{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,25]],"date-time":"2026-10-25T03:48:47Z","timestamp":1792900127357,"version":"4.5.0"},"reference-count":37,"publisher":"Massachusetts Medical Society","issue":"21","license":[{"start":{"date-parts":[[2025,11,27]],"date-time":"2025-11-27T00:00:00Z","timestamp":1764201600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.nejmgroup.org\/legal\/terms-of-use.htm"}],"funder":[{"name":"Vaderis Therapeutics"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["N Engl J Med"],"published-print":{"date-parts":[[2025,11,27]]},"DOI":"10.1056\/nejmoa2504411","type":"journal-article","created":{"date-parts":[[2025,11,26]],"date-time":"2025-11-26T22:00:32Z","timestamp":1764194432000},"page":"2131-2141","source":"Crossref","is-referenced-by-count":13,"title":["Engasertib versus Placebo for Bleeding in Hereditary Hemorrhagic Telangiectasia"],"prefix":"10.1056","volume":"393","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6175-1383","authenticated-orcid":false,"given":"Hanny","family":"Al-Samkari","sequence":"first","affiliation":[{"name":"Division of Hematology\u2013Oncology, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Josefien","family":"Hessels","sequence":"additional","affiliation":[{"name":"Department of Pulmonology, St. Antonius Hospital, Nieuwegein, the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9411-804X","authenticated-orcid":false,"given":"Antoni","family":"Riera-Mestre","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Hospital Universitari de Bellvitge\u2013Institut d\u2019Investigaci\u00f3 Biom\u00e8dica de Bellvitge, L\u2019Hospitalet de Llobregat, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Dupuis-Girod","sequence":"additional","affiliation":[{"name":"Genetic Department, H\u00f4pital Femme\u2013M\u00e8re\u2013Enfant, Hospices Civils de Lyon, Bron, France"},{"name":"Biosant\u00e9 Unit 1292, Grenoble Alpes University, INSERM, Commissariat \u00e0 L\u2019\u00c9nergie Atomique et aux \u00c9nergies Alternatives, Grenoble, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thibaut","family":"Van Zele","sequence":"additional","affiliation":[{"name":"Department of Otorhinolaryngology, Ghent University Hospital, Ghent, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vicente","family":"G\u00f3mez del Olmo","sequence":"additional","affiliation":[{"name":"Internal Medicine Department, Ram\u00f3n y Cajal University Hospital, Instituto Ram\u00f3n y Cajal de Investigaci\u00f3n Sanitaria, Madrid"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pamela G.","family":"Hodges","sequence":"additional","affiliation":[{"name":"Division of Hematology\u2013Oncology, Massachusetts General Hospital, Harvard Medical School, Boston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raquel","family":"Torres-Iglesias","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Hospital Universitari de Bellvitge\u2013Institut d\u2019Investigaci\u00f3 Biom\u00e8dica de Bellvitge, L\u2019Hospitalet de Llobregat, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto","family":"Bert\u00e8","sequence":"additional","affiliation":[{"name":"Gastroenterology Department, Azienda Socio-Sanitaria Territoriale, Ospedale Maggiore Crema, Crema, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Saint-Mezard","sequence":"additional","affiliation":[{"name":"Vaderis Therapeutics, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hedvika","family":"Lazar","sequence":"additional","affiliation":[{"name":"Vaderis Therapeutics, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicholas","family":"Benedict","sequence":"additional","affiliation":[{"name":"Vaderis Therapeutics, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Debra","family":"Barker","sequence":"additional","affiliation":[{"name":"Vaderis Therapeutics, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1019-5468","authenticated-orcid":false,"given":"Corrado","family":"Bernasconi","sequence":"additional","affiliation":[{"name":"Vaderis Therapeutics, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Damien","family":"Picard","sequence":"additional","affiliation":[{"name":"Vaderis Therapeutics, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elisabetta","family":"Buscarini","sequence":"additional","affiliation":[{"name":"Gastroenterology Department, Azienda Socio-Sanitaria Territoriale, Ospedale Maggiore Crema, Crema, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hans-Jurgen","family":"Mager","sequence":"additional","affiliation":[{"name":"Department of Pulmonology, St. Antonius Hospital, Nieuwegein, the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"150","reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.3389\/fgene.2014.00456"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1017\/cjn.2018.360"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-024-00585-z"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000001538"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1136\/jnis-2022-019162"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2023011961"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2018.193003"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1288\/00005537-199109000-00008"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.7326\/M20-1443"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.thromres.2017.03.003"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.1186\/s12955-017-0586-z"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1572-0241.2000.01792.x"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.24832"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2023021765"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejmg.2021.104370"},{"issue":"3","key":"e_1_3_5_17_2","first-page":"djv342","article-title":"Maintenance therapy with immunomodulatory drugs in multiple myeloma: a meta-analysis and systematic review.","volume":"108","author":"Wang Y","year":"2015","unstructured":"Wang Y, Yang F, Shen Y, et al. Maintenance therapy with immunomodulatory drugs in multiple myeloma: a meta-analysis and systematic review. J Natl Cancer Inst 2015;108(3):djv342-djv342.26582244","journal-title":"J Natl Cancer Inst"},{"key":"e_1_3_5_18_2","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15354"},{"key":"e_1_3_5_19_2","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCULATIONAHA.118.033842"},{"key":"e_1_3_5_20_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI176379"},{"key":"e_1_3_5_21_2","doi-asserted-by":"publisher","DOI":"10.1002\/(SICI)1096-8628(20000306)91:1<66::AID-AJMG12>3.0.CO;2-P"},{"key":"e_1_3_5_22_2","doi-asserted-by":"publisher","DOI":"10.1002\/lary.20818"},{"key":"e_1_3_5_23_2","unstructured":"National Cancer Institute. Common Terminology Criteria for Adverse Events (CTCAE) (https:\/\/ctep.cancer.gov\/protocoldevelopment\/electronic_applications\/docs\/CTCAE_v5_Quick_Reference_5x7.pdf)."},{"key":"e_1_3_5_24_2","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2009.25.3641"},{"key":"e_1_3_5_25_2","unstructured":"Thalgott J Pollenus T Goujon GPE et al. An evaluation of the efficacy and safety of AKT inhibitors in the prevention of hereditary hemorrhagic telangiectasia. Angiogenesis 2023;26:Suppl 1:3-4. abstract."},{"key":"e_1_3_5_26_2","doi-asserted-by":"publisher","DOI":"10.1161\/ATVBAHA.118.310760"},{"key":"e_1_3_5_27_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2214131"},{"key":"e_1_3_5_28_2","unstructured":"Novartis. Piqray (alpelisib): highlights of prescribing information. January 2024 (https:\/\/www.novartis.com\/us-en\/sites\/novartis_us\/files\/piqray.pdf)."},{"key":"e_1_3_5_29_2","unstructured":"AstraZeneca. Truqap (capivasertib): highlights of prescribing information. February 2025 (https:\/\/drd9vrdh9yh09.cloudfront.net\/50fd68b9-106b-4550-b5d0-12b045f8b184\/841065f2-fcba-4795-b92a-3afc2ba47325\/841065f2-fcba-4795-b92a-3afc2ba47325_viewable_rendition__v.pdf)."},{"key":"e_1_3_5_30_2","doi-asserted-by":"publisher","DOI":"10.3390\/genes12020174"},{"key":"e_1_3_5_31_2","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12654"},{"key":"e_1_3_5_32_2","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2020.261859"},{"key":"e_1_3_5_33_2","doi-asserted-by":"publisher","DOI":"10.1111\/joim.13714"},{"key":"e_1_3_5_34_2","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2012.250"},{"key":"e_1_3_5_35_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2312749"},{"key":"e_1_3_5_36_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(15)00195-7"},{"key":"e_1_3_5_37_2","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14034"},{"key":"e_1_3_5_38_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-018-0982-4"}],"container-title":["New England Journal of Medicine"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.nejm.org\/doi\/pdf\/10.1056\/NEJMoa2504411","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,26]],"date-time":"2025-11-26T22:00:46Z","timestamp":1764194446000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.nejm.org\/doi\/10.1056\/NEJMoa2504411"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,11,27]]},"references-count":37,"journal-issue":{"issue":"21","published-print":{"date-parts":[[2025,11,27]]}},"alternative-id":["10.1056\/NEJMoa2504411"],"URL":"https:\/\/doi.org\/10.1056\/nejmoa2504411","relation":{},"ISSN":["0028-4793","1533-4406"],"issn-type":[{"value":"0028-4793","type":"print"},{"value":"1533-4406","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,11,27]]}}}