{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Nakamae H"],"funding":["Japan Society for the Promotion of Science"],"pagination":["9636897221112098"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC9340897"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["31"],"pubmed_abstract":["The optimal dose of posttransplant cyclophosphamide (PTCy) for use in patients undergoing HLA-haploidentical hematopoietic cell transplantation with posttransplant cyclophosphamide (PTCy-haplo) has not been sufficiently examined. This study evaluates the safety and efficacy of HLA-haploidentical hematopoietic cell transplantation with a reduced dose of PTCy for patients with a poor prognosis or those with refractory hematological malignancies. We conducted a prospective clinical study of PTCy-haplo with peripheral blood stem cells (PBSCs) using a modified PTCy dosage regimen consisting of 50 mg/kg on day 3 posttransplantation and a reduced dose of 25 mg/kg on day 4. The cumulative incidences of grades II to III and IV acute graft-versus-host disease (GVHD) at day 100 posttransplantation were 30% and 0%, respectively. The cumulative incidence of moderate-to-severe chronic GVHD after transplantation was 7.0%. The cumulative incidence of nonrelapse mortality at 1 year posttransplantation was 6.1%. Overall survival (OS) at 1 year was 66%. In addition, the restricted cubic-spline Cox regression analysis showed nonlinear relationship between the number of infused CD34<sup>+</sup> cells and CD3<sup>+</sup> cells, and OS. A graft composition of >4.54 × 10<sup>6</sup>/kg CD34<sup>+</sup> cells and >1.85 × 10<sup>8</sup>/kg but ≤3.70 × 10<sup>8</sup>/kg CD3<sup>+</sup> cells was significantly associated with better survival, irrespective of the disease status (hazard ratio, 0.13; 95% confidence interval, 0.04-0.41; <i>P</i> < 0.001). These results suggest that PTCy-haplo with PBSCs using a de-escalated dose of 50 mg/kg on day 3 and 25 mg/kg on day 4 posttransplantation is a feasible option."],"journal":["Cell transplantation"],"pubmed_title":["A Prospective Study of an HLA-Haploidentical Peripheral Blood Stem Cell Transplantation Regimen Based on Modification of the Dose of Posttransplant Cyclophosphamide for Poor Prognosis or Refractory Hematological Malignancies."],"pmcid":["PMC9340897"],"funding_grant_id":["17K09017"],"pubmed_authors":["Makuuchi Y","Nishimoto M","Nakashima Y","Kuno M","Hino M","Koh H","Nakamae H","Okamura H","Takakuwa T","Hirose A","Harada N","Nakamae M"],"additional_accession":[]},"is_claimable":false,"name":"A Prospective Study of an HLA-Haploidentical Peripheral Blood Stem Cell Transplantation Regimen Based on Modification of the Dose of Posttransplant Cyclophosphamide for Poor Prognosis or Refractory Hematological Malignancies.","description":"The optimal dose of posttransplant cyclophosphamide (PTCy) for use in patients undergoing HLA-haploidentical hematopoietic cell transplantation with posttransplant cyclophosphamide (PTCy-haplo) has not been sufficiently examined. This study evaluates the safety and efficacy of HLA-haploidentical hematopoietic cell transplantation with a reduced dose of PTCy for patients with a poor prognosis or those with refractory hematological malignancies. We conducted a prospective clinical study of PTCy-haplo with peripheral blood stem cells (PBSCs) using a modified PTCy dosage regimen consisting of 50 mg/kg on day 3 posttransplantation and a reduced dose of 25 mg/kg on day 4. The cumulative incidences of grades II to III and IV acute graft-versus-host disease (GVHD) at day 100 posttransplantation were 30% and 0%, respectively. The cumulative incidence of moderate-to-severe chronic GVHD after transplantation was 7.0%. The cumulative incidence of nonrelapse mortality at 1 year posttransplantation was 6.1%. Overall survival (OS) at 1 year was 66%. In addition, the restricted cubic-spline Cox regression analysis showed nonlinear relationship between the number of infused CD34<sup>+</sup> cells and CD3<sup>+</sup> cells, and OS. A graft composition of >4.54 × 10<sup>6</sup>/kg CD34<sup>+</sup> cells and >1.85 × 10<sup>8</sup>/kg but ≤3.70 × 10<sup>8</sup>/kg CD3<sup>+</sup> cells was significantly associated with better survival, irrespective of the disease status (hazard ratio, 0.13; 95% confidence interval, 0.04-0.41; <i>P</i> < 0.001). These results suggest that PTCy-haplo with PBSCs using a de-escalated dose of 50 mg/kg on day 3 and 25 mg/kg on day 4 posttransplantation is a feasible option.","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Jan-Dec","modification":"2024-10-17T18:59:08.996Z","creation":"2024-10-17T18:59:08.996Z"},"accession":"S-EPMC9340897","cross_references":{"pubmed":["35906755"],"doi":["10.1177/09636897221112098"]}}