In the largest prospective clinical trial to date of treatment-naïve and previously-treated patients with BPDCN, ELZONRIS was evaluated in an open-label, multicenter clinical study (N=44). The pivotal cohort consisted of 13 treatment-naïve patients.1,2

*Complete response (CR) criteria: normalization of blast percentage (≤ 5%) in the bone marrow; normalization of neutrophil count (≥ 1,000/µL) and platelet count (≥ 100,000/µL) in the peripheral blood; absence of leukemic blasts in the peripheral blood; 100% clearance of all skin lesions from baseline; no new lesions in patients without lesions at baseline; regression of nodal masses to normal size on CT; no palpable nodules on the spleen or liver.3
†Clinical complete response with minimal residual skin abnormality (CRc) criteria: marked clearance of all skin lesions from baseline; residual hyperpigmentation or abnormality with BPDCN identified on biopsy (or no biopsy performed).3
‡Dosing period may be extended for dose delays up to day 10 of the cycle.2
Baseline patient characteristics
Key baseline demographics in the study reflect the patient population and variable presentation of BPDCN.1-3
Baseline characteristics: Treatment-naïve patients2,3§
Characteristics | Pivotal cohort (n=13) |
Other cohorts (n=16) |
All cohorts (n=29) |
---|---|---|---|
Male, % (n) | 84.6% (11) | 75.0% (12) | 79.3% (23) |
Female, % (n) | 15.4% (2) | 25.0% (4) | 20.7% (6) |
Median age | (min, max)65.0 (22, 84) | 67.5 (28, 84) | 67.0 (22, 84) |
ECOG PS, % (n) | |||
0 | 61.5% (8) | 43.8% (7) | 51.7% (15) |
1 | 38.5% (5) | 56.3% (9) | 48.3% (14) |
BPDCN, % (n) | |||
Skin | 100.0% (13) | 93.8% (15) | 96.6% (28) |
Bone marrow | 53.8% (7) | 43.8% (7) | 48.3% (14) |
Peripheral blood | 23.1% (3) | 25.0% (4) | 24.1% (7) |
Lymph nodes | 46.2% (6) | 43.8% (7) | 44.8% (13) |
Viscera | 15.4% (2) | 12.5% (2) | 13.8% (4) |
Baseline characteristics: Previously-treated patients1-3
Characteristics | Previously-treated patients | (n=15)
---|---|
Male, % (n) | 86.7% (13) |
Female, % (n) | 13.3% (2) |
Median age | (min, max)72 (44, 80) |
ECOG PS, % (n) | |
0 | 33.3% (5) |
1 | 66.7% (10) |
BPDCN, % (n) | |
Skin | 86.7% (13) |
Bone marrow | 60.0% (9) |
Peripheral blood | 6.7% (1) |
Lymph nodes | 53.3% (8) |
Viscera | 26.7% (4) |
Prior treatments, % (n) | |
Radiation therapy | 33.3% (5) |
Stem cell transplantation | 26.7% (4) |
Previous lines of therapy: Previously-treated patients1
Number of lines | Previously-treated patients, % (n) |
---|---|
1 | 60% (9) |
2-3 | 27% (4) |
≥ 4 | 13% (2) |
§All-cohorts population (N=29) includes patients from the pivotal cohort (N=13).
CT = computed tomography; ECOG PS = Eastern Cooperative Oncology Group performance status.
- References:
- Pemmaraju N, Lane AA, Sweet KL, et al. Tagraxofusp in blastic plasmacytoid dendritic-cell neoplasm. N Engl J Med. 2019;380(17):1628-1637.
- ELZONRIS [prescribing information]. New York, NY, US: Stemline Therapeutics, Inc.; December 2018.
- Data on file. Stemline Therapeutics, Inc.