{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,4]],"date-time":"2026-07-04T00:03:06Z","timestamp":1783123386614,"version":"3.54.6"},"reference-count":23,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2025,7,10]],"date-time":"2025-07-10T00:00:00Z","timestamp":1752105600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,7,10]],"date-time":"2025-07-10T00:00:00Z","timestamp":1752105600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Clin Exp Med"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Congenital dyserythropoietic anemia type I (CDA-I) is a rare hereditary anemia caused by\n                    <jats:italic>CDAN1<\/jats:italic>\n                    or\n                    <jats:italic>C15orf41<\/jats:italic>\n                    mutations, with\n                    <jats:italic>CDAN1<\/jats:italic>\n                    -related cases responding to interferon-alpha (IFN-\u03b1) therapy. However, traditional IFN-\u03b1 requires frequent injections and often causes flu-like symptoms, which can hinder long-term adherence. Here, we present the first documented use of ropeginterferon alfa-2b, a next-generation pegylated interferon, in two patients with CDA-I. Both patients, who were previously transfusion dependent, achieved transfusion independence and improved hemoglobin levels after transitioning from recombinant IFN-\u03b1 to ropeginterferon alfa-2b with biweekly dosing. This treatment preserved efficacy while minimizing adverse effects and the injection burden. Our findings suggest that ropeginterferon alfa-2b may serve as a more tolerable and effective long-term treatment. Further prospective large-scale studies are needed to validate its broader clinical applicability.\n                  <\/jats:p>","DOI":"10.1007\/s10238-025-01785-x","type":"journal-article","created":{"date-parts":[[2025,7,10]],"date-time":"2025-07-10T17:05:36Z","timestamp":1752167136000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Ropeginterferon alfa-2b as a promising alternative to conventional interferon in CDA type 1: a case report of two siblings"],"prefix":"10.1007","volume":"25","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2631-9477","authenticated-orcid":false,"given":"Tyng-Wei","family":"Yang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4174-8766","authenticated-orcid":false,"given":"Xavier Cheng-Hong","family":"Tsai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5917-2961","authenticated-orcid":false,"given":"Yu-Hsuan","family":"Fu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3561-1093","authenticated-orcid":false,"given":"Liang-In","family":"Lin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2780-4845","authenticated-orcid":false,"given":"Hsin-An","family":"Hou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,7,10]]},"reference":[{"issue":"11","key":"1785_CR1","doi-asserted-by":"publisher","first-page":"1274","DOI":"10.1182\/blood.2019000948","volume":"136","author":"A Iolascon","year":"2020","unstructured":"Iolascon A, Andolfo I, Russo R. Congenital dyserythropoietic anemias. Blood. 2020;136(11):1274\u201383. https:\/\/doi.org\/10.1182\/blood.2019000948.","journal-title":"Blood"},{"issue":"3","key":"1785_CR2","doi-asserted-by":"publisher","first-page":"436","DOI":"10.1111\/bjh.15817","volume":"185","author":"NBA Roy","year":"2019","unstructured":"Roy NBA, Babbs C. The pathogenesis, diagnosis and management of congenital dyserythropoietic anaemia type I. Br J Haematol. 2019;185(3):436\u201349. https:\/\/doi.org\/10.1111\/bjh.15817.","journal-title":"Br J Haematol"},{"issue":"4","key":"1785_CR3","doi-asserted-by":"publisher","first-page":"929","DOI":"10.1111\/j.1365-2141.1995.tb08442.x","volume":"89","author":"T Lavabre-Bertrand","year":"1995","unstructured":"Lavabre-Bertrand T, Blanc P, Navarro R, Saghroun M, Vannereau H, Braun M, Wagner A, Taib J, Lavabre-Bertrand C, Navarro M. Alpha-interferon therapy for congenital dyserythropoiesis type I. Br J Haematol. 1995;89(4):929\u201332. https:\/\/doi.org\/10.1111\/j.1365-2141.1995.tb08442.x.","journal-title":"Br J Haematol"},{"issue":"5","key":"1785_CR4","doi-asserted-by":"publisher","first-page":"672","DOI":"10.1002\/ajh.25058","volume":"93","author":"R Russo","year":"2018","unstructured":"Russo R, Andolfo I, Manna F, Gambale A, Marra R, Rosato BE, Caforio P, Pinto V, Pignataro P, Radhakrishnan K, Unal S, Tomaiuolo G, Forni GL, Iolascon A. Multi-gene panel testing improves diagnosis and management of patients with hereditary anemias. Am J Hematol. 2018;93(5):672\u201382. https:\/\/doi.org\/10.1002\/ajh.25058.","journal-title":"Am J Hematol"},{"issue":"2","key":"1785_CR5","doi-asserted-by":"publisher","first-page":"318","DOI":"10.1111\/bjh.14221","volume":"175","author":"NB Roy","year":"2016","unstructured":"Roy NB, Wilson EA, Henderson S, Wray K, Babbs C, Okoli S, Atoyebi W, Mixon A, Cahill MR, Carey P, Cullis J, Curtin J, Dreau H, Ferguson DJ, Gibson B, Hall G, Mason J, Morgan M, Proven M, Qureshi A, Sanchez Garcia J, Sirachainan N, Teo J, Tedgard U, Higgs D, Roberts D, Roberts I, Schuh A. A novel 33-gene targeted resequencing panel provides accurate, clinical-grade diagnosis and improves patient management for rare inherited anaemias. Br J Haematol. 2016;175(2):318\u201330. https:\/\/doi.org\/10.1111\/bjh.14221.","journal-title":"Br J Haematol"},{"issue":"2","key":"1785_CR6","doi-asserted-by":"publisher","first-page":"133","DOI":"10.1080\/08880010590907221","volume":"22","author":"RK Marwaha","year":"2005","unstructured":"Marwaha RK, Bansal D, Trehan A, Garewal G. Interferon therapy in congenital dyserythropoietic anemia type I\/II. Pediatr Hematol Oncol. 2005;22(2):133\u20138. https:\/\/doi.org\/10.1080\/08880010590907221.","journal-title":"Pediatr Hematol Oncol"},{"issue":"2","key":"1785_CR7","doi-asserted-by":"publisher","first-page":"295","DOI":"10.1046\/j.1365-2141.1997.1993016.x","volume":"98","author":"SN Wickramasinghe","year":"1997","unstructured":"Wickramasinghe SN, Hasan R, Smythe J. Reduced interferon-alpha production by Epstein-Barr virus transformed B-lymphoblastoid cell lines and lectin-stimulated lymphocytes in congenital dyserythropoietic anaemia type I. Br J Haematol. 1997;98(2):295\u20138. https:\/\/doi.org\/10.1046\/j.1365-2141.1997.1993016.x.","journal-title":"Br J Haematol"},{"issue":"4","key":"1785_CR8","doi-asserted-by":"publisher","first-page":"621","DOI":"10.1007\/s12288-016-0765-9","volume":"33","author":"V Agrigento","year":"2017","unstructured":"Agrigento V, Barone R, Sclafani S, Di Maggio R, Sacco M, Maggio A, D\u2019Alcamo E. Response to alpha-interferon treatment of the congenital dyserythropoietic anemia type I in two Sicilian beta thalassemia carriers. Indian J Hematol Blood Transfus. 2017;33(4):621\u20133. https:\/\/doi.org\/10.1007\/s12288-016-0765-9.","journal-title":"Indian J Hematol Blood Transfus"},{"issue":"5","key":"1785_CR9","doi-asserted-by":"publisher","first-page":"380","DOI":"10.1111\/j.1600-0609.2004.00310.x","volume":"73","author":"T Lavabre-Bertrand","year":"2004","unstructured":"Lavabre-Bertrand T, Ramos J, Delfour C, Henry L, Guiraud I, Carillo S, Wagner A, Bureau JP, Blanc P. Long-term alpha interferon treatment is effective on anaemia and significantly reduces iron overload in congenital dyserythropoiesis type I. Eur J Haematol. 2004;73(5):380\u20133. https:\/\/doi.org\/10.1111\/j.1600-0609.2004.00310.x.","journal-title":"Eur J Haematol"},{"issue":"3","key":"1785_CR10","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.26866","volume":"65","author":"M Rathe","year":"2018","unstructured":"Rathe M, Moller MB, Greisen PW, Fisker N. Successful management of transfusion-dependent congenital dyserythropoietic anemia type 1b with interferon alfa-2a. Pediatr Blood Cancer. 2018;65(3): e26866. https:\/\/doi.org\/10.1002\/pbc.26866.","journal-title":"Pediatr Blood Cancer"},{"issue":"2","key":"1785_CR11","doi-asserted-by":"publisher","first-page":"121","DOI":"10.1111\/j.1600-0609.1997.tb00935.x","volume":"58","author":"SN Wickramasinghe","year":"1997","unstructured":"Wickramasinghe SN. Response of CDA type I to alpha-interferon. 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