Blood cancer journal (New York), 2019-01-22, Vol.9 (2), p.10-10
Outcomes of pediatric and young adult patients diagnosed with acute lymphoblastic leukemia (ALL) have improved significantly in the past few decades. Treatment advances have provided 5-year survival rates ranging from 78 to 91% depending on the age at diagnosis. However, approximately 2-3% of patients will present with refractory disease that is unresponsive to chemotherapy, and 10-15% of patients will relapse. Outcomes post-relapse show significantly reduced 5-year survival rates that continue to decrease with each subsequent relapse. Despite our increased understanding of risk factors and disease predictors, treatment strategies for patients with relapsed or refractory (r/r) disease, including variations of chemotherapy and stem cell transplant, remain ineffective for many patients. To improve outcomes of patients with r/r disease, immunotherapies targeting specific B cell antigens are being developed. Tisagenlecleucel is an autologous anti-CD19 chimeric antigen receptor (CAR) T cell therapy recently approved by the US Food and Drug Administration for patients with refractory leukemia or those with second or later relapse. In this treatment strategy, a patient's own T cells are transduced to express an anti-CD19 CAR that, when reintroduced into the patient, directs specific binding and killing of CD19+ B cells. In a phase 2, single-arm, multicenter, global study, tisagenlecleucel resulted in a remission rate of 81% in pediatric and adolescent patients with r/r B cell ALL. This review article summarizes four typical cases of pediatric and adolescent r/r B-cell ALL, focusing on the patient's journey from initial diagnosis to treatment with CAR T cell therapy.
Recurrence ; Immunotherapy, Adoptive - methods ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - immunology ; Receptors, Antigen, T-Cell - metabolism ; Humans ; Treatment Outcome ; Combined Modality Therapy ; Stem Cell Transplantation ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy ; T-Lymphocytes - metabolism ; Receptors, Chimeric Antigen - genetics ; T-Lymphocytes - immunology ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - pathology ; Receptors, Chimeric Antigen - metabolism ; Pediatrics ; Chemotherapy ; Young adults ; Lymphocytes ; Patients ; Leukemia ; Index Medicus ; Review
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