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Combined intensive immunosuppression and eculizumab for aplastic anemia in the context of hemolytic paroxysmal nocturnal hemoglobinuria: a retrospective analysis

Bone Marrow Transplantation volume 53, pages 105–107 (2018)Cite this article

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Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by complement-mediated intravascular hemolysis, thromboembolic complications and bone marrow failure (BMF), which may range from mild cytopenia to severe aplastic anemia (SAA).1, 2 Even if the clinical manifestations may change over time,3 most, if not all PNH patients, have a degree of aplastic anemia (AA) with or without significant hemolysis that may require a treatment according to its severity. While classical hemolytic PNH patients without significant cytopenia other than anemia may respond well to eculizumab,4, 5 concomitant treatment for patients with associated severe thrombocytopenia or neutropenia is still unknown. Thus, in a small proportion of patients, AA and hemolysis may be present at the same time. To date, to the best of our knowledge, beyond some single case experience,6, 7 no data are available in the literature concerning the management of this rare condition. Sequential treatment with IST followed by eculizumab seems a logical option, but the majority of PNH patients who develop marrow failure are already on anti-complement treatment or may exhibit clinically meaningful hemolysis, raising the question of a possible concomitant therapy with IST and eculizumab.

Here we retrospectively assessed characteristics and outcomes of patients who have received ‘intensive IST’ (defined as any lymphocyte-depleting IST regimen based on anti-lymphocyte antibodies, such as horse- or rabbit-antithymocyte globulin,8 or anti-CD52 monoclonal antibody alemtuzumab9) in combination with eculizumab (concomitantly or sequentially administered), from a cohort of PNH patients seen at our reference centers in France (Saint Louis Hospital of Paris), and in Italy (Federico II University of Naples) between 2007 and 2016.

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Authors and Affiliations

  1. Department of Hematology and Transplant Unit, APHP Saint Louis Hospital, Paris, France

    S Pagliuca, F S De Fontbrune, M Robin, D Michonneau, G Socié & R P De Latour

  2. Department of Hematology and Transplant Unit, University Federico II of Naples, Naples, Italy

    A M Risitano & S Marotta

  3. Department of Bone marrow transplantion, University La Sapienza, Rome, Italy

    A P Iori

  4. Department of Heamtology Centre Hospitalier Régional Universitaire de Tours, Tours, France,

    A Villate

  5. Department of Hematology Centre Hospitalier Universitaire de Poitiers, Poitiers, France,

    D Desmier

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  1. S Pagliuca
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  4. M Robin
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  10. G Socié
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  11. R P De Latour
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Corresponding author

Correspondence to S Pagliuca.

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Author contributions

Conception and design: SP, AMR and RPL. Data collection and assembly: SP, FS, AMR, AI, SM, FS, DM, AV, DD and RPL. Data analysis and interpretation: SP, FS, RPL, AMR. Manuscript: SP, RPL, AMR. Final approval of manuscript: SP, AMR, AI, SM, FS, DM, AV, DD, MR, GS, RPL.

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Pagliuca, S., Risitano, A., De Fontbrune, F. et al. Combined intensive immunosuppression and eculizumab for aplastic anemia in the context of hemolytic paroxysmal nocturnal hemoglobinuria: a retrospective analysis. Bone Marrow Transplant 53, 105–107 (2018). https://doi.org/10.1038/bmt.2017.220

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