Pediatric Hematology Oncology Journal · Published 2026-02-26 · DOI 10.1016/j.phoj.2026.100812
Background: Paediatric autoimmune haemolytic anaemia (AIHA) is a rare but potentially life-threatening disorder. The clinical course ranges from acute, self-limiting episodes to chronic, relapsing disease. First-line therapy with corticosteroids works in most cases but some patients relapse and require prolonged administration of immunomodulators. A subset of children remain refractory to commonly used immunomodulators, posing significant management challenges, and novel agents such as proteasome inhibitors, including bortezomib have been explored to target autoantibody-producing plasma cells. We report two cases of chronic refractory AIHA where bortezomib achieved hemoglobin improvement and transfusion independence, supporting its role as a salvage option. Case presentation: We report two cases of refractory AIHA, a 6-year-old boy and a 10-year-old girl with relapsing AIHA for 2 years and 2.5 years respectively, who initially responded to methylprednisolone and intravenous immunoglobulin (IVIG), but later became steroid-resistant. They subsequently received rituximab, mycophenolate mofetil (MMF), and azathioprine sequentially, but still required transfusions. Evaluation for inborn errors of immunity were negative in both cases, and they eventually received bortezomib with an excellent response. After 10 doses in the first case and 8 doses in the second case, hemoglobin stabilized at 11 g/dL and 10.6 g/dL remaining transfusion-independent for 3 months and 2 months respectively. Conculsion: This report illustrates the successful use of bortezomib in two children with chronic, refractory AIHA unresponsive to conventional therapies. In the absence of long-term follow up, the durability of such response and potential side effects of this therapy in children remains unknown, and a prospective clinical trial or registry would greatly clarify the role of this agent in pediatric refractory AIHA.
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