Citation: | Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital National Rare Diseases Committee. Expert Consensus on Clobazam in the Treatment of Refractory Epilepsy (2022)[J]. Medical Journal of Peking Union Medical College Hospital, 2022, 13(5): 768-782. DOI: 10.12290/xhyxzz.2022-0421 |
[1] |
Song P, Liu Y, Yu X, et al. Prevalence of epilepsy in China between 1990 and 2015: A systematic review and meta-analysis[J]. J Glob Health, 2017, 7: 020706. DOI: 10.7189/jogh.07.020706
|
[2] |
Kalilani L, Sun X, Pelgrims B, et al. The epidemiology of drug-resistant epilepsy: A systematic review and meta-analysis[J]. Epilepsia, 2018, 59: 2179-2193. DOI: 10.1111/epi.14596
|
[3] |
Kwan P, Arzimanoglou A, Berg AT, et al. Definition of drug resistant epilepsy: consensus proposal by the ad hoc Task Force of the ILAE Commission on Therapeutic Strategies[J]. Epilepsia, 2010, 51: 1069-1077.
|
[4] |
中国抗癫痫协会. 临床诊疗指南: 癫痫病分册[M]. 北京: 人民卫生出版社, 2015.
|
[5] |
Ng YT, Collins SD. Clobazam[J]. Neurotherapeutics, 2007, 4: 138-144. DOI: 10.1016/j.nurt.2006.11.002
|
[6] |
Nakajima H. A pharmacological profile of clobazam (Mystan), a new antiepileptic drug[J]. Nihon Yakurigaku Zasshi, 2001, 118: 117-122. DOI: 10.1254/fpj.118.117
|
[7] |
Humayun MJ, Samanta D, Carson RP. Clobazam[M]. StatPearls. Treasure Island (FL): StatPearls Publishing, 2022.
|
[8] |
FDA Approves Onfi. FDA approves onfi for seizures associated with lennox-gastaut syndrome[EB/OL]. (2022-01-02)[2022-08-11]. https://www.drugs.com/newdrugs/fda-approves-onfi-seizuresassociated-lennox-gastaut-syndrome-2921.html.
|
[9] |
Scottish Intercollegiate Guidelines Network. Epilepsies in children and young people: investigative procedures and managemen[EB/OL]. (2021-05)[2022-08-11]. https://www.sign.ac.uk/our-guidelines/epilepsies-in-children-and-young-people-investigative-procedures-and-manage-ment/.
|
[10] |
Joshi C, Nickels K, Demarest S, et al. Results of an international Delphi consensus in epilepsy with myoclonic atonic seizures/ Doose syndrome[J]. Seizure, 2021, 85: 12-18. DOI: 10.1016/j.seizure.2020.11.017
|
[11] |
Montouris G, Aboumatar S, Burdette D, et al. Expert opinion: Proposed diagnostic and treatment algorithms for Lennox-Gastaut syndrome in adult patients[J]. Epilepsy Behav, 2020, 110: 107146. DOI: 10.1016/j.yebeh.2020.107146
|
[12] |
Wirrell EC, Laux L, Franz DN, et al. Stiripentol in Dravet syndrome: results of a retrospective U.S. study[J]. Epilepsia, 2013, 54: 1595-1604. DOI: 10.1111/epi.12303
|
[13] |
Dressler A, Trimmel-Schwahofer P, Reithofer E, et al. Efficacy and tolerability of the ketogenic diet in Dravet syndrome-Comparison with various standard antiepileptic drug regimen[J]. Epilepsy Res, 2015, 109: 81-89. DOI: 10.1016/j.eplepsyres.2014.10.014
|
[14] |
Trivisano M, Striano P, Sartorelli J, et al. CHD2 mutations are a rare cause of generalized epilepsy with myoclonic-atonic seizures[J]. Epilepsy Behav, 2015, 51: 53-56. DOI: 10.1016/j.yebeh.2015.06.029
|
[15] |
Vlaskamp DR, Rump P, Callenbach PM, et al. Haploinsufficiency of the STX1B gene is associated with myoclonic astatic epilepsy[J]. Eur J Paediatr Neurol, 2016, 20: 489-492. DOI: 10.1016/j.ejpn.2015.12.014
|
[16] |
Song L, Liu F, Liu Y, et al. Clonazepam add-on therapy for drug-resistant epilepsy[J]. Cochrane Database Syst Rev, 2020, 4: Cd012253.
|
[17] |
国家药品监督管理局. 氯巴占临时进口工作方案[EB/OL]. (2022-06-29)[2022-08-11]. http://www.nhc.gov.cn/yaozs/s7653/202206/be5e83d15642445f89685af454e1557a.shtml.
|
[18] |
Centre for Evidence-Based Medicine. Oxford Center for Evidence-Based Medicine: Levels of Evidence(March 2009)[EB/OL]. (2009-03)[2022-08-11]. https://www.cebm.ox.ac.uk/resources/levels-of-evidence/oxford-centre-for-evidence-based-medicine-levels-of-evidence-march-2009.
|
[19] |
翟菲菲, 卢强. 氯巴占在癫痫治疗中的研究进展[J]. 癫痫杂志, 2022, 8: 133-137. https://www.cnki.com.cn/Article/CJFDTOTAL-DXZA202202006.htm
|
[20] |
Sankar R. GABA(A) receptor physiology and its relationship to the mechanism of action of the 1, 5-benzodiazepine clobazam[J]. CNS Drugs, 2012, 26: 229-244. DOI: 10.2165/11599020-000000000-00000
|
[21] |
Strzelczyk A, Schubert-Bast S. Expanding the Treatment Landscape for Lennox-Gastaut Syndrome: Current and Future Strategies[J]. CNS Drugs, 2021, 35: 61-83. DOI: 10.1007/s40263-020-00784-8
|
[22] |
Trevathan E, Murphy CC, Yeargin-Allsopp M. Prevalence and descriptive epidemiology of Lennox-Gastaut syndrome among Atlanta children[J]. Epilepsia, 1997, 38: 1283-1288. DOI: 10.1111/j.1528-1157.1997.tb00065.x
|
[23] |
European Commission. Rare Disease Registries in Europe. September 2020[EB/OL]. (2020-09)[2022-08-11]. https://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf.
|
[24] |
National Organization for Rare Disorders. Rare Disease Database[EB/OL]. [2022-08-11]. https://rarediseases.org/for-patients-and-families/information-resources/rare-disease-information/.
|
[25] |
Ostendorf AP, Ng YT. Treatment-resistant Lennox-Gastaut syndrome: therapeutic trends, challenges and future directions[J]. Neuropsychiatr Dis Treat, 2017, 13: 1131-1140. DOI: 10.2147/NDT.S115996
|
[26] |
Cross JH, Auvin S, Falip M, et al. Expert Opinion on the Management of Lennox-Gastaut Syndrome: Treatment Algorithms and Practical Considerations[J]. Front Neurol, 2017, 8: 505. DOI: 10.3389/fneur.2017.00505
|
[27] |
National Institute for Health and Care Excellence. Clinical Guidelines. Epilepsies: diagnosis and management[M]. London: National Institute for Health and Care Excellence, 2021.
|
[28] |
季涛云, 姜玉武, 蒋莉, 等. Lennox-Gastaut综合征诊断治疗的中国专家共识[J]. 癫痫杂志, 2022, 8: 187-195. https://www.cnki.com.cn/Article/CJFDTOTAL-DXZA202203014.htm
|
[29] |
Conry JA, Ng YT, Paolicchi JM, et al. Clobazam in the treatment of Lennox-Gastaut syndrome[J]. Epilepsia, 2009, 50: 1158-1166. DOI: 10.1111/j.1528-1167.2008.01935.x
|
[30] |
Ng YT, Conry JA, Drummond R, et al. Randomized, phase Ⅲ study results of clobazam in Lennox-Gastaut syndrome[J]. Neurology, 2011, 77: 1473-1481. DOI: 10.1212/WNL.0b013e318232de76
|
[31] |
Ng YT, Conry J, Paolicchi J, et al. Long-term safety and efficacy of clobazam for Lennox-Gastaut syndrome: interim results of an open-label extension study[J]. Epilepsy Behav, 2012, 25: 687-694. DOI: 10.1016/j.yebeh.2012.09.039
|
[32] |
Conry JA, Ng YT, Kernitsky L, et al. Stable dosages of clobazam for Lennox-Gastaut syndrome are associated with sustained drop-seizure and total-seizure improvements over 3 years[J]. Epilepsia, 2014, 55: 558-567. DOI: 10.1111/epi.12561
|
[33] |
Cramer JA, Sapin C, François C. Indirect comparison of clobazam and other therapies for Lennox-Gastaut syndrome[J]. Acta Neurol Scand, 2013, 128: 91-99. DOI: 10.1111/ane.12086
|
[34] |
Devi N, Madaan P, Ameen R, et al. Short-term and long-term efficacy and safety of antiseizure medications in Lennox Gastaut syndrome: A network meta-analysis[J]. Seizure, 2022, 99: 164-175. DOI: 10.1016/j.seizure.2022.04.004
|
[35] |
Zhang L, Wang J, Wang C. Efficacy and safety of antisei-zure medication for Lennox-Gastaut syndrome: a systematic review and network meta-analysis[J]. Dev Med Child Neurol, 2022, 64: 305-313. DOI: 10.1111/dmcn.15072
|
[36] |
Engel J Jr, International League Against Epilepsy. A proposed diagnostic scheme for people with epileptic seizures and with epilepsy: report of the ILAE Task Force on Classification and Terminology[J]. Epilepsia, 2001, 42: 796-803. DOI: 10.1046/j.1528-1157.2001.10401.x
|
[37] |
Sun H, Zhang Y, Liu X, et al. Analysis of SCN1A mutation and parental origin in patients with Dravet syndrome[J]. J Hum Genet, 2010, 55: 421-427. DOI: 10.1038/jhg.2010.39
|
[38] |
Connolly MB. Dravet Syndrome: Diagnosis and Long-Term Course[J]. Can J Neurol Sci, 2016, 43: S3-S8.
|
[39] |
刘家雯, 操德智. Dravet综合征临床治疗新进展[J]. 中华实用儿科临床杂志, 2018, 33: 1913-1917. DOI: 10.3760/cma.j.issn.2095-428X.2018.24.020
|
[40] |
Lagae L. Dravet syndrome[J]. Curr Opin Neurol, 2021, 34: 213-218. DOI: 10.1097/WCO.0000000000000902
|
[41] |
Tanabe T, Awaya Y, Matsuishi T, et al. Management of and prophylaxis against status epilepticus in children with severe myoclonic epilepsy in infancy (SMEI, Dravet syndrome)--a nationwide questionnaire survey in Japan[J]. Brain Dev, 2008, 30: 629-635. DOI: 10.1016/j.braindev.2008.03.002
|
[42] |
Wirrell EC, Laux L, Donner E, et al. Optimizing the Diagnosis and Management of Dravet Syndrome: Recommenda-tions From a North American Consensus Panel[J]. Pediatric Neurol, 2017, 68: 18-34. e3. DOI: 10.1016/j.pediatrneurol.2017.01.025
|
[43] |
Cross JH, Caraballo RH, Nabbout R, et al. Dravet syndrome: Treatment options and management of prolonged seizures[J]. Epilepsia, 2019, 60: S39-S48.
|
[44] |
Brigo F, Igwe SC, Bragazzi NL. Antiepileptic drugs for the treatment of infants with severe myoclonic epilepsy[J]. Cochrane Database Syst Rev, 2017, 5: CD010483.
|
[45] |
Doose H, Gerken H, Leonhardt R, et al. Centrencephalic myoclonic-astatic petit mal. Clinical and genetic investigation[J]. Neuropadiatrie, 1970, 2: 59-78. DOI: 10.1055/s-0028-1091841
|
[46] |
Kelley SA, Kossoff EH. Doose syndrome (myoclonic-astatic epilepsy): 40 years of progress[J]. Dev Med Child Neurol, 2010, 52: 988-993. DOI: 10.1111/j.1469-8749.2010.03744.x
|
[47] |
陈娇阳, 张月华. 癫痫伴肌阵挛-失张力发作的分子遗传学研究进展[J]. 中华儿科杂志, 2019, 57: 384-387. DOI: 10.3760/cma.j.issn.0578-1310.2019.05.015
|
[48] |
Nickels K, Kossoff EH, Eschbach K, et al. Epilepsy with myoclonic-atonic seizures (Doose syndrome): Clarification of diagnosis and treatment options through a large retrospective multicenter cohort[J]. Epilepsia, 2021, 62: 120-127. DOI: 10.1111/epi.16752
|
[49] |
Kilaru S, Bergqvist AGC. Current treatment of myoclonic astatic epilepsy: clinical experience at the Children's Hospital of Philadelphia[J]. Epilepsia, 2007, 48: 1703-1707. DOI: 10.1111/j.1528-1167.2007.01186.x
|
[50] |
Stephani U. The natural history of myoclonic astatic epilepsy (Doose syndrome) and Lennox-Gastaut syndrome[J]. Epilepsia, 2006, 47: 53-55. DOI: 10.1111/j.1528-1167.2006.00690.x
|
[51] |
McTague A, Cross JH. Treatment of epileptic encephalopathies[J]. CNS Drugs, 2013, 27: 175-184. DOI: 10.1007/s40263-013-0041-6
|
[52] |
邓劼, 张月华, 杨志仙, 等. 肌阵挛失张力癫痫48例治疗及预后随访[J]. 中国实用儿科杂志, 2012, 27: 57-61. https://www.cnki.com.cn/Article/CJFDTOTAL-ZSEK201201019.htm
|
[53] |
Nickels K, Thibert R, Rau S, et al. How do we diagnose and treat epilepsy with myoclonic-atonic seizures (Doose syndrome)? Results of the Pediatric Epilepsy Research Consortium survey[J]. Epilepsy Res, 2018, 144: 14-19. DOI: 10.1016/j.eplepsyres.2018.04.010
|
[54] |
Ito S, Nagumo K, Nishikawa A, et al. Low-dose phenobarbital for epilepsy with myoclonic absences: A case report[J]. Brain Dev, 2021, 43: 666-668. DOI: 10.1016/j.braindev.2020.12.018
|
[55] |
Wallace A, Wirrell E, Kenney-Jung DL. Pharmacotherapy for Dravet Syndrome[J]. Paediatric Drugs, 2016, 18: 197-208. DOI: 10.1007/s40272-016-0171-7
|
[56] |
Anon. Clobazam in treatment of refractory epilepsy: the Canadian experience. A retrospective study. Canadian Cloba-zam Cooperative Group[J]. Epilepsia, 1991, 32: 407-416. DOI: 10.1111/j.1528-1157.1991.tb04670.x
|
[57] |
Contin M, Sangiorgi S, Riva R, et al. Evidence of polymorphic CYP2C19 involvement in the human metabolism of N-desmethylclobazam[J]. Ther Drug Monit, 2002, 24: 737-741. DOI: 10.1097/00007691-200212000-00009
|
[58] |
Walzer M, Bekersky I, Blum RA, et al. Pharmacokinetic drug interactions between clobazam and drugs metabolized by cytochrome P450 isoenzymes[J]. Pharmacotherapy, 2012, 32: 340-353. DOI: 10.1002/j.1875-9114.2012.01028.x
|
[59] |
Hiemke C, Bergemann N, Clement HW, et al. Consensus Guidelines for Therapeutic Drug Monitoring in Neuropsychopharmacology: Update 2017[J]. Pharmacopsychiatry, 2018, 51: 9-62. DOI: 10.1055/s-0043-116492
|
[60] |
Rupp W, Badian M, Christ O, et al. Pharmacokinetics of single and multiple doses of clobazam in humans[J]. Br J Clin Pharmacol, 1979, 7: 51S-57S. DOI: 10.1111/j.1365-2125.1979.tb04665.x
|
[61] |
Patsalos PN, Berry DJ, Bourgeois BF, et al. Antiepileptic drugs--best practice guidelines for therapeutic drug monitoring: a position paper by the subcommission on therapeutic drug monitoring, ILAE Commission on Therapeutic Strategies[J]. Epilepsia, 2008, 49: 1239-1276. DOI: 10.1111/j.1528-1167.2008.01561.x
|
[62] |
Guberman A, Couture M, Blaschuk K, et al. Add-on trial of clobazam in intractable adult epilepsy with plasma level correlations[J]. Can J Neurol Sci, 1990, 17: 311-316. DOI: 10.1017/S031716710003064X
|
[63] |
Aylett SE, Cross H, Berry D. Eye rolling as a manifestation of clobazam toxicity in a child with epilepsy[J]. Dev Med Child Neurol, 2006, 48: 612-615. DOI: 10.1017/S0012162206001289
|
[64] |
Montenegro MA, Arif H, Nahm EA, et al. Efficacy of clobazam as add-on therapy for refractory epilepsy: experience at a US epilepsy center[J]. Clin Neuropharmacol, 2008, 31: 333-338. DOI: 10.1097/WNF.0b013e31815cd960
|
[65] |
Naccarato M, Yoong D, Kovacs C, et al. A case of a potential drug interaction between clobazam and etravirine-based antiretroviral therapy[J]. Antivir ther, 2012, 17: 589-592. DOI: 10.3851/IMP1953
|
[66] |
Parmeggiani A, Posar A, Sangiorgi S, et al. Unusual side-effects due to clobazam: a case report with genetic study of CYP2C19[J]. Brain Dev, 2004, 26: 63-66. DOI: 10.1016/S0387-7604(03)00074-3
|
[67] |
Pok PR, Mauras M, De Saint Léger MN, et al. Blood concentrations of clobazam and norclobazam in a lethal case involving clobazam, meprobamate and clorazepate[J]. Legal Med (Tokyo), 2010, 12: 300-304. DOI: 10.1016/j.legalmed.2010.08.002
|
[68] |
Proença P, Teixeira H, Pinheiro J, et al. Forensic intoxication with clobazam: HPLC/DAD/MSD analysis[J]. Forensic Sci Int, 2004, 143: 205-209. DOI: 10.1016/j.forsciint.2004.03.029
|
[69] |
Dean L. Clobazam Therapy and CYP2C19 Genotype[M]. US: National Center for Biotechnology Information, 2012.
|
[70] |
Johannessen Landmark C, Heger K, Lund C, et al. Pharmacokinetic Variability During Long-Term Therapeutic Drug Monitoring of Valproate, Clobazam, and Levetiracetam in Patients With Dravet Syndrome[J]. Ther Drug Monit, 2020, 42: 744-753. DOI: 10.1097/FTD.0000000000000781
|
[71] |
Burns ML, Baftiu A, Opdal MS, et al. Therapeutic Drug Monitoring of Clobazam and Its Metabolite-Impact of Age and Comedication on Pharmacokinetic Variability[J]. Ther Drug Monit, 2016, 38: 350-357. DOI: 10.1097/FTD.0000000000000272
|
[72] |
De Leon J, Spina E, Diaz FJ. Clobazam therapeutic drug monitoring: a comprehensive review of the literature with proposals to improve future studies[J]. Thera Drug Monit, 2013, 35: 30-47. DOI: 10.1097/FTD.0b013e31827ada88
|
[73] |
Spina E, Pisani F, de Leon J. Clinically significant pharmacokinetic drug interactions of antiepileptic drugs with new antidepressants and new antipsychotics[J]. Pharmacol Res, 2016, 106: 72-86. DOI: 10.1016/j.phrs.2016.02.014
|
[74] |
Anon. Thioridazine[J]. Tuberculosis (Edinb), 2008, 88: 164-167. DOI: 10.1016/S1472-9792(08)70028-3
|
[75] |
Pasupuleti B, Gone V, Baddam R, et al. Clinical Impact of Co-medication of Levetiracetam and Clobazam with Proton Pump Inhibitors: A Drug Interaction Study[J]. Curr Drug Metab, 2020, 21: 126-131. DOI: 10.2174/1389200221666200218121050
|
[76] |
Monjanel-Mouterde S, Antoni M, Bun H, et al. Pharmacokinetics of a single oral dose of clobazam in patients with liver disease[J]. Pharmacol Toxicol, 1994, 74: 345-350. DOI: 10.1111/j.1600-0773.1994.tb01371.x
|
[77] |
Tolbert D, Bekersky I, Chu HM, et al. An integrative population pharmacokinetics approach to the characterization of the effect of hepatic impairment on clobazam pharmacokine-tics[J]. J Clin Pharmacol, 2016, 56: 213-222. DOI: 10.1002/jcph.586
|
[78] |
Kamitaki BK, Minacapelli CD, Zhang P, et al. Drug-induced liver injury associated with antiseizure medications from the FDA Adverse Event Reporting System (FAERS)[J]. Epilepsy Behav, 2021, 117: 107832. DOI: 10.1016/j.yebeh.2021.107832
|
[79] |
Iqbal MM, Sobhan T, Ryals T. Effects of commonly used benzodiazepines on the fetus, the neonate, and the nursing infant[J]. Psychiatr Serv, 2002, 53: 39-49. DOI: 10.1176/appi.ps.53.1.39
|
[80] |
Noh Y, Lee H, Choi A, et al. First-trimester exposure to benzodiazepines and risk of congenital malformations in offspring: A population-based cohort study in South Korea[J]. PLoS Med, 2022, 19: e1003945. DOI: 10.1371/journal.pmed.1003945
|
[81] |
Suresh L, Radfar L. Pregnancy and lactation[J]. Oral Surg Oral Med Oral Pathol Oral Radiol Endod, 2004, 97: 672-682. DOI: 10.1016/j.tripleo.2004.02.002
|
[82] |
Greenblatt DJ, Divoll M, Puri SK, et al. Reduced single-dose clearance of clobazam in elderly men predicts increased multiple-dose accumulation[J]. Clin Pharmacokinet, 1983, 8: 83-94. DOI: 10.2165/00003088-198308010-00005
|
[83] |
Greenblatt DJ, Divoll M, Puri SK, et al. Clobazam kinetics in the elderly[J]. Br J Clin Pharmacol, 1981, 12: 631-636. DOI: 10.1111/j.1365-2125.1981.tb01281.x
|
[84] |
Tolbert D, Chu HM, Ette EI. Pharmacometrics of clobazam in pediatrics: Prediction of effective clobazam doses for Dravet syndrome[J]. Epilepsy Res, 2019, 157: 106182. DOI: 10.1016/j.eplepsyres.2019.106182
|
[85] |
Hahn J, Lee H, Kang HC, et al. Clobazam as an adjunctive treatment for infantile spasms[J]. Epilepsy Behav, 2019, 95: 161-165. DOI: 10.1016/j.yebeh.2019.03.040
|
[86] |
Mahmoud SH, Rans C. Systematic review of clobazam use in patients with status epilepticus[J]. Epilepsia Open, 2018, 3: 323-330. DOI: 10.1002/epi4.12230
|
[87] |
Redondo P, Vicente J, España A, et al. Photo-induced toxic epidermal necrolysis caused by clobazam[J]. Br J Dermatol, 1996, 135: 999-1002. DOI: 10.1046/j.1365-2133.1996.d01-1111.x
|
[88] |
Yapici AK, Fidanci MK, Kilic S, et al. Stevens-Johnson Syndrome triggered by a combination of clobazam, lamotrig-ine and valproic acid in a 7-year-old child[J]. Ann Burns Fire Disasters, 2014, 27: 121-125.
|
[89] |
Caramaschi P, Biasi D, Carletto A, et al. Clobazam-induced systemic lupus erythematosus[J]. Clin Rheumatol, 1995, 14: 116.
|
[90] |
US. Food & Durg. FDA Drug Safety Communication: FDA warns of serious skin reactions with the anti-seizure drug Onfi (clobazam) and has approved label changes[EB/OL]. (2016-01-15)[2022-08-11]. https://www.fda.gov/drugs/drug-safety-and-availability/fda-drug-safety-communication-fda-warns-serious-skin-reactions-anti-seizure-drug-onfi-cloba-zam-and#:~:text=%5B12-32013%5D%20The%20U.S.%20Food%20and%20Drug%20Administration%20%28FDA%29,describe%20the%20risk%20of%20these%20serious%20skin%20reactions.
|
[91] |
Chen B, Choi H, Hirsch LJ, et al. Psychiatric and behavioral side effects of antiepileptic drugs in adults with epilepsy[J]. Epilepsy Behav, 2017, 76: 24-231. DOI: 10.1016/j.yebeh.2017.08.039
|
[92] |
Kalra V, Seth R, Mishra D, et al. Clobazam in refractory childhood epilepsy[J]. Indian J Pediatr, 2010, 77: 263-266. DOI: 10.1007/s12098-010-0035-z
|
[93] |
Gedela S, Freedman DA, Gedela S, et al. Safety and Efficacy of Supratherapeutic Doses of Clobazam[J]. J Child Neurol, 2019, 34: 735-738. DOI: 10.1177/0883073819856834
|
[94] |
Schmidt D. Clobazam for treatment of intractable epilepsy: a critical assessment[J]. Epilepsia, 1994, 35: S92-S95. DOI: 10.1111/j.1528-1157.1994.tb05979.x
|
[1] | LIU Qingyang, LIU Xin, WANG Shaohong, SHANG Junmei, TANG Yan, ZHANG Bo. Research of Accessibility of Rare Disease Drugs Based on the China's First List of Rare Diseases[J]. Medical Journal of Peking Union Medical College Hospital, 2023, 14(6): 1208-1216. DOI: 10.12290/xhyxzz.2023-0163 |
[2] | ZHANG Lu, LI Jian. Castleman Disease in China: State-of-the-art Technology Before the Era of IL-6 Targeted Therapy[J]. Medical Journal of Peking Union Medical College Hospital, 2023, 14(5): 911-914. DOI: 10.12290/xhyxzz.2023-0227 |
[3] | ZHOU Qi, LI Qinyuan, LIU Yali, LUO Zhengxiu, ZHANG Weishe, CHEN Tong, LI Guobao, SHANG Hongcai, YANG Kehu, ZHANG Bo, CHEN Yaolong, ZHANG Shuyang. The Development of Guidelines for Rare Diseases: Past, Present and Future[J]. Medical Journal of Peking Union Medical College Hospital, 2023, 14(3): 621-628. DOI: 10.12290/xhyxzz.2022-0360 |
[4] | CHEN Chen, HAN Xiaohong. Clinical Trials of Rare Diseases in China over One Decade: Based on the Chinese First List of Rare Diseases[J]. Medical Journal of Peking Union Medical College Hospital, 2022, 13(6): 1028-1035. DOI: 10.12290/xhyxzz.2022-0010 |
[5] | Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital, National Rare Diseases Committee. Expert Consensus on the Application of Multi-criteria Decision Analysis in Clinical Comprehensive Evaluation of Orphan Drugs(2022)[J]. Medical Journal of Peking Union Medical College Hospital, 2022, 13(2): 235-254. DOI: 10.12290/xhyxzz.2022-0013 |
[6] | Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital. Consensus on the Treatment of Adrenocortical Carcinoma with Mitotane(2021)[J]. Medical Journal of Peking Union Medical College Hospital, 2021, 12(5): 674-683. DOI: 10.12290/xhyxzz.2021-0554 |
[7] | YU Songlin, WANG Danchen, ZOU Yutong, MA Xiaoli, QIU Ling. The Clinical Application of Liquid Chromatography-Tandem Mass Spectrometry in the Diagnosis of Rare Diseases[J]. Medical Journal of Peking Union Medical College Hospital, 2021, 12(4): 450-455. DOI: 10.12290/xhyxzz.2021-0324 |
[8] | Liang SHI, Ya-zhou CUI, Jin-xiang HAN. Models of Rare Diseases Based on Induced Pluripotent Stem Cells and Their Applications[J]. Medical Journal of Peking Union Medical College Hospital, 2018, 9(3): 261-270. DOI: 10.3969/j.issn.1674-9081.2018.03.014 |
[9] | Zhaoyun Jiang, Yulan lU, Le Yu, Mengchun Gong, Wenzhao Shi, Shuyang Zhang, Wenhao Zhou. Progress and Application of Medical Informatics in the Diagnosis and Treatment of Rare Diseases[J]. Medical Journal of Peking Union Medical College Hospital, 2018, 9(2): 165-171. DOI: 10.3969/j.issn.1674-9081.2018.02.012 |
[10] | Hao-peng XU, Chong ZHU, Meng-chun GONG, Shu-yang ZHANG. Research of Rare Diseases in China: from the Past to the Future[J]. Medical Journal of Peking Union Medical College Hospital, 2018, 9(1): 5-9. DOI: 10.3969/j.issn.1674-9081.2018.01.002 |