Long-term safety and efficacy of ELZONRIS, the first and only targeted FDA-approved treatment for BPDCN, were evaluated in the long-term analysis, which included all patients from the initial analysis and those enrolled in the continued access cohort.1
The primary outcome of the study was the combined rate of CR and CRc among treatment-naive patients. All other data presented here were secondary outcomes.2,3
The primary outcome was CR/CRc1
Long-term analysis
The long-term analysis included all 44 patients from the initial analysis, as well as 40 others enrolled in the continued access cohort1,3,4
- 65 treatment-naive patients and 19 previously treated patients were efficacy evaluable1
- Patients with CR/CRc received ELZONRIS until disease progression, unacceptable toxicity, or allogeneic (allo)/autologous (auto) SCT2,5
- Median follow-up was ~3 years1
Long-term efficacy of ELZONRIS in treatment-naive patients with BPDCN
Data from the largest prospective study to date in treatment-naive patients with BPDCN (n=65) with a median follow-up of ~3 years1,2






Explore the baseline characteristics of treatment-naive patients in the long-term analysis
Baseline characteristics of treatment-naive patients1
Characteristics | Long-term analysis (n=65) |
||
---|---|---|---|
Male, % (n) | 80% (52) | ||
Female, % (n) | 20% (13) | ||
Median age | (min, max)68 (22, 84) | ||
ECOG PS, % (n) | |||
0 | 48% (31) | ||
1 | 48% (31) | ||
BPDCN, % (n) | |||
Skin | 92% (60) | ||
Bone marrow | 49% (32) | ||
Peripheral blood | 26% (17) | ||
Lymph nodes | 51% (33) | ||
Visceral | 14% (9) |
ECOG PS, Eastern Cooperative Oncology Group performance status.
Long-term efficacy of ELZONRIS in previously treated patients with BPDCN
In previously treated patients with BPDCN (n=19) who were efficacy evaluable, ORR was 58% (95% CI, 33.5 to 79.7) with 1 patient achieving CR and 2 patients achieving CRc.1
Explore the baseline characteristics of previously treated patients in the long-term analysis
Baseline characteristics of previously treated patients1,2
Characteristics | Long-term analysis (n=19) |
||
---|---|---|---|
Male, % (n) | 84% (16) | ||
Female, % (n) | 16% (3) | ||
Median age | (min, max)72 (44, 87) | ||
ECOG PS, % (n) | |||
0 | 37% (7) | ||
1 | 63% (12) | ||
BPDCN, % (n) | |||
Skin | 79% (15) | ||
Bone marrow | 63% (12) | ||
Peripheral blood | 5% (1) | ||
Lymph nodes | 47% (9) | ||
Visceral | 21% (4) | ||
Prior treatments, % (n) | |||
Radiation therapy | 32% (6) | ||
Stem cell transplantation | 32% (6) |
ECOG PS, Eastern Cooperative Oncology Group performance status.
*n=19/37.
BPDCN, blastic plasmacytoid dendritic cell neoplasm; CI, confidence interval; CR, complete response; CRc, CR with residual skin abnormalities not indicative of the active disease; NR, not reached; ORR, overall response rate; SCT, stem cell transplant.
- References:
- Pemmaraju N, et al. Long-term benefits of tagraxofusp for patients with blastic plasmacytoid dendritic cell neoplasm. J Clin Oncol. 2022;40(26):3032-3036.
- Data on file. Stemline Therapeutics, Inc.
- Pemmaraju N, et al. Tagraxofusp in blastic plasmacytoid dendritic-cell neoplasm. N Engl J Med. 2019;380(17):1628-1637.
- Pemmaraju N, et al. Long-term benefits of tagraxofusp for patients with blastic plasmacytoid dendritic cell neoplasm. J Clin Oncol. 2022;40(26):3032-3036 [supplementary appendix].
- ELZONRIS [prescribing information]. New York, NY: Stemline Therapeutics, Inc.; November 2022.