Table 5.
Summary of clinical investigations of novel therapies.
| Reference | Regimen | Disease Status | Study Type | n | Result |
|---|---|---|---|---|---|
| [74] | Tofacitinib and ruxolitinib |
Salvage | Case Report | 1 | Patient had 10 months of partial response followed by eventual progression |
| [75] | Olaparib | Salvage | Phase I clinical trial | 2 | Patient A: treated for 133 days w/disease progression. Patient B withdrew after 14 days given dose limiting toxicity |
| [76] | Venetoclax | Salvage | Case Report | 2 | Patient A: partial response however died with fulminant sepsis 18 days after treatment initiation. Patient B: partial response, at 131 days on venetoclax, disease relapse |
| [77] | Venetoclax and ibrutinib |
Salvage | Case Report | 2 | Patient A: treated for 40 days. Course was complicated by influenza A infection and patient transitioned to comfort care. Patient B: treated for 80 days; however, patient died of bacterial pneumonia. |
| [78] | Venetoclax and ruxolitinib |
Salvage | Case Report | 2 | Patient A: 10 months of partial response. Patient B: progression on day 127 following therapy, then lost to follow-up. |
| [78] | Venetoclax and romidepsin |
Salvage | Case Report | 1 | Partial response ongoing, 9 months, at time of publication of case. |
| [79] | Venetoclax and ruxolitinib |
Salvage (n = 14) Frontline (n = 1) |
Multicenter retrospective study | 15 | ORR 73% (all were partial response), median PFS 1.8 months in wild-type JAK/STAT vs. 5.6 months in mutated JAK/STAT pathway ‡ |
| [80] | Venetoclax; 5 patients received bendamustine | Salvage | Single-center retrospective study | 9 | Disease control rate 56% (best response was PR in 4 and stable disease in 1) |
| [81] | Alemtuzumab and cladribine, some received vorinostat, romidepsin, and brentuximab | Salvage (n = 6) Frontline (n = 2) |
Single-center prospective study | 8 | ORR 100% (88% CR, 12% PR), median OS 25.7 months, 38% were alive at time of publication. |
| NCT03873493 | Venetoclax and Ibrutinib | Salvage | Phase II Clinical Trial | 14 | ORR 7.1%, median PFS 2.7 months |
| NCT01186640 | FMC-subcutaneous alemtuzumab induction followed by alemtuzumab | Salvage (n = 4) Frontline (n = 12) |
Phase II Clinical Trial | 16 | ORR 68.8% (31.3% CR, 37.5% PR), median OS 16.7 months, median PFS 11.2 months |
| NCT04496349 | APG-115 (MDM2 inhibitor) +/− APG-2575 (Bcl-2 inhibitor) | Salvage | Phase II Clinical Trial | 36 † | Recruiting; estimated study completion date: 31 May 2024 |
| NCT03989466 | Alemtuzumab and Itacitinib (JAK1 inhibitor) |
Frontline or salvage | Phase I Clinical Trial | 15 † | Recruiting; estimated study completion date: 21 December 2023 |
‡ p < 0.05; † estimated enrollment.