2019 Volume 56 Issue 3 Pages 348-352
A 9-month-old male infant with congenital amegakaryocytic thrombocytopenia underwent unrelated bone marrow transplantation (BMT). Hematological engraftment was prompt, and serial chimerism analyses showed sustained complete chimerism on and after day 91. He developed autoimmune hemolytic anemia (AIHA) on day 56. Prednisolone and intravenous immunoglobulin (IVIG) did not improve his symptoms with persistent transfusion dependence. Treatment with rituximab on day 69, followed by the addition of the mTOR inhibitor everolimus on day 102, resulted in the gradual improvement of his anemia. The incidence of steroid- and IVIG-refractory AIHA has recently been increasing among pediatric patients undergoing hematopoietic stem cell transplantation. Therefore, it is necessary to reassess the treatment algorithm for these patients, such as the incorporation of rituximab or mTOR inhibitors into first-line treatment.
