New England Journal of Medicine最新文献

{"title":"On-Table Reanimation of a Pediatric Heart from Donation after Circulatory Death.","authors":"John A Kucera,Douglas M Overbey,Joseph W Turek","doi":"10.1056/nejmoa2503487","DOIUrl":"https://doi.org/10.1056/nejmoa2503487","url":null,"abstract":"Cardiac allotransplantation is warranted in children with end-stage heart failure or irreparable congenital heart disease. A dearth of organs for transplantation has contributed to long wait-list times and resultant deaths in this population. Donation after circulatory death (DCD) with normothermic regional perfusion has the potential to increase the donor pool by up to 30%. Ethical concerns have limited adoption of this technique in the United States and abroad. Consequently, a method facilitating ex vivo resuscitation of hearts from deceased donors after circulatory death (i.e., DCD hearts) is needed. We describe an on-table reanimation of a pediatric DCD heart transplanted into a 3-month-old recipient. Adverse events are reported.","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"7 1","pages":"275-280"},"PeriodicalIF":158.5,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645954","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multidose Ondansetron after Emergency Visits in Children with Gastroenteritis.","authors":"Stephen B Freedman,Sarah Williamson-Urquhart,Amy C Plint,Andrew Dixon,Darcy Beer,Gary Joubert,Petros Pechlivanoglou,Yaron Finkelstein,Anna Heath,Jasper Zhongyuan Zhang,Angela Wallace,Martin Offringa,Terry P Klassen, ","doi":"10.1056/nejmoa2503596","DOIUrl":"https://doi.org/10.1056/nejmoa2503596","url":null,"abstract":"BACKGROUNDOndansetron improves outcomes when administered in emergency departments to children with acute gastroenteritis-associated vomiting. It is commonly prescribed at discharge to reduce symptoms, but evidence to support this practice is limited.METHODSWe conducted a double-blind, randomized superiority trial involving children 6 months to less than 18 years of age with acute gastroenteritis-associated vomiting in six pediatric emergency departments. Caregivers were provided with six doses of oral ondansetron or placebo to administer in response to ongoing vomiting during the first 48 hours after enrollment. The primary outcome was moderate-to-severe gastroenteritis, defined by a score of 9 or higher on the modified Vesikari scale (scores range from 0 to 20, with higher scores indicating greater severity), during the 7 days after enrollment. Secondary outcomes included the presence of vomiting, the duration of vomiting (defined as the time from enrollment to the last vomiting episode), the number of vomiting episodes within 48 hours after enrollment, unscheduled physician visits within 7 days after enrollment, and receipt of intravenous fluids.RESULTSA total of 1030 children underwent randomization. Moderate-to-severe gastroenteritis occurred in 5.1% (23 of 452 participants for whom data were available) in the ondansetron group and 12.5% (55 of 441) in the placebo group (unadjusted risk difference, -7.4 percentage points; 95% confidence interval [CI], -11.2 to -3.7). After adjustment for site, weight, and missing data, ondansetron was associated with a lower risk of moderate-to-severe gastroenteritis than placebo (adjusted odds ratio, 0.50; 95% CI, 0.40 to 0.60). Although we did not observe any meaningful difference between the groups in the presence or median duration of vomiting, the total number of vomiting episodes within 48 hours after enrollment was lower with ondansetron than with placebo (adjusted rate ratio, 0.76; 95% CI, 0.67 to 0.87). The percentage of children who had unscheduled health care visits and the percentage who received intravenous fluids after enrollment did not differ substantially between the groups. The incidence of adverse events also did not differ meaningfully between the groups (odds ratio, 0.99; 95% CI, 0.61 to 1.61).CONCLUSIONSAmong children with gastroenteritis-associated vomiting, the provision of ondansetron after an emergency department visit led to lower risk of moderate-to-severe gastroenteritis during the subsequent 7 days than the provision of placebo. (Funded by the Canadian Institutes of Health Research and others; ClinicalTrials.gov number, NCT03851835.).","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"7 1","pages":"255-266"},"PeriodicalIF":158.5,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645957","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Monoclonal Anti-Platelet Factor 4 Antibodies in Recurrent Pregnancy Loss.","authors":"Natalie R Bavli,Adam J Kanack,Yoshito Nishimura,Noah P Splinter,Emily E Mauch,Alessandra J Ainsworth,Ravi Sarode,Robert D McBane,Mindy C Kohlhagen,David Murray,Dong Chen,Rajiv K Pruthi,Anand Padmanabhan","doi":"10.1056/nejmc2506014","DOIUrl":"https://doi.org/10.1056/nejmc2506014","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"15 1","pages":"306-309"},"PeriodicalIF":158.5,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645955","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Apixaban for Cancer-Associated Venous Thromboembolism.","authors":"Gujie Wu, Weiwei Liu, Lee Sheung","doi":"10.1056/NEJMc2506526","DOIUrl":"10.1056/NEJMc2506526","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"393 3","pages":"310"},"PeriodicalIF":96.2,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144651225","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Apixaban for Cancer-Associated Venous Thromboembolism.","authors":"Vincenza Conteduca,Guido Giordano,Matteo Landriscina","doi":"10.1056/nejmc2506526","DOIUrl":"https://doi.org/10.1056/nejmc2506526","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"37 1","pages":"310-311"},"PeriodicalIF":158.5,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645953","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Advances in Preventing Transmission of Mitochondrial DNA Diseases.","authors":"Julie Steffann","doi":"10.1056/nejme2507755","DOIUrl":"https://doi.org/10.1056/nejme2507755","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"17 1","pages":""},"PeriodicalIF":158.5,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mitochondrial Donation in a Reproductive Care Pathway for mtDNA Disease.","authors":"Robert McFarland, Louise A Hyslop, Catherine Feeney, Rekha N Pillai, Emma L Blakely, Eilis Moody, Matthew Prior, Anita Devlin, Robert W Taylor, Mary Herbert, Meenakshi Choudhary, Jane A Stewart, Douglass M Turnbull","doi":"10.1056/NEJMoa2503658","DOIUrl":"https://doi.org/10.1056/NEJMoa2503658","url":null,"abstract":"<p><p>Pathogenic variants in mitochondrial DNA (mtDNA) are a common cause of severe, often fatal, inherited metabolic disease. A reproductive care pathway was implemented to provide women carrying pathogenic mtDNA variants with reproductive options. A total of 22 women with pathogenic mtDNA variants have commenced or completed pronuclear transfer (and thus receipt of a mitochondrial donation), and there have been 8 live births. All 8 children were healthy at birth, with no or low levels of mtDNA heteroplasmy in blood. Hyperlipidemia and cardiac arrhythmia developed in a child whose mother had hyperlipidemia during pregnancy; both of the child's conditions responded to treatment. Infant myoclonic epilepsy developed in another child, with spontaneous remission. At the time of this report, all the children have made normal developmental progress. (Funded by the U.K. National Health Service and others.).</p>","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":" ","pages":""},"PeriodicalIF":96.2,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144676573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mitochondrial Donation and Preimplantation Genetic Testing for mtDNA Disease.","authors":"Louise A Hyslop,Emma L Blakely,Magomet Aushev,Jordan Marley,Yuko Takeda,Angela Pyle,Eilis Moody,Catherine Feeney,Jan Dutton,Carol Shaw,Sarah J Smith,Kate Craig,Charlotte L Alston,Lisa Lister,Karina Endacott,Samantha Byerley,Helen McDermott,Kathryn Wilson,Lynne Botham,Beth Matthew,Nilendran Prathalingam,Matthew Prior,Alison Murdoch,Douglass M Turnbull,Gavin Hudson,Meenakshi Choudhary,Robert W Taylor,Rekha N Pillai,Jane A Stewart,Robert McFarland,Mary Herbert","doi":"10.1056/nejmoa2415539","DOIUrl":"https://doi.org/10.1056/nejmoa2415539","url":null,"abstract":"BACKGROUNDChildren born to women who carry pathogenic variants in mitochondrial DNA (mtDNA) are at risk for a range of clinical syndromes collectively known as mtDNA disease. Mitochondrial donation by pronuclear transfer involves transplantation of nuclear genome from a fertilized egg from the affected woman to an enucleated fertilized egg donated by an unaffected woman. Thus, pronuclear transfer offers affected women the potential to have a genetically related child with a reduced risk of mtDNA disease.METHODSWe offered mitochondrial donation (by pronuclear transfer) or preimplantation genetic testing (PGT) to a series of women with pathogenic mtDNA variants who sought to reduce the transmission of these variants to their children. Patients with heteroplasmy (variants present in a proportion of copies of mtDNA) were offered PGT, and patients with homoplasmy (variants present in all copies of mtDNA) or elevated heteroplasmy were offered pronuclear transfer.RESULTSClinical pregnancies were confirmed in 8 of 22 patients (36%) and 16 of 39 patients (41%) who underwent an intracytoplasmic sperm injection procedure for pronuclear transfer or for PGT, respectively. Pronuclear transfer resulted in 8 live births and 1 ongoing pregnancy. PGT resulted in 18 live births. Heteroplasmy levels in the blood of the 8 infants whose mothers underwent pronuclear transfer ranged from undetectable to 16%. Levels of the maternal pathogenic mtDNA variant were 95 to 100% lower in 6 newborns and 77 to 88% lower in 2 newborns than in the corresponding enucleated zygotes. Heteroplasmy levels were known for 10 of the 18 infants whose mothers underwent PGT and ranged from undetectable to 7%.CONCLUSIONSWe found that mitochondrial donation through pronuclear transfer was compatible with human embryo viability. An integrated program involving pronuclear transfer and PGT was effective in reducing the transmission of homoplasmic and heteroplasmic pathogenic mtDNA variants. (Funded by NHS England and others.).","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"9 1","pages":""},"PeriodicalIF":158.5,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645962","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Brain Death in Pregnancy - Abortion, Advance-Directive, or End-of-Life Law?","authors":"Katie Watson","doi":"10.1056/nejmp2507544","DOIUrl":"https://doi.org/10.1056/nejmp2507544","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"13 1","pages":""},"PeriodicalIF":158.5,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645964","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reducing the Risks of Mitochondrial Disease in Children.","authors":"Robin Lovell-Badge","doi":"10.1056/nejme2507753","DOIUrl":"https://doi.org/10.1056/nejme2507753","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"15 1","pages":""},"PeriodicalIF":158.5,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645963","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}