Background: The prognosis of primary intraocular lymphoma (PIOL) is poor. Due to frequent ocular and/or CNS relapses, the optimal treatment for PIOL has not yet been defined. This is the first report of PIOL treatment with trofosfamide.
Methods: The first patient with a newly diagnosed PIOL was not considered eligible for high-dose methotrexate-based systemic chemotherapy due to comorbidities. The second patient had a primary central nervous system lymphoma, which was resistant to three chemotherapy regimens and responded to whole-brain irradiation. A tumor relapse in the eye was treated with local radiotherapy with initial success; however, the tumor subsequently recurred. The first patient received oral trofosfamide 150 mg daily, the second 400 mg daily (reduced after 5 months of therapy to 300 mg daily) over 5 consecutive days, followed by a 5-day drug-free interval.
Results: In both patients, complete remission was achieved, which lasted for 8+ and 18 months, respectively. Except for a temporary asymptomatic decrease in hemoglobin in the first patient, no toxicity was observed.
Conclusions: Trofosfamide may offer an alternative treatment option for PIOL with a very favorable side effect profile. The exact role of trofosfamide in the treatment of PIOL remains to be defined.