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).

Research output: Contribution to journalArticle

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 languageEnglish
JournalBiology of Blood and Marrow Transplantation
DOIs
Publication statusPublished - 2019

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Thiotepa
Busulfan
Primary Myelofibrosis
Hematopoietic Stem Cell Transplantation
Multicenter Studies
Disease-Free Survival
Arm
Confidence Intervals
Splenomegaly
Splenectomy
Siblings
Neutrophils
Blood Platelets
Transplantation
Alleles
Tissue Donors
Transplants
Recurrence
Survival
Mortality

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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 journalArticle

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. {\circledC} 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 -