TY - JOUR
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
PY - 2019/5
Y1 - 2019/5
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.
KW - Allogeneic stem cell transplantation
KW - Busulfan
KW - Myelofibrosis
KW - Reduced-intensity conditioning regimen
KW - Thiotepa
UR - http://www.scopus.com/inward/record.url?scp=85060088415&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85060088415&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2018.12.064
DO - 10.1016/j.bbmt.2018.12.064
M3 - Article
AN - SCOPUS:85060088415
SN - 1083-8791
SP - 932
EP - 940
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
ER -