Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Conditions
Keywords
BPDCN, Retrospective, STML-401-0521, ELZONRIS, Chemotherapy
Brief summary
The main goal of this study is to investigate the effectiveness and safety of chemotherapy agents in the treatment of participants with BPDCN and to provide comparator arm data to the prospective ELZONRIS registry study (STML-401-0521).
Interventions
DRUGChemotherapy
Several chemotherapy regimens can be considered (acute myeloid leukemia-like, acute lymphocytic leukemia-like, lymphoma-like). The choice of the regimen, as well as dosing and schedules, are based on the institution's and the health care provider's preferences. No study medication will be provided by the sponsor during this study.
Sponsors
Stemline Therapeutics, Inc.
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Alive, deceased, or unreachable participants (as permitted by local regulation), aged ≥18 years at the start of first-line treatment for BPDCN, and treated with chemotherapy regimens (at a minimum) as first-line treatment for BPDCN from May 2016 * Diagnosed with BPDCN, based on clinical, laboratory, and imaging (where performed per institutional practice) assessments, with a demonstration of a specific immunophenotype (either by immunohistochemistry or flow cytometry), particularly CD4, CD56, and CD123 * Participants (or their legally authorized representatives) who have signed the informed consent and privacy form, where required as per local regulation
Exclusion criteria
* Participant has received an investigational agent as first-line therapy for BPDCN * Participant has received first-line therapy for BPDCN in an interventional clinical trial
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Objective Response Rate | Baseline through Month 18 |
| Composite Complete Response | Baseline through Month 18 |
| Number of Participants Bridged to Stem Cell Transplant | Baseline through Month 18 |
| Duration of Response | Baseline through Month 18 |
| Real-world Progression-free Survival | Baseline through Month 18 |
| Overall Survival | Baseline through Month 18 |
| Number of Participants with Treatment-emergent Adverse Events | Baseline through Month 18 |
| Number of Participants Requiring Transfusions | Baseline through Month 18 |
| Duration of Treatments Received and Hospitalization | Baseline through Month 18 |
Secondary
| Measure | Time frame |
|---|---|
| Time to Next Treatment | Baseline through Month 26 |
| Number of Different First-line Treatment Regimens and Sequences Received | Baseline through Month 26 |
Countries
Denmark, France, Germany, Greece, Italy, Netherlands, Poland, Portugal, Spain, Sweden, United Kingdom
Contacts
CONTACTStemline Trials
STUDY_DIRECTORMedical Director
Stemline Therapeutics, Inc.
Outcome results
None listed