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Medline ® Abstract for Reference 45

of 'Treatment of relapsed or refractory acute lymphoblastic leukemia in adults'

High single-drug activity of nelarabine in relapsed T-lymphoblastic leukemia/lymphoma offers curative option with subsequent stem cell transplantation.
Gökbuget N, Basara N, Baurmann H, Beck J, Brüggemann M, Diedrich H, Güldenzoph B, Hartung G, Horst HA, Hüttmann A, Kobbe G, Naumann R, Ratei R, Reichle A, Serve H, Stelljes M, Viardot A, Wattad M, Hoelzer D
Blood. 2011;118(13):3504. Epub 2011 Jun 28.
Nelarabine, a purine analog with T-cell specific action, has been approved for relapsed/refractory T-cell acute lymphoblastic leukemia/lymphoma (ALL/LBL). This is a report of a single-arm phase 2 study conducted in adults (18-81 years of age) with relapsed/refractory T-ALL/LBL. After 1 or 2 cycles, 45 of 126 evaluable patients (36%) achieved complete remission (CR), 12 partial remission (10%), and 66 (52%) were refractory. One treatment-related death was observed, and 2 patients were withdrawn before evaluation. A total of 80% of the CR patients were transferred to stem cell transplantation (SCT). Overall survival was 24% at 1 year (11% at 6 years). After subsequent SCT in CR, survival was 31% and relapse-free survival 37% at 3 years. Transplantation-related mortality was 11%. Neurologic toxicities of grade I-IV/grade III-IV were observed in 13%/4% of the cycles and 16%/7% of the patients. This largest study so far with nelarabine in adults showed impressive single-drug activity in relapsed T-ALL/T-LBL. The drug was well tolerated, even in heavily pretreated patients. A high proportion of CR patients were transferred to SCT with low mortality but a high relapse rate. Exploration of nelarabine in earlier stages of relapse (eg, increasing minimal residual disease), in front-line therapy, and in combination is warranted.
Department of Medicine II, Hematology/Oncology, Goethe University Hospital, Frankfurt, Germany. goekbuget@em.uni-frankfurt.de