Peripheral Blood Hematopoietic Stem Cells for Transplantation of Hematological Diseases from Related, Haploidentical Donors after Reduced-Intensity Conditioning

Abstract

In a multi-center collaboration, we carried out T-replete, peripheral blood stem cell (PBSC) transplants from related, HLA-haploidentical donors with reduced-intensity conditioning (RIC) and post-transplant cyclophosphamide (Cy) as GvHD prophylaxis in 55 patients with high-risk hematologic disorders. Patients received 2 doses of Cy 50mg/kg IV on days 3 and 4 post-infusion of PBSC (mean 6.4×106/kg CD34+ cells, mean 2.0 ×108/kg CD3+ cells). The median times to neutrophil (500/mcL) and platelet (>20,000/mcL) recovery were 17 and 21 days respectively. All but two of the patients achieved full engraftment. The one-year cumulative incidences of grade II and grade III acute GVHD were 53% and 8% respectively. There were no cases of grade IV GvHD. The two-year cumulative incidence of chronic GHVD was 18%. With a median follow up of 509 days, overall survival (OS) and event free survival (EFS) at two years were 48% and 51%, respectively. The two-year cumulative incidences of non-relapse mortality (NRM) and relapse were 23% and 28% respectively. Our results suggest that PBSC can be substituted safely and effectively for bone marrow (BM) as the graft source for haploidentical transplantation following reduced intensity conditioning.

Unrelated cord blood compared with haploidentical grafts in patients with hematological malignancies

“…This is in accordance with our previous published data 33 and with data from Raj et al 34 The current study has some limitations, mainly due to its retrospective nature. Essentially, diseases and the period of allo-HSCT were significantly different between the 2 cohorts; because the haplo program was initiated after UCB, the follow-up is different and some late events Abbreviations: aGVHD, aGVHD, acute graft-versus-host disease; ANC, absolute neutrophil count; cGVHD, chronic graft-versus-host disease; haplo-HSCT, haploidentical donor allogeneic hematopoietic stem cell transplantation; NRM, nonrecurrence mortality; OS, overall survival; PFS, progression-free survival; PLT, platelet; PT-Cy, posttransplant cyclophosphamide; UCB-HSCT, umbilical cord blood allogeneic hematopoietic stem cell transplantation.…”

Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study

“…Full chimerism was defined as 495% donor cells and mixed chimerism as 5% to 95% donor cells. 29 The presence of donor HLAspecific Abs were assessed by solid-phase immunoassays on the Luminex platform, according to the manufacturer's instructions. Sera were tested using polled HLA Ags, HLA class I or class II phenotypes and single HLA Ags panels (One Lambda Inc., Canoga Park, CA, USA).…”

Haploidentical hematopoietic SCT for acquired severe aplastic anemia using post-transplant high-dose CY

“…5,6 This strategy has been used in patients with sickle cell anemia 7 and recently in four adults with refractory aplastic anemia. 8 This strategy has not yet been reported in children with aplastic anemia. We report herein our experience of PT/CY haploSCT in two pediatric patients with refractory aplastic anemia.…”

“…One of the patients failed to engraft but was salvaged by doing a second transplant with a high CD34+ stem cell dose. In a multicentre study reported by Raj et al, 8 55 patients with different hematologic diseases, including 4 patients with refractory aplastic anemia, underwent PBSC haploSCT using PT/CY regimen. Grade II and III acute GVHD was seen in 53% and 8% patients, respectively, but none developed grade IV acute GVHD.…”