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Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party

Bone Marrow Transplantation volume 55, pages 1050–1058 (2020)Cite this article

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Abstract

In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (n = 33). The cumulative incidence of neutrophil engraftment was 67% (CI95%: 51–83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II–III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0–20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64–93), and 2-year graft-versus-host disease-free survival was 63% (46–81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81–100) versus 64% (41–87), p = 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.

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Fig. 1: Stacked cumulative incidence of neutrophil engraftment.
Fig. 2: Cumulative incidence of platelet engraftment.
Fig. 3: Cumulative incidence of separate aGvHD grades.
Fig. 4: Overall survival.

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Acknowledgements

The authors are grateful to the Fondation HPN France—Aplasie Médullaire (Paroxysmal Nocturnal Hemoglobinuria—Aplastic Anemia Foundation in France) for helping fund this study.

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Author notes

  1. These author contributed equally: Carlo Dufour, Régis Peffault de Latour

Authors and Affiliations

  1. Hematology-Transplantation Department, Saint-Louis Hospital, Paris, France

    Pedro H. Prata, Gérard Socié & Régis Peffault de Latour

  2. EBMT Statistical Unit, Leiden, The Netherlands

    Dirk-Jan Eikema

  3. First State Pavlov Medical University, St Petersburg, Russia

    Boris Afansyev

  4. EBMT Data Office, Leiden, The Netherlands

    Paul Bosman

  5. Leiden University Hospital, Leiden, The Netherlands

    Frans Smiers

  6. Departamento de Medicina, Gregorio Maranon G.U. Hospital, Instituto de Investigación Sanitaria Gregorio Marañón, Universidad Complutense, Madrid, Spain

    José L. Diez-Martin

  7. Sírio-Libanês Hospital, Sao Paulo, Brazil

    Celso Arrais-Rodrigues & Vanderson Rocha

  8. Medical Park Hospitals, Antalya, Turkey

    Yener Koc

  9. Clinique Universitaire St. Luc, Brussels, Belgium

    Xavier Poiré

  10. CHU Lapeyronie, Montpellier, France

    Anne Sirvent

  11. University Hospital Eppendorf, Hamburg, Germany

    Nicolaus Kröger

  12. Ospedale dei Bambini Spedali Civili, Brescia, Italy

    Fulvio Porta

  13. St. Anna Kinderspital, Vienna, Austria

    Wolfgang Holter

  14. Christie NHS Trust Hospital, Manchester, UK

    Adrian Bloor

  15. Groupe Hospitalier Pellegrin-Enfants, Bordeaux, France

    Charlotte Jubert

  16. Hannover Medical School, Hannover, Germany

    Arnold Ganser

  17. Fundeni Clinical Institute, Bucharest, Romania

    Alina Tanase

  18. Centre Henri Becquerel, Rouen, France

    Anne-Lise Ménard

  19. Ospedale San Gerardo, Monza, Italy

    Pietro Pioltelli

  20. Hospital Universitario Virgen del Rocio, Sevilla, Spain

    José A. Pérez-Simón

  21. Singapore General Hospital, Singapore, Singapore

    Aloysius Ho

  22. King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia

    Mahmoud Aljurf

  23. Nottingham University, Nottingham, UK

    Nigel Russell

  24. Centre Hospitalier Lyon Sud, Lyon, France

    Helene Labussiere-Wallet

  25. Ghent University Hospital, Ghent, Belgium

    Tessa Kerre

  26. Université de Paris, INSERM U976, Paris, France

    Gérard Socié

  27. Federico II University of Naples, Naples, Italy

    Antonio Risitano

  28. Giannina Gaslini Children’s Hospital, Genoa, Italy

    Carlo Dufour

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on behalf of the SAA WP of the EBMT

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PHP, RPL, CD and AR planned this study. PHP, DJE and RPL wrote the paper with support of GS. PB was responsible for data collection and processing. DJE performed statistical analyses. All authors provided critical feedback and helped shape the research, analyses, and paper.

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Correspondence to Pedro H. Prata.

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Prata, P.H., Eikema, DJ., Afansyev, B. et al. Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party. Bone Marrow Transplant 55, 1050–1058 (2020). https://doi.org/10.1038/s41409-019-0773-0

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