{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T14:55:38Z","timestamp":1788274538897,"version":"build-2803163510"},"reference-count":20,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2020,5,19]],"date-time":"2020-05-19T00:00:00Z","timestamp":1589846400000},"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":[[2020,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>Pegylated IFN\u2010\u03b12a has been reported in two case reports as being efficacious in treating CDA\u2010I patients. This study aims to assess its efficacy on a series of CDA\u2010I patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Study sample consisted of seven CDA type 1 transfusion\u2010dependent patients. They received pegylated interferon alpha\u20102a at an initial dose of 90\u2010180\u00a0\u00b5g once a week, tapered according to clinical response and side effects. Good response was defined as Hb\u00a0\u2265\u00a010\u00a0g\/dL for \u22653\u00a0months, partial response was defined as 7\u00a0\u2264\u00a0Hb&lt;10\u00a0g\/dL for \u22653\u00a0months, and no response was defined as HB\u00a0&lt;\u00a07\u00a0g\/dL for over 3\u00a0months on treatment. Time to response was defined as the time needed to achieve hemoglobin levels\u00a0\u2265\u00a010\u00a0g\/dL without transfusion. Patients were evaluated periodically by abdominal ultrasounds to rule out liver adenomas.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Five patients (71%) had a good response to treatment. One patient stopped treatment due to side effects. One patient had partial response. One patient, with more severe phenotype and poor compliance, had poor response to treatment. No abnormal findings were found in ultrasound examination. No effect on serum ferritin level could be established.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Pegylated interferon \u03b12a therapy is efficacious in CDA\u2010I patients with a reasonable safety profile.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.13428","type":"journal-article","created":{"date-parts":[[2020,4,17]],"date-time":"2020-04-17T19:40:25Z","timestamp":1587152425000},"page":"216-222","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Treatment of transfusion\u2010dependent congenital dyserythropoietic anemia Type I patients with pegylated interferon alpha\u20102a"],"prefix":"10.1111","volume":"105","author":[{"given":"Abed","family":"Abu\u2010Quider","sequence":"first","affiliation":[{"name":"Pediatric Hemato\u2010oncology Department Saban Pediatric Medical Center Soroka University Medical Center Beer\u2010Sheva 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