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Similar outcome of upfront‐unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party,Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT |
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| Authors: | Carlo Dufour Paul Veys Elisa Carraro Neha Bhatnagar Marta Pillon Rob Wynn Brenda Gibson Ajay J. Vora Colin G. Steward Anna M. Ewins Rachael E. Hough Josu de la Fuente Mark Velangi Persis J. Amrolia Roderick Skinner Andrea Bacigalupo Antonio M. Risitano Gerard Socie Regis Peffault de Latour Jakob Passweg Alicia Rovo André Tichelli Hubert Schrezenmeier Britta Hochsmann Peter Bader Anja van Biezen Mahmoud D. Aljurf Austin Kulasekararaj Judith C. Marsh Sujith Samarasinghe |
| Affiliation: | 1. Clinical and Experimental Haematology Unit, Giannina Gaslini Children's Hospital, Genova, Italy;2. Department of Haematology & Bone Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK;3. Paediatric Haematology and Oncology, University of Padova, Padova, Italy;4. Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK;5. Department of Paediatric Haematology & Oncology, Royal Hospital for Sick Children, Glasgow, UK;6. Department of Paediatric Haematology, The Children's Hospital, Sheffield, UK;7. BMT Unit, Royal Hospital for Children, Bristol, UK;8. University College Hospital, London, UK;9. Division of Paediatrics, Imperial College Healthcare NHS Trust, London, UK;10. Birmingham Children's Hospital, Birmingham, UK;11. Department of Paediatric and Adolescent Haematology/Oncology and BMT, Great North Children's Hospital & Northern Institute for Cancer Research, Newcastle upon Tyne, UK;12. Haematology and Oncology Department, IRCCS A.O.U. San Martino Hospital, IST, Genoa, Italy;13. Haematology, Federico II, Naples, Italy;14. H?pital Saint‐Louis, Paris, France;15. Stem Cell Transplant Team, Division of Haematology, University Hospital Basel, Basel, Switzerland;16. Haematology, University Hospital of Basel, Basel, Switzerland;17. Institute for Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Transfusion Service Baden‐Württemberg‐Hessen und University Hospital Ulm, Ulm, Germany;18. Division for Stem Cell Transplantation and Immunology, Hospital for Children and Adolescents, University Hospital Frankfurt, Goethe University, Frankfurt am Main, Germany;19. EBMT Data Office, University Medical Centre, Leiden, The Netherlands;20. Adult Haematology/HSCT Oncology Centre, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia;21. Department of Haematological Medicine, King's College Hospital NHS Foundation Trust, London, UK |
| Abstract: | We explored the feasibility of unrelated donor haematopoietic stem cell transplant (HSCT) upfront without prior immunosuppressive therapy (IST) in paediatric idiopathic severe aplastic anaemia (SAA). This cohort was then compared to matched historical controls who had undergone first‐line therapy with a matched sibling/family donor (MSD) HSCT (n = 87) or IST with horse antithymocyte globulin and ciclosporin (n = 58) or second‐line therapy with unrelated donor HSCT post‐failed IST (n = 24). The 2‐year overall survival in the upfront cohort was 96 ± 4% compared to 91 ± 3% in the MSD controls (P = 0·30) and 94 ± 3% in the IST controls (P = 0·68) and 74 ± 9% in the unrelated donor HSCT post‐IST failure controls (P = 0·02).The 2‐year event‐free survival in the upfront cohort was 92 ± 5% compared to 87 ± 4% in MSD controls (P = 0·37), 40 ± 7% in IST controls (P = 0·0001) and 74 ± 9% in the unrelated donor HSCT post‐IST failure controls (n = 24) (P = 0·02). Outcomes for upfront‐unrelated donor HSCT in paediatric idiopathic SAA were similar to MSD HSCT and superior to IST and unrelated donor HSCT post‐IST failure. Front‐line therapy with matched unrelated donor HSCT is a novel treatment approach and could be considered as first‐line therapy in selected paediatric patients who lack a MSD. |
| Keywords: | paediatric aplastic anaemia aplastic anaemia transplantation |