Retrospective study of patients with BPDCN treated with chemotherapy
Research type
Research Study
Full title
Retrospective Study of Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Treated with Chemotherapy Agents
IRAS ID
354217
Contact name
Jennifer Vidler
Contact email
Sponsor organisation
Menarini Stemline
Duration of Study in the UK
1 years, 3 months, 30 days
Research summary
The approval of tagraxofusp was granted under exceptional circumstances with limited data, due to the rarity of BPDCN (Blastic Plasmacytoid Dendritic Cell Neoplasm), with the specific obligation to conduct a Post-Authorization Safety and Efficacy Registry Study aiming to assess the clinical outcomes of tagraxofusp treatment in adult (≥ 18 years old) patients with BPDCN in a real-world clinical setting across Europe. The study is sponsored by Stemline Therapeutics and managed by IQVIA. In the UK, it will be conducted at multiple centres.
The purpose of this retrospective, non-interventional cohort study is to create an adequate, external (historical) control group to the ELZONRIS Registry Study (STML-401-0521) and thereby allow assessment of comparative effectiveness and safety profile of ELZONRIS and previously used historical chemotherapy alternatives in a real-world practice setting.
This research aims to improve understanding of how adults with a rare blood cancer called BPDCN respond to chemotherapy treatments in real-world clinical practice. BPDCN is an aggressive disease with limited treatment options and poor survival rates. The study will look back at medical records of at least 140 adult patients across Europe who started chemotherapy for BPDCN since May 2016.
Researchers will collect information about how well patients responded to treatment, how long they lived, and any side effects they experienced.
All data will be collected from existing medical records, no new treatments or tests will be given as part of this research. Patient privacy will be protected by removing personal details and following strict data protection laws.
By analysing real-world outcomes, this study hopes to provide valuable information to doctors, patients, and policymakers about the best ways to treat BPDCN and improve care for people affected by this rare cancer.
Countries being assessed are the United Kingdom, Denmark, France, Germany, Greece, Italy, Netherlands, Poland, Portugal, Spain and Sweden.
The study end of data collection is planned for May 2027.REC name
London - Harrow Research Ethics Committee
REC reference
26/PR/0103
Date of REC Opinion
27 Mar 2026
REC opinion
Further Information Favourable Opinion