title: 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.
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.
authors.affiliation: Clinical and Experimental Haematology Unit, Giannina Gaslini Children's Hospital, Genova, Italy.
authors.forename: Carlo
authors.initials: C
authors.lastname: Dufour
authors.affiliation: Department of Haematology & Bone Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
authors.affiliation: Paediatric Haematology and Oncology, University of Padova, Padova, Italy.
authors.forename: Elisa
authors.initials: E
authors.lastname: Carraro
authors.affiliation: Department of Haematology & Bone Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
authors.forename: Neha
authors.initials: N
authors.lastname: Bhatnagar
authors.affiliation: Paediatric Haematology and Oncology, University of Padova, Padova, Italy.
authors.forename: Marta
authors.initials: M
authors.lastname: Pillon
authors.affiliation: Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK.
authors.forename: Rob
authors.initials: R
authors.lastname: Wynn
authors.affiliation: Department of Paediatric Haematology & Oncology, Royal Hospital for Sick Children, Glasgow, UK.
authors.forename: Brenda
authors.initials: B
authors.lastname: Gibson
authors.affiliation: Department of Paediatric Haematology, The Children's Hospital, Sheffield, UK.
authors.forename: Ajay J
authors.initials: AJ
authors.lastname: Vora
authors.affiliation: BMT Unit, Royal Hospital for Children, Bristol, UK.
authors.forename: Colin G
authors.initials: CG
authors.lastname: Steward
authors.affiliation: Department of Paediatric Haematology & Oncology, Royal Hospital for Sick Children, Glasgow, UK.
authors.forename: Anna M
authors.initials: AM
authors.lastname: Ewins
authors.affiliation: University College Hospital, London, UK.
authors.forename: Rachael E
authors.initials: RE
authors.lastname: Hough
authors.affiliation: Division of Paediatrics, Imperial College Healthcare NHS Trust, London, UK.
authors.forename: Josu
authors.initials: J
authors.lastname: de la Fuente
authors.affiliation: Birmingham Children's Hospital, Birmingham, UK.
authors.forename: Mark
authors.initials: M
authors.lastname: Velangi
authors.affiliation: Department of Haematology & Bone Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
authors.forename: Persis J
authors.initials: PJ
authors.lastname: Amrolia
authors.affiliation: Department of Paediatric and Adolescent Haematology/Oncology and BMT, Great North Children's Hospital & Northern Institute for Cancer Research, Newcastle upon Tyne, UK.
authors.forename: Roderick
authors.initials: R
authors.lastname: Skinner
authors.affiliation: Haematology and Oncology Department, IRCCS A.O.U. San Martino Hospital, IST, Genoa, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Bacigalupo
authors.affiliation: Haematology, Federico II, Naples, Italy.
authors.forename: Antonio M
authors.initials: AM
authors.lastname: Risitano
authors.affiliation: Hôpital Saint-Louis, Paris, France.
authors.forename: Gerard
authors.initials: G
authors.lastname: Socie
authors.affiliation: Hôpital Saint-Louis, Paris, France.
authors.forename: Regis
authors.initials: R
authors.lastname: Peffault de Latour
authors.affiliation: Stem Cell Transplant Team, Division of Haematology, University Hospital Basel, Basel, Switzerland.
authors.forename: Jakob
authors.initials: J
authors.lastname: Passweg
authors.affiliation: Haematology, University Hospital of Basel, Basel, Switzerland.
authors.forename: Alicia
authors.initials: A
authors.lastname: Rovo
authors.affiliation: Haematology, University Hospital of Basel, Basel, Switzerland.
authors.forename: André
authors.initials: A
authors.lastname: Tichelli
authors.affiliation: Institute for Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Transfusion Service Baden-Württemberg-Hessen und University Hospital Ulm, Ulm, Germany.
authors.forename: Hubert
authors.initials: H
authors.lastname: Schrezenmeier
authors.affiliation: Institute for Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Transfusion Service Baden-Württemberg-Hessen und University Hospital Ulm, Ulm, Germany.
authors.forename: Britta
authors.initials: B
authors.lastname: Hochsmann
authors.affiliation: Division for Stem Cell Transplantation and Immunology, Hospital for Children and Adolescents, University Hospital Frankfurt, Goethe University, Frankfurt am Main, Germany.
authors.forename: Peter
authors.initials: P
authors.lastname: Bader
authors.affiliation: EBMT Data Office, University Medical Centre, Leiden, The Netherlands.
authors.forename: Anja
authors.initials: A
authors.lastname: van Biezen
authors.affiliation: Adult Haematology/HSCT Oncology Centre, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.
authors.forename: Mahmoud D
authors.initials: MD
authors.lastname: Aljurf
authors.affiliation: Department of Haematological Medicine, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Austin
authors.initials: A
authors.lastname: Kulasekararaj
authors.affiliation: Department of Haematological Medicine, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Judith C
authors.initials: JC
authors.lastname: Marsh
authors.affiliation: Department of Haematology & Bone Marrow Transplantation, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
authors.forename: Sujith
authors.initials: S
authors.lastname: Samarasinghe
mesh_terms: D000257:Adenoviridae Infections / Q000188:drug therapy / Q000453:epidemiology; D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D000961:Antilymphocyte Serum; D001803:Blood Transfusion / Q000706:statistics & numerical data; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000706:statistics & numerical data*; D016022:Case-Control Studies; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000453:epidemiology / Q000517:prevention & control; D006566:Herpesviridae Infections / Q000188:drug therapy / Q000453:epidemiology; D006648:Histocompatibility; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D053208:Kaplan-Meier Estimate; D007902:Length of Stay / Q000706:statistics & numerical data; D019520:Living Donors; D008297:Male; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects / Q000706:statistics & numerical data*; D011183:Postoperative Complications / Q000188:drug therapy / Q000453:epidemiology; D055031:Primary Graft Dysfunction / Q000453:epidemiology; D011788:Quality of Life; D012189:Retrospective Studies; D035781:Siblings; D015996:Survival Rate; D013601:T-Lymphocytes; D016896:Treatment Outcome; D014775:Virus Activation; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine
keywords: aplastic anaemia; paediatric aplastic anaemia; transplantation