Clinical trial NCT05430971: Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry
Official title: Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Lead sponsor: Immune Oncology Research Institute
Sponsor class: OTHER
First posted: 2022-06-24
Enrollment: 200
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients with BPDCN diagnosed since 01.01.2010

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.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of BPDCN
* Signed informed consent form for prospective patients

Exclusion Criteria:

-

Primary outcomes:
measure: Overall survival
timeFrame: 5 years
Secondary outcomes:
measure: Complete remission rate
timeFrame: 5 years
measure: Mean duration of the first remission
timeFrame: 5 years
measure: Event-free survival
timeFrame: 5 years
Other outcomes:
Locations:
city: Miami
country: United States
facility: Sylvester Comprehensive Cancer Center, University of Miami
state: Florida
city: Tampa
country: United States
facility: Moffitt Cancer Center
state: Florida
city: Seattle
country: United States
facility: Seattle Children's Cancer and Blood Disorders Center
state: Washington
city: Yerevan
country: Armenia
facility: Hematology Center named after prof. R. Yeolyan
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario, University of Ottawa
state: Ontario
city: Nicosia
country: Cyprus
facility: Cyprus Society of Haematology
city: Mansoura
country: Egypt
facility: Oncology Center, Mansoura University Faculty of Medicine
city: Tbilisi
country: Georgia
facility: M. Iashvili Children's Central Hospital
city: New Delhi
country: India
facility: Department of Medical Oncology, Dr. B.R.A Institute Rotary Cancer Hospital, All India Institute of Medical Sciences
city: Baghdad
country: Iraq
facility: Pediatric Hematology Oncology, Children's Welfare Teaching Hospital, Medical City, College of Medicine, University of Baghdad
city: Perugia
country: Italy
facility: University of Perugia - Azienda Ospedaliera Perugia
city: Roma
country: Italy
facility: Department of Biomedicine and Prevention, University of Rome Tor Vergata
city: Kuwait
country: Kuwait
facility: Department of hematology, Kuwait Cancer Control Center
city: Bucharest
country: Romania
facility: Fundeni Clinical Institute, Department of Acute Leukemia
city: Taichung
country: Taiwan
facility: China Medical University Children's Hospital
city: Ankara
country: Turkey
facility: Turkish Pediatric Cancer Registry
city: Istanbul
country: Turkey
facility: Istanbul University, Oncology Institute
city: Istanbul
country: Turkey
facility: University of Health Sciences, Umraniye Research and Education Hospital, Pediatric Hematology and Oncology Department, Pediatric Bone Marrow Transplantation Unit
city: Chelmsford
country: United Kingdom
facility: Broomfield Hospital, Haematology Mid and South Essex University Hospitals Group
state: Essex
Study officials:
affiliation: Immune Oncology Research Institute, Yerevan, Armenia
name: Astghik Voskanyan, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Immune Oncology Research Institute, Yerevan, Armenia
name: Gevorg Tamamyan, MD, PhD, DSc
role: STUDY_DIRECTOR
References: