{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T23:50:33Z","timestamp":1794009033837,"version":"4.8.0"},"reference-count":94,"publisher":"Oxford University Press (OUP)","issue":"1","license":[{"start":{"date-parts":[[2020,7,15]],"date-time":"2020-07-15T00:00:00Z","timestamp":1594771200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2020,10,14]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Introduction or background<\/jats:title>\n                  <jats:p>For many primary immunodeficiencies (PIDs), haematopoietic stem cell transplantation (HSCT) offers treatment to cure disease. However, patients with PID present a unique set of challenges when considering HSCT.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Sources of data<\/jats:title>\n                  <jats:p>Review of recent literature.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Areas of agreement<\/jats:title>\n                  <jats:p>The most significant recent impact on successful outcome is introduction of newborn screening programmes for diagnosis of severe combined immunodeficiency\u2014wider adoption of screening in an increasing number of countries will see further improvements. Other PIDs have better outcomes when treated earlier, before development of co-morbidities\u2014early referral for consideration of HSCT is important. Evolution of conditioning regimens is improving short- and long-term toxicities\u2014targeted busulfan and low-toxicity myeloablative treosulfan regimens deliver good survival with reduced short-term toxicities.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Areas of controversy<\/jats:title>\n                  <jats:p>The most radical development, still in clinical trials, is the use of mono-antibody-based conditioning, which eliminates the requirement for chemotherapy and is likely to become much more important in HSCT for non-malignant disease in the future.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Growing points<\/jats:title>\n                  <jats:p>Multidisciplinary working for optimum care is essential.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Areas timely for developing research<\/jats:title>\n                  <jats:p>International collaborations are important to learn about rare presentations and complications, and to formulate the most effective and safe treatment strategies.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1093\/bmb\/ldaa017","type":"journal-article","created":{"date-parts":[[2020,5,29]],"date-time":"2020-05-29T11:08:07Z","timestamp":1590750487000},"page":"4-15","source":"Crossref","is-referenced-by-count":4,"title":["The challenges presented by haematopoietic stem cell transplantation in children with primary immunodeficiency"],"prefix":"10.1093","volume":"135","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6218-1324","authenticated-orcid":false,"given":"A R","family":"Gennery","sequence":"first","affiliation":[{"name":"Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Great North Children\u2019s Hospital, Newcastle upon Tyne NE1 4LP, UK"},{"name":"Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne NE1 7RU, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2020,7,15]]},"reference":[{"key":"2020102607504342800_ref1","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1007\/s10875-019-00737-x","article-title":"Human inborn errors of immunity: 2019 update on the classification from the International Union of Immunological Societies Expert Committee","volume":"40","author":"Tangye","year":"2020","journal-title":"J Clin Immunol"},{"key":"2020102607504342800_ref2","doi-asserted-by":"crossref","first-page":"1080","DOI":"10.1016\/S0140-6736(86)90477-0","article-title":"Bone-marrow transplantation for immunodeficiencies and osteopetrosis: European survey, 1968-1985","volume":"2","author":"Fischer","year":"1986","journal-title":"Lancet"},{"key":"2020102607504342800_ref3","doi-asserted-by":"crossref","first-page":"508","DOI":"10.1056\/NEJM199902183400703","article-title":"Hematopoietic stem-cell transplantation for the treatment of 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