RETROSPECTIVE STUDY OF PATIENTS WITH BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM (BPDCN) TREATED WITH CHEMOTHERAPY AGENTS
Objectif(s) de la recherche et intérêt pour la santé publique
Finalité de l'étude
Objectifs poursuivis
Domaines médicaux investigués
Bénéfices attendus
The approval of tagraxofusp (ELZONRIS®) was granted under exceptional circumstances with limited data, due to the rarity of BPDCN, with the specific obligation by EMA (European Medicines Agency) to conduct a Post Authorization Safety and Efficacy Registry Study aiming to assess the clinical outcomes of ELZONRIS treatment in adult (≥ 18 years old) patients with BPDCN in a real-world clinical setting across Europe.
In the setting of this EMA request, the purpose of this retrospective, non-interventional cohort study is to create an adequate, external (historical) control group to the ELZONRIS Registry Study (STML-401-0521) and there by allow assessment of comparative effectiveness and safety profile of ELZONRIS and previously used historical chemotherapy alternatives in a real-world practice setting.
The primary objective of this study is to describe the effectiveness of first line (1L) chemotherapy treatments.
The cohort of patients included in the study will be identified from medical charts of patients treated at sites participating in the prospective. Registry or additional existing data sources which capture medical charts and relevant variables of patients with BPDCN treated with several regimens of chemotherapy (acute myeloid leukemia [AML]-like, acute lymphocytic leukemia [ALL]-like, and Lymphoma-like) from May 2016, the date of the first publication of the World Health Organization (WHO) classification for BPDCN as a unique myeloid neoplasm
Données utilisées
Catégories de données utilisées
Source de données utilisées
Autre(s) source(s) de donnée(s) mobilisée(s)
Appariement entre les sources de données mobilisées
Variables sensibles utilisées
Justification du recours à cette(ces) variable(s) sensible(s)
This information will ensure accurate and comprehensive data analysis, while maintaining privacy and confidentiality in compliance with data protection regulations.
Recours au numéro d'identification des professionnels de santé
Justification de l'utilisation du numéro d'identification des professionnels de santé
Collection of RPPS numbers to facilitate the completion of preliminary formalities with the relevant orders, as part of the framework for overseeing benefits.
Plateforme utilisée pour l'analyse des données
Acteurs finançant et participant à l'étude
Responsable(s) de traitement
Type de responsable de traitement 1
Responsable de traitement 1
Localisation du responsable de traitement 1
Représentant du responsable de traitement 1
Responsable(s) de mise en oeuvre non cités comme responsable de traitement
Responsable de mise en oeuvre non cité comme responsable de traitement 1
Calendrier du projet
Base légale pour accéder aux données
Encadrement réglementaire
Durée de conservation aux fins du projet (en années)
25
Existence d'une prise de décision automatisée
Fondement juridique
Article 6 du RGPD (Licéité du traitement)
Article 9 du RGPD (Exception permettant de traiter des données de santé)
Transfert de données personnelles vers un pays hors UE
The Sponsor will rely on mechanism of transfer, such as the Standard Contractual Clauses.
Droits des personnes
The centers participating in the study provide the concerned individuals with an information notice in accordance with Article 14 of the General Data Protection Regulation. Participants can access their data and request that it be rectified or completed.
They can also request the restriction of data processing (i.e., request a temporary freeze of their data). Living patients will sign an informed consent form to confirm their participation in the study and allow the processing of their data for this purpose. Even if patients do not initially refuse to participate in the study, they can object to the processing of their data at any time thereafter. In such cases, no further information about them will be collected.
Patients can also exercise their right to erasure of the data already collected; however, this data may not be erased if doing so would make it impossible or seriously compromise the achievement of the study's objectives.
Participants can exercise their rights at any time, without needing to provide justification.