Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Plasmacytoid Dendritic Cells
Conditions
Interventions
tagraxofusp (ELZONRIS)
Sponsors
Stemline Therapeutics Inc.
Eligibility
Inclusion criteria
Inclusion criteria: Male or female, alive, deceased, or unreachable patients (as permitted by local regulation), aged = 18 years at the start of 1L treatment for BPDCN, and treated with chemotherapy regimens (at a minimum) as 1L treatment for BPDCN from May 2016Diagnosed 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 CD123Patients (or their legally authorized representatives) who have signed the informed consent and privacy form (ICF), where required as per local regulation
Exclusion criteria
Exclusion criteria: • Patient has received an investigational agent as 1L therapy for BPDCN • Patient has received 1L therapy for BPDCN in an interventional clinical trial
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary: 1. To describe the effectiveness of first-line (1L) chemotherapy treatments measured by: o Objective Response Rate (ORR) (complete response [CR] + complete response with minimal residual skin abnormality [CRc] + complete response with incomplete blood count recovery [CRi] + partial response [PR]) in 1L following the prescribed treatment o Rate of composite CR (CR + CRc) o Rate of patients bridged to Stem Cell Transplant (SCT) o Duration of Response (DOR) o Real-world Progression-Free Survival (PFS) o Overall Survival (OS) 2. To describe the safety of 1L chemotherapy treatments measured by: o Incidence rate and severity of treatment related adverse events (AEs), and all AEs of interest regardless of causality, defined in Section 8.4 o Incidence rate of Grade 5 AEs and Grade 5 AEs of interest, defined in Section 8.4 o Incidence of red blood cell (RBC), whole blood, or platelet transfusions o Duration of treatments and hospitalization | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary: 1. To describe the treatment practices for adult patients with BPDCN who received 1L treatments other than ELZONRIS® (tagraxofusp), including: o Time to next treatment (TTNT) o Treatment regimens and sequence o Treatment type o Number of cycles Exploratory: 1. To perform a comparative analysis of the effectiveness and safety (according to primary and secondary outcomes) of ELZONRIS versus chemotherapy as 1L treatment for adults with BPDCN using data from the ELZONRIS Registry Study (STML-401-0521) and the present study (STML-401-0623) 2. To further describe the effectiveness of 1L chemotherapy treatments measured by: o Time to Response (TTR) o Time to SCT o Rate of relapse post SCT 3. To describe the effectiveness and safety of treatment options and to describe treatment practices for patients with relapsed/refractory (R/R) BPDCN | — |
Countries
Denmark, France, Germany, Greece, Italy, Netherlands, Poland, Portugal, Spain, Sweden, Switzerland, United Kingdom
Contacts
Public ContactSylvie Fernandez
IQVIA Solutions Italy S.r.l.
Outcome results
None listed