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

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

Eleni Gavriilaki  1 , Athanasios Tragiannidis  2 , Maria Papathanasiou  1 , Sotiria Besikli  1 , Paraskevi Karvouni  3 , Vassiliki Douka  1 , Eleni Paphianou  1 , Emmanuel Hatzipantelis  2 , Giorgos Papaioannou  1 , Anastasia Athanasiadou  1 , Anastasia Marvaki  1 , Alkistis-Kira Panteliadou  1 , Anna Vardi  1 , Ioannis Batsis  1 , Antonia Syrigou  1 , Despina Mallouri  1 , Chrysavgi Lalayanni  1 , Ioanna Sakellari  1
Affiliations

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

Eleni Gavriilaki et al. Front Oncol. .

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.

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Conflict of interest statement

EG has received honoraria from Alexion and Omeros Pharmaceuticals. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
Kaplan Meier curves for overall survival in aplastic anemia patients. (A) From disease diagnosis in patients that did not receive allogeneic hematopoietic cell transplantation (alloHCT) (B) From alloHCT.
See this image and copyright information in PMC

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