Busulfan- or Thiotepa-Based Conditioning in Myelofibrosis: A Phase II Multicenter Randomized Study from the GITMO Group |
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| Authors: | Francesca Patriarca Arianna Masciulli Andrea Bacigalupo Stefania Bregante Chiara Pavoni Maria Chiara Finazzi Alberto Bosi Domenico Russo Franco Narni Giuseppe Messina Emilio Paolo Alessandrino Angelo Michele Carella Giuseppe Milone Benedetto Bruno Sonia Mammoliti Barbara Bruno Renato Fanin Francesca Bonifazi Alessandro Rambaldi |
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| Affiliation: | 1. Udine University Hospital, DAME, University of Udine, Udine, Italy;2. “Papa Giovanni XXIII” Hospital, Bergamo, Italy;3. “Fondazione A. Gemelli”, University Hospital, Rome, Italy;4. IRCSS “San Martino” Hospital, Genoa, Italy;5. Hematology, University of Florence, Florence, Italy;6. ASST Hospital of Brescia, DSCS, Brescia University, Brescia, Italy;7. University Hospital of Modena, Modena, Italy;8. “Bianchi-Melacrino-Morelli” Hospital, Reggio Calabria, Italy;9. IRCSS San Matteo Hospital, Pavia, Italy;10. IRCSS, San Giovanni Rotondo Hospital (FG), San Giovanni Rotondo, Italy;11. Ferrarotto Hospital, Catania, Italy;12. “Citta’ della Salute e della Scienza” University Hospital, DBMSS, University of Torino, Torino, Italy;13. Trial Clinical Office, Gruppo Italiano Trapianto Midollo Osseo (GITMO), Genoa, Italy;14. Institute of Hematology “Seragnoli”, University Hospital “S. Orsola Malpighi”, Bologna, Italy;15. Department of Hematology-Oncology, University of Milano, Milan, Italy |
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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. |
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| Keywords: | Gruppo Italiano Trapianti di Midollo Osseo. (GITMO). Myelofibrosis Allogeneic stem cell transplantation Reduced-intensity conditioning regimen Busulfan Thiotepa |
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