{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,2]],"date-time":"2026-09-02T05:28:28Z","timestamp":1788326908192,"version":"build-2803163510"},"reference-count":18,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2018,5,10]],"date-time":"2018-05-10T00:00:00Z","timestamp":1525910400000},"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":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2018,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Hematopoietic stem cell transplantation (HSCT) is the curative treatment for Wiskott\u2013Aldrich syndrome (WAS). However, it is difficult to find a matched donor for patients. Therefore, haploidentical donors should be considered for patients lacking a suitable donor. Our pilot study evaluated whether HSCT with posttransplantation cyclophosphamide (PTCy) is an effective treatment for WAS.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Haploidentical family donors were selected as donor sources for a total of five patients without a suitable donor between March 2015 and March 2017. A modified transplant protocol using PTCy (50\u00a0mg\/kg\/day on days +3 and +4) was performed, including busulfan (16\u00a0mg\/kg), fludarabine (150\u00a0mg\/m<jats:sup>2<\/jats:sup>), and rabbit antihuman thymocyte globulin (7.5\u00a0mg\/kg).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The median time for neutrophil recovery over 1,000 \u00d7 10<jats:sup>3<\/jats:sup>\/mm<jats:sup>3<\/jats:sup> was 15 days (range, 12\u201318 days), and that for keeping platelets counts over 50,000\/mm<jats:sup>3<\/jats:sup> was 27.5 days (range, 20\u201335 days). The median follow\u2010up was 2.1 years (range, 1.4\u20132.5 years). Two patients developed grade I acute graft\u2010versus\u2010host disease (GVHD), and one patient had limited chronic GVHD. All five patients are alive and independent of platelet infusion with 100% donor chimerism.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Our pilot study suggests that HSCT with modified PTCy is a safe and effective treatment for WAS, which needs further clinical practice and research.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.27092","type":"journal-article","created":{"date-parts":[[2018,5,10]],"date-time":"2018-05-10T07:25:33Z","timestamp":1525937133000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Posttransplant cyclophosphamide for haploidentical stem cell transplantation in children with Wiskott\u2013Aldrich syndrome"],"prefix":"10.1002","volume":"65","author":[{"given":"Yan","family":"Yue","sequence":"first","affiliation":[{"name":"Department of Hematology and Oncology Hospital of Capital Institute of Pediatrics  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1184-2321","authenticated-orcid":false,"given":"Xiaodong","family":"Shi","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Hospital of Capital Institute of Pediatrics  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zeliang","family":"Song","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Hospital of Capital Institute of Pediatrics  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiayue","family":"Qin","sequence":"additional","affiliation":[{"name":"Acorndx Biotechnology Co., Ltd.  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Junhui","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Hospital of Capital Institute of Pediatrics  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shunqiao","family":"Feng","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Hospital of Capital Institute of Pediatrics  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rong","family":"Liu","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Hospital of Capital Institute of Pediatrics  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2018,5,10]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-05-1592"},{"key":"e_1_2_7_3_1","first-page":"26","article-title":"Wiskott\u2013Aldrich syndrome: a comprehensive review","volume":"1285","author":"Massaad MJ","year":"2013","journal-title":"Ann"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-12-115253"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1097\/00130832-200312000-00003"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2008.10.007"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.iac.2010.02.001"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2016.02.001"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1586\/1744666X.2015.1062366"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2008.03.005"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2016.12.628"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1097\/00007890-197410000-00001"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2005.09.004"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1097\/MPH.0000000000000959"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2017.25"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2012.31"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1705946"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2016.158808"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V96.8.2703.h8002703_2703_2711"}],"container-title":["Pediatric Blood &amp; Cancer"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpbc.27092","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.27092","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,23]],"date-time":"2023-09-23T07:05:51Z","timestamp":1695452751000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pbc.27092"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,5,10]]},"references-count":18,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2018,8]]}},"alternative-id":["10.1002\/pbc.27092"],"URL":"https:\/\/doi.org\/10.1002\/pbc.27092","archive":["Portico"],"relation":{},"ISSN":["1545-5009","1545-5017"],"issn-type":[{"value":"1545-5009","type":"print"},{"value":"1545-5017","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,5,10]]},"assertion":[{"value":"2017-12-05","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2018-03-20","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2018-05-10","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e27092"}}