2020 Volume 57 Issue 2 Pages 80-84
[Introduction] Challenges are associated with treating acute leukemia that relapsed in the central nervous system (CNS) after conventional modalities have been administered. [Case] A 10-year-old girl was diagnosed with mixed-phenotype acute leukemia with BCR-ABL1. The disease was resistant to induction chemotherapy, and re-emerged after a short success with dasatinib. She underwent bone marrow transplantation (BMT) from her father. The disease relapsed in the CNS when she was 12 years old. The CNS overt relapse was successfully treated by cerebrospinal irradiation, and minimal residual disease in the bone marrow went into molecular remission following the intravenous infusion of peripheral blood lymphocytes from her mother (a non-BMT donor). When she was 13 years old, the disease relapsed in the CNS again. Intrathecal infusion of maternal lymphocytes was initiated. Maternally derived cells in the cerebrospinal fluid (CSF) were replaced with paternal-type lymphocytes, BCR-ABL1-positive leukemic cells were no longer detected 1 week after the first trial, and symptoms disappeared. These effects lasted for 2 months. [Discussion] Paternal-type lymphocytes in the CSF were activated and presumably recruited from the peripheral blood of the patient. This intrathecal cell therapy exerts similar effects to cytokine therapy and may overcome the anergy of BMT-donor-derived lymphocytes against leukemic cells. [Conclusions] The current treatment was safe and effective. It also provides insights into the mechanisms underlying the significance of cell-immune therapy.