Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry: Tagraxofusp Cohort Treatment Results
Notice bibliographique
Résumé
Background: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an orphan malignancy defined by the WHO as affection fewer than 65 per 100,000 people and by the U.S. FDA as affecting less than 200,000 people in the U.S.. The BPDCN international registry was launched in July 2022 with the aim to create a large database for this rare disease, to generate data-driven diagnostic and treatment recommendations, and provide real-world evidence. Aim:The aim of this abstract is to evaluate the outcomes of BPDCN patients treated with tagraxofusp, an immunotoxin targeting CD123 that was approved by the U.S. FDA in December 2018 and the European EMA in January 2021 for the treatment of BPDCN. Methods: The registry collects retrospective and prospective data of patients diagnosed with BPDCN after January 1st, 2010. We assessed baseline characteristics, response to treatment and outcomes of BPDCN patients who received tagraxofusp. Results: As of June 2024, 70 patients from 13 countries (USA, Egypt, Canada, Italy, Georgia, Turkey, UK, India, Armenia, Iraq, Kuwait, Cyprus and Romania) were included in the registry. Among those, 22 patients (from USA, UK and Italy) have received treatment with tagraxofusp. Seventeen patients were treated with tagraxofusp as a first line therapy and 5 patients as a second line. The median age was 76years (range 40-87) with only one female patient. Fifteen patients had received tagraxofusp before the U.S. FDA approval in the setting of clinical trials. In the first line therapy setting, the overall response rate was 82% (14/17 patients) where 9 patients had a complete response (CR), 5 pts partial response (PR), 2 stable disease (SD), and one patient was not evaluable(no information was available). Of the 9 CR patients, 2 passed away from other reasons besides BPDCN, one was lost to follow up, the other 6 relapsed. Median time to relapse was 3 months. Among the relapsed patients, 4 experienced progressive or stable disease on subsequent chemotherapy regimens, 2 were treated with HyperCVAD followed allogeneic stem cell transplantation (allo-SCT) and were alive at last follow-up. Of the 7 patients with PR and SD as first line therapy, 6 experienced progressive disease, 1 patient who was treated with HyperCVAD followed by allo-SCT was alive at last follow-up. None of 5 patients treated with tagraxofusp as a second line therapy experienced CR, two patients experienced short SD, the other three progressive disease. At the last follow-up information was available for 19 of 22 patients of which 4 were alive. Conclusion: Tagraxofusp yields high response rates with more than 50% CR in the upfront setting; however, the relapse rates without allo-SCT remains high. This therapy is currently only available in selected countries and only 3 out of the 13 countries participating in this registry reported its use. The treatment strategy of BPDCN should be a total therapy approach incorporating novel agents like tagraxofusp with intrathecal chemotherapy and allo-SCT to prevent the relapse.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,002 | 0,002 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,001 |
| Bibliométrie | 0,001 | 0,001 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,001 | 0,000 |
| Science ouverte | 0,000 | 0,001 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,002 | 0,001 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».