title: Improved clinical outcomes of high risk β thalassemia major patients undergoing a HLA matched related allogeneic stem cell transplant with a treosulfan based conditioning regimen and peripheral blood stem cell grafts.
abstract: Improving clinical outcomes among high risk Class III β thalassemia major patients (Class IIIHR) receiving an allogeneic SCT remains a challenge. From October, 2009 a treosulfan based regimen (TreoFluT) was used for all consecutive Class III patients (n = 50). The clinical outcomes were compared with the historical conventional busulfan (BuCy) based regimen (n = 139). Use of TreoFluT was associated with a significantly reduced incidence of sinusoidal obstruction syndrome (SOS) among Class IIIHR cases (78% to 30%; P = 0.000) and early TRM (46% to 13%; p = 0.005). There was also a trend towards better engraftment in the Class IIIHR subset (P = 0.055). However, the use of bone marrow (BM) as source of stem cells along with the TreoFluT regimen was associated with 50% early mixed chimerism which reduced to 8.5% with the use of a peripheral blood stem cell graft (PBSC). Use of a PBSC graft was not associated with a significant increase in the incidence of acute or chronic graft versus host disease (GVHD). The overall and event free survival was significantly better among the Class IIIHR subset with the use of TreoFluT Vs. BuCy (86.6 ± 7.3 Vs. 39.4 ± 6.8%; P = 0.002 and 77.8 ± 8.8 Vs. 32.4 ± 6.5%; P = 0.003 respectively). A TreoFluT conditioning regimen with a PBSC graft can significantly improve clinical outcomes of Class IIIHR patients.
authors: Department of Haematology, Christian Medical College, Vellore, India. vikram@cmcvellore.ac.in
authors: Vikram
authors: V
authors: Mathews
authors: Biju
authors: B
authors: George
authors: Auro
authors: A
authors: Viswabandya
authors: Aby
authors: A
authors: Abraham
authors: Rayaz
authors: R
authors: Ahmed
authors: Abhijeet
authors: A
authors: Ganapule
authors: Eunice
authors: E
authors: Sindhuvi
authors: Kavitha M
authors: KM
authors: Lakshmi
authors: Alok
authors: A
authors: Srivastava
mesh_terms: D000293:Adolescent; D001854:Bone Marrow Cells / Q000166:cytology; D002066:Busulfan / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D046528:Chimerism; D003710:Demography; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D006504:Hepatic Veno-Occlusive Disease / Q000209:etiology; D006650:Histocompatibility Testing; D006801:Humans; D008297:Male; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects; D012307:Risk Factors; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000009:adverse effects; D014184:Transplantation, Homologous / Q000009:adverse effects; D016896:Treatment Outcome; D055815:Young Adult; D017086:beta-Thalassemia / Q000188:drug therapy*
chemical_list: C018404:treosulfan; D002066:Busulfan
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