Peripheral blood stem cell for haploidentical transplantation with post-transplant high dose cyclophosphamide: detailed analysis of 181 consecutive patients

• Published in: Bone marrow transplantation (Basingstoke) 2019-11, Vol.54 (11), p.1730-1737
• Main Authors: Granata, Angela, Fürst, Sabine, Bramanti, Stefania, Legrand, Faezeh, Sarina, Barbara, Harbi, Samia, De Philippis, Chiara, Faucher, Catherine, Chabannon, Christian, Lemarie, Claude, Calmels, Boris, Mariotti, Jacopo, Maisano, Valerio, Weiller, Pierre-Jean, Mokart, Djamel, Vey, Norbert, Bouabdallah, Reda, Castagna, Luca, Blaise, Didier, Devillier, Raynier
• Format: Article
• Language: English
• Subjects: 692/308; 692/699/1541/1990; Adult; Aged; Allografts; Blood; Blood diseases; Blood Platelets - metabolism; Bone marrow; Bone marrow transplantation; Cell Biology; Chronic Disease; Cyclophosphamide; Cyclophosphamide - administration & dosage; Diagnosis; Disease control; Disease-Free Survival; Dosage and administration; Female; Graft vs Host Disease - blood; Graft vs Host Disease - mortality; Graft vs Host Disease - prevention & control; Graft-versus-host reaction; Grafting; Grafts; Hematological diseases; Hematology; Hematopoietic stem cells; Histocompatibility antigen HLA; Humans; Incidence; Internal Medicine; Male; Medicine; Medicine & Public Health; Middle Aged; Neutrophils - metabolism; Peripheral blood; Peripheral Blood Stem Cell Transplantation; Public Health; Quality of life; Risk analysis; Risk factors; Stem cell transplantation; Stem Cells; Survival Rate; Transplantation; Transplants & implants
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/s41409-019-0500-x

While bone marrow (BM) grafts were initially used for T-replete HLA-haploidentical related donors transplantation (Haplo-SCT) with post-transplantation cyclophosphamide (PT-Cy), the use of peripheral blood stem cell (PBSC) remains debated. We thus conducted a detailed analysis evaluating the incidence, risk factors, and prevalence of GVHD after PBSC Haplo-SCT with PT-Cy. One hundred and eighty-one patients with hematological diseases were included. Median time for neutrophil and platelet recovery was 21 and 30 days, respectively. The cumulative incidence of grade 3–4 acute GVHD and severe chronic GVHD were 8% and 4%, respectively, approaching what was observed after BM Haplo-SCT. NRM at 2 years was 21%, and 41% of the non-relapse deaths were caused by GVHD. The cumulative incidence of relapse at 2 years was 17% in the whole cohort, and 13% among AML patients ( n  = 54), suggesting a high GVL effect. As surrogate markers for good quality of life, we observed a 2-year GVHD-relapse-free survival probability of 50% and found that 6% and 2% of disease-free patients at 2 years were still living with GVHD and immunosuppressive treatments, respectively. Haplo-SCT with PT-Cy using PBSC grafts results in low incidence GVHD and promising disease control, making PBSCs a valuable alternative to BM graft in this setting.