TY - JOUR
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 -