{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T23:59:40Z","timestamp":1791417580148,"version":"4.3.3"},"reference-count":9,"publisher":"American Academy of Pediatrics (AAP)","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2013,9,1]]},"abstract":"<jats:p>MYH9-related disease (MYH9-RD) is one of the most frequent autosomal-dominant forms of inherited macrothrombocytopenias and is caused by mutations in MYH9 (nonmuscle myosin IIA), the gene coding for the heavy chain of the nonmuscle myosin IIA. Affected individuals can present with isolated thrombocytopenia, and whereas only some will have bleeding events requiring intervention, nearly all will require the use of prophylactic platelet transfusions before surgery. Here we report the first prophylactic use of eltrombopag before surgery in a child with MYH9-RD. Our patient was a 13-year-old girl with an MYH9 S96L missense mutation who required a tympanoplasty due to chronic otitis media. Pretreatment microscopic platelet count was 10 \u00d7 109\/L. The child was treated with eltrombopag starting 4 weeks before her planned surgery. On the day of surgery her platelet count was 70 \u00d7 109\/L. She required no platelet transfusions and no abnormal bleeding was reported either during surgery or postoperatively. Given these results, the first reported in a child, we suggest that the use of this thrombopoietic agent should be further evaluated as a useful presurgical prophylactic option in this hereditary thrombocytopenia, thus avoiding the use of platelet transfusions and their associated risks, which include alloimmunization and the transmission of infectious agents.<\/jats:p>","DOI":"10.1542\/peds.2012-3807","type":"journal-article","created":{"date-parts":[[2013,8,13]],"date-time":"2013-08-13T00:25:48Z","timestamp":1376353548000},"page":"e793-e795","source":"Crossref","is-referenced-by-count":46,"title":["First Successful Use of Eltrombopag Before Surgery in a Child With MYH9-Related Thrombocytopenia"],"prefix":"10.1542","volume":"132","author":[{"given":"Remi","family":"Favier","sequence":"first","affiliation":[{"name":"Assistance Publique-H\u00f4pitaux de Paris, Armand Trousseau Children\u2019s Hospital, French Reference Centre for Inherited Platelet Disorders, Paris, France;"},{"name":"INSERM U1009, Villejuif, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joffrey","family":"Feriel","sequence":"additional","affiliation":[{"name":"Assistance Publique-H\u00f4pitaux de Paris, Armand Trousseau Children\u2019s Hospital, French Reference Centre for Inherited Platelet Disorders, Paris, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie","family":"Favier","sequence":"additional","affiliation":[{"name":"Department of Biopathology, GHPSO, Senlis, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fran\u00e7oise","family":"Denoyelle","sequence":"additional","affiliation":[{"name":"Department of Pediatric Otolaryngology and Head and Neck Surgery, Armand Trousseau Children\u2019s Hospital, Paris, France; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John A.","family":"Martignetti","sequence":"additional","affiliation":[{"name":"Departments of Genetics and Genomic Sciences, Pediatrics and Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, New York"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"417","published-online":{"date-parts":[[2013,9,1]]},"reference":[{"issue":"1","key":"2021120902580880800_B1","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1038\/79063","article-title":"Mutations in MYH9 result in the May-Hegglin anomaly, and Fechtner and Sebastian syndromes.","volume":"26","author":"Seri","year":"2000","journal-title":"Nat Genet"},{"issue":"2","key":"2021120902580880800_B2","doi-asserted-by":"crossref","first-page":"189","DOI":"10.1055\/s-0029-1220327","article-title":"MYH9-related platelet disorders.","volume":"35","author":"Althaus","year":"2009","journal-title":"Semin Thromb Hemost"},{"issue":"3","key":"2021120902580880800_B3","doi-asserted-by":"crossref","first-page":"409","DOI":"10.1002\/humu.20661","article-title":"Position of nonmuscle myosin heavy chain IIA (NMMHC-IIA) mutations predicts the natural history of MYH9-related disease.","volume":"29","author":"Pecci","year":"2008","journal-title":"Hum Mutat"},{"issue":"3","key":"2021120902580880800_B4","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1053\/j.seminhematol.2010.02.005","article-title":"Biology and chemistry of thrombopoietic agents.","volume":"47","author":"Kuter","year":"2010","journal-title":"Semin Hematol"},{"issue":"26","key":"2021120902580880800_B5","doi-asserted-by":"crossref","first-page":"5832","DOI":"10.1182\/blood-2010-08-304725","article-title":"Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations.","volume":"116","author":"Pecci","year":"2010","journal-title":"Blood"},{"issue":"6","key":"2021120902580880800_B6","doi-asserted-by":"crossref","first-page":"1188","DOI":"10.1160\/TH12-01-0005","article-title":"Short-term eltrombopag for surgical preparation of a patient with inherited thrombocytopenia deriving from MYH9 mutation.","volume":"107","author":"Pecci","year":"2012","journal-title":"Thromb Haemost"},{"issue":"9664","key":"2021120902580880800_B7","doi-asserted-by":"crossref","first-page":"641","DOI":"10.1016\/S0140-6736(09)60402-5","article-title":"Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial.","volume":"373","author":"Bussel","year":"2009","journal-title":"Lancet"},{"issue":"9763","key":"2021120902580880800_B8","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1016\/S0140-6736(10)60959-2","article-title":"Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study.","volume":"377","author":"Cheng","year":"2011","journal-title":"Lancet"},{"issue":"9","key":"2021120902580880800_B9","doi-asserted-by":"crossref","first-page":"e33","DOI":"10.3324\/haematol.2011.048819","article-title":"The European Medicines Agency review of eltrombopag (Revolade) for the treatment of adult chronic immune (idiopathic) thrombocytopenic purpura: summary of the scientific assessment of the Committee for Medicinal Products for Human Use.","volume":"96","author":"Nieto","year":"2011","journal-title":"Haematologica"}],"container-title":["Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/publications.aap.org\/pediatrics\/article-pdf\/132\/3\/e793\/1091053\/peds_2012-3807.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/publications.aap.org\/pediatrics\/article-pdf\/132\/3\/e793\/1091053\/peds_2012-3807.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,12,9]],"date-time":"2021-12-09T16:20:25Z","timestamp":1639066825000},"score":1,"resource":{"primary":{"URL":"https:\/\/publications.aap.org\/pediatrics\/article\/132\/3\/e793\/31603\/First-Successful-Use-of-Eltrombopag-Before-Surgery"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,9,1]]},"references-count":9,"journal-issue":{"issue":"3","published-online":{"date-parts":[[2013,9,1]]},"published-print":{"date-parts":[[2013,9,1]]}},"URL":"https:\/\/doi.org\/10.1542\/peds.2012-3807","relation":{},"ISSN":["0031-4005","1098-4275"],"issn-type":[{"value":"0031-4005","type":"print"},{"value":"1098-4275","type":"electronic"}],"subject":[],"published":{"date-parts":[[2013,9,1]]}}}