Table 1.
Summary of studies and ongoing clinical trials on BPDCN patients evaluating conventional and targeted therapies.
| Conventional and Approved Therapies | ||||||
|---|---|---|---|---|---|---|
| Treatments Methods | n | CR n (%) | Relapse n (%) | OS (Mon) | PFS (%) | References |
| ALL-type | 35 | n.r. | n.r. | n.r. | at 2 years: 40 | Taylor et al. [5] |
| AML-type | 9 | n.r. | n.r. | n.r. | at 2 years: 11 | |
| ALL-type + allo-HSCT | 33 | 31 (94) | 4 (13) | n.r. | n.r. | Laribi et al. [7] |
| NHL-type + allo-HSCT | 12 | 12 (100) | 4 (33) | n.r. | n.r. | |
| AML-type + allo-HSCT | 16 | 14 (88) | 2 (58) | n.r. | n.r. | |
| NHL/ALL/AML-type + auto-HSCT | 16 | n.r. | 5 (31) | n.r. | n.r. | |
| AML-type | 19 | 13 (68.4) | 4 (28.5) | 18 | n.r. | Garnache-Ottou et al. [11] |
| ALL-type | 15 | 15 (78.9) | 5 (33.3) | 15 | n.r. | |
| Aspa-MTX | 16 | 12 (75) | 4 (33.3) | 15 | n.r. | |
| CHOP-type | 16 | 6 (37.5) | 4 (66.7) | 11 | n.r. | |
| allo-HSCT | 30 | n.r. | 10 (33) | 49 | n.r. | |
| auto-HSCT | 4 | n.r. | 4 (100) | n.r. | ||
| NHL-type | 10 | 5 (50) | n.r. | n.r. | n.r. | Yun et al. [6] |
| ALL-type | 11 | 10 (91) | n.r. | n.r. | n.r. | |
| AML-type | 1 | n.a. | n.r. | n.r. | n.r. | |
| SL-401 | 12 | 6 (50) | n.r. | n.r. | n.r. | |
| allo-HSCT (in CR1) | 110 | n.r. | n.r. | pooled: 67% | pooled: 53 | Kharfan-Dajaba et al. [17] |
| auto-HSCT (in CR1) | 19 | n.r. | n.r. | pooled: 7% | pooled: 7 | |
| allo-HSCT (in CR1) | n.r. | n.r. | at 4 years: 69% | at 4 years: 60 | Aoki et al. [18] | |
| auto-HSCT (in CR1) | 25 | n.r. | n.r. | at 4 years: 82% | at 4 years: 73 | |
| SL-401 | 9 | 5 (55%) [2 (22%) PRs] | n.r. | n.r. | n.r. | Frankel et al. [43] |
| SL-401 | 32 FL | 90% of ORR | n.r. | at 2 years: 52% | n.r. | Pemmaraju et al. [44] |
| 15 R/R | 67% of ORR | n.r. | 8.5 | n.r. | ||
| Under evaluation targeted therapies | ||||||
| Treatment methods | n | Type of study | Results | Status | References | |
| Venetoclax | 2 | Case report | PR at 4 weeks | / | Montero et al. [19] | |
| 1 | Case report | CR at 5 months, no new cutaneous lesions at 10 months | / | Grushchak et al. [20] | ||
| / | Phase 1 | n.a. | Recruiting | NCT03113643 | ||
| / | Phase 2 | n.a. | Recruiting | NCT03404193 | ||
| 5-azacytidine | / | Phase 1 | n.a. | Recruiting | NCT03113643 | |
| / | Phase 2 | n.a. | Recruiting | NCT04216524 | ||
| Lenalidomide/bortezomib/dexamethasone | 3 | Case report | 2 CR and 1 clinical remission | / | Marmouset et al. [40] | |
| IMGN632 | / | Phase 1/2 | n.a. | Recruiting | NCT03386513 | |
| CAR-T cells | / | Phase 1 | n.a. | Recruiting | NCT04318678, NCT02159495 | |
| / | Phase 1/2 | n.a. | Recruiting | NCT04109482 | ||
BPDCN, blastic dendritic cell neoplasm; ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; NHL, non-Hodgkin’s lymphoma; Aspa-MTX, asparaginase-methotrexate; CHOP, cyclophosphamide, doxorubicin, vincristine, prednisone; allo-HSCT, allogeneic hematopoietic stem cell transplantation; auto-HSCT, autologous hematopoietic stem cell transplantation; CAR-T, T cells expressing a chimeric antigen receptor; CR, complete remission; OS, overall survival; PFS, progression-free survival; Mon, month; n.r., not reported; n.a., not available.