Yang, Tyng-WeiTyng-WeiYangTsai, Xavier Cheng-HongXavier Cheng-HongTsaiFu, Yu-HsuanYu-HsuanFuLIANG-IN LINHSIN-AN HOU2025-09-232025-09-232025-07-1015918890https://scholars.lib.ntu.edu.tw/handle/123456789/732277Congenital dyserythropoietic anemia type I (CDA-I) is a rare hereditary anemia caused by CDAN1 or C15orf41 mutations, with CDAN1-related cases responding to interferon-alpha (IFN-α) therapy. However, traditional IFN-α 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-α 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.entrueCDACDAN1Ropeginterferon alfa-2b[SDGs]SDG3journal article10.1007/s10238-025-01785-x406404732-s2.0-105010534018