作者: Heim, Dominik ; Bauer, Michael ; Medinger, Michael ; Tzankov, Alexandar ; Vigolo, Michele ; Lehal, Rajwinder ; Passweg, Jakob ; Vogl, Florian D. ; Bobadilla, Maria ; Junker, Till ; Jermann, Philip M.
AbstractRelapsed 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.