{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,3]],"date-time":"2026-09-03T05:33:51Z","timestamp":1788413631939,"version":"build-2803163510"},"reference-count":22,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2021,3,16]],"date-time":"2021-03-16T00:00:00Z","timestamp":1615852800000},"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":["European J of Haematology"],"published-print":{"date-parts":[[2021,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Evans syndrome is a rare but challenging disorder in children; and despite rapidly growing evidence for targetable systemic immune dysregulation driving these \u201cidiopathic\u201d autoimmune cytopenias, precision diagnosis and management remains sub\u2010optimal among these patients. We analyzed retrospective clinical data for 60 pediatric ES patients followed at 3 large tertiary referral centers in the United States over a recent 6\u2010year period and found that definable underlying systemic immune dysregulation was identified in only 42% of these patients throughout the course of clinical care. Median time from ES diagnosis to identification of the underlying systemic immune dysregulation disorder was 1.3\u00a0years (&lt;1\u00a0month for rheumatologic disease, 2.3\u00a0years for CVID, 3.4\u00a0years for ALPS, and 7.4\u00a0years for monogenic disorders of immune regulation). Notably, a significantly higher percentage of patients in whom a definitive immune dysregulation disorder was ultimately identified required \u22653 cytopenia\u2010directed therapies (92%) and also second\u2010 and third\u2010line immunomodulatory agents (84%), vs those in whom no unifying immune dysregulation was diagnosed (65%, and 35%, respectively)\u2014indicating that autoimmune cytopenias as a manifestation of systemic immune dysregulation are more treatment\u2010refractory and severe. These data underline the importance of identifying the underlying systemic immune dysregulation and providing targeted therapy in pediatric ES.<\/jats:p>","DOI":"10.1111\/ejh.13600","type":"journal-article","created":{"date-parts":[[2021,2,11]],"date-time":"2021-02-11T13:53:44Z","timestamp":1613051624000},"page":"783-787","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":17,"title":["Refractory autoimmune cytopenias in pediatric Evans syndrome with underlying systemic immune dysregulation"],"prefix":"10.1111","volume":"106","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2842-9266","authenticated-orcid":false,"given":"Amanda B.","family":"Grimes","sequence":"first","affiliation":[{"name":"Section of Hematology\/Oncology Department of Pediatrics Baylor College of Medicine Houston TX USA"},{"name":"Texas Children's Cancer and Hematology Centers Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Taylor O.","family":"Kim","sequence":"additional","affiliation":[{"name":"Section of Hematology\/Oncology Department of Pediatrics Baylor College of Medicine Houston TX USA"},{"name":"Texas Children's Cancer and Hematology Centers Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan E.","family":"Kirk","sequence":"additional","affiliation":[{"name":"Section of Hematology\/Oncology Department of Pediatrics Baylor College of Medicine Houston TX USA"},{"name":"Texas Children's Cancer and Hematology Centers Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Flanagan","sequence":"additional","affiliation":[{"name":"Section of Hematology\/Oncology Department of Pediatrics Baylor College of Medicine Houston TX USA"},{"name":"Texas Children's Cancer and Hematology Centers Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michele P.","family":"Lambert","sequence":"additional","affiliation":[{"name":"Perelman School of Medicine at the University of Pennsylvania Philadelphia PA USA"},{"name":"Department of Pediatrics Children's Hospital of Philadelphia Philadelphia PA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachael F.","family":"Grace","sequence":"additional","affiliation":[{"name":"Harvard Medical School Boston MA USA"},{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Boston MA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenny M.","family":"Despotovic","sequence":"additional","affiliation":[{"name":"Section of Hematology\/Oncology Department of Pediatrics Baylor College of Medicine Houston TX USA"},{"name":"Texas Children's Cancer and Hematology Centers Houston TX 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