Fludarabine/busulfan versus busulfan/cyclophosphamide as myeloablative conditioning for myelodysplastic syndrome: a propensity score-matched analysis

• Published in: Bone marrow transplantation (Basingstoke) 2021-12, Vol.56 (12), p.3008-3015
• Main Authors: Kurosawa, Shuhei, Shimomura, Yoshimitsu, Itonaga, Hidehiro, Najima, Yuho, Kobayashi, Takeshi, Ozawa, Yukiyasu, Kanda, Yoshinobu, Kako, Shinichi, Kawakita, Toshiro, Matsuoka, Ken-ichi, Maruyama, Yumiko, Ota, Shuichi, Nakazawa, Hideyuki, Imada, Kazunori, Kanda, Junya, Fukuda, Takahiro, Atsuta, Yoshiko, Aoki, Jun
• Format: Article
• Language: English
• Subjects: 692/499; 692/699/1541/1990/1673; 96/100; Acute myeloid leukemia; Antimitotic agents; Antineoplastic agents; Busulfan; Busulfan - therapeutic use; Care and treatment; Cell Biology; Conditioning; Confidence intervals; Cyclophosphamide; Cyclophosphamide - therapeutic use; Fludarabine; Graft vs Host Disease; Health risks; Hematology; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Humans; Internal Medicine; Leukemia; Leukemia, Myeloid, Acute - therapy; Medicine; Medicine & Public Health; Myelodysplastic syndrome; Myelodysplastic syndromes; Myelodysplastic Syndromes - therapy; Myeloid leukemia; Propensity Score; Public Health; Retrospective Studies; Stem cell transplantation; Stem Cells; Subgroups; Survival; Testing; Transplantation; Transplantation Conditioning; Transplants & implants; Vidarabine - analogs & derivatives; Vidarabine - therapeutic use
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/s41409-021-01447-y

Myeloablative conditioning with fludarabine/busulfan (Flu/Bu4) prior to allogeneic hematopoietic stem cell transplantation (allo-HSCT) is effective for acute myeloid leukemia. However, the effectiveness of Flu/Bu4 for myelodysplastic syndrome (MDS) remains poorly understood. Therefore, we retrospectively analyzed nationwide registry data in Japan from 2006 to 2018 and compared transplant outcomes of adult MDS patients receiving Flu/Bu4 and busulfan/cyclophosphamide (Bu4/Cy) using propensity score (PS) matching. The primary endpoint was overall survival (OS). Among 2,482 MDS patients, 153 patients were assigned each to the Flu/Bu4 and Bu4/Cy groups. The 3-year OS rates were 52.7% (95% confidence interval [CI], 43.8–60.8%) and 49.5% (95% CI, 40.8–57.6%) in the Flu/Bu4 and Bu4/Cy group, respectively ( P  = 0.548). The 3-year progression-free survival ( P  = 0.858), the cumulative incidence of relapse ( P  = 0.536), and cumulative incidence of non-relapse mortality ( P  = 0.684) were not significantly different between the two groups. According to the findings of subgroup analyses, no patient had a favorable OS when using either of the two regimens. In conclusion, although our PS-matched cohort mainly comprised older patients who had a low hematopoietic cell transplantation-comorbidity index and low-risk disease status, Flu/Bu4 could be an alternative to Bu4/Cy for MDS patients prior to allo-HSCT.