Table 1.

Early-phase investigations in relapsed PCNSL/SCNSL

Agent/studyDesignResponse ratePFSComments
Ibrutinib NCT02315326 MSKCC phase 1 in relapsed PCNSL/SCNSL 75% 4.6 mo. One case pulmonary aspergillosis; ibrutinib resistance with CARD11 mutation. 
Ibrutinib NCT02542514 French LOC network phase 2 in relapsed PCNSL and IOL 56%  One case pulmonary aspergillosis; final analysis pending 
DA-TEDDi-R with ibrutinib monotherapy NCT02203526 NCI phase 1 in newly diagnosed and relapsed PCNSL 94% ibrutinib 86% CR rate with TEDDiR  39% rate of invasive aspergillosis in lungs and brain; 28% treatment-related mortality. 
Temsirolimus NCT00942747 Phase 1/2 in relapsed PCNSL 54% 2.1 mo. 13.5% treatment-related mortality (pneumonia) 
Nivolumab NCT02857426 Phase 2 in recurrent, PCNSL and primary testicular NHL   In progress 
Lenalidomide/rituximab NCT01542918 UCSF phase 1 in relapsed PCNSL/SCNSL 64% 6 mo. Independent, retrospective cohort: 12-fold increase in response duration in CR2-4 with lenalidomide maintenance compared with CR1 in relapsed PCNSL/SCNSL 
Lenalidomide/rituximab NCT01956695 French LOC network phase 2 in relapsed PCNSL/PVRL 63% 8.9 mo. Final analysis pending 
Pomalidomide/dex NCT01722305 Phase 1 in relapsed PCNSL or newly diagnosed or relapsed IOL   In Progress 
Agent/studyDesignResponse ratePFSComments
Ibrutinib NCT02315326 MSKCC phase 1 in relapsed PCNSL/SCNSL 75% 4.6 mo. One case pulmonary aspergillosis; ibrutinib resistance with CARD11 mutation. 
Ibrutinib NCT02542514 French LOC network phase 2 in relapsed PCNSL and IOL 56%  One case pulmonary aspergillosis; final analysis pending 
DA-TEDDi-R with ibrutinib monotherapy NCT02203526 NCI phase 1 in newly diagnosed and relapsed PCNSL 94% ibrutinib 86% CR rate with TEDDiR  39% rate of invasive aspergillosis in lungs and brain; 28% treatment-related mortality. 
Temsirolimus NCT00942747 Phase 1/2 in relapsed PCNSL 54% 2.1 mo. 13.5% treatment-related mortality (pneumonia) 
Nivolumab NCT02857426 Phase 2 in recurrent, PCNSL and primary testicular NHL   In progress 
Lenalidomide/rituximab NCT01542918 UCSF phase 1 in relapsed PCNSL/SCNSL 64% 6 mo. Independent, retrospective cohort: 12-fold increase in response duration in CR2-4 with lenalidomide maintenance compared with CR1 in relapsed PCNSL/SCNSL 
Lenalidomide/rituximab NCT01956695 French LOC network phase 2 in relapsed PCNSL/PVRL 63% 8.9 mo. Final analysis pending 
Pomalidomide/dex NCT01722305 Phase 1 in relapsed PCNSL or newly diagnosed or relapsed IOL   In Progress 

IOL, intraocular lymphoma; NHL, non-Hodgkin lymphoma; PVRL, primary vitreoretinal lymphoma.

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