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

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.

Original languageEnglish
JournalBiology of Blood and Marrow Transplantation
DOIs
Publication statusE-pub ahead of print - Dec 20 2018

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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. / Gruppo Italiano Trapianti di Midollo Osseo. (GITMO).

In: Biology of Blood and Marrow Transplantation, 20.12.2018.

Research output: Contribution to journalArticle

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title = "Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group",
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.",
author = "{Gruppo Italiano Trapianti di Midollo Osseo. (GITMO).} and Francesca Patriarca and Arianna Masciulli and Andrea Bacigalupo and Stefania Bregante and Chiara Pavoni and Finazzi, {Maria Chiara} and Alberto Bosi and Domenico Russo and Franco Narni and Giuseppe Messina and Alessandrino, {Emilio Paolo} and Carella, {Angelo Michele} and Giuseppe Milone and Benedetto Bruno and Sonia Mammoliti and Barbara Bruno and Renato Fanin and Francesca Bonifazi and Alessandro Rambaldi",
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T1 - Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis

T2 - A Phase II Multicenter Randomized Study from the GITMO Group

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

AU - Patriarca, Francesca

AU - Masciulli, Arianna

AU - Bacigalupo, Andrea

AU - Bregante, Stefania

AU - Pavoni, Chiara

AU - Finazzi, Maria Chiara

AU - Bosi, Alberto

AU - Russo, Domenico

AU - Narni, Franco

AU - Messina, Giuseppe

AU - Alessandrino, Emilio Paolo

AU - Carella, Angelo Michele

AU - Milone, Giuseppe

AU - Bruno, Benedetto

AU - Mammoliti, Sonia

AU - Bruno, Barbara

AU - Fanin, Renato

AU - Bonifazi, Francesca

AU - Rambaldi, Alessandro

N1 - Copyright © 2019. Published by Elsevier Inc.

PY - 2018/12/20

Y1 - 2018/12/20

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.

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.

U2 - 10.1016/j.bbmt.2018.12.064

DO - 10.1016/j.bbmt.2018.12.064

M3 - Article

C2 - 30579966

JO - Biology of Blood and Marrow Transplantation

JF - Biology of Blood and Marrow Transplantation

SN - 1083-8791

ER -