title: T Cell-Depleted and T Cell-Replete HLA-Haploidentical Stem Cell Transplantation for Non-malignant Disorders.
abstract: Hematopoietic stem cell transplantation (HSCT) is a treatment option for children with malignant and non-malignant disorders as well as an expanding number of inherited disorders. However, only a limited portion of patients in the need of an allograft have an HLA-compatible, either related or unrelated, donor. Haploidentical HSCT is now considered a valid treatment option, especially in view of the recent insights in terms of graft manipulation. This review will offer an overview of clinical results obtained through the use of haploidentical HSCT in non-malignant diseases. We will analyze major advantages and drawbacks of both T cell depleted and unmanipulated HSCT, discussing future challenges for further improving patients' outcome.
  
  
  
  T cell depletion (TCD) aims to reduce the morbidity and mortality associated with graft-versus-host disease (GvHD). However, the delayed immune recovery and the risk of graft failure still remain potential problems. In the last years, the use of post-transplant cyclophosphamide has been shown to be an alternative effective strategy to prevent GvHD in recipients of haploidentical HSCT. Recent data suggest that both T cell depleted and T cell-replete haplo-HSCT are suitable options to treat children with several types of non-malignant disorders lacking an HLA-identical donor.
authors.affiliation: Department of Pediatric Hematology-Oncology, IRCCS, Bambino Gesù Children's Hospital, Piazza Sant'Onofrio, 4 - 00165, Rome, Italy. alice.bertaina@opbg.net.
authors.forename: Alice
authors.initials: A
authors.lastname: Bertaina
authors.affiliation: Department of Pediatric Hematology-Oncology, IRCCS, Bambino Gesù Children's Hospital, Piazza Sant'Onofrio, 4 - 00165, Rome, Italy.
authors.forename: Angela
authors.initials: A
authors.lastname: Pitisci
authors.affiliation: Department of Pediatric Hematology-Oncology, IRCCS, Bambino Gesù Children's Hospital, Piazza Sant'Onofrio, 4 - 00165, Rome, Italy.
authors.forename: Matilde
authors.initials: M
authors.lastname: Sinibaldi
authors.affiliation: Department of Pediatric Hematology-Oncology, IRCCS, Bambino Gesù Children's Hospital, Piazza Sant'Onofrio, 4 - 00165, Rome, Italy.
authors.forename: Mattia
authors.initials: M
authors.lastname: Algeri
mesh_terms: D006086:Graft vs Host Disease / Q000517:prevention & control; D016179:Granulocyte Colony-Stimulating Factor / Q000494:pharmacology; D006680:HLA Antigens / Q000276:immunology*; D006801:Humans; D008212:Lymphocyte Depletion; D009369:Neoplasms / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000009:adverse effects; D013234:Stem Cells / Q000187:drug effects / Q000276:immunology / Q000378:metabolism; D013601:T-Lymphocytes / Q000276:immunology; D014184:Transplantation, Homologous
chemical_list: D006680:HLA Antigens; D016179:Granulocyte Colony-Stimulating Factor
keywords: Graft-versus-host disease; Haploidentical stem cell transplantation; Immune recovery; Non-malignant disorders; T cell depletion