{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,26]],"date-time":"2026-02-26T15:10:42Z","timestamp":1772118642081,"version":"3.50.1"},"reference-count":23,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2022,8,29]],"date-time":"2022-08-29T00:00:00Z","timestamp":1661731200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Hematological Oncology"],"published-print":{"date-parts":[[2022,12]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Advanced Systemic Mastocytosis (Adv\u2010SM) is rare and has a poor prognosis. Midostaurin (Rydapt\n                    <jats:sup>\u00ae<\/jats:sup>\n                    ) is one of the few treatments for Adv\u2010SM in Europe. The study aims were to describe the characteristics of patients treated with midostaurin, their treatment modalities, outcomes, and serious events requiring hospitalization. This retrospective observational study was conducted using the French National Healthcare Database (SNDS) and included adult Adv\u2010SM patients treated with midostaurin between 01\u201001\u20102012 and 09\u201030\u20102018. Kaplan\u2010Meier method was used to assess survival and treatment duration. Eighty\u2010one patients were included: 37 with Aggressive Systemic Mastocytosis (SM) (46%), 31 with SM with an Associated Hematological Neoplasm (38%), and 4 with Mast Cell Leukemia (5%). The SM subtype was undetermined in 9 patients (11%). The median age was 67\u00a0years; 64% of patients were male. Over the mean follow\u2010up of 11.4\u00a0months, median midostaurin treatment duration was 8.4\u00a0months and 28 patients (35%) were still under treatment at the end of the study. One\u2010year and 5\u2010year overall survival rates estimated since the time of diagnosis were 83% and 56%, respectively. Twelve serious events (among those frequently reported during clinical trials and compassionate use) requiring hospitalization were identified; a causal association with Adv\u2010SM treatment could neither be excluded nor established. In this first real\u2010life study on patients treated with midostaurin for Adv\u2010SM, the patients' characteristics, their management, treatment discontinuation, and survival were in line with previous results (compassionate use and clinical trials).\n                  <\/jats:p>","DOI":"10.1002\/hon.3062","type":"journal-article","created":{"date-parts":[[2022,8,11]],"date-time":"2022-08-11T01:07:06Z","timestamp":1660180026000},"page":"1030-1040","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Therapeutic management and outcome of patients with advanced systemic mastocytosis treated with midostaurin: A\u00a0comprehensive real\u2010life study in the French national healthcare database"],"prefix":"10.1002","volume":"40","author":[{"given":"Julien","family":"Rossignol","sequence":"first","affiliation":[{"name":"CEREMAST H\u00f4pital Necker\u2010Enfants Malades  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sandra","family":"Nizard","sequence":"additional","affiliation":[{"name":"Novartis Pharma S.A.S  Rueil Malmaison France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anne\u2010Sandrine","family":"Blanc","sequence":"additional","affiliation":[{"name":"Novartis Pharma S.A.S  Rueil Malmaison France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anne","family":"Filipovics","sequence":"additional","affiliation":[{"name":"Novartis Pharma S.A.S  Rueil Malmaison France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8816-7475","authenticated-orcid":false,"given":"Joannie","family":"Lortet\u2010Tieulent","sequence":"additional","affiliation":[{"name":"HEVA  Lyon France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hassiba","family":"Bouktit","sequence":"additional","affiliation":[{"name":"CEREMAST H\u00f4pital Necker\u2010Enfants Malades  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gwendoline","family":"Poinsot","sequence":"additional","affiliation":[{"name":"HEVA  Lyon France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Aur\u00e9lie","family":"Schmidt","sequence":"additional","affiliation":[{"name":"HEVA  Lyon France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fanny","family":"Raguideau","sequence":"additional","affiliation":[{"name":"HEVA  Lyon France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Olivier","family":"Hermine","sequence":"additional","affiliation":[{"name":"CEREMAST H\u00f4pital Necker\u2010Enfants Malades  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2022,8,29]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.12916"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1097\/md.0000000000001414"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102016\u201003\u2010643544"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.2147\/blctt.s87186"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.iac.2014.01.003"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jid.2020.12.030"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaip.2020.05.005"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1136\/esmoopen\u20102019\u2010000606"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/pds.4233"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.respe.2017.05.004"},{"key":"e_1_2_13_12_1","unstructured":"Centre des liaisons europ\u00e9ennes et internationales de s\u00e9curit\u00e9 sociale R\u00e9publique Fran\u00e7aise. The French Social Security System. I\u2010Health maternity paternity disability and death [Internet]. Vous informer sur la protection sociale \u00e0 l\u2019international.2020[cited 2020 Oct 12].https:\/\/www.cleiss.fr\/docs\/regimes\/regime_france\/an_1.html"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1186\/1471\u20102458\u20109\u201033"},{"key":"e_1_2_13_14_1","unstructured":"ameli.fr \u2010 Cartographie des pathologies et des d\u00e9penses [Internet].2019[cited 2019 Jun 12].https:\/\/www.ameli.fr\/l\u2010assurance\u2010maladie\/statistiques\u2010et\u2010publications\/etudes\u2010en\u2010sante\u2010publique\/cartographie\u2010des\u2010pathologies\u2010et\u2010des\u2010depenses\/index.php"},{"key":"e_1_2_13_15_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102012\u201009\u2010458521"},{"key":"e_1_2_13_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.24490"},{"key":"e_1_2_13_17_1","doi-asserted-by":"publisher","DOI":"10.7150\/thno.51872"},{"key":"e_1_2_13_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.24382"},{"key":"e_1_2_13_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaip.2018.09.024"},{"key":"e_1_2_13_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25371"},{"key":"e_1_2_13_21_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1513098"},{"key":"e_1_2_13_22_1","doi-asserted-by":"publisher","DOI":"10.1056\/nejmc1515403"},{"key":"e_1_2_13_23_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102009\u201002\u2010205237"},{"key":"e_1_2_13_24_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102014\u201012\u2010614743"}],"container-title":["Hematological Oncology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/hon.3062","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/hon.3062","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/hon.3062","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,19]],"date-time":"2023-08-19T10:10:13Z","timestamp":1692439813000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/hon.3062"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,8,29]]},"references-count":23,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2022,12]]}},"alternative-id":["10.1002\/hon.3062"],"URL":"https:\/\/doi.org\/10.1002\/hon.3062","archive":["Portico"],"relation":{"has-review":[{"id-type":"doi","id":"10.1002\/HON.3062\/v1\/decision1","asserted-by":"object"},{"id-type":"doi","id":"10.1002\/HON.3062\/v2\/decision1","asserted-by":"object"},{"id-type":"doi","id":"10.1002\/HON.3062\/v1\/review1","asserted-by":"object"}]},"ISSN":["0278-0232","1099-1069"],"issn-type":[{"value":"0278-0232","type":"print"},{"value":"1099-1069","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,8,29]]},"assertion":[{"value":"2022-02-22","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-07-30","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-08-29","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}