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Table 3 Novel Targeted Therapies for CNS Lymphoma

From: Current and emerging therapies for primary central nervous system lymphoma

Author Year Diagnosis N Study Age (year) Regimen Outcome
BTKi
 Grommes C. et al [70] 2017 PCNSL/SCNSL, r/r 20 phase I 21-85 ibrutinib monotherapy ORR: 10/13, CR 5, PR 5
 Lionakis MS. et al [14] 2017 PCNSL, ND and r/r 18 phase Ib 49-87 ibrutinib + DA-TEDDi-R CR+CRu 16/18
Soussain C. et al [71] 2019 PCNSL/PVRL, r/r 52 phase II 47-82 ibrutinib monotherapy ORR: 59%, mean PFS 4.8m, mean OS 19.2m
 Grommes C. et al [72] 2019 PCNSL/SCNSL, r/r 15 phase Ib 23-74 ibrutinib + MTX+R ORR: 12/15
 Chen F. et al [73] 2020 PCNSL, ND 11 retrospective 41-68 Ibrutinib+MTX ORR 81.8%
 Narita Y. et al [74] 2021 PCNSL, r/r 44 phase I/II 29-86 tirabrutinib monotherapy >60% at 320 mg, 100% at 480 mg, and 53% at 480 mg (fasted)
IMids
 Rubenstein JL. et al [42] 2018 PCNSL/SCNSL, r/r 14 phase I 47-79 lenalidomide or lenalidomide +R ORR: 64%
 Tun HW. et al [15] 2018 PCNSL/PVRL, r/r 25 phase I adults pomalidomide+DXM ORR: 48%
 Vu K. et al [75] 2019 PCNSL/SCNSL 13 retrospective 70-86 lenalidomide in maintenance mean PFS: not reached
 Ghesquieres H.et al [76] 2019 PCNSL/PVRL, r/r 50 phase II 46-86 lenalidomide+R ORR: 35.6%
PD-1 antibody
 Nayak L. et al [77] 2017 PCNSL/SCNSL, r/r 5 case report 54-85 nivolumab+R/WBRT CR 4, PR 1
BBB permeabilization
 Ferreri AJM. et al [78] 2020 PCNSL, r/r 28 phase II 26-78 NGR-hTNF/R-CHOP ORR: 75%; CR 11, PR 10
CAR-T
 Tu S. et al [79] 2019 PCNSL, r/r 1 case report 67 CD19-CD70 dual CART CR
 Abbasi A. et al [80] 2020 SCNSL, r/r 2 retrospective NA axicabtagene ciloleucel CR 2
 Abramson JS. et al [81] 2020 SCNSL, r/r 7 prospective NA lisocabtagene maraleucel CR 3 (of 6 evaluated)
  1. Abbreviations: CART chimeric antigen receptor T cells; CNS central nervous system; CR complete remission; CRu complete remission unconfirmed; DA-TEDDi-R etoposide, temozolomide, liposomal doxorubicin, dexamethasone, intrathecal cytarabinem; DXM dexamethasone; MTX methotrexate; NA not available; ND newly diagnosed; NGR-hTNF tumor necrosis factor-α coupled with CNGRCG peptide; ORR overall response rate, OS overall survival, PCNSL primary central nervous system lymphoma; PFS progression-free survival; PR partial remission, PVRL primary vitreoretinal lymphoma; R rituximab; r/r refractory/relapse; SCNSL secondary central nervous system lymphoma; WBRT whole brain radiotherapy