Durable Survival Outcomes in Primary and Secondary Central Nervous System Lymphoma Following High-Dose Chemotherapy and Autologous Stem Cell Transplant Using a Thiotepa, Busulfan, and Cyclophosphamide Conditioning Regimen

2020 
Abstract Background High-dose chemotherapy with autologous stem cell transplantation (HDC/ASCT) has been investigated in patients with primary central nervous system lymphoma (PCNSL) or non-Hodgkin lymphoma (NHL) with CNS involvement with promising results. Patients and Methods A retrospective analysis was performed among 48 consecutive patients undergoing HDC/ASCT with thiotepa, busulfan, and cyclophosphamide (TBC) conditioning for PCNSL (27 patients) or secondary CNS lymphoma (SCNSL)/CNS relapse (21 patients) between July 2006 and December 2017. Twenty-one of the patients with PCNSL underwent ASCT in CR1 Results The 2-year PFS rate was 80.5% (95% CI 69.9-92.9) and 2-year OS rate was 80.1% (95% CI 69.2-92.7) among all patients. The 2-year PFS and OS rate for PCNSL patients in CR1 was 95.2% (95% CI 86.6-100) and 95.2% (95% CI 86.6-100), respectively. In univariate analysis of PCNSL patients, ASCT in CR1 was the only variable noted to be significant for outcome (p = 0.007 for PFS; p = 0.008 for OS). Among patients with SCNSL/CNS relapse, the 2-year PFS and OS rate were comparable at 75.9% (95% CI 59.5-96.8) and 75.3% (95% CI 58.6-98.6), respectively. The most common side effects were febrile neutropenia (89.6%), of which 66.7% had an infectious etiology identified, nausea/vomiting (85.4%), diarrhea (93.8%), mucositis (89.6%), and electrolyte abnormalities (89.6%). Four patients (8.3%) experienced treatment-related mortality from overwhelming infection, of which 3 had SCNSL Conclusion High-dose chemotherapy and autologous stem cell transplant using TBC conditioning for both PCNSL and secondary CNS non-Hodgkin lymphoma appears to have encouraging long term efficacy with manageable side effects.
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