BPDCN International Registry: A Transformative Step Towards Advancing Rare Disease Research

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a rare, aggressive hematologic malignancy characterized by CD4+ CD56+ plasmacytoid dendritic cell proliferation. It often presents with nonspecific cutaneous lesions and can rapidly progress to leukemia, with a median survival of 8–14 months if untreated. To address the challenges of BPDCN, the BPDCN International Registry, launched on July 1, 2022 by the Immune Oncology Research Institute (IMMONC) and listed on ClinicalTrials.gov, aims to collect global patient data.

In this article, we discuss the importance of rare disease registries, particularly how they support research, improve patient outcomes, and provide valuable insights for clinicians. Additionally, we highlight scientific publications from the registry team, demonstrating its contributions to BPDCN research. We also provide guidance on how patients, doctors, and research centers can join the registry and contribute to advancing knowledge and treatment for this complex disease.

International BPDCN Day is observed annually to raise awareness about Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), a rare and aggressive hematologic malignancy. In 2025, it was celebrated on January 23rd, focusing on advancing awareness, research, and hope for patients and families affected by this challenging disease.

The Impact of Rare Disease Registries

Rare disease registries have a profound human impact by addressing the unique challenges faced by individuals with these conditions and their families. These registries play a crucial role in improving diagnosis and treatment by collecting and organizing data on disease symptoms, progression, and treatment outcomes, enabling healthcare providers to make more accurate diagnoses and informed treatment decisions. They also help identify risk factors and guidelines for managing diseases like Fabry disease, Gaucher disease, and Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN).

Additionally, registries facilitate research by providing real-world evidence crucial for understanding the biology of rare diseases, which often lack sufficient research due to small patient populations. By aggregating global data, they overcome geographical limitations and accelerate the development of new therapies and clinical trials.

Beyond research, registries empower patients and families by offering a platform to share experiences, fostering a sense of community, and contributing to scientific advancements. They also highlight the psychosocial and economic burdens faced by families, ensuring these are considered in healthcare planning. On a broader scale, rare diseases collectively affect 3.5–5.9% of the global population, impacting over 300 million people worldwide. Registries bring attention to this public health challenge, advocating for better policies, resource allocation, and supporting universal health coverage (UHC) initiatives by addressing systemic inequalities in access to care.

Many rare diseases lack effective treatments, and registries fill critical gaps left by traditional clinical trials, providing long-term data that guides therapeutic advancements. They also document patient-reported outcomes, ensuring that research aligns with real-world needs and ultimately improves healthcare strategies for rare disease patients.

BPDCN International Registry

The BPDCN International Registry stands as a crucial initiative to tackle the significant challenges posed by Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), a rare and aggressive hematologic malignancy. This registry, as indicated by its listing on ClinicalTrials.gov, aims to systematically collect data on patients diagnosed with BPDCN, fostering a deeper understanding of the disease’s natural history, treatment responses, and long-term outcomes. BPDCN International Registry officially launched in 2022 by the Immune Oncology Research Institute (IMMONC), the registry’s primary objective is to collect comprehensive data on patients diagnosed with BPDCN globally, with the ultimate goal of improving diagnostic and treatment strategies for this complex disease.

This prospective and retrospective registry aims to build a robust database that will facilitate a deeper understanding of BPDCN, identify unmet clinical needs, and accelerate the development of more effective therapiesBy consolidating information from around the globe, the BPDCN International Registry facilitates collaborative research efforts, enhances the ability to identify prognostic factors, and ultimately strives to improve the lives of individuals affected by this complex and uncommon neoplasm.

Who are the Study Director and Principal Investigator of the BPDCN International Registry?

The Study Director is Professor Gevorg Tamamyan, who is the President-Elect of the Pediatric Oncology East and Mediterranean (POEM) Group and the President-Elect of SIOP Asia (International Society of Pediatric Oncology, Asian Division).

The Principal Investigator of the BPDCN International Registry is Astghik Voskanyan, MD, who serves as the Head of the Adult Blood Disorders Clinic and the Deputy Director of Scientific Affairs at the Yeolyan Hematology and Oncology Center.

Why is This Registry Important?

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) presents significant challenges in clinical management, primarily due to the lack of standardized treatment approaches. Currently, there is no consensus on the optimal therapy for BPDCN, leading to a heterogeneous array of treatment strategies that vary widely among institutions. This inconsistency complicates patient care and outcomes, highlighting the urgent need for a more unified approach. The BPDCN International Registry aims to address this gap by generating data-driven recommendations that can guide clinical practice and improve treatment protocols for this rare malignancy.

Conducting large clinical trials for rare cancers like BPDCN is inherently difficult due to the limited patient population. This scarcity often results in insufficient data to establish robust treatment guidelines, making registries a vital tool for research and progress. Cancer registries systematically collect comprehensive data on patients, which is then analyzed to identify patterns, monitor trends, and inform future research directions. Specially trained cancer registrars play a crucial role in this process, gathering detailed information about patient demographics, treatment responses, and outcomes. The data collected not only aids in understanding the disease but also helps in planning effective interventions and advancing research efforts aimed at improving patient care and outcomes in BPDCN.

Details of the BPDCN International Registry

The BPDCN International Registry is designed as a multicenter, international initiative that encompasses both retrospective and prospective data collection. This registry welcomes patients of all ages diagnosed with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) since 2010, aiming to gather extensive information on clinical presentation, treatment regimens, and patient outcomes. Utilizing electronic case report forms (eCRF), the registry facilitates efficient data collection and analysis.

Currently, the registry involves collaboration among 15 participating centers across 11 countries, including the United States, Armenia, Canada, Cyprus, Egypt, Georgia, India, Iraq, Italy, Kuwait, Romania, Taiwan, Turkey, and the United Kingdom, highlighting the global effort to address the challenges posed by this rare disease. One of the primary objectives of the BPDCN International Registry is to generate data-driven recommendations that can guide clinical practice, particularly in light of the lack of standardized treatment approaches for BPDCN.

The hospitals participating in the BPDCN International Registry

The BPDCN International Registry involves a network of leading medical institutions globally, working together to collect vital data on Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN).

Sylvester Comprehensive Cancer Center, University of Miami in Miami, Florida, USA; Moffitt Cancer Center in Tampa, Florida, USA; Seattle Children’s Cancer and Blood Disorders Center in Seattle, Washington, USA; Hematology Center named after Prof. R. Yeolyan in Yerevan, Armenia; University of Calgary in Calgary, Alberta, Canada; Children’s Hospital of Eastern Ontario, University of Ottawa in Ottawa, Ontario, Canada; Cyprus Society of Haematology in Nicosia, Cyprus; Oncology Center, Mansoura University Faculty of Medicine in Mansoura, Egypt; M. Iashvili Children’s Central Hospital in Tbilisi, Georgia; Department of Medical Oncology, Dr. B.R.A Institute Rotary Cancer Hospital, All India Institute of Medical Sciences in New Delhi, India; Pediatric Hematology Oncology, Children’s Welfare Teaching Hospital, Medical City, College of Medicine,

University of Baghdad in Baghdad, Iraq; University of Perugia – Azienda Ospedaliera Perugia in Perugia, Italy; Department of Biomedicine and Prevention, University of Rome Tor Vergata in Rome, Italy; Department of Hematology, Kuwait Cancer Control Center in Kuwait; Fundeni Clinical Institute, Department of Acute Leukemia in Bucharest, Romania; China Medical University Children’s Hospital in Taichung, Taiwan; Turkish Pediatric Cancer Registry in Ankara, Turkey; Istanbul University, Oncology Institute in Istanbul, Turkey; University of Health Sciences, Umraniye Research and Education Hospital, Pediatric Hematology and Oncology Department, Pediatric Bone Marrow Transplantation Unit in Istanbul, Turkey; and Broomfield Hospital, Haematology Mid and South Essex University Hospitals Group in Chelmsford, Essex, United Kingdom.

Conducting large clinical trials for rare cancers like BPDCN is inherently difficult due to limited patient populations, making registries a vital tool for research and progress. Cancer registries systematically collect comprehensive data on patients, with specially trained cancer registrars responsible for gathering detailed information about demographics, treatment responses, and outcomes. This data is then analyzed to determine cancer patterns, monitor trends, guide planning, and advance research efforts aimed at improving patient care and outcomes in BPDCN.

A Call to Action: Join the BPDCN International Registry

The BPDCN International Registry offers a unique opportunity for clinicians and centers to contribute to meaningful advancements in the diagnosis and treatment of this rare malignancy.

For Doctors: Join the BPDCN International Registry and Empower Your Practice

Contributing data to the BPDCN International Registry can directly impact patient care by helping to establish evidence-based guidelines for BPDCN management. By sharing your clinical experiences, you contribute to a comprehensive understanding of the disease, which can lead to improved diagnostic accuracy and more effective treatment strategies. You’ll have the opportunity to collaborate with international experts, share insights, and contribute to cutting-edge research that will ultimately benefit patients worldwide. Rest assured, data security and patient privacy are paramount: each participating hospital has a designated data custodian who authorizes the use of de-identified data, ensuring that patient information remains secure and confidential.

For Centers: Join the BPDCN Registry: Elevate Your Center’s Impact on Research and Care

Becoming a part of the BPDCN International Registry’s global network offers numerous benefits for your center. Participation enhances your center’s reputation as a leader in BPDCN research and positions you at the forefront of efforts to improve patient outcomes. Your center will gain access to a wealth of shared data and expertise, fostering collaboration and accelerating research progress. Data ownership concerns are addressed through a stringent data tenancy framework: each hospital has a data custodian who must authorize the use of any de-identified data.

This framework ensures that data access is strictly controlled and that data security and privacy are maintained at all times. By joining the BPDCN International Registry, your center will play a pivotal role in shaping the future of BPDCN research and treatment.

We invite you to contribute by sharing patient data from your institution even if you have only a patient. Your participation offers the opportunity to collaborate with international experts, contribute to evidence-based treatment recommendations, and be recognized as a co-author in our future publications. Enclosed are our recent publications for your review.

Your involvement would be invaluable in advancing BPDCN research. If you require further information or wish to discuss this initiative in more detail, please do not hesitate to contact us. We would greatly appreciate the opportunity to collaborate with you.

For more details and collaboration, you can contact Immune Oncology Research Institute Fellow, Aren Karapetyan, MD – [email protected]

Dr. Astghik Voskanyan presented a poster at the 66th Annual Meeting of the American Society of Hematology (ASH) in 2024.

How the Registry Data will be Used?

The BPDCN International Registry’s comprehensive data collection efforts will enable a wide range of impactful research endeavors. By gathering detailed clinical information, treatment regimens, and patient outcomes, the registry facilitates the identification of prognostic factors that can predict disease progression and treatment response. This knowledge is crucial for developing personalized treatment strategies tailored to individual patient characteristics, ultimately improving patient outcomes and survival rates.

Moreover, linking registry data with external datasets, such as genomic databases or electronic health records, and integrating it with tissue samples will maximize the opportunity for impactful research output. This integrated approach allows for a deeper understanding of the molecular mechanisms driving BPDCN, identification of novel therapeutic targets, and the development of innovative diagnostic tools. The combined power of clinical data, genomic information, and tissue analysis will accelerate the translation of research findings into clinical practice, ultimately transforming the landscape of BPDCN management and patient care.

Publications Already Done by the BPDCN International Registry Team

The BPDCN International Registry team has already begun publishing valuable data, as exemplified by their presentation at the American Society of Hematology (ASH) Annual Meeting in December 2024. This presentation, focused on evaluating outcomes of BPDCN patients treated with tagraxofusp (a CD123-targeting immunotoxin approved for BPDCN), highlighted the registry’s ability to generate real-world evidence for this rare disease. As of June 2024, the analysis included 70 patients from 13 countries, with 22 having received tagraxofusp.

• Tagraxofusp Cohort Treatment Results
• Diagnosing and Treating BPDCN in a Resource-Limited Setting
• Tagraxofusp Cohort Treatment Results
• AML-396 BPDCN: Initial Results of a Novel, Ongoing BPDCN International Registry Program

The findings demonstrated high response rates with tagraxofusp in the upfront setting, but also emphasized the high relapse rates without subsequent allogeneic stem cell transplantation (allo-SCT), suggesting a total therapy approach incorporating novel agents, intrathecal chemotherapy, and allo-SCT may be optimal. This publication underscores the registry’s commitment to providing data-driven insights into the treatment of BPDCN and its potential to influence clinical practice globally.