CB‐103: A novel CSL‐NICD inhibitor for the treatment of NOTCH‐driven T‐cell acute lymphoblastic leukemia: A case report of complete clinical response in a patient with relapsed and refractory T‐ALL

Relapsed T cell acute lymphoblastic leukaemia (T‐ALL) has a very poor prognosis. A 24‐year‐old patient with relapsed high‐risk T‐ALL (PTEN gene deletion; NOTCH1 mutation), was treated with the NOTCH inhibitor CB‐103. Within 1 week of starting CB‐103, the bone marrow was free of T‐ALL blast infiltrat...

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Detalles Bibliográficos
Autores principales: Medinger, Michael, Junker, Till, Heim, Dominik, Tzankov, Alexandar, Jermann, Philip M., Bobadilla, Maria, Vigolo, Michele, Lehal, Rajwinder, Vogl, Florian D., Bauer, Michael, Passweg, Jakob
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Case Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9421963/
https://www.ncbi.nlm.nih.gov/pubmed/36051082
http://dx.doi.org/10.1002/jha2.510
Descripción
Sumario:Relapsed T cell acute lymphoblastic leukaemia (T‐ALL) has a very poor prognosis. A 24‐year‐old patient with relapsed high‐risk T‐ALL (PTEN gene deletion; NOTCH1 mutation), was treated with the NOTCH inhibitor CB‐103. Within 1 week of starting CB‐103, the bone marrow was free of T‐ALL blast infiltration (MRD+) and successfully underwent allogeneic hematopoietic stem cell transplantation (allo‐HSCT). Sequential samples of ctDNA to monitor the disease after allo‐HSCT showed a decrease of circulating Notch1 and PTEN alterations. This is the first T‐ALL patient treated with CB‐103. The observed clinical response encourages further exploration of CB‐103 in ALL.

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