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

F. Patriarca; A. Masciulli; Andrea Bacigalupo; S. Bregante; C. Pavoni; M.C. Finazzi; A. Bosi; D. Russo; F. Narni; G. Messina; E.P. Alessandrino; A.M. Carella; G. Milone; B. Bruno; S. Mammoliti; B. Bruno; R. Fanin; F. Bonifazi; A. Rambaldi; Gruppo Italiano Trapianti di Midollo Osseo. (GITMO).

doi:10.1016/j.bbmt.2018.12.064

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

F. Patriarca, A. Masciulli, Andrea Bacigalupo, S. Bregante, C. Pavoni, M.C. Finazzi, A. Bosi, D. Russo, F. Narni, G. Messina, E.P. Alessandrino, A.M. Carella, G. Milone, B. Bruno, S. Mammoliti, B. Bruno, R. Fanin, F. Bonifazi, A. Rambaldi, Gruppo Italiano Trapianti di Midollo Osseo. (GITMO).

IRCCS Fondazione Policlinico Universitario A. Gemelli

Research output: Contribution to journal › Article

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. © 2019

Original language	English
Journal	Biology of Blood and Marrow Transplantation
DOIs	https://doi.org/10.1016/j.bbmt.2018.12.064
Publication status	Published - 2019

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Patriarca, F., Masciulli, A., Bacigalupo, A., Bregante, S., Pavoni, C., Finazzi, M. C., Bosi, A., Russo, D., Narni, F., Messina, G., Alessandrino, E. P., Carella, A. M., Milone, G., Bruno, B., Mammoliti, S., Bruno, B., Fanin, R., Bonifazi, F., Rambaldi, A., & (GITMO)., G. I. T. D. M. O. (2019). Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group. Biology of Blood and Marrow Transplantation. https://doi.org/10.1016/j.bbmt.2018.12.064

Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group. / Patriarca, F.; Masciulli, A.; Bacigalupo, Andrea; Bregante, S.; Pavoni, C.; Finazzi, M.C.; Bosi, A.; Russo, D.; Narni, F.; Messina, G.; Alessandrino, E.P.; Carella, A.M.; Milone, G.; Bruno, B.; Mammoliti, S.; Bruno, B.; Fanin, R.; Bonifazi, F.; Rambaldi, A.; (GITMO)., Gruppo Italiano Trapianti di Midollo Osseo.

In: Biology of Blood and Marrow Transplantation, 2019.

Research output: Contribution to journal › Article

Patriarca, F, Masciulli, A, Bacigalupo, A, Bregante, S, Pavoni, C, Finazzi, MC, Bosi, A, Russo, D, Narni, F, Messina, G, Alessandrino, EP, Carella, AM, Milone, G, Bruno, B, Mammoliti, S, Bruno, B, Fanin, R, Bonifazi, F, Rambaldi, A & (GITMO)., GITDMO 2019, 'Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group', Biology of Blood and Marrow Transplantation. https://doi.org/10.1016/j.bbmt.2018.12.064

Patriarca, F. ; Masciulli, A. ; Bacigalupo, Andrea ; Bregante, S. ; Pavoni, C. ; Finazzi, M.C. ; Bosi, A. ; Russo, D. ; Narni, F. ; Messina, G. ; Alessandrino, E.P. ; Carella, A.M. ; Milone, G. ; Bruno, B. ; Mammoliti, S. ; Bruno, B. ; Fanin, R. ; Bonifazi, F. ; Rambaldi, A. ; (GITMO)., Gruppo Italiano Trapianti di Midollo Osseo. / Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group. In: Biology of Blood and Marrow Transplantation. 2019.

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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. {\textcopyright} 2019",

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T1 - Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group

AU - Patriarca, F.

AU - Masciulli, A.

AU - Bacigalupo, Andrea

AU - Bregante, S.

AU - Pavoni, C.

AU - Finazzi, M.C.

AU - Bosi, A.

AU - Russo, D.

AU - Narni, F.

AU - Messina, G.

AU - Alessandrino, E.P.

AU - Carella, A.M.

AU - Milone, G.

AU - Bruno, B.

AU - Mammoliti, S.

AU - Bruno, B.

AU - Fanin, R.

AU - Bonifazi, F.

AU - Rambaldi, A.

AU - (GITMO)., Gruppo Italiano Trapianti di Midollo Osseo.

N1 - cited By 0; Article in Press

PY - 2019

Y1 - 2019

N2 - 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. © 2019

AB - 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. © 2019

U2 - 10.1016/j.bbmt.2018.12.064

DO - 10.1016/j.bbmt.2018.12.064

M3 - Article

JO - Biology of Blood and Marrow Transplantation

JF - Biology of Blood and Marrow Transplantation

SN - 1083-8791

ER -