# Ropeginterferon alfa-2b as a promising alternative to conventional interferon in CDA type 1: a case report of two siblings

## TL;DR

A new long-acting interferon improved treatment for a rare anemia in two siblings, reducing the need for blood transfusions and side effects.

## Contribution

First documented use of ropeginterferon alfa-2b in CDA-I patients, showing improved efficacy and tolerability.

## Key findings

- Two CDA-I patients achieved transfusion independence after switching to ropeginterferon alfa-2b.
- Biweekly dosing reduced injection burden and adverse effects compared to conventional interferon.
- Hemoglobin levels improved while maintaining treatment efficacy.

## Abstract

Congenital 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.