Prospective Studies on New Agents in PCNSL Identified by a Systematic Literature Search
| Reference . | Year . | Type of publication . | NCT identifier . | Study design . | Sample size . | Population . | Intervention . | Outcome . | Comment . |
|---|---|---|---|---|---|---|---|---|---|
| 19 | 2017 | Article | NCT02315326 | Prospective, single center, no RCT | 20 | r/r PCNSL and SCNSL | Ibrutinib | 77% ORR, median PFS 4.6 mo, median OS 15 mo | First full publication on single-agent ibrutinib in r/r PCNSL and SCNSL and comprehensive analyses on mutational landscape in PCNSL |
| 28 | 2017 | Article | NCT02203526 | Prospective, single center, no RCT | 18 | De novo PCNSL, r/r PCNSL and SCNSL | Ibrutinib + chemotherapy (TEDDI-R) | 89% ORR, median PFS 15.3 mo, median OS not reached | Novel polyimmunochemotherapy combined with ibrutinib; high morbidity rate, especially fungal infections |
| 51 | 2017 | Abstract | Unclear | Prospective, single center, no RCT | 9 | r/r PCNSL and SCNSL | Lenalidomide + rituximab maintenance | NA | Small series on lenalidomide maintenance after HD-MTX–based induction |
| 52 | 2017 | Abstract | NCT01722305 | Prospective multicenter, no RCT | 25 | r/r PCNSL and IOL | Pomalidomide + dexamethasone followed by pomalidomide maintenance | 43% ORR, PFS and OS not reported yet | First study investigating pomalidomide in r/r PCNSL; full study publication needs to be awaited regarding appraisal of long-term remissions |
| 31 | 2017 | Abstract | NCT02315326 | Prospective bicentric, no RCT | 6 | r/r PCNSL and SCNSL | Ibrutinib + HD-MTX + rituximab | Four of 6 patients showed a response | Study investigating ibrutinib in combination with HD-MTX and rituximab in r/r CNS lymphoma |
| 50 | 2016 | Abstract | NCT01542918 | Prospective, single center, no RCT | 13 | r/r PCNSL and SCNSL | Lenalidomide | 8 of 13 achieved a response | Study showing activity of lenalidomide in r/r PCNSL |
| 26 | 2016 | Abstract | NCT02542514 | Prospective multicenter, no RCT | 52 | r/r PCNSL and IOL | Ibrutinib | 50% ORR, median PFS 8.1 mo, median OS 19.2 mo | Largest prospective study investigating ibrutinib single agent in r/r PCNSL; benchmark for future ibrutinib studies |
| 36 | 2016 | Article | NCT00942747 | Prospective multicenter, no RCT | 37 | r/r PCNSL | Temsirolimus | 54% ORR, PFS and OS not reported | Only prospective study that investigated mTOR inhibition in PCNSL, but results are not promising |
| 49 | 2016 | Abstract | NCT01956695 | Prospective multicenter, no RCT | 50 | r/r PCNSL and IOL | Lenalidomide + rituximab | 67% ORR, median PFS 8.1 mo, median OS 19.2 mo | Largest prospective study investigating this combination with promising activity, benchmark for future lenalidomide containing approached in r/r PCNSL |
| 37 | 2016 | Abstract | NCT02301364 | Prospective single center, no RCT | 4 | r/r PCNSL and SCNSL | Buparlisib single agent | One patient with PR | Buparlisib did not reach meaningful concentrations in the CNS |
| Reference . | Year . | Type of publication . | NCT identifier . | Study design . | Sample size . | Population . | Intervention . | Outcome . | Comment . |
|---|---|---|---|---|---|---|---|---|---|
| 19 | 2017 | Article | NCT02315326 | Prospective, single center, no RCT | 20 | r/r PCNSL and SCNSL | Ibrutinib | 77% ORR, median PFS 4.6 mo, median OS 15 mo | First full publication on single-agent ibrutinib in r/r PCNSL and SCNSL and comprehensive analyses on mutational landscape in PCNSL |
| 28 | 2017 | Article | NCT02203526 | Prospective, single center, no RCT | 18 | De novo PCNSL, r/r PCNSL and SCNSL | Ibrutinib + chemotherapy (TEDDI-R) | 89% ORR, median PFS 15.3 mo, median OS not reached | Novel polyimmunochemotherapy combined with ibrutinib; high morbidity rate, especially fungal infections |
| 51 | 2017 | Abstract | Unclear | Prospective, single center, no RCT | 9 | r/r PCNSL and SCNSL | Lenalidomide + rituximab maintenance | NA | Small series on lenalidomide maintenance after HD-MTX–based induction |
| 52 | 2017 | Abstract | NCT01722305 | Prospective multicenter, no RCT | 25 | r/r PCNSL and IOL | Pomalidomide + dexamethasone followed by pomalidomide maintenance | 43% ORR, PFS and OS not reported yet | First study investigating pomalidomide in r/r PCNSL; full study publication needs to be awaited regarding appraisal of long-term remissions |
| 31 | 2017 | Abstract | NCT02315326 | Prospective bicentric, no RCT | 6 | r/r PCNSL and SCNSL | Ibrutinib + HD-MTX + rituximab | Four of 6 patients showed a response | Study investigating ibrutinib in combination with HD-MTX and rituximab in r/r CNS lymphoma |
| 50 | 2016 | Abstract | NCT01542918 | Prospective, single center, no RCT | 13 | r/r PCNSL and SCNSL | Lenalidomide | 8 of 13 achieved a response | Study showing activity of lenalidomide in r/r PCNSL |
| 26 | 2016 | Abstract | NCT02542514 | Prospective multicenter, no RCT | 52 | r/r PCNSL and IOL | Ibrutinib | 50% ORR, median PFS 8.1 mo, median OS 19.2 mo | Largest prospective study investigating ibrutinib single agent in r/r PCNSL; benchmark for future ibrutinib studies |
| 36 | 2016 | Article | NCT00942747 | Prospective multicenter, no RCT | 37 | r/r PCNSL | Temsirolimus | 54% ORR, PFS and OS not reported | Only prospective study that investigated mTOR inhibition in PCNSL, but results are not promising |
| 49 | 2016 | Abstract | NCT01956695 | Prospective multicenter, no RCT | 50 | r/r PCNSL and IOL | Lenalidomide + rituximab | 67% ORR, median PFS 8.1 mo, median OS 19.2 mo | Largest prospective study investigating this combination with promising activity, benchmark for future lenalidomide containing approached in r/r PCNSL |
| 37 | 2016 | Abstract | NCT02301364 | Prospective single center, no RCT | 4 | r/r PCNSL and SCNSL | Buparlisib single agent | One patient with PR | Buparlisib did not reach meaningful concentrations in the CNS |
IOL, intraocular lymphoma; NA, not applicable; ORR, overall response rate; RCT, randomized clinical trial; SCNSL, secondary CNS lymphoma.