{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T16:33:38Z","timestamp":1773419618653,"version":"3.50.1"},"reference-count":53,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2024,4,12]],"date-time":"2024-04-12T00:00:00Z","timestamp":1712880000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2024,4,12]],"date-time":"2024-04-12T00:00:00Z","timestamp":1712880000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2024,7]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Hematopoietic cell transplantation (HCT) is an established curative therapy for transfusion\u2010dependent thalassemia (TDT) and sickle cell disease (SCD). The latest American Society of Hematology guidelines recommend myeloablative preparative regimen in patients under 18\u00a0years of age.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Procedure<\/jats:title>\n                    <jats:p>The objective was to demonstrate safety and efficacy of a reduced intensity conditioning (RIC) regimen including high\u2010dose fludarabine, anti\u2010thymocyte globulin, and targeted busulfan as a single alkylator to sub\u2010myeloablative exposures.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Between 2012 and 2021, 11 patients with SCD and five patients with TDT and matched related donor (MRD) HCT were included. The median age at transplantation was 8.3\u00a0years (range: 3.7\u201318.8\u00a0years). The median administered busulfan AUC was 67.4\u00a0mg\/L\u00d7h (range: 60.7\u201380\u00a0mg\/L\u00d7h). Overall survival was 93.8% and event\u2010free survival 87.5% with one engrafted SCD patient with pre\u2010existing moyamoya disease succumbing after drainage of a subdural hematoma. One SCD patient developed a secondary graft failure and was treated with a second HCT. Myeloid chimerism was full in all other patients with a median follow\u2010up time of 4.1\u00a0years (range: 2.0\u201311.1\u00a0years), whereas T\u2010cell donor chimerism was frequently mixed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>This RIC conditioning followed by MRD HCT is sufficiently myeloablative to cure pediatric patients with hemoglobinopathies without the need for additional total body irradiation or thiotepa.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/pbc.30988","type":"journal-article","created":{"date-parts":[[2024,4,13]],"date-time":"2024-04-13T01:58:45Z","timestamp":1712973525000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Good engraftment after reduced intensity targeted busulfan\u2010based conditioning and matched related donor hematopoietic cell transplantation in hemoglobinopathies"],"prefix":"10.1002","volume":"71","author":[{"given":"Maximilian","family":"Klink","sequence":"first","affiliation":[{"name":"Medical Faculty University of Z\u00fcrich  Z\u00fcrich Switzerland"},{"name":"Division of Stem Cell Transplantation and Children's Research Center University Children's Hospital Zurich University of Z\u00fcrich  Z\u00fcrich Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Matthias","family":"Felber","sequence":"additional","affiliation":[{"name":"Division of Stem Cell Transplantation and Children's Research Center University Children's Hospital Zurich University of Z\u00fcrich  Z\u00fcrich Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ulrike","family":"Zeilhofer","sequence":"additional","affiliation":[{"name":"Division of Stem Cell Transplantation and Children's Research Center University Children's Hospital Zurich University of Z\u00fcrich  Z\u00fcrich Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mathias","family":"Hauri\u2010Hohl","sequence":"additional","affiliation":[{"name":"Division of Stem Cell Transplantation and Children's Research Center University Children's Hospital Zurich University of Z\u00fcrich  Z\u00fcrich Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Markus","family":"Schmugge","sequence":"additional","affiliation":[{"name":"Division of Hematology and Children's Research Center University Children's Hospital Zurich University of Z\u00fcrich  Z\u00fcrich Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3010-7525","authenticated-orcid":false,"given":"Daniel","family":"Drozdov","sequence":"additional","affiliation":[{"name":"Division of Stem Cell Transplantation and Children's Research Center University Children's Hospital Zurich University of Z\u00fcrich  Z\u00fcrich Switzerland"},{"name":"Division of Pediatric Hematology and Oncology Department of Pediatrics Kantonsspital Aarau  Aarau Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3261-1186","authenticated-orcid":false,"given":"Tayfun","family":"G\u00fcng\u00f6r","sequence":"additional","affiliation":[{"name":"Division of Stem Cell Transplantation and Children's Research Center University Children's Hospital Zurich University of Z\u00fcrich  Z\u00fcrich Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2024,4,12]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019001142"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.10937"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102009\u201007\u2010233700"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102010\u201006\u2010293514"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2018.213207"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1177\/003335491312800206"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.18631"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.216788"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352\u20103026(19)30154\u20101"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcyt.2021.09.003"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2031054"},{"key":"e_1_2_11_13_1","unstructured":"The ASCO Post Staff.FDA approves first cell\u2010based gene therapy to treat adult and pediatric patients with beta\u2010thalassemia who require regular blood transfusions.FDA; August 17 2022. Accessed April 11 2023.https:\/\/www.fda.gov\/news\u2010events\/press\u2010announcements\/fda\u2010approves\u2010first\u2010cell\u2010based\u2010gene\u2010therapy\u2010treat\u2010adult\u2010and\u2010pediatric\u2010patients\u2010beta\u2010thalassemia\u2010who"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2117175"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2021004394C"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140\u20106736(13)62069\u20103"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102018\u201001\u2010828277"},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2020001748"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10875\u2010020\u201000888\u20102"},{"key":"e_1_2_11_20_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41409\u2010021\u201001378\u20108"},{"key":"e_1_2_11_21_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2014.7192"},{"key":"e_1_2_11_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2019.02.011"},{"key":"e_1_2_11_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.19122"},{"key":"e_1_2_11_24_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.30322"},{"key":"e_1_2_11_25_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.24183"},{"key":"e_1_2_11_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2015.01.015"},{"key":"e_1_2_11_27_1","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2020.551170"},{"key":"e_1_2_11_28_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2008.08.016"},{"key":"e_1_2_11_29_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375\u2010022\u201001693\u2010z"},{"key":"e_1_2_11_30_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V93.10.3267.410k22_3267_3275"},{"key":"e_1_2_11_31_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2016.206"},{"key":"e_1_2_11_32_1","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.154344"},{"key":"e_1_2_11_33_1","doi-asserted-by":"publisher","DOI":"10.2165\/11598180\u2010000000000\u201000000"},{"key":"e_1_2_11_34_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470\u20102045(20)30582\u20109"},{"key":"e_1_2_11_35_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470\u20102045(20)30594\u20105"},{"key":"e_1_2_11_36_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020007905"},{"key":"e_1_2_11_37_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41409\u2010022\u201001594\u2010w"},{"key":"e_1_2_11_38_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102013\u201003\u2010489112"},{"key":"e_1_2_11_39_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277\u2010021\u201004732\u20104"},{"key":"e_1_2_11_40_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102016\u201010\u2010745711"},{"key":"e_1_2_11_41_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.01791"},{"key":"e_1_2_11_42_1","doi-asserted-by":"publisher","DOI":"10.1080\/16078454.2022.2163357"},{"key":"e_1_2_11_43_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102017\u201003\u2010772392"},{"key":"e_1_2_11_44_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V91.3.756"},{"key":"e_1_2_11_45_1","doi-asserted-by":"publisher","DOI":"10.3390\/pharmaceutics14112462"},{"key":"e_1_2_11_46_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2017.06.021"},{"key":"e_1_2_11_47_1","doi-asserted-by":"publisher","DOI":"10.3390\/pharmaceutics16010013"},{"key":"e_1_2_11_48_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.22.01203"},{"key":"e_1_2_11_49_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.227561"},{"key":"e_1_2_11_50_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.13248"},{"key":"e_1_2_11_51_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2017.231"},{"key":"e_1_2_11_52_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41409\u2010022\u201001786\u20104"},{"key":"e_1_2_11_53_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.hemonc.2019.12.011"},{"key":"e_1_2_11_54_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2022009051"}],"container-title":["Pediatric Blood &amp; Cancer"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.30988","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/pbc.30988","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.30988","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T14:23:59Z","timestamp":1773411839000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pbc.30988"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,4,12]]},"references-count":53,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2024,7]]}},"alternative-id":["10.1002\/pbc.30988"],"URL":"https:\/\/doi.org\/10.1002\/pbc.30988","archive":["Portico"],"relation":{},"ISSN":["1545-5009","1545-5017"],"issn-type":[{"value":"1545-5009","type":"print"},{"value":"1545-5017","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,4,12]]},"assertion":[{"value":"2023-11-15","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-20","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-04-12","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e30988"}}