We report a randomized study comparing fludarabine in combination with busulfan (FB) or thiotepa (FT), as conditioning regimen for hematopoietic stem cell transplantation (HSCT) in patients with myelofibrosis. The primary study endpoint was progression-free survival (PFS). Sixty patients were enrolled with a median age of 56 years and an intermediate-2 or high-risk score in 65%, according to the Dynamic International Prognostic Staging System (DIPSS). Donors were HLA-identical sibling (n = 25), matched unrelated (n = 25) or single allele mismatched unrelated (n = 10). With a median follow-up of 22 months (range, 1 to 68 months), outcomes at 2 years after HSCT in the FB arm versus the FT arm were as follows: PFS, 43% versus 55% (P =.28); overall survival (OS), 54% versus 70% (P =.17); relapse/progression, 36% versus 24% (P =.24); nonrelapse mortality (NRM), 21% in both arms (P =.99); and graft failure, 14% versus 10% (P =.96). A better PFS was observed in patients with intermediate-1 DIPSS score (P =.03). Both neutrophil engraftment and platelet engraftment were significantly influenced by previous splenectomy (hazard ratio [HR], 2.28; 95% confidence interval [CI], 1.16 to 4.51; P =.02) and splenomegaly at transplantation (HR, 0.51; 95% CI, 0.27 to 0.94; P =.03). In conclusion, the clinical outcome after HSCT was comparable when using either a busulfan or thiotepa based conditioning regimen.

Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group / Patriarca, Francesca; Masciulli, Arianna; Bacigalupo, Andrea; Bregante, Stefania; Pavoni, Chiara; Finazzi, Maria Chiara; Bosi, Alberto; Russo, Domenico; Narni, Franco; Messina, Giuseppe; Alessandrino, Emilio Paolo; Carella, Angelo Michele; Milone, Giuseppe; Bruno, Benedetto; Mammoliti, Sonia; Bruno, Barbara; Fanin, Renato; Bonifazi, Francesca; Rambaldi, Alessandro. - In: BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION. - ISSN 1083-8791. - 25:5(2019), pp. 932-940. [10.1016/j.bbmt.2018.12.064]

Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group

Narni, Franco;
2019

Abstract

We report a randomized study comparing fludarabine in combination with busulfan (FB) or thiotepa (FT), as conditioning regimen for hematopoietic stem cell transplantation (HSCT) in patients with myelofibrosis. The primary study endpoint was progression-free survival (PFS). Sixty patients were enrolled with a median age of 56 years and an intermediate-2 or high-risk score in 65%, according to the Dynamic International Prognostic Staging System (DIPSS). Donors were HLA-identical sibling (n = 25), matched unrelated (n = 25) or single allele mismatched unrelated (n = 10). With a median follow-up of 22 months (range, 1 to 68 months), outcomes at 2 years after HSCT in the FB arm versus the FT arm were as follows: PFS, 43% versus 55% (P =.28); overall survival (OS), 54% versus 70% (P =.17); relapse/progression, 36% versus 24% (P =.24); nonrelapse mortality (NRM), 21% in both arms (P =.99); and graft failure, 14% versus 10% (P =.96). A better PFS was observed in patients with intermediate-1 DIPSS score (P =.03). Both neutrophil engraftment and platelet engraftment were significantly influenced by previous splenectomy (hazard ratio [HR], 2.28; 95% confidence interval [CI], 1.16 to 4.51; P =.02) and splenomegaly at transplantation (HR, 0.51; 95% CI, 0.27 to 0.94; P =.03). In conclusion, the clinical outcome after HSCT was comparable when using either a busulfan or thiotepa based conditioning regimen.
20-dic-2018
25
5
932
940
Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group / Patriarca, Francesca; Masciulli, Arianna; Bacigalupo, Andrea; Bregante, Stefania; Pavoni, Chiara; Finazzi, Maria Chiara; Bosi, Alberto; Russo, Domenico; Narni, Franco; Messina, Giuseppe; Alessandrino, Emilio Paolo; Carella, Angelo Michele; Milone, Giuseppe; Bruno, Benedetto; Mammoliti, Sonia; Bruno, Barbara; Fanin, Renato; Bonifazi, Francesca; Rambaldi, Alessandro. - In: BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION. - ISSN 1083-8791. - 25:5(2019), pp. 932-940. [10.1016/j.bbmt.2018.12.064]
Patriarca, Francesca; Masciulli, Arianna; Bacigalupo, Andrea; Bregante, Stefania; Pavoni, Chiara; Finazzi, Maria Chiara; Bosi, Alberto; Russo, Domenico; Narni, Franco; Messina, Giuseppe; Alessandrino, Emilio Paolo; Carella, Angelo Michele; Milone, Giuseppe; Bruno, Benedetto; Mammoliti, Sonia; Bruno, Barbara; Fanin, Renato; Bonifazi, Francesca; Rambaldi, Alessandro
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11380/1171304
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