title: Feasibility and Outcome of Haploidentical Hematopoietic Stem Cell Transplantation with Post-Transplant High-Dose Cyclophosphamide for Children and Adolescents with Hematologic Malignancies: An AIEOP-GITMO Retrospective Multicenter Study.
abstract: Post-transplant high-dose cyclophosphamide (PTCy) is a novel approach to prevent graft-versus-host disease (GVHD) and rejection in patients given haploidentical hematopoietic stem cell transplantation (HSCT). Thirty-three patients with high-risk hematologic malignancies and lacking a match-related or -unrelated donor were treated with PTCy haploidentical HSCT in 5 Italian AIEOP centers. Nineteen patients had a nonmyeloablative preparative regimen (57%), and 14 patients received a full myeloablative conditioning regimen (43%). No patients received serotherapy; GVHD prophylaxis was based on PTCy (50 mg/kg on days +3 and +4) combined with mycophenolate plus tacrolimus or cyclosporine A. Neutrophil and platelet engraftment was achieved on days +17 (range, 14 to 37) and +27 (range, 16 to 71). One patient had autologous reconstitution for anti-HLA antibodies. Acute GVHD grades II to IV and III to IV and chronic GVHD developed in 22% (95% CI, 11 to 42), 3% (95% CI, 0 to 21), and 4% (95% CI, 0 to 27) of cases, respectively. The 1-year overall survival rate was 72% (95% CI, 56 to 88), progression-free survival rate was 61% (95% CI, 43 to 80), cumulative incidence of relapse was 24% (95% CI, 13 to 44), and transplant-related mortality was 9% (95% CI, 3 to 26). The univariate analysis for risk of relapse incidence showed how 3 significant variables, mother as donor (P = .02), donor gender as female (P = .04), and patient gender as female (P = .02), were significantly associated with a lower risk of relapse. Disease progression was the main cause of death. PTCy is a safe procedure also for children and adolescents who have already received several lines of chemotherapy. Among the different diseases, a trend for better 1-year rates of overall survival was obtained for nonacute leukemia patients.
authors: Pediatric Onco-Hematology and Stem Cell Transplant Division, City of Health and Science, Regina Margherita Children's Hospital, Turin, Italy. Electronic address: massimo.berger@unito.it.
authors: Massimo
authors: M
authors: Berger
authors: Department of Pediatric Hematology-Oncology, IRCCS G. Gaslini, Genoa, Italy.
authors: Edoardo
authors: E
authors: Lanino
authors: Pediatric Hematology-Oncology, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
authors: Simone
authors: S
authors: Cesaro
authors: Pediatric Hematology-Oncology, IRCCS Policlinico San Matteo Foundation, Pavia, Italy.
authors: Marco
authors: M
authors: Zecca
authors: Pediatric Onco-Hematology and Stem Cell Transplant Division, City of Health and Science, Regina Margherita Children's Hospital, Turin, Italy.
authors: Elena
authors: E
authors: Vassallo
authors: Department of Pediatric Hematology-Oncology, IRCCS G. Gaslini, Genoa, Italy.
authors: Maura
authors: M
authors: Faraci
authors: Pediatric Hematology-Oncology, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
authors: Massimiliano
authors: M
authors: De Bortoli
authors: Pediatric Onco-Hematology and Stem Cell Transplant Division, City of Health and Science, Regina Margherita Children's Hospital, Turin, Italy.
authors: Veronica
authors: V
authors: Barat
authors: Pediatric Oncology and Hematology Unit "Lalla Seràgnoli", Department of Pediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Bologna, Italy.
authors: Arcangelo
authors: A
authors: Prete
authors: Pediatric Onco-Hematology and Stem Cell Transplant Division, City of Health and Science, Regina Margherita Children's Hospital, Turin, Italy.
authors: Franca
authors: F
authors: Fagioli
mesh_terms: D000293:Adolescent; D000328:Adult; D064591:Allografts; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage*; D018572:Disease-Free Survival; D005260:Female; D019337:Hematologic Neoplasms / Q000401:mortality* / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D015996:Survival Rate; D013997:Time Factors; D019172:Transplantation Conditioning
chemical_list: D003520:Cyclophosphamide
keywords: Hematological malignancies; PTCy-haplo HSCT; Pediatric