Aplastic anemia and paroxysmal nocturnal hemoglobinuria in children and adults in two centers of Northern Greece

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

doi:10.3389/fonc.2022.947410

Aplastic anemia and paroxysmal nocturnal hemoglobinuria in children and adults in two centers of Northern Greece

Front Oncol. 2022 Nov 10:12:947410. doi: 10.3389/fonc.2022.947410. eCollection 2022.

Authors

Eleni Gavriilaki¹, Athanasios Tragiannidis², Maria Papathanasiou¹, Sotiria Besikli¹, Paraskevi Karvouni³, Vassiliki Douka¹, Eleni Paphianou¹, Emmanuel Hatzipantelis², Giorgos Papaioannou¹, Anastasia Athanasiadou¹, Anastasia Marvaki¹, Alkistis-Kira Panteliadou¹, Anna Vardi¹, Ioannis Batsis¹, Antonia Syrigou¹, Despina Mallouri¹, Chrysavgi Lalayanni¹, Ioanna Sakellari¹

Affiliations

¹ Hematology Department and Bone Marrow Transplant (BMT) Unit, G Papanicolaou Hospital, Thessaloniki, Greece.
² 2ndPaediatric Department, American Hellenic Educational Progressive Association (AHEPA) Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece.
³ School of Medicine, University of Thessaloniki, Thessaloniki, Greece.

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.

Keywords: bone marrow failure (BMF); bone marrow failure syndromes (BMFs); fanconi anaemia; hematopoietic (stem) cell transplantation (HCST); paroxysmal nocturnal hemoglobinuria.

Copyright © 2022 Gavriilaki, Tragiannidis, Papathanasiou, Besikli, Karvouni, Douka, Paphianou, Hatzipantelis, Papaioannou, Athanasiadou, Marvaki, Panteliadou, Vardi, Batsis, Syrigou, Mallouri, Lalayanni and Sakellari.