Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry
- Conditions
- Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
- Registration Number
- NCT05430971
- Lead Sponsor
- Immune Oncology Research Institute
- Brief Summary
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 200
- Diagnosis of BPDCN
- Signed informed consent form for prospective patients
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Overall survival 5 years Time from diagnosis to death
- Secondary Outcome Measures
Name Time Method Event-free survival 5 years Time from diagnosis to occurrence of a complication
Complete remission rate 5 years The proportion of patients with complete remission
Mean duration of the first remission 5 years Time from first remission to disease progression or death
Trial Locations
- Locations (20)
Sylvester Comprehensive Cancer Center, University of Miami
🇺🇸Miami, Florida, United States
Moffitt Cancer Center
🇺🇸Tampa, Florida, United States
Seattle Children's Cancer and Blood Disorders Center
🇺🇸Seattle, Washington, United States
Hematology Center named after prof. R. Yeolyan
🇦🇲Yerevan, Armenia
University of Calgary
🇨🇦Calgary, Alberta, Canada
Children's Hospital of Eastern Ontario, University of Ottawa
🇨🇦Ottawa, Ontario, Canada
Cyprus Society of Haematology
🇨🇾Nicosia, Cyprus
Oncology Center, Mansoura University Faculty of Medicine
🇪🇬Mansoura, Egypt
M. Iashvili Children's Central Hospital
🇬🇪Tbilisi, Georgia
Department of Medical Oncology, Dr. B.R.A Institute Rotary Cancer Hospital, All India Institute of Medical Sciences
🇮🇳New Delhi, India
Pediatric Hematology Oncology, Children's Welfare Teaching Hospital, Medical City, College of Medicine, University of Baghdad
🇮🇶Baghdad, Iraq
University of Perugia - Azienda Ospedaliera Perugia
🇮🇹Perugia, Italy
Department of Biomedicine and Prevention, University of Rome Tor Vergata
🇮🇹Roma, Italy
Department of hematology, Kuwait Cancer Control Center
🇰🇼Kuwait, Kuwait
Fundeni Clinical Institute, Department of Acute Leukemia
🇷🇴Bucharest, Romania
China Medical University Children's Hospital
🇨🇳Taichung, Taiwan
Turkish Pediatric Cancer Registry
🇹🇷Ankara, Turkey
Istanbul University, Oncology Institute
🇹🇷Istanbul, Turkey
University of Health Sciences, Umraniye Research and Education Hospital, Pediatric Hematology and Oncology Department, Pediatric Bone Marrow Transplantation Unit
🇹🇷Istanbul, Turkey
Broomfield Hospital, Haematology Mid and South Essex University Hospitals Group
🇬🇧Chelmsford, Essex, United Kingdom