• Jens Panse.
• Klinik für Onkologie, Hämatologie, Hämostaseologie und Stammzelltransplantation, Uniklinik RWTH Aachen.
• Dtsch. Med. Wochenschr. 2021 Apr 1; 146 (7): 451-454.

AbstractThe landscape of aplastic anemia (AA) is changing as new therapeutic options become available and sophistic diagnostics enable us to decipher various subgroups of bone marrow failure syndromes (BMFS) such as telomeropathies and other constitutional diseases with manifestations beyond childhood.This article briefly summarizes developments of the last few years with potential clinical impact and puts it into perspective. Focus is given to the growing list of inherited BMFS and the need to e. g. screen patients at all ages for telomeropathies before initiation of treatment as part of routine evaluation of AA. The usage of next generation sequencing (NGS) to differentiate between AA and malignancies such as myelodysplatic syndrome (MDS) and the need to watch out for potential clonal evolution during and after treatment is also briefly discussed. Recent data on combinations of immunosuppressive therapy (IST) with thrombopoietin receptor agonists (TRA) for patients with severe AA as well as newer data with TRA mono-therapy for patients with moderate AA are presented. Finally the importance of supportive measures, structures and quality of life aspects are highlighted.Thieme. All rights reserved.

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