{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T02:35:25Z","timestamp":1793673325715,"version":"4.5.1"},"reference-count":43,"publisher":"American Society of Hematology","issue":"23","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2020,6,4]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Wiskott-Aldrich syndrome (WAS) is an X-linked disease caused by mutations in the WAS gene, leading to thrombocytopenia, eczema, recurrent infections, autoimmune disease, and malignancy. Hematopoietic cell transplantation (HCT) is the primary curative approach, with the goal of correcting the underlying immunodeficiency and thrombocytopenia. HCT outcomes have improved over time, particularly for patients with HLA-matched sibling and unrelated donors. We report the outcomes of 129 patients with WAS who underwent HCT at 29 Primary Immune Deficiency Treatment Consortium centers from 2005 through 2015. Median age at HCT was 1.2 years. Most patients (65%) received myeloablative busulfan-based conditioning. With a median follow-up of 4.5 years, the 5-year overall survival (OS) was 91%. Superior 5-year OS was observed in patients &amp;lt;5 vs \u22655 years of age at the time of HCT (94% vs 66%; overall P = .0008). OS was excellent regardless of donor type, even in cord blood recipients (90%). Conditioning intensity did not affect OS, but was associated with donor T-cell and myeloid engraftment after HCT. Specifically, patients who received fludarabine\/melphalan-based reduced-intensity regimens were more likely to have donor myeloid chimerism &amp;lt;50% early after HCT. In addition, higher platelet counts were observed among recipients who achieved full (&amp;gt;95%) vs low-level (5%-49%) donor myeloid engraftment. In summary, HCT outcomes for WAS have improved since 2005, compared with prior reports. HCT at a younger age continues to be associated with superior outcomes supporting the recommendation for early HCT. High-level donor myeloid engraftment is important for platelet reconstitution after either myeloablative or busulfan-containing reduced intensity conditioning. (This trial was registered at www.clinicaltrials.gov as #NCT02064933.)<\/jats:p>","DOI":"10.1182\/blood.2019002939","type":"journal-article","created":{"date-parts":[[2020,4,8]],"date-time":"2020-04-08T22:58:10Z","timestamp":1586386690000},"page":"2094-2105","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":141,"title":["Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report"],"prefix":"10.1182","volume":"135","author":[{"given":"Lauri M.","family":"Burroughs","sequence":"first","affiliation":[{"name":"Fred Hutchinson Cancer Research Center, Seattle, WA;"},{"name":"Department of Pediatrics, University of Washington\u2013Seattle Children\u2019s Hospital, Seattle, WA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aleksandra","family":"Petrovic","sequence":"first","affiliation":[{"name":"Fred Hutchinson Cancer Research Center, Seattle, WA;"},{"name":"Department of Pediatrics, University of Washington\u2013Seattle Children\u2019s Hospital, Seattle, WA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruta","family":"Brazauskas","sequence":"first","affiliation":[{"name":"Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuerong","family":"Liu","sequence":"first","affiliation":[{"name":"Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda M.","family":"Griffith","sequence":"first","affiliation":[{"name":"Division of Allergy, Immunology, and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hans D.","family":"Ochs","sequence":"first","affiliation":[{"name":"Department of Pediatrics, University of Washington\u2013Seattle Children\u2019s Hospital, Seattle, WA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jack J.","family":"Bleesing","sequence":"first","affiliation":[{"name":"Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children\u2019s Hospital Medical Center, University of Cincinnati, Cincinnati, OH;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie","family":"Edwards","sequence":"first","affiliation":[{"name":"Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children\u2019s Hospital Medical Center, University of Cincinnati, Cincinnati, OH;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6146-3952","authenticated-orcid":false,"given":"Christopher C.","family":"Dvorak","sequence":"first","affiliation":[{"name":"Pediatric Allergy, Immunology, and Blood and Marrow Transplant Division, University of California, San Francisco Benioff Children\u2019s Hospital, San Francisco, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonali","family":"Chaudhury","sequence":"first","affiliation":[{"name":"Division of Hematology, Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children\u2019s Hospital of Chicago\u2013Northwestern University Feinberg School of Medicine, Chicago, IL;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan E.","family":"Prockop","sequence":"first","affiliation":[{"name":"Bone Marrow Transplant Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ralph","family":"Quinones","sequence":"first","affiliation":[{"name":"Pediatric Bone Marrow Transplant (BMT) and Cellular Therapy Section, Department of Pediatrics, The University of Colorado School of Medicine, Aurora, CO;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0764-4883","authenticated-orcid":false,"given":"Frederick D.","family":"Goldman","sequence":"first","affiliation":[{"name":"Department of Pediatrics, The University of Alabama at Birmingham, Birmingham, AL;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Troy C.","family":"Quigg","sequence":"first","affiliation":[{"name":"Texas Transplant Institute, Methodist Children\u2019s Hospital, San Antonio, TX;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shanmuganathan","family":"Chandrakasan","sequence":"first","affiliation":[{"name":"Aflac Cancer and Blood Disorders Center, Emory\/Children\u2019s Healthcare of Atlanta, Atlanta, GA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela R.","family":"Smith","sequence":"first","affiliation":[{"name":"Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6066-9852","authenticated-orcid":false,"given":"Suhag","family":"Parikh","sequence":"first","affiliation":[{"name":"Duke University Medical Center, Durham, NC;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8281-3627","authenticated-orcid":false,"given":"Blachy J.","family":"D\u00e1vila Salda\u00f1a","sequence":"first","affiliation":[{"name":"Division of Blood and Marrow Transplantation, Children's National Hospital\u2013George Washington University School of Medicine and Health Sciences, Washington, DC;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monica S.","family":"Thakar","sequence":"first","affiliation":[{"name":"Center for Blood and Marrow Transplant Research\u2013Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, WI;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachel","family":"Phelan","sequence":"first","affiliation":[{"name":"Center for Blood and Marrow Transplant Research\u2013Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, WI;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shalini","family":"Shenoy","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Washington University School of Medicine, St. Louis, MO;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9402-1934","authenticated-orcid":false,"given":"Lisa R.","family":"Forbes","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Baylor College of Medicine Section of Immunology, Allergy, and Retrovirology, Texas Children\u2019s Hospital, Baylor, TX;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caridad","family":"Martinez","sequence":"first","affiliation":[{"name":"Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children\u2019s Hospital Cancer Center, Baylor, TX;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deepak","family":"Chellapandian","sequence":"first","affiliation":[{"name":"Blood and Marrow Transplant, Johns Hopkins All Children's Hospital, St. Petersburg, FL;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evan","family":"Shereck","sequence":"first","affiliation":[{"name":"Division of Pediatric Hematology\/Oncology, Oregon Health and Science University, Portland, OR;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Holly K.","family":"Miller","sequence":"first","affiliation":[{"name":"Phoenix Children\u2019s Hospital, Phoenix, AZ;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neena","family":"Kapoor","sequence":"first","affiliation":[{"name":"Transplantation and Cellular Therapy Program, Cancer and Blood Disease Institute, Children\u2019s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessie L.","family":"Barnum","sequence":"first","affiliation":[{"name":"UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hey","family":"Chong","sequence":"first","affiliation":[{"name":"UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David C.","family":"Shyr","sequence":"first","affiliation":[{"name":"Division of Pediatric Hematology\/Oncology, Primary Children\u2019s Hospital, University of Utah School of Medicine, Salt Lake City, UT;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5593-436X","authenticated-orcid":false,"given":"Karin","family":"Chen","sequence":"first","affiliation":[{"name":"Division of Allergy and Immunology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rolla","family":"Abu-Arja","sequence":"first","affiliation":[{"name":"Nationwide Children\u2019s Hospital, Columbus, OH;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ami J.","family":"Shah","sequence":"first","affiliation":[{"name":"Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine Pediatric Stem Cell Transplantation, Stanford University, Stanford, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katja G.","family":"Weinacht","sequence":"first","affiliation":[{"name":"Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford School of Medicine Pediatric Stem Cell Transplantation, Stanford University, Stanford, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Theodore B.","family":"Moore","sequence":"first","affiliation":[{"name":"Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Avni","family":"Joshi","sequence":"first","affiliation":[{"name":"Mayo Clinic Children\u2019s Center, Rochester, MN;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenneth B.","family":"DeSantes","sequence":"first","affiliation":[{"name":"American Family Children's Hospital, University of Wisconsin, Madison, WI;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alfred P.","family":"Gillio","sequence":"first","affiliation":[{"name":"Institute for Pediatric Cancer and Blood Disorders, Hackensack University Medical Center, Hackensack, NJ;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Geoffrey D. E.","family":"Cuvelier","sequence":"first","affiliation":[{"name":"Cancer Care Manitoba, University of Manitoba, Winnipeg, MB, Canada;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael D.","family":"Keller","sequence":"first","affiliation":[{"name":"Division of Allergy & Immunology, Children\u2019s National Hospital, Washington, DC.;"},{"name":"GW Cancer Center, George Washington University, Washington, DC.;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacob","family":"Rozmus","sequence":"first","affiliation":[{"name":"Children\u2019s & Women\u2019s Health Centre of British Columbia, Vancouver, BC, Canada;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Troy","family":"Torgerson","sequence":"first","affiliation":[{"name":"Department of Pediatrics, University of Washington\u2013Seattle Children\u2019s Hospital, Seattle, WA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3030-8420","authenticated-orcid":false,"given":"Michael A.","family":"Pulsipher","sequence":"first","affiliation":[{"name":"Transplantation and Cellular Therapy Program, Cancer and Blood Disease Institute, Children\u2019s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2446-6879","authenticated-orcid":false,"given":"Elie","family":"Haddad","sequence":"first","affiliation":[{"name":"Pediatric Immunology and Rheumatology Division, CHU Sainte-Justine, Department of Pediatrics, University of Montreal, Montreal, QC, Canada;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen E.","family":"Sullivan","sequence":"first","affiliation":[{"name":"Allergy and Immunology, The Children's Hospital of Philadelphia, Philadelphia, PA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brent R.","family":"Logan","sequence":"first","affiliation":[{"name":"Division of Biostatistics, Medical College of Wisconsin, Milwaukee, WI;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1840-6087","authenticated-orcid":false,"given":"Donald B.","family":"Kohn","sequence":"first","affiliation":[{"name":"Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer M.","family":"Puck","sequence":"first","affiliation":[{"name":"Pediatric Allergy, Immunology, and Blood and Marrow Transplant Division, University of California, San Francisco Benioff Children\u2019s Hospital, San Francisco, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8335-0262","authenticated-orcid":false,"given":"Luigi D.","family":"Notarangelo","sequence":"first","affiliation":[{"name":"Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0158-8147","authenticated-orcid":false,"given":"Sung-Yun","family":"Pai","sequence":"first","affiliation":[{"name":"Division of Hematology-Oncology, Boston Children's Hospital, Boston, MA; and"},{"name":"Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David J.","family":"Rawlings","sequence":"first","affiliation":[{"name":"Department of Pediatrics, University of Washington\u2013Seattle Children\u2019s Hospital, Seattle, WA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1088-8045","authenticated-orcid":false,"given":"Morton J.","family":"Cowan","sequence":"first","affiliation":[{"name":"Pediatric Allergy, Immunology, and Blood and Marrow Transplant Division, University of California, San Francisco Benioff Children\u2019s Hospital, San Francisco, CA;"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"issue":"6","key":"2020060411210480100_B1","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1023\/A:1026416017763","article-title":"Primary immunodeficiency diseases in Norway","volume":"20","author":"Stray-Pedersen","year":"2000","journal-title":"J Clin Immunol"},{"issue":"6 Pt 1","key":"2020060411210480100_B2","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1016\/S0022-3476(05)82002-5","article-title":"A multiinstitutional survey of the Wiskott-Aldrich syndrome","volume":"125","author":"Sullivan","year":"1994","journal-title":"J Pediatr"},{"issue":"1","key":"2020060411210480100_B3","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1016\/0888-7543(91)90480-3","article-title":"Localization of the gene for the Wiskott-Aldrich syndrome between two flanking markers, TIMP and DXS255, on Xp11.22-Xp11.3","volume":"10","author":"Kwan","year":"1991","journal-title":"Genomics"},{"issue":"4","key":"2020060411210480100_B4","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1016\/0092-8674(94)90528-2","article-title":"Isolation of a novel gene mutated in Wiskott-Aldrich syndrome [published correction appears in Cell. 1994;79(5): following 922]","volume":"78","author":"Derry","year":"1994","journal-title":"Cell"},{"issue":"7","key":"2020060411210480100_B5","doi-asserted-by":"crossref","first-page":"2680","DOI":"10.1182\/blood.V90.7.2680","article-title":"Wiskott-Aldrich syndrome\/X-linked thrombocytopenia: WASP gene mutations, protein expression, and phenotype","volume":"90","author":"Zhu","year":"1997","journal-title":"Blood"},{"issue":"3-4","key":"2020060411210480100_B6","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1155\/2010\/781523","article-title":"The Wiskott-Aldrich syndrome: The actin cytoskeleton and immune cell function [review]","volume":"29","author":"Blundell","year":"2010","journal-title":"Disease Markers"},{"issue":"2","key":"2020060411210480100_B7","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/S1074-7613(02)00360-6","article-title":"Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation","volume":"17","author":"Dupr\u00e9","year":"2002","journal-title":"Immunity"},{"issue":"13","key":"2020060411210480100_B8","doi-asserted-by":"crossref","first-page":"4010","DOI":"10.1182\/blood-2003-05-1592","article-title":"Mutations of the Wiskott-Aldrich Syndrome Protein (WASP): hotspots, effect on transcription, and translation and phenotype\/genotype correlation","volume":"104","author":"Jin","year":"2004","journal-title":"Blood"},{"issue":"1","key":"2020060411210480100_B9","doi-asserted-by":"crossref","first-page":"42","DOI":"10.1097\/MOH.0b013e32834114bc","article-title":"Clinical spectrum, pathophysiology and treatment of the Wiskott-Aldrich syndrome","volume":"18","author":"Albert","year":"2011","journal-title":"Curr Opin Hematol"},{"issue":"4","key":"2020060411210480100_B10","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1111\/bjh.15831","article-title":"How I manage patients with Wiskott Aldrich syndrome","volume":"185","author":"Rivers","year":"2019","journal-title":"Br J Haematol"},{"issue":"7583","key":"2020060411210480100_B11","doi-asserted-by":"crossref","first-page":"1364","DOI":"10.1016\/S0140-6736(68)92672-X","article-title":"Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome","volume":"292","author":"Bach","year":"1968","journal-title":"Lancet"},{"issue":"10","key":"2020060411210480100_B12","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1038\/sj.bmt.1705512","article-title":"Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation","volume":"38","author":"Pai","year":"2006","journal-title":"Bone Marrow Transplant"},{"issue":"6","key":"2020060411210480100_B13","doi-asserted-by":"crossref","first-page":"1598","DOI":"10.1182\/blood.V97.6.1598","article-title":"Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program","volume":"97","author":"Filipovich","year":"2001","journal-title":"Blood"},{"issue":"1","key":"2020060411210480100_B14","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1182\/blood-2007-03-076679","article-title":"Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation","volume":"111","author":"Ozsahin","year":"2008","journal-title":"Blood"},{"issue":"3","key":"2020060411210480100_B15","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1007\/s12026-008-8063-8","article-title":"Results and long-term outcome in 39 patients with Wiskott-Aldrich syndrome transplanted from HLA-matched and -mismatched donors","volume":"44","author":"Friedrich","year":"2009","journal-title":"Immunol Res"},{"issue":"6","key":"2020060411210480100_B16","doi-asserted-by":"crossref","first-page":"1675","DOI":"10.1182\/blood-2010-11-319376","article-title":"Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study","volume":"118","author":"Moratto","year":"2011","journal-title":"Blood"},{"issue":"11","key":"2020060411210480100_B17","doi-asserted-by":"crossref","first-page":"1428","DOI":"10.1038\/bmt.2012.31","article-title":"Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome","volume":"47","author":"Shin","year":"2012","journal-title":"Bone Marrow Transplant"},{"issue":"2","key":"2020060411210480100_B18","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1016\/j.jaci.2016.01.051","article-title":"Primary Immune Deficiency Treatment Consortium (PIDTC) update","volume":"138","author":"Griffith","year":"2016","journal-title":"J Allergy Clin Immunol"},{"issue":"10","key":"2020060411210480100_B19","doi-asserted-by":"crossref","first-page":"3797","DOI":"10.1182\/blood.V86.10.3797.bloodjournal86103797","article-title":"The Wiskott-Aldrich syndrome and X-linked congenital thrombocytopenia are caused by mutations of the same gene","volume":"86","author":"Zhu","year":"1995","journal-title":"Blood"},{"issue":"1-2","key":"2020060411210480100_B20","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1016\/S0022-1759(01)00549-X","article-title":"Flow cytometric determination of intracytoplasmic Wiskott-Aldrich syndrome protein in peripheral blood lymphocyte subpopulations","volume":"260","author":"Kawai","year":"2002","journal-title":"J Immunol Methods"},{"issue":"3","key":"2020060411210480100_B21","doi-asserted-by":"crossref","first-page":"857","DOI":"10.1182\/blood-2004-02-0414","article-title":"Once-daily intravenous busulfan and fludarabine: clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS","volume":"104","author":"de Lima","year":"2004","journal-title":"Blood"},{"issue":"5","key":"2020060411210480100_B22","doi-asserted-by":"crossref","first-page":"434","DOI":"10.1056\/NEJMoa1401177","article-title":"Transplantation outcomes for severe combined immunodeficiency, 2000-2009","volume":"371","author":"Pai","year":"2014","journal-title":"N Engl J Med"},{"issue":"17","key":"2020060411210480100_B23","doi-asserted-by":"crossref","first-page":"1737","DOI":"10.1182\/blood-2018-03-840702","article-title":"SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery","volume":"132","author":"Haddad","year":"2018","journal-title":"Blood"},{"issue":"12","key":"2020060411210480100_B24","doi-asserted-by":"crossref","first-page":"1628","DOI":"10.1016\/j.bbmt.2009.07.004","article-title":"Defining the intensity of conditioning regimens: working definitions","volume":"15","author":"Bacigalupo","year":"2009","journal-title":"Biol Blood Marrow Transplant"},{"issue":"9915","key":"2020060411210480100_B25","doi-asserted-by":"crossref","first-page":"436","DOI":"10.1016\/S0140-6736(13)62069-3","article-title":"Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study","volume":"383","author":"G\u00fcng\u00f6r","year":"2014","journal-title":"Lancet"},{"issue":"6","key":"2020060411210480100_B26","first-page":"825","article-title":"1994 Consensus Conference on Acute GVHD Grading","volume":"15","author":"Przepiorka","year":"1995","journal-title":"Bone Marrow Transplant"},{"issue":"6","key":"2020060411210480100_B27","doi-asserted-by":"crossref","first-page":"1112","DOI":"10.3324\/haematol.2016.158808","article-title":"A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome","volume":"102","author":"Shekhovtsova","year":"2017","journal-title":"Haematologica"},{"issue":"22","key":"2020060411210480100_B28","doi-asserted-by":"crossref","first-page":"4285","DOI":"10.1182\/blood-2012-07-438408","article-title":"HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease","volume":"120","author":"Bola\u00f1os-Meade","year":"2012","journal-title":"Blood"},{"issue":"2","key":"2020060411210480100_B29","doi-asserted-by":"crossref","first-page":"310","DOI":"10.1016\/j.bbmt.2016.11.006","article-title":"Haploidentical bone marrow transplantation with post-transplant cyclophosphamide for children and adolescents with Fanconi anemia","volume":"23","author":"Bonfim","year":"2017","journal-title":"Biol Blood Marrow Transplant"},{"issue":"3","key":"2020060411210480100_B30","doi-asserted-by":"crossref","first-page":"498","DOI":"10.1016\/j.bbmt.2016.12.628","article-title":"Alternative donor transplantation with high-dose post-transplantation cyclophosphamide for refractory severe aplastic anemia","volume":"23","author":"DeZern","year":"2017","journal-title":"Biol Blood Marrow Transplant"},{"issue":"5","key":"2020060411210480100_B31","doi-asserted-by":"crossref","first-page":"895","DOI":"10.1016\/j.bbmt.2016.02.001","article-title":"Alternative-donor hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for nonmalignant disorders","volume":"22","author":"Klein","year":"2016","journal-title":"Biol Blood Marrow Transplant"},{"issue":"4","key":"2020060411210480100_B32","doi-asserted-by":"crossref","first-page":"570","DOI":"10.1038\/bmt.2016.301","article-title":"Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia","volume":"52","author":"Thakar","year":"2017","journal-title":"Bone Marrow Transplant"},{"issue":"8","key":"2020060411210480100_B33","doi-asserted-by":"crossref","first-page":"e27092","DOI":"10.1002\/pbc.27092","article-title":"Posttransplant cyclophosphamide for haploidentical stem cell transplantation in children with Wiskott-Aldrich syndrome","volume":"65","author":"Yue","year":"2018","journal-title":"Pediatr Blood Cancer"},{"issue":"7","key":"2020060411210480100_B34","doi-asserted-by":"crossref","first-page":"1432","DOI":"10.1016\/j.bbmt.2018.03.006","article-title":"A Conditioning Regimen With Plerixafor Is Safe and Improves the Outcome of TCRalphabeta(+) and CD19(+) Cell-Depleted Stem Cell Transplantation in Patients with Wiskott-Aldrich Syndrome","volume":"24","author":"Balashov","year":"2018","journal-title":"Biol Blood Marrow Transplant"},{"issue":"5","key":"2020060411210480100_B35","doi-asserted-by":"crossref","first-page":"822","DOI":"10.1182\/blood-2014-03-563817","article-title":"HLA-haploidentical stem cell transplantation after removal of \u03b1\u03b2+ T and B cells in children with nonmalignant disorders","volume":"124","author":"Bertaina","year":"2014","journal-title":"Blood"},{"issue":"4","key":"2020060411210480100_B36","doi-asserted-by":"crossref","first-page":"1417","DOI":"10.1016\/j.jaci.2017.07.008","article-title":"T-cell receptor \u03b1\u03b2+ and CD19+ cell-depleted haploidentical and mismatched hematopoietic stem cell transplantation in primary immune deficiency [published correction appears in J Allery Clin Immunol. 2019;143(5):1977","volume":"141","author":"Shah","year":"2018","journal-title":"J Allergy Clin Immunol"},{"issue":"20","key":"2020060411210480100_B37","doi-asserted-by":"crossref","first-page":"1918","DOI":"10.1056\/NEJMoa1003548","article-title":"Stem-cell gene therapy for the Wiskott-Aldrich syndrome","volume":"363","author":"Boztug","year":"2010","journal-title":"N Engl J Med"},{"issue":"227","key":"2020060411210480100_B38","doi-asserted-by":"crossref","first-page":"227ra33","DOI":"10.1126\/scitranslmed.3007280","article-title":"Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity","volume":"6","author":"Braun","year":"2014","journal-title":"Sci Transl Med"},{"issue":"15","key":"2020060411210480100_B39","doi-asserted-by":"crossref","first-page":"1550","DOI":"10.1001\/jama.2015.3253","article-title":"Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome","volume":"313","author":"Hacein-Bey Abina","year":"2015","journal-title":"JAMA"},{"issue":"5","key":"2020060411210480100_B40","doi-asserted-by":"crossref","first-page":"e239","DOI":"10.1016\/S2352-3026(19)30021-3","article-title":"Lentiviral haemopoietic stem\/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1\/2 clinical study","volume":"6","author":"Ferrua","year":"2019","journal-title":"Lancet Haematol"},{"issue":"5","key":"2020060411210480100_B41","doi-asserted-by":"crossref","first-page":"1654","DOI":"10.1016\/j.jaci.2018.06.042","article-title":"One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years","volume":"142","author":"Elfeky","year":"2018","journal-title":"J Allergy Clin Immunol"},{"issue":"3","key":"2020060411210480100_B42","doi-asserted-by":"crossref","first-page":"364","DOI":"10.1007\/s12185-019-02686-y","article-title":"Long-term outcome and chimerism in patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation: a retrospective nationwide survey","volume":"110","author":"Iguchi","year":"2019","journal-title":"Int J Hematol"},{"issue":"8","key":"2020060411210480100_B43","doi-asserted-by":"crossref","first-page":"786","DOI":"10.1007\/s10875-019-00689-2","article-title":"Quality of Life of Patients with Wiskott Aldrich Syndrome and X-Linked Thrombocytopenia: a Study of the Primary Immune Deficiency Consortium (PIDTC), Immune Deficiency Foundation, and the Wiskott-Aldrich Foundation","volume":"39","author":"Shah","year":"2019","journal-title":"J Clin Immunol"}],"container-title":["Blood"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/ashpublications.org\/blood\/article-pdf\/135\/23\/2094\/1743290\/bloodbld2019002939.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"http:\/\/ashpublications.org\/blood\/article-pdf\/135\/23\/2094\/1743290\/bloodbld2019002939.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,6,4]],"date-time":"2020-06-04T23:19:57Z","timestamp":1591312797000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/blood\/article\/135\/23\/2094\/454301\/Excellent-outcomes-following-hematopoietic-cell"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,6,4]]},"references-count":43,"journal-issue":{"issue":"23","published-print":{"date-parts":[[2020,6,4]]}},"URL":"https:\/\/doi.org\/10.1182\/blood.2019002939","relation":{},"ISSN":["0006-4971","1528-0020"],"issn-type":[{"value":"0006-4971","type":"print"},{"value":"1528-0020","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,6,4]]}}}