence alignment analysis showed that tyrosine at codon 124 of GDAP1 protein was highly conserved. All the 3 prediction software predicted that the mutation was harmful. Molecular structure simulation showed a weakened interaction force between the amino acid residues at codon 124 and the surrounding amino acid residues to affect the overall stability of the protein.@*CONCLUSIONS@#The mutation of gene may be related to the pathogenesis of autosomal dominant AD-CMT in this pedigree. The newly discovered c.371A>G mutation (p.Y124C) expands the mutation spectrum of gene, but further study is needed to clarify the underlying pathogenesis.

Clinical and CLB1 gene mutations analysis of GM1 gangliosidosis in a patient / 中华神经科杂志

Objective@#To investigate the clinical and CLB1 gene mutation characteristics of GM1 gangliosidosis patient.@*Methods@#The clinical data of one GM1 gangliosidosis patient from Children′s Hospital Affiliated to Zhengzhou University in March 2018 were reviewed and analyzed. The patient was diagnosed by gene detection and enzymatic activity.@*Results@#The patient is a 4 years and 1 month old boy, mainly presented psychomotor retrogression. His β-galactosidase activity was low (8.0 nmol·g-1·min-1). Two splice site mutations (c.458-2A(IVS4)>G and c.1068+5G(IVS10)>A) of patient′s CLB1 gene were screened by targeted next generation sequencing. The results of Sanger sequencing showed that the mutations are compound heterozygous and both are first reported. The mutation c.1068+5G(IVS10)>A was derived from patient′s mother, and the other one is de nove.@*Conclusion@#GM1 gangliosidosis is a rare neurodegenerative disease, which could be accurately diagnosed by the next generation sequencing and enzyme assay.

Clinical and CLB1 gene mutations analysis of GM1 gangliosidosis in a patient / 中华神经科杂志

Objective To investigate the clinical and CLB1 gene mutation characteristics of GM1 gangliosidosis patient. Methods The clinical data of one GM1 gangliosidosis patient from Children′s Hospital Affiliated to Zhengzhou University in March 2018 were reviewed and analyzed. The patient was diagnosed by gene detection and enzymatic activity. Results The patient is a 4 years and 1 month old boy, mainly presented psychomotor retrogression. His β?galactosidase activity was low (8.0 nmol·g-1·min-1). Two splice site mutations (c.458?2A(IVS4)>G and c.1068+5G(IVS10)>A) of patient′s CLB1 gene were screened by targeted next generation sequencing. The results of Sanger sequencing showed that the mutations are compound heterozygous and both are first reported. The mutation c.1068+5G(IVS10)>A was derived from patient′s mother, and the other one is de nove. Conclusion GM1 gangliosidosis is a rare neurodegenerative disease, which could be accurately diagnosed by the next generation sequencing and enzyme assay.

Effect of ganglioside preconditioning on endoplasmic reticulum stress-dependent apoptosis during spinal cord injury induced by bupivacaine in rats / 中华麻醉学杂志

Objective To evaluate the effect of ganglioside ( GM-1) preconditioning on endoplas-mic reticulum stress ( ERS)-dependent apoptosis during spinal cord injury induced by bupivacaine in rats. Methods One hundred and eight clean-grade healthy male Sprague-Dawley rats, aged 8 weeks, weighing 250-300 g, were divided into 3 groups ( n=36 each) using a random number table method: control group (group C), bupivacaine group (group B) and GM-1 pretreatment group (group G). In group B, 5％bupivacaine 0. 12 μl/g was intrathecally injected at 1. 5 h intervals, 3 times intotal. In group G, GM-120μl ( 30 mg/kg) was intrathecally injected, and 24 h later bupivacaine was intrathecally injected according to the method previously described in group B. Immediately after intrathecal injection and at 1, 3, 5, 7 and 14 days after intrathecal injection, the maximum percentage of anti-nociceptive effects (％MPE) was detected, the hindlimb motor function score ( BBB score) was evaluated, and spinal cord tissues were har-vested for determination of cell apoptosis ( using TUNEL) and expression of caspase-12 and CCAAT/enhan-cer-binding protein homologous protein ( CHOP ) protein and mRNA ( by Western blot or quantitative real-time polymerase chain reaction) . The apoptosis rate was calculated. Results Compared with group C, the％MPE and apoptosis rate were significantly increased, the BBB score was decreased, and the expression of CHOP and caspase-12 protein and mRNA was up-regulated in group B ( P＜0. 05) . Compared with group B, the ％MPE and apoptosis rate were significantly decreased, the BBB score was increased, and the ex-pression of CHOP and caspase-12 protein and mRNA was down-regulated in group G ( P＜0. 05) . Conclu-sion GM-1 preconditioning reduces bupivacaine-induced spinal cord injury possibly through inhibiting ERS-dependent apoptosis in rats.

Clinical Heterogeneity of Anti-GM2-Ganglioside-Antibody Syndrome

BACKGROUND AND PURPOSE: Antiganglioside antibodies are known to play a pathogenic role in Guillain-Barré syndrome (GBS). Either an immunoglobulin (Ig)G- or IgM-type anti-GM2 antibody is detected in rare cases in GBS patients. However, the specific pathogenic role of these antibodies in GBS has not been reported previously. This study aimed to define and characterize the clinical spectrum of GBS with anti-GM2 positivity. METHODS: We reviewed the database of the Dong-A University Neuroimmunology Team, which has collected sera of GBS and its variants from more than 40 general and university-based hospitals in Korea. Detailed information about the involved patients was often obtained and then corrected by the charge doctor applying additional questionnaires. RESULTS: Four patients with acute monophasic peripheral neuropathy or cranial neuropathy with isolated IgM-type anti-GM2-antibody positivity were recruited. In addition, IgG-type anti-GM2 antibody was solely detected in the sera of another four patients. The IgM-positive group comprised heterogeneous syndromes: two cases of acute motor axonal neuropathy, one of acute inflammatory demyelinating polyneuropathy, and one of isolated facial diplegia. In contrast, all of the cases enrolled in the IgG-positive group manifested with dizziness with or without oculomotor palsy due to cranial neuropathy syndrome. CONCLUSIONS: This study has identified that anti-GM2 antibody can be found in various subtypes of GBS and its variants in rare cases. Compared to the clinical heterogeneity of the IgM-positive group, the IgG-positive group can be characterized by cranial-dominant GBS variants presenting mainly with oculomotor and vestibular dysfunctions.

Ganglioside GD3 synthase (GD3S), a novel cancer drug target

Gangliosides are a class of important glycosphingolipids containing sialic acid that are widely distributed on the outer surface of cells and are abundantly distributed in brain tissue. Disialoganglioside with three glycosyl groups (GD3) and disialoganglioside with two glycosyl groups (GD2) are markedly increased in pathological conditions such as cancers and neurodegenerative diseases. GD3 and GD2 were found to play important roles in cancers by mediating cell proliferation, migration, invasion, adhesion, angiogenesis and in preventing immunosuppression of tumors. GD3 synthase (GD3S) is the regulatory enzyme of GD3 and GD2 synthesis, and is important in tumorigenesis and the development of cancers. The study of GD3S as a drug target may be of great significance for the discovery of new drugs for cancer treatment. This review will describe the gangliosides and their roles in physiological and pathological conditions; the roles of GD3 and GD2 in cancers; the expression, functions and mechanisms of GD3S, and its potential as a drug target in cancers.

Analysis of risk factors of outcomes of radiation induced-brain injury after IMRT in nasopharyngeal carcinoma / 中华放射肿瘤学杂志

Objective To explore the risk factors influencing the outcomes of radiation brain injury after intensity-modulated radiotherapy (IMRT) in patients diagnosed with nasopharyngeal carcinoma.Methods Clinical data of 1 300 nasopharyngeal carcinoma patients treated with IMRT in our hospital during 2006 and 2013 were retrospectively analyzed.Fifty-eight patients presented with radiation brain injury after IMRT.MRI data of these patients during 3-24 months follow-up were collected.The clinical efficacy in the treatment of radiation brain injury was evaluated according to RECIST guidelines.Results Forty-six patients with intact follow-up data were enrolled.The median latency of radiation brain injury was 34 months.Patients were divided into the response (CR+PR) and non-response groups.The risk factors influencing the response rate during 10-12 months and 18-24 months were identified and analyzed.Univariate analysis demonstrated that gender,age,smoking history,T stage,and high-intensity treatment exerted no significant effect upon the objective remission rate during these two time intervals periods.Patients treated with gangliosides obtained high response rate.The response rate was 68.8％ in 10-12 months (P=0.000),and 81.8％ in 18-24 months (P=0.008).Multivariate analysis revealed that use of gangliosides was a favorable factor for mitigating radiation brain injury in two time intervals (OR=19.8 and 13.5;P=0.001 and 0.005).Conclusions Use of gangliosides probably accelerates the healing of radiation brain injury,whereas the clinical efficacy remains to be elucidated by prospective clinical trials.

Gangliosidose GM1 infantil ­ relato de caso / Child GM1 Gangliosidosis ­ Case Report

Introdução: A gangliosidose é uma doença caracterizada pelo acúmulo do substrato gangliosídeo nos lisossomos devido à deficiência da enzima betagalactosidase. É uma desordem rara, estimando-se uma incidência na população de 1:100.000 a 200.000. Clinicamente os pacientes apresentam graus variados de neurodegeneração e alterações esqueléticas, categorizadas pela gravidade e atividade residual da beta-galactosidase, podendo ocorrer dismorfismo facial, hepatoesplenomegalia, displasia esquelética, manchas maculares vermelhas, cegueira e até morte precoce. O atraso no desenvolvimento neuropsicomotor associada à degeneração do sistema nervoso central, pode levar o paciente a um quadro de hipotonia muscular generalizada progressiva, evoluindo para espasticidade e crises convulsivas. Objetivo: Relata-se caso de paciente masculino, apresentando alterações no desenvolvimento neuropsicomotor desde os oito meses, com elucidação diagnóstica através da clínica, exames de imagem e laboratoriais. Método: Busca em bancos de dados digitais artigos científicos que discorram sobre a gangliosidose. Resultados/Conclusão: A gangliosiodose é uma disordem rara, o que torna o relato de caso importante como fonte de pesquisa. (AU)

Introduction: Gangliosidosis is a disease characterized by accumulation of the ganglioside substrate in lysosomes due to beta-galactosidase enzyme deficiency. It is a rare disorder, with an incidence of 1: 100,000 to 200,000. Clinically the patients pres- ent varying degrees of neurodegeneration and skeletal changes, categorized by the gravity and residual activity of beta-galactosidase, being able to occur facial dysmorphism, hepatosplenomegaly, skeletal dysplasia, red macular spots, blind- ness and early death. Objective: Delayed neuropsychomotor development associated with degeneration of the central nervous system may lead the patient to progressive generalized muscular hypotonia, evolving into seizures and convulsive seizures. We report a case of male patient, presenting changes in neuropsychomotor devel- opment since eight months of age, with diagnostic elucidation through clinical exam, imaging and laboratory tests. Method: Search in digital databases for scientific articles that discuss gangliosidoses. Results/ conclusion: Gangliosidosis is rare disorder, which makes reporting an important source of research. (AU)

Clinical Observation of Mouse Nerve Growth Factor Combined with Ganglioside in the Treatment of Hy-poxic-ischemic Encephalopathy / 中国药房

OBJECTIVE:To evaluate clinical efficacy and safety of mouse nerve growth factor combined with ganglioside in the treatment of hypoxic-ischemic encephalopathy (HIE). METHODS:A total of 150 HIE children in pediatric department of our hospital during Jan. 2013-Jan. 2015 were divided into control group and observation group according to random number table,with 75 cases in each group. Both groups received routine treatment as correcting hypotension,reducing intracranial pressure,etc. Con-trol group was additionally given Monosialotetrahexosylganglioside sodium injection 20 mg added into 10% Glucose injection 30-50 mL,ivgtt,qd. Observation group was additionally given Mouse nerve growth factor for injection 30 μg added into Water for injection 2 mL,im,qd,on the basis of control group. A treatment course lasted for 10 days,and both groups received 2 courses of treatment. Clinical efficacies of 2 groups were compared as well as NBNA score,the levels of related lab test indexes (IL-10, TNF-α,SOD,NSE,VEGF) before and after treatment,the occurrence of ADR and sequela (following up to 1 year old). RE-SULTS:The response rate of observation group was 86.7%,which was significantly higher than 72.0% of control group,with sta-tistical significance(P0.05). On 4th,7th,10th day after treatment,NBNA scores of 2 groups were increased significantly, compared to before treatment;the observation group was significantly higher than the control group,with statistical significance (P<0.05). After treatment,serum levels of IL-10,TNF-α,NSE and VEGF in 2 groups were decreased significantly,compared to before treatment,SOD levels were increased significantly,and the observation group was significantly better than the control group,with statistical significance(P<0.05). No obvious ADR was found in 2 groups during treatment. Totally 64 children in ob-servation group and 60 in control group completed follow-up. The total incidence of sequela in observation group was 10.9%, which was significantly lower than 25.0% of control group,with statistical significance(P<0.05). CONCLUSIONS:For neonatal HIE,mouse nerve growth factor combined with ganglioside can effectively relieve brain tissue inflammatory reaction and oxidative stress injury,accelerate the recovery of cerebral tissue and reduce the occurrence of sequela with good safety.

Protective effects of Shuxuening Injection combined with ganglioside on patients with acute severe carbon monoxide poisoning / 中成药

AIM To investigate the effects of Shuxuening Injection (Ginkgo biloba extract) combined with ganglioside on cerebral oxygen utilization coefficient (O2UCc) and lactate clearance rate (LCR) in patients with acute severe carbon monoxide poisoning (ASCOP).METHODS Two hundred and sixty-two patients with ASCOP treated in emergency department of Harrison International Peace Hospital Affiliated to Hebei Medical University from Mar 2011 to Mar 2016 were randomly divided into control group,Shuxuening Injection group,ganglioside group and Shuxuening Injection combined with ganglioside (combined medication) group.The changes were detected in oxygen saturations of carotid blood and jugular blood,arterial blood lactic acid,O2UCc and LCR in all patients before and 6,24 hours,3 days after the treatment.The clinical symptoms,and the mortality and morbidity of delayed encephalopathy after acute carbon monoxide poisoning after two weeks were observed.RESULTS Total effective rate of the combined medication group was higher than that of the other groups.O2 Ucc at 6,24 hours after the treatment in the combined medication group were significantly lower than those in the other groups,so did the mortality and morbidity of delayed encephalopathy after acute carbon monoxide poisoning,but LCR was significantly higher than that in the other groups.All the differences had statistical significances.CONCLUSION The efficacy of combined medication of Shuxuening Injection and ganglioside in ASCOP patients is better than that of applying single administration separately.

Effect of cognitive dysfunction of gangliosides on in the treatment of the patients with traumatic brain injury intraoperative and postoperative / 中国生化药物杂志

Objective To study the effect of cognitive dysfunction of gangliosides on in the treatment of the patients with traumatic brain injury intraoperative and postoperative. Methods From February 2015 to August 2016, 100 patients with cognitive dysfunction after operation were randomly divided into two groups, the control group and the experimental group, 50 cases in each group. The control group were received routine treatment, including medication, brain rehabilitation, and dehydration and intracranial pressure reduction. At this basis, the experimental group were treated with gangliosides. The clinical indexes in the experimental group and the control group were compared and analyzed. Results The cure rate was 90% in the experimental group, and 54% in the control group. The difference in the two groups has statistically significant (P<0.05). Before treatment, there was no significant difference of the MMSE in the two groups. After treatment, the MMSE score in the experimental group was (28.34 ± 4.12) points, and the score of MMSE in the control group was (22.19 ± 5.49) points. The difference in the two groups has statistically significant (P<0.05). Conclusion The effect of cognitive dysfunction is better which ganglioside was used on the treatment of the patients with traumatic brain injury intraoperative and postoperative, and the cure rate is higher. It has the significance of further popularization and application.