Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the Literature

Congenital or acquired severe aplastic anaemia (SAA) is cured by bone marrow transplantation (BMT) from a histocompatible leukocyte antigen- (HLA-) identical sibling. The best conditioning regimen is cyclophosphamide (CTX) with or without antithymocyte globulin (ATG), followed by short-term methotre...

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Main Authors: Katia Perruccio, Elena Mastrodicasa, Francesco Arcioni, Ilaria Capolsini, Carla Cerri, Grazia Gurdo, Maurizio Caniglia
Format: Article
Language:English
Published: Wiley 2015-01-01
Series:Case Reports in Hematology
Online Access:http://dx.doi.org/10.1155/2015/321602
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author Katia Perruccio
Elena Mastrodicasa
Francesco Arcioni
Ilaria Capolsini
Carla Cerri
Grazia Gurdo
Maurizio Caniglia
author_facet Katia Perruccio
Elena Mastrodicasa
Francesco Arcioni
Ilaria Capolsini
Carla Cerri
Grazia Gurdo
Maurizio Caniglia
author_sort Katia Perruccio
collection DOAJ
description Congenital or acquired severe aplastic anaemia (SAA) is cured by bone marrow transplantation (BMT) from a histocompatible leukocyte antigen- (HLA-) identical sibling. The best conditioning regimen is cyclophosphamide (CTX) with or without antithymocyte globulin (ATG), followed by short-term methotrexate (MTX) and cyclosporine A (CsA) to prevent graft-versus-host disease (GvHD). In our pediatric oncology-hematology unit, a 5-year-old girl with SAA was treated with two BMT from the same HLA-identical sibling donor. Severe CsA-induced adverse events (severe hypertension and PRES) after the first BMT led necessarily to CSA withdrawal. Alternative immunosuppressive treatment for GvHD prevention as tacrolimus and mycophenolate were not tolerated by our patient because toxicity > grade II. For this reason we decided to administrate sirolimus alone as GvHD prophylaxis and to prevent disease relapse after the rescue BMT. Here we report the successful use of sirolimus alone for GvHD prophylaxis after the second transplant in a pediatric BMT setting for SAA.
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spelling doaj-art-dbe08da73faa41b6a2cbcffddf6f003d2025-08-20T03:23:56ZengWileyCase Reports in Hematology2090-65602090-65792015-01-01201510.1155/2015/321602321602Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the LiteratureKatia Perruccio0Elena Mastrodicasa1Francesco Arcioni2Ilaria Capolsini3Carla Cerri4Grazia Gurdo5Maurizio Caniglia6Pediatric Oncology Hematology Unit, Perugia General Hospital, Località Sant’Andrea delle Fratte, 06156 Perugia, ItalyPediatric Oncology Hematology Unit, Perugia General Hospital, Località Sant’Andrea delle Fratte, 06156 Perugia, ItalyPediatric Oncology Hematology Unit, Perugia General Hospital, Località Sant’Andrea delle Fratte, 06156 Perugia, ItalyPediatric Oncology Hematology Unit, Perugia General Hospital, Località Sant’Andrea delle Fratte, 06156 Perugia, ItalyPediatric Oncology Hematology Unit, Perugia General Hospital, Località Sant’Andrea delle Fratte, 06156 Perugia, ItalyPediatric Oncology Hematology Unit, Perugia General Hospital, Località Sant’Andrea delle Fratte, 06156 Perugia, ItalyPediatric Oncology Hematology Unit, Perugia General Hospital, Località Sant’Andrea delle Fratte, 06156 Perugia, ItalyCongenital or acquired severe aplastic anaemia (SAA) is cured by bone marrow transplantation (BMT) from a histocompatible leukocyte antigen- (HLA-) identical sibling. The best conditioning regimen is cyclophosphamide (CTX) with or without antithymocyte globulin (ATG), followed by short-term methotrexate (MTX) and cyclosporine A (CsA) to prevent graft-versus-host disease (GvHD). In our pediatric oncology-hematology unit, a 5-year-old girl with SAA was treated with two BMT from the same HLA-identical sibling donor. Severe CsA-induced adverse events (severe hypertension and PRES) after the first BMT led necessarily to CSA withdrawal. Alternative immunosuppressive treatment for GvHD prevention as tacrolimus and mycophenolate were not tolerated by our patient because toxicity > grade II. For this reason we decided to administrate sirolimus alone as GvHD prophylaxis and to prevent disease relapse after the rescue BMT. Here we report the successful use of sirolimus alone for GvHD prophylaxis after the second transplant in a pediatric BMT setting for SAA.http://dx.doi.org/10.1155/2015/321602
spellingShingle Katia Perruccio
Elena Mastrodicasa
Francesco Arcioni
Ilaria Capolsini
Carla Cerri
Grazia Gurdo
Maurizio Caniglia
Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the Literature
Case Reports in Hematology
title Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the Literature
title_full Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the Literature
title_fullStr Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the Literature
title_full_unstemmed Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the Literature
title_short Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the Literature
title_sort sirolimus based immunosuppression as gvhd prophylaxis after bone marrow transplantation for severe aplastic anaemia a case report and review of the literature
url http://dx.doi.org/10.1155/2015/321602
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