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A multicentre ambispective observational study into the incidence and clinical management of aplastic anaemia in Spain (IMAS study)

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Abstract

Aplastic anemia (AA) is a rare, life-threatening hematological disease, with a poorly defined incidence. As the data available on AA varies substantially worldwide, a multicenter, ambispective, observational study was carried out between 2010 and 2019 to assess the incidence, clinical management and survival of AA at seven Spanish hospitals. The incidence of AA was 2.83 per million inhabitants per year, consistent with that reported previously in Europe, with a median age at diagnosis of 61 years-old (range 12–86), and a similar number of males and females. The initial diagnosis was severe or very severe AA in 55.8% of cases and 93.7% required transfusion. The most frequent first line therapy was anti-thymocyte globulin (ATG) plus cyclosporin A (CsA, 44.2%), followed by other CsA-based regimes (46.3%), with hematopoietic stem cell transplantation an infrequent 1st line therapy. The 6-month response rate was 68.2%, which then increased over a median follow-up of 3.9 years. The 5-year overall survival (5OS) was 73.6%, similar in severe (78.6%) and very severe AA patients (74.6%) but lower in moderate AA (MAA) patients (68.4%). The 5OS was 100% in 0–25 year-old patients but dropping to 58.3% in patients ≥ 60 years-old. At the last contact, 75.8% of the patients were alive. In conclusion, the incidence, characteristics and management of AA in our study are consistent with that reported previously. In terms of survival, although the global long-term OS rate was good, there is room for improvement, particularly in older patients. Finally, what appears to be a worse long-term survival of MAA patients, as reported previously, reinforces the importance of not underestimating this condition when diagnosed as MAA.

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Acknowledgements

The authors would like to acknowledge the efforts of M. Paz Regalado (Biomedred) and of Itxaso Calbano (Instituto de Investigación Sanitaria Biodonostia) in the analysis of the data, Lourdes Aguirre (Hospital Universitario Donostia) in the data collection, and of M. Paz Regalado and Mark Sefton (Biomedred) in the preparation of the manuscript. This study was promoted by the Instituto de Investigación Sanitaria Biodonostia.

Funding

The funds to carry out this study were provided by Novartis S.A. through a grant awarded to Dr C Vallejo.

Author information

Authors and Affiliations

  1. Complejo Asistencial Universitario de Salamanca, Salamanca, Spain

    Carlos Vallejo

  2. Hospital Universitario Donostia, San Sebastián, Spain

    Carlos Vallejo

  3. PETHEMA Cooperative Group, Madrid, Spain

    Carlos Vallejo

  4. Instituto de Investigación Sanitaria Biodonostia, Donostia-San Sebastián, Spain

    Carlos Vallejo, Brígida Esteban & M. Lorena Codesido

  5. Hospital Universitario Virgen de La Victoria, Málaga, Spain

    Ana Rosell

  6. Institut Català d’Oncologia, Hospital Universitari Germans Trias i Pujol, Josep Carreras Leukemia Research Institute, Universitat Autònoma de Barcelona, Badalona, Spain

    Blanca Xicoy

  7. Hospital General Universitario de Alicante, Alicante, Spain

    Carmen García

  8. Hospital Universitario Álvaro Cunqueiro, Vigo, Spain

    Carmen Albo

  9. Hospital Clínico Universitario San Carlos, Madrid, Spain

    Marta Polo

  10. Hospital Universitario La Fe, Valencia, Spain

    Isidro Jarque

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  1. Carlos Vallejo
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  2. Ana Rosell
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  3. Blanca Xicoy
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Contributions

All authors contributed to the data collection at the participating centres, and they revised and approved the final version of the manuscript submitted for publication.

Corresponding author

Correspondence to Carlos Vallejo.

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Ethics approval

The present study was approved by the institutional review board at the coordinating center, the Hospital Universitario Donostia, San Sebastián, Spain (Basque Country Ethics Committee Ref.: EPA2017043-SP) and it was carried out in accordance with the Helsinki declaration regarding research on humans.

Consent to participate

All patients provided their written informed consent to participate in the study.

Competing interests

CV received research funding from Novartis S.A. However, the funding body did not influence the performance of the study in any way, nor the preparation of the manuscript or the decision to publish. AR, BX, CG, CA, MP, IJ, BE, and MLC have no competing interests to declare that are relevant to the content of this article.

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Vallejo, C., Rosell, A., Xicoy, B. et al. A multicentre ambispective observational study into the incidence and clinical management of aplastic anaemia in Spain (IMAS study). Ann Hematol 103, 705–713 (2024). https://doi.org/10.1007/s00277-023-05602-x

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