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Biol Blood Marrow Transplant. 2018 Jun;24(6):1223-1231. doi: 10.1016/j.bbmt.2018.01.022. Epub 2018 Feb 2.

Outcomes of Children with Hemophagocytic Lymphohistiocytosis Given Allogeneic Hematopoietic Stem Cell Transplantation in Italy.

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Department of Women's and Children's Health, Pediatric Hemato-Oncology, University Hospital of Padova, Padova, Italy.
Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Pediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Torino, Italy.
Bone Marrow Transplantation Unit, Pediatric Department of Milano-Bicocca University, MBBM Foundation, Monza, Italy.
Bone Marrow Transplantation Unit, IRCCS G. Gaslini, Genova, Italy.
Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy.
Oncology-Hematology and BMT Unit, Ospedale dei Bambini, Spedali Civili, Brescia, Italy.
Azienda Sanitaria Provinciale, Ragusa, Italy.
Pediatric Hematology-Oncology, Meyer Children's Hospital, Firenze, Italy.
Department of Hemato-Oncology, Santobono-Pausilipon Hospital, BMT Unit, Napoli, Italy.
Department of Pediatrics, Institute of Maternal and Child Health, IRCCS Burlo Garofolo Trieste, Trieste, Italy.
Pediatric Hematology Oncology, Azienda Ospedaliera Universitaria Integrata Verona, Verona, Italy.
Pediatric Hematology and Oncology, SM della Misericordia Hospital, Perugia, Italy.
Department of Hematology, Bone Marrow Transplant Center, Pescara, Italy.
Pediatric Hematology and Oncology, ARNAS Ospedale Civico di Palermo, Palermo, Italy.
Department of Pediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Pediatric Oncology and Hematology Unit, Bologna, Italy.
Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy; Department of Pediatric Sciences, University of Pavia, Italy. Electronic address:


We report on 109 patients with hemophagocytic lymphohistiocytosis (HLH) undergoing 126 procedures of allogeneic hematopoietic stem cell transplantation (HSCT) between 2000 and 2014 in centers associated with the Italian Pediatric Hematology Oncology Association. Genetic diagnosis was FHL2 (32%), FHL3 (33%), or other defined disorders known to cause HLH (15%); in the remaining patients no genetic abnormality was found. Donor for first transplant was an HLA-matched sibling for 25 patients (23%), an unrelated donor for 73 (67%), and an HLA-partially matched family donor for 11 children (10%). Conditioning regimen was busulfan-based for 61 patients (56%), treosulfan-based for 21 (20%), and fludarabine-based for 26 children (24%). The 5-year probabilities of overall survival (OS) and event-free survival (EFS) were 71% and 60%, respectively. Twenty-six patients (24%) died due to transplant-related causes, whereas 14 (13%) and 10 (9%) patients experienced graft rejection and/or relapse, respectively. Twelve of 14 children given a second HSCT after graft failure/relapse are alive and disease-free. Use of HLA-partially matched family donors was associated with higher risk of graft failure and thus with lower EFS (but not with lower OS) in multivariable analysis. Active disease at transplantation did not significantly affect prognosis. These data confirm that HSCT can cure most HLH patients, active disease not precluding successful transplantation. Because in HLH patients HLA-haploidentical HSCT performed through CD34+ cell positive selection was found to be associated with poor sustained engraftment of donor cells, innovative approaches able to guarantee a more robust engraftment are warranted in patients given this type of allograft.


Allogeneic HSCT; Cytotoxicity; Graft failure; Hemophagocytic lymphohistiocytosis; Perforin; Second allograft


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