Haploidentical, unmanipulated, G-CSF–primed bone marrow transplantation for patients with high-risk hematologic malignancies

• Published in: Blood 2013-01, Vol.121 (5), p.849-857
• Main Authors: Di Bartolomeo, Paolo, Santarone, Stella, De Angelis, Gottardo, Picardi, Alessandra, Cudillo, Laura, Cerretti, Raffaella, Adorno, Gaspare, Angelini, Stefano, Andreani, Marco, De Felice, Lidia, Rapanotti, Maria Cristina, Sarmati, Loredana, Bavaro, Pasqua, Papalinetti, Gabriele, Di Nicola, Marta, Papola, Franco, Montanari, Mauro, Nagler, Arnon, Arcese, William
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; Bone Marrow Transplantation; Child; Child, Preschool; Disease-Free Survival; Female; Follow-Up Studies; Graft Survival; Graft vs Host Disease - blood; Graft vs Host Disease - mortality; Graft vs Host Disease - therapy; Granulocyte Colony-Stimulating Factor - administration & dosage; Hematologic Neoplasms - blood; Hematologic Neoplasms - mortality; Hematologic Neoplasms - therapy; Humans; Living Donors; Male; Middle Aged; Neutrophils - metabolism; Risk Factors; Survival Rate; Time Factors; Transplantation, Homologous
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2012-08-453399

Eighty patients with high-risk hematologic malignancies underwent unmanipulated, G-CSF–primed BM transplantation from an haploidentical family donor. Patients were transplanted in first or second complete remission (CR, standard-risk: n = 45) or in > second CR or active disease (high-risk: n = 35). The same regimen for GVHD prophylaxis was used in all cases. The cumulative incidence (CI) of neutrophil engraftment was 93% ± 0.1%. The 100-day CIs for II-IV and III-IV grade of acute GVHD were 24% ± 0.2% and 5% ± 0.6%, respectively. The 2-year CI of extensive chronic GVHD was 6% ± 0.1%. The 1-year CI of treatment-related mortality was 36% ± 0.3%. After a median follow-up of 18 months, 36 of 80 (45%) patients are alive in CR. The 3-year probability of overall and disease-free survival for standard-risk and high-risk patients was 54% ± 8% and 33% ± 9% and 44% ± 8% and 30% ± 9%, respectively. In multivariate analysis, disease-free survival was significantly better for patients who had standard-risk disease and received transplantations after 2007. We conclude that unmanipulated, G-CSF–primed BM transplantation from haploidentical family donor provides very encouraging results in terms of engraftment rate, incidence of GVHD and survival and represents a feasible, valid alternative for patients with high-risk malignant hematologic diseases, lacking an HLA identical sibling and in need to be urgently transplanted. •Haploidentical, unmanipulated, G-CSF–primed bone marrow transplantation.•Haploidentical hematopoietic stem cell transplantation for hematologic malignancies.