학술논문
Aplastic anemia and paroxysmal nocturnal hemoglobinuria in children and adults in two centers of Northern Greece
Document Type
article
Author
Eleni Gavriilaki; Athanasios Tragiannidis; Maria Papathanasiou; Sotiria Besikli; Paraskevi Karvouni; Vassiliki Douka; Eleni Paphianou; Emmanuel Hatzipantelis; Giorgos Papaioannou; Anastasia Athanasiadou; Anastasia Marvaki; Alkistis-Kira Panteliadou; Anna Vardi; Ioannis Batsis; Antonia Syrigou; Despina Mallouri; Chrysavgi Lalayanni; Ioanna Sakellari
Source
Frontiers in Oncology, Vol 12 (2022)
Subject
Language
English
ISSN
2234-943X
Abstract
Bone marrow failure (BMF) syndromes are a group of various hematological diseases with cytopenia as a main common characteristic. Given their rarity and continuous progress in the field, we aim to provide data considering the efficiency and safety of the therapeutic methods, focusing on the treatment of aplastic anemia(AA) and paroxysmal nocturnal hemoglobinuria (PNH). We enrolled consecutive patients diagnosed with BMF in two referral centers of Northern Greece from 2008 to 2020. We studied 43 patients with AA (37 adults and 6 children/adolescents) and 6 with classical PNH. Regarding classical PNH, 4 patients have received eculizumab treatment with 1/4 presenting extravascular hemolysis. Among 43 patients with aplastic anemia, PNH clones were detected in 11. Regarding patients that did not receive alloHCT (n=15), 14/15 were treated with ATG and cyclosporine as first line, with the addition of eltrombopag in patients treated after its approval (n=9). With a median follow-up of 16.7 (1.8-56.2) months from diagnosis, 12/14 (85.7%) are alive (4-year OS: 85.1%). AlloHCT was performed in 28 patients. Five patients developed TA-TMA which did not resolve in 3/5 (all with a pre-transplant PNH clone). With the follow-up among survivors reaching 86.3 (6.3-262.4) months, 10-year OS was 56.9%, independently associated with PNH clones after adjusting for age (p=0.024). In conclusion, our real-world experience confirms that novel treatments are changing the field of BMF syndromes. Nevertheless, there is still an unmet need to personalize algorithms in this field.