Reduced-intensity conditioning using fludarabine, melphalan and thiotepa for adult patients undergoing haploidentical SCT

S. O. Ciurea, R. Saliba, G. Rondon, S. Pesoa, P. Cano, M. Fernandez-Vina, S. Qureshi, L. L. Worth, J. McMannis, P. Kebriaei, R. B. Jones, M. Korbling, M. Qazilbash, E. J. Shpall, S. Giralt, M. De Lima, R. E. Champlin, J. Gajewski

    Research output: Contribution to journalArticlepeer-review

    47 Scopus citations


    Haploidentical SCT (HaploSCT) has been most commonly performed using a myeloablative, TBI-based preparative regimen; however, the toxicity with this approach remains very high. We studied the feasibility of a reduced-intensity conditioning regimen in a phase II clinical trial using fludarabine, melphalan and thiotepa and antithymocyte globulin (ATG) for patients with advanced hematological malignancies undergoing T-cell depleted HaploSCT. Twenty-eight patients were entered in the study. Engraftment with donor-derived hematopoiesis was achieved in 78 of patients after a median of 13 days. Six patients experienced primary graft failure, three out of four tested patients had donor-specific anti-HLA antibodies (DSA) (P0.001). Toxicity included mostly infections. A total of 21 out of 22 patients with AMLmyelodysplastic syndrome (MDS) achieved remission after transplant (16 with relapsedrefractory AML). Five out of the 12 patients (42) with AMLMDS with <15 BM blasts survived long term as compared with none with more advanced disease (P0.03). HaploSCT with this fludarabine, melphalan and thiotepa and ATG RIC is an effective, well-tolerated conditioning regimen for patients with AMLMDS with low disease burden at the time of transplant and allowed a high rate of engraftment in patients without DSA. Patients with overt relapse fared poorly and require novel treatment strategies.

    Original languageEnglish (US)
    Pages (from-to)429-436
    Number of pages8
    JournalBone marrow transplantation
    Issue number3
    StatePublished - 2010


    • AML
    • Haploidentical SCT
    • Myelodysplastic syndromes
    • T-cell depletion

    ASJC Scopus subject areas

    • Hematology
    • Transplantation


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