Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
- Кому может быть актуально
- Состояния в реестре: Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). Базовые параметры: Без ограничений · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- США, Армения, Канада, Cyprus, Египет +10
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Обзор
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.
Подробное описание
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.
Первичные конечные точки
- Overall survival [Срок оценки: 5 years]
Вторичные конечные точки (3)
- Complete remission rate [Срок оценки: 5 years]
- Mean duration of the first remission [Срок оценки: 5 years]
- Event-free survival [Срок оценки: 5 years]
Критерии участия
Критерии включения
- Diagnosis of BPDCN
- Signed informed consent form for prospective patients
Критерии исключения
\-
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Только случаи
Центры проведения
США · 3 центра
- Sylvester Comprehensive Cancer Center, University of Miami — Miami
- Moffitt Cancer Center — Tampa
- Seattle Children's Cancer and Blood Disorders Center — Seattle
Turkey (Türkiye) · 3 центра
- Turkish Pediatric Cancer Registry — Ankara
- Istanbul University, Oncology Institute — Istanbul
- University of Health Sciences, Umraniye Research and Education Hospital, Pediatric Hematol — Istanbul
Канада · 2 центра
- University of Calgary — Calgary
- Children's Hospital of Eastern Ontario, University of Ottawa — Ottawa
Италия · 2 центра
- University of Perugia - Azienda Ospedaliera Perugia — Perugia
- Department of Biomedicine and Prevention, University of Rome Tor Vergata — Roma
Великобритания · 2 центра
- Broomfield Hospital, Haematology Mid and South Essex University Hospitals Group — Chelmsford
- United Lincolnshire Teaching Hospital NHS Trust — Lincoln
Армения · 1 центр
- Hematology Center named after prof. R. Yeolyan — Yerevan
Cyprus · 1 центр
- Cyprus Society of Haematology — Nicosia
Египет · 1 центр
- Oncology Center, Mansoura University Faculty of Medicine — Al Mansurah
Грузия · 1 центр
- M. Iashvili Children's Central Hospital — Tbilisi
Индия · 1 центр
- Department of Medical Oncology, Dr. B.R.A Institute Rotary Cancer Hospital, All India Inst — New Delhi
Ирак · 1 центр
- Pediatric Hematology Oncology, Children's Welfare Teaching Hospital, Medical City, College — Baghdad
Иордания · 1 центр
- King Hussein Cancer Center (KHCC) — Amman
Кувейт · 1 центр
- Department of hematology, Kuwait Cancer Control Center — Kuwait City
Румыния · 1 центр
- Fundeni Clinical Institute, Department of Acute Leukemia — Bucharest
Тайвань · 1 центр
- China Medical University Children's Hospital — Taichung
Идентификаторы
NCT: NCT05430971 · IMMONC0002