title: Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors.
abstract: Thalassemia-free survival after allogeneic stem cell transplantation (SCT) is about 80-90% with either matched-related or -unrelated donors. We explored the use of a mismatched-related ('haplo- ') donor. All patients received two courses of pretransplant immunosuppressive therapy (PTIS) with fludarabine (Flu) and dexamethasone (Dxm). After two courses of PTIS, a conditioning regimen of rabbit antithymocyte globulin, Flu and IV busulfan (Bu) was given followed by T-cell-replete peripheral blood progenitor cells. GvHD prophylaxis consisted of cyclophosphamide (Cy) on days SCT +3 and +4 (post-Cy), and on day SCT +5 tacrolimus or sirolimus was started together with a short course of mycophenolate mofetil. Thirty-one patients underwent haplo-SCT. Their median age was 10 years (range, 2-20 years). Twenty-nine patients engrafted with 100% donor chimerism. Two patients suffered primary graft failure. Median time to neutrophil engraftment was 14 days (range, 11-18 days). Five patients developed mild to moderate, reversible veno-occlusive disease, while nine patients developed acute GvHD grade II. Only five patients developed limited-chronic GvHD. Projected overall and event-free survival rates at 2 years are 95% and 94%, respectively. The median follow up time is 12 months (range, 7-33 months).
authors.affiliation: Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: U
authors.initials: U
authors.lastname: Anurathapan
authors.affiliation: Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: S
authors.initials: S
authors.lastname: Hongeng
authors.affiliation: Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: S
authors.initials: S
authors.lastname: Pakakasama
authors.affiliation: Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: N
authors.initials: N
authors.lastname: Sirachainan
authors.affiliation: Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: D
authors.initials: D
authors.lastname: Songdej
authors.affiliation: Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: A
authors.initials: A
authors.lastname: Chuansumrit
authors.affiliation: Department of Pediatrics, Chiangmai University Hospital, Chiangmai, Thailand.
authors.forename: P
authors.initials: P
authors.lastname: Charoenkwan
authors.affiliation: Department of Pediatrics, Khonkaen University, Khonkaen, Thailand.
authors.forename: A
authors.initials: A
authors.lastname: Jetsrisuparb
authors.affiliation: Department of Pediatrics, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: K
authors.initials: K
authors.lastname: Sanpakit
authors.affiliation: Department of Pediatrics, Phramongkutklao Hospital, Bangkok, Thailand.
authors.forename: P
authors.initials: P
authors.lastname: Rujkijyanont
authors.affiliation: Queen Sirikit National Institute of Child Health, Bangkok, Thailand.
authors.forename: A
authors.initials: A
authors.lastname: Meekaewkunchorn
authors.affiliation: Sunpasitthiprasong Hospital, Ubon Ratchathani, Thailand.
authors.forename: Y
authors.initials: Y
authors.lastname: Lektrakul
authors.affiliation: Samitivej Hospital, Bangkok, Thailand.
authors.forename: P
authors.initials: P
authors.lastname: Iamsirirak
authors.affiliation: Department of Pediatrics, Thammasat University, Pathum Thani, Thailand.
authors.forename: P
authors.initials: P
authors.lastname: Surapolchai
authors.affiliation: Department of Pediatrics, Thammasat University, Pathum Thani, Thailand.
authors.forename: W
authors.initials: W
authors.lastname: Satayasai
authors.affiliation: Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: S
authors.initials: S
authors.lastname: Sirireung
authors.affiliation: Department of Pharmacy Practice, Center of Pharmaceutical Outcomes Research, Naresuan University, Phitsanulok, Thailand.
authors.forename: R
authors.initials: R
authors.lastname: Sruamsiri
authors.affiliation: Department of Child Health, Cipto Mangunkusumo Hospital, Universitas Indonesia, Jakarta, Indonesia.
authors.forename: P A
authors.initials: PA
authors.lastname: Wahidiyat
authors.affiliation: Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: A
authors.initials: A
authors.lastname: Ungkanont
authors.affiliation: Department of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: S
authors.initials: S
authors.lastname: Issaragrisil
authors.affiliation: Department of Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: B S
authors.initials: BS
authors.lastname: Andersson
mesh_terms: D000293:Adolescent; D001781:Blood Component Removal; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006446:Hemoglobin E; D006720:Homozygote; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D000075442:Transplantation, Haploidentical / Q000379:methods* / Q000401:mortality; D055815:Young Adult; D017086:beta-Thalassemia / Q000628:therapy*
chemical_list: D007166:Immunosuppressive Agents; D006446:Hemoglobin E