2009

ABSTRACT Methods Study design and end points In this prospective multicenter phase II study, the effect of three courses of vincristine, doxorubicin, dexamethasone (VAD) administered as rapid infusion 9,10 followed by HDM with ASCT was evaluated in AL amyloidosis. Patients who did not meet the eligibility criteria for HDM after VAD went off protocol treatment and were followed for survival. Study end points were efficacy with regard to response rate, overall survival, feasibility and the value of risk factors at diagnosis. The trial was carried out in accordance with the Declaration of Helsinki. The protocol was approved by the Institutional Review Boards of all participating hospitals, and all patients provided written informed consent before inclusion in the study

Treatment of secondary central nervous system lymphoma with intrathecal rituximab, high-dose methotrexate, and R-DHAP followed by autologous stem cell transplantation: Results of the HOVON 80 phase 2 study

The prognosis of central nervous system (CNS) relapse of systemic non-Hodgkin lymphoma is poor with 1-year survival historically at 0% to 20%. Aiming to improve these results, we performed a multicenter phase 2 study in patients with a CNS relapse, with or without concurrent systemic relapse. Treatment consisted of 2 cycles of R-DHAP alternating with high-dose methotrexate (MTX) and was combined with intrathecal rituximab. Responding patients received a third R-DHAP-MTX cycle followed by busulfan and cyclophosphamide myeloablative therapy and autologous stem cell transplantation. In patients with persistent cerebrospinal fluid lymphoma after cycle 1, the intrathecal rituximab was replaced by intrathecal triple therapy, with MTX, cytarabine, and dexamethasone. Thirty-six patients were included. Eighteen had evidence of cerebrospinal fluid lymphoma, 24 had brain parenchymal disease, and 20 (56%) had concurrent systemic disease. The overall response rate after 2 R-DHAP-MTX was 53% (19/36), with 22% (8/36) complete remission. Fifteen patients (42%) underwent a transplant. One-year progression-free survival was 19% (95% confidence interval, 9-34): 25% in patients without and 15% in patients with systemic disease. One-year overall survival was 25% (95% confidence interval, 12-40). This treatment regimen did not result in a major improvement of outcome of secondary CNS lymphoma, especially when concurrent systemic disease was present