Comments

Is hematopoietic stem cell transplantation indicated in THPO-mutated congenital amegakaryocytic thrombocytopenia? Comment on: "Avatrombopag as a novel salvage therapy for refractory thrombocytopenia in THPO-mutated congenital amegakaryocytic thrombocytopenia after allogeneic hematopoietic stem cell transplantation"

Aix Marseille Univ, APHM, INSERM, INRAe, C2VN, La Timone Children’s Hospital, Department of pediatric hematology, immunology and oncology, Marseille
Dipartimento Medico, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy; Dipartimento di Medicina Interna, Università di Pavia, Pavia
Haematologica Early view Oct 15, 2026 https://doi.org/10.3324/haematol.2026.302090