Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Armenia, Canada, Cyprus, Egypt +10
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a very rare hematologic malignancy. Despite recent advances, at present there is no consensus on the optimal treatment of BPDCN. The optimal therapy of disease remains to be determined, and due to the rarity of cases, there is a need for international collaboration to collect data on BPDCN clinical presentations, diagnostics, treatment regimens and outcomes. Therefore, the objectives of this study are: (1) to build a large database of patients with BPDCN, (2) to investigate the characteristics and outcome of the disease with different treatment regimens, (3) to evaluate prognostic factors, and (4) to generate data-based prospective treatment recommendations.
Detailed description
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy. In 2008, it was recognized by the WHO as a distinct entity and separately listed in the group of acute myeloid leukemias and related precursor neoplasms.
The final diagnosis of BPDCN relies on a compatible immunophenotype. The triple positive CD4+CD56+CD123+ phenotype associated with negativity for lineage-specific markers is a minimum requirement for defining BPDCN. The highly specific marker BDCA2/CD303, as well as other plasmacytoid dendritic cell-associated antigens (e.g. TCL1 and CD2AP), might be of great support to exclude potential mimickers of BPDCN (acute myeloid and monocytic leukemias, precursor lymphoblastic T-cell leukemia/lymphomas and T- and NK/T cell lymphomas.
At present, there is no consensus on the optimal treatment of BPDCN. The majority of patients receive multi-agent chemotherapy with AML or ALL treatment regimens, while a few patients undergo allogeneic haematopoietic stem cell transplantation (HSCT). In recent years, different novel and innovative therapies are in development to target surface molecules in BPDCN. The patients are still in need of better treatments and the optimal therapy of disease remains to be determined.
This is a multicenter, international prospective and retrospective registry with the aim of collecting data of patients with a diagnosis of BPDCN globally.
Patients will be recruited directly by the national study groups / participating centers.
Participating centers will collect and verify informed consent of all prospective patients enrolled at their center.
The following data will be collected through questionnaires:
1. Patient characteristics 2. BPDCN characteristics 3. Treatment details 4. Outcomes 5. Cause of death 6. End of data collection
Quality control and data management will be conducted by the Immune Oncology Research Institute.
Primary outcome measures
- Overall survival [Time frame: 5 years]
Secondary outcome measures (3)
- Complete remission rate [Time frame: 5 years]
- Mean duration of the first remission [Time frame: 5 years]
- Event-free survival [Time frame: 5 years]
Eligibility criteria
Inclusion criteria
- Diagnosis of BPDCN
- Signed informed consent form for prospective patients
Exclusion criteria
\-
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
United States · 3 centers
- Sylvester Comprehensive Cancer Center, University of Miami — Miami
- Moffitt Cancer Center — Tampa
- Seattle Children's Cancer and Blood Disorders Center — Seattle
Turkey (Türkiye) · 3 centers
- Turkish Pediatric Cancer Registry — Ankara
- Istanbul University, Oncology Institute — Istanbul
- University of Health Sciences, Umraniye Research and Education Hospital, Pediatric Hematol — Istanbul
Canada · 2 centers
- University of Calgary — Calgary
- Children's Hospital of Eastern Ontario, University of Ottawa — Ottawa
Italy · 2 centers
- University of Perugia - Azienda Ospedaliera Perugia — Perugia
- Department of Biomedicine and Prevention, University of Rome Tor Vergata — Roma
United Kingdom · 2 centers
- Broomfield Hospital, Haematology Mid and South Essex University Hospitals Group — Chelmsford
- United Lincolnshire Teaching Hospital NHS Trust — Lincoln
Armenia · 1 center
- Hematology Center named after prof. R. Yeolyan — Yerevan
Cyprus · 1 center
- Cyprus Society of Haematology — Nicosia
Egypt · 1 center
- Oncology Center, Mansoura University Faculty of Medicine — Al Mansurah
Georgia · 1 center
- M. Iashvili Children's Central Hospital — Tbilisi
India · 1 center
- Department of Medical Oncology, Dr. B.R.A Institute Rotary Cancer Hospital, All India Inst — New Delhi
Iraq · 1 center
- Pediatric Hematology Oncology, Children's Welfare Teaching Hospital, Medical City, College — Baghdad
Jordan · 1 center
- King Hussein Cancer Center (KHCC) — Amman
Kuwait · 1 center
- Department of hematology, Kuwait Cancer Control Center — Kuwait City
Romania · 1 center
- Fundeni Clinical Institute, Department of Acute Leukemia — Bucharest
Taiwan · 1 center
- China Medical University Children's Hospital — Taichung
Identifiers
NCT: NCT05430971 · IMMONC0002