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Haploidentical, G-CSF-primed, unmanipulated bone marrow transplantation for patients with high-risk hematological malignancies: an update

Bone Marrow Transplantation volume 50, pages S24–S30 (2015)Cite this article

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Abstract

Ninety-seven patients affected by high-risk hematological malignancies underwent G-CSF primed, unmanipulated bone marrow (BM) transplantation from a related, haploidentical donor. All patients were prepared with an identical conditioning regimen including Thiotepa, Busilvex, Fludarabine (TBF) and antithymocyte globulin given at myeloablative (MAC=68) or reduced (reduced intensity conditioning (RIC)=29) dose intensity and received the same GvHD prophylaxis consisting of the combination of methotrexate, cyclosporine, mycofenolate-mofetil and basiliximab. Patients were transplanted in 1st or 2nd CR (early phase: n=60) or in >2nd CR or active disease (advanced phase: n=37). With a median time of 21 days (range 12–38 days), the cumulative incidence (CI) of neutrophil engraftment was 94±3%. The 100-day CI of III–IV grade acute GvHD and the 2-year CI of extensive chronic GvHD were 9±3% and 12±4%, respectively. Overall, at a median follow-up of 2.2 years (range 0.3–5.6), 44 out of 97 (45%) patients are alive in CR. The 5-year probability of overall survival (OS) and disease-free survival (DFS) for patients in early and advanced phase was 53±7 vs 24±8% (P=0.006) and 48±7 vs 22±8% (P=0.01), respectively. By comparing MAC with RIC patient groups, the transplant-related mortality was equivalent (36±6 vs 28±9%) while the relapse risk was lower for the MAC patients (22±6 vs 45±11%), who showed higher OS (48±7 vs 29±10%) and DFS (43±7 vs 26±10%). However, all these differences did not reach a statistical significance. In multivariate analysis, diagnosis and recipient age were significant factors for OS and DFS. In conclusion, this analysis confirms, on a longer follow-up and higher number of patients, our previous encouraging results obtained by using MAC and RIC TBF regimen as conditioning for G-CSF primed, unmanipulated BM transplantation from related, haploidentical donor in patients with high-risk hematological malignancies, lacking an HLA-identical sibling or unrelated donor and in need to be urgently transplanted.

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Acknowledgements

This work was supported in part by grants from the Agenzia Regionale del Lazio per i Trapianti e le Patologie Connesse and from the “Matteo Fabrizio” Onlus-Association.

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Authors and Affiliations

  1. Department of Hematology, Stem Cell Transplant Unit, Rome Transplant Network, "Tor Vergata" University Hospital, Rome, Italy

    W Arcese, A Picardi, G De Angelis, R Cerretti, L Cudillo & F Di Piazza

  2. Department of Hematology, Bone Marrow Transplant Center, Spirito Santo Hospital, Pescara, Italy

    S Santarone, E Pennese, P Bavaro, P Olioso & P Di Bartolomeo

  3. Department of Specialties, Hematology, Sant’Andrea Hospital, Rome, Italy

    T Dentamaro & L Cupelli

  4. Department of Specialties, Hematology, San Giovanni Hospital, Rome, Italy

    A Chierichini

  5. Department of Oncology, Hematology, Sant’Andrea Hospital, Rome, Italy

    A Ferrari

  6. Department of Oncology, Regima Elena National Cancer Institute, Rome, Italy

    A Mengarelli

  7. Hematology Stem Cell Transplant Transfusion Medicine and Cellular Therapy, Rome, Italy

    M C Tirindelli

  8. Laboratory of Immunogenetics and Transplant Biology—IME Foundation, Policlinic of Tor Vergata, Rome, Italy

    M Testi

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This article was published as part of a supplement, supported by WIS-CSP Foundation, in collaboration with Gilead, Milteny Biotec, Gamida cell, Adienne Pharma and Biotech, Medac hematology, Kiadis Pharma and Almog Diagnostic.

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Arcese, W., Picardi, A., Santarone, S. et al. Haploidentical, G-CSF-primed, unmanipulated bone marrow transplantation for patients with high-risk hematological malignancies: an update. Bone Marrow Transplant 50 (Suppl 2), S24–S30 (2015). https://doi.org/10.1038/bmt.2015.91

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