Article
Steroid Responsiveness and Clinical Outcomes in Diamond-Blackfan Anemia: Analysis From the Canadian Inherited Marrow Failure Registry.
European journal of haematology - 1 Jul 2026
Choi Young Bae, Mathew Rinu, Li Hongbing, Cada Michaela, Klaassen Robert J, Villeneuve Stephanie, Abish Sharon, Corriveau-Bourque Catherine, Robitaille Nancy, Steele MacGregor, Sinha Roona, Rayar Meera, Wheaton Laura, Breakey Vicky, Goodyear Lisa, Crooks Bruce, Tole Soumitra, Dror Yigal
Abstract excerpt
Diamond-Blackfan anemia (DBA) is a rare inherited bone marrow failure syndrome characterized by erythroid aplasia, congenital anomalies, and cancer predisposition. Although corticosteroids are the standard first-line therapy for transfusion-dependent patients, treatment response varies, and reliable predictors remain undefined. We retrospectively analyzed data from the Canadian Inherited Marrow Failure Registry...
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