{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,4]],"date-time":"2026-03-04T16:47:36Z","timestamp":1772642856001,"version":"3.50.1"},"reference-count":15,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2017,6,23]],"date-time":"2017-06-23T00:00:00Z","timestamp":1498176000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2017,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Wiskott\u2013Aldrich syndrome (WAS) is an inherited X\u2010linked disorder characterized by microthrombocytopenia, immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) is the treatment of choice. Eltrombopag, a thrombopoietin receptor agonist, may be useful to prevent bleeding while awaiting HSCT. We present a case of a male with WAS, profound thrombocytopenia, and bleeding diathesis successfully managed with eltrombopag before HSCT. Eltrombopag was given for 32 weeks obtaining a stable platelet count without any platelet transfusion. The patient did not experience any bleeding symptom. Eltrombopag may be a suitable therapeutic option for patients with WAS and severe thrombocytopenia as \u201cbridge\u201d to definitive cure.<\/jats:p>","DOI":"10.1002\/pbc.26692","type":"journal-article","created":{"date-parts":[[2017,6,23]],"date-time":"2017-06-23T07:38:33Z","timestamp":1498203513000},"source":"Crossref","is-referenced-by-count":30,"title":["Eltrombopag use in a patient with Wiskott\u2013Aldrich syndrome"],"prefix":"10.1002","volume":"64","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6226-9895","authenticated-orcid":false,"given":"Maria","family":"Gabelli","sequence":"first","affiliation":[{"name":"Department of Women's and Children's Health, Pediatric Onco\u2010Hematology Unit University of Padova  Padova Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Antonio","family":"Marzollo","sequence":"additional","affiliation":[{"name":"Department of Women's and Children's Health, Pediatric Onco\u2010Hematology Unit University of Padova  Padova Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lucia Dora","family":"Notarangelo","sequence":"additional","affiliation":[{"name":"Pediatric Onco\u2010Hematology and BMT Unit Children's Hospital Spedali Civili of Brescia  Brescia Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Giuseppe","family":"Basso","sequence":"additional","affiliation":[{"name":"Department of Women's and Children's Health, Pediatric Onco\u2010Hematology Unit University of Padova  Padova Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria Caterina","family":"Putti","sequence":"additional","affiliation":[{"name":"Department of Women's and Children's Health, Pediatric Onco\u2010Hematology Unit University of Padova  Padova Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2017,6,23]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(05)82002-5"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-08-448118"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-01-475913"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2012.31"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2015.3253"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1136\/adc.75.5.436"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-03-076679"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-04-425512"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(15)61107-2"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-09-602573"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-08-304725"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1080\/09537104.2016.1190446"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.24345"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.3109\/09537104.2012.693991"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/cyto.a.22520"}],"container-title":["Pediatric Blood &amp; Cancer"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpbc.26692","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.26692","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,29]],"date-time":"2023-09-29T08:55:40Z","timestamp":1695977740000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pbc.26692"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,6,23]]},"references-count":15,"journal-issue":{"issue":"12","published-print":{"date-parts":[[2017,12]]}},"alternative-id":["10.1002\/pbc.26692"],"URL":"https:\/\/doi.org\/10.1002\/pbc.26692","archive":["Portico"],"relation":{},"ISSN":["1545-5009","1545-5017"],"issn-type":[{"value":"1545-5009","type":"print"},{"value":"1545-5017","type":"electronic"}],"subject":[],"published":{"date-parts":[[2017,6,23]]},"article-number":"e26692"}}