Refractory severe aplastic anemia in a child: treatment failure and access barriers in a low-resource setting

Aplastic anemia (AA) is a rare but potentially fatal hematologic disorder in children, marked by bone marrow failure and pancytopenia. Early diagnosis and access to curative treatment such as hematopoietic stem cell transplantation (HSCT) are crucial. In low-resource settings, limited access to HSCT poses significant challenges.

We present a 7-year-old girl with spontaneous ecchymoses and purpura following an upper respiratory infection. Laboratory tests revealed severe pancytopenia, and bone marrow biopsy showed <5% cellularity, consistent with severe AA. Infectious, autoimmune, and malignant causes were excluded. She received immunosuppressive therapy with rabbit anti-thymocyte globulin, cyclosporine A, and corticosteroids but failed to show hematologic recovery. HSCT was not an option due to financial constraints, and she remained transfusion-dependent.

AA is often immune-mediated, and IST is a standard option when HSCT is unavailable. However, responses vary, particularly in pediatric cases. This case illustrates the limited effectiveness of IST in some patients and highlights how socioeconomic barriers hinder access to definitive treatment, impacting outcomes.

This case underscores the urgent need for affordable curative therapies like HSCT in low-income settings. It also emphasizes the variable response to IST and the role of systemic inequities in limiting care for children with life-threatening hematologic diseases.