Adding melphalan to fludarabine and a myeloablative dose of busulfan improved survival after allogeneic hematopoietic stem cell transplantation in a propensity score-matched cohort of hematological malignancies

Fludarabine and a myeloablative dose of busulfan (Flu/Bu4) can improve prognosis after allogeneic hematopoietic stem cell transplantation (HSCT) with melphalan (Mel). We investigated the prognostic impact of adding Mel to Flu/Bu4 by comparing between Flu/Bu4/Mel and Flu/Bu4 groups. This study includ...

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Veröffentlicht in:Bone marrow transplantation (Basingstoke) 2021-07, Vol.56 (7), p.1691-1699
Hauptverfasser: Shimomura, Yoshimitsu, Hara, Masahiko, Yamamoto, Hisashi, Uchida, Naoyuki, Kawakita, Toshiro, Ashida, Takashi, Takada, Satoru, Ikeda, Takashi, Morishige, Satoshi, Maruyama, Yumiko, Wake, Atsushi, Ichinohe, Tatsuo, Fukuda, Takahiro, Takanashi, Minoko, Atsuta, Yoshiko, Ishikawa, Takayuki
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Sprache:eng
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Zusammenfassung:Fludarabine and a myeloablative dose of busulfan (Flu/Bu4) can improve prognosis after allogeneic hematopoietic stem cell transplantation (HSCT) with melphalan (Mel). We investigated the prognostic impact of adding Mel to Flu/Bu4 by comparing between Flu/Bu4/Mel and Flu/Bu4 groups. This study included 846 propensity score (PS)-matched patients who received either Flu/Bu4/Mel ( n  = 423) or Flu/Bu4 ( n  = 423) from 2394 patients enrolled in a multicenter prospective registry, from January 2010 to December 2016. The primary endpoint (5-year overall survival [OS]), and the prognostic impact of adding Mel was evaluated using Cox regression analysis. The study population median age was 58 (interquartile 50–64) years and 61.0% were male. Patient characteristics were well-balanced between groups. Five-year OS was 34.2% (95% confidence interval [CI]: 27.3–41.1%) and 30.1% (24.8–35.6%) in the Flu/Bu4/Mel and Flu/Bu4 groups, respectively (log-rank P  = 0.019). The adjusted hazard ratio of adding Mel was 0.77 (95% CI: 0.62–0.96) ( P  = 0.022) for the 5-year OS, and this attributed to a lower incidence of 5-year relapse (0.71, 0.56–0.90, P  = 0.005) and relapse associated mortality (0.73, 0.57–0.95, P  = 0.018). There was no statistical difference in 5-year non-relapse mortality between groups (log-rank P  = 0.855). Flu/Bu4/Mel was associated with better 5-year OS compared to Flu/Bu4 in a PS-matched cohort after allogeneic HSCT.
ISSN:0268-3369
1476-5365
DOI:10.1038/s41409-021-01217-w