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Haploidentical hematopoietic stem cell transplantation in aplastic anemia: a systematic review and meta-analysis of clinical outcome on behalf of the severe aplastic anemia working party of the European group for blood and marrow transplantation (SAAWP of EBMT)

Bone Marrow Transplantation volume 55, pages 1906–1917 (2020)Cite this article

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Abstract

Aplastic anemia (AA) is a serious hematological disorder, which is solely cured by hematopoietic stem cell transplantation (HSCT). Haploidentical HSCT is an emerging modality with encouraging outcomes in several blood conditions. The present study aims to comprehensively assess the feasibility and safety of haploidentical HSCT in patients with severe and very severe AA. It is a systematic review and meta-analysis of studies related to haploidentical stem cell transplantation in idiopathic AA investigating rates of successful engraftment, acute graft-versus-host disease (aGvHD), chronic GvHD (cGvHD), transplant-related mortality (TRM), and posttransplantation viral infections (including cytomegalovirus [CMV]) in patients with AA. The effects of reduced-intensity conditioning (RIC) and nonmyeloablative conditioning (NMA), as well as various GvHD prophylaxis regimens on these outcomes were evaluated. In total 15 studies were identified, (577 patients, 58.9% males), successful engraftment was observed in 97.3% of patients (95% CI, 95.9–98.7) while grades II–IV aGvHD and cGvHD were reported in 26.6% and 25.0%, respectively. The pooled incidence of TRM was 6.7% per year (95% CI, 4.0–9.4). RIC regimens were associated with higher proportions of successful engraftment (97.7% vs 91.7%, P = 0.03) and aGvHD (29.5% vs 18.7%, P = 0.008) when compared with NMA regimens with no differences in cGvHD or mortality incidence. When compared with methotrexate-containing regimens and other regimens, posttransplant cyclophosphamide-containing regimens reduced the rates of aGvHD (28.6%, 27.8%, and 12.8%, respectively, P = 0.02), CMV viremia (55.7%, 38.6%, and 10.4%, respectively, P < 0.001), and CMV disease in initially viremic patients (2.1%, 33.0%, and 0%, respectively, P < 0.001). We have concluded that Haploidentical HSCT was associated with promising outcomes in terms of successful engraftment and reduced complications. Future prospective trials are needed to identify the preferred conditioning regimen, GvHD prophylaxis, and graft source in the setting of haploidentical transplant for AA.

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Authors and Affiliations

  1. King Khalid University Hospital, Riyadh, Saudi Arabia

    Ghada ElGohary

  2. Faculty of Medicine Ain Shams University, Cairo, Egypt

    Ghada ElGohary

  3. King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia

    Riad El Fakih, Hazzaa Alzahrani, Ali Alahmari, Syed Osman Ahmed & Mahmoud Aljurf

  4. Saint-Louis Hospital, Paris, France

    Regis de Latour

  5. Federico II University, Naples, Italy

    Antonio Risitano

  6. King’s College Hospital/King’s College London, London, UK

    Judith Marsh

  7. University Hospital Ulm, Ulm, Germany

    Hubert Schrezenmeier & Britta Höchsmann

  8. Eurocord, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris, France

    Eliane Gluckman

  9. Monacord, Centre Scientifique de Monaco, 8 Quai Antoine 1er, 98000, Principauté de Monaco, Monaco

    Eliane Gluckman

  10. Hematology Unit, G Gaslini Children Research Hospital, Genova, Italy

    Filomena Pierri & Carlo Dufour

  11. Leiden University Medical Centre, Leiden, The Netherlands

    Constantijn Halkes

  12. Imperial College Healthcare/Imperial College London, London, UK

    Josu De la Fuente

  13. Pediatric Hematology Oncology, Azienda Ospedaliera Universitaria Integrata, Verona, Italy

    Simone Cesaro

  14. University Hospital Basel, Basel, Switzerland

    Jakob Passweg

  15. Universita’ Cattolica del Sacro Cuore, Roma, Italy

    Andrea Bacigalupo

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  1. Ghada ElGohary
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The first author wrote the initial draft, the second and last author thoroughly revised the initial draft, the rest of coauthors did the subsequent revisions.

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Correspondence to Ghada ElGohary or Riad El Fakih.

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ElGohary, G., El Fakih, R., de Latour, R. et al. Haploidentical hematopoietic stem cell transplantation in aplastic anemia: a systematic review and meta-analysis of clinical outcome on behalf of the severe aplastic anemia working party of the European group for blood and marrow transplantation (SAAWP of EBMT). Bone Marrow Transplant 55, 1906–1917 (2020). https://doi.org/10.1038/s41409-020-0897-2

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