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<p>Allogeneic hematopoietic cell transplantation (ASCT) is the only curative option for patients with myelodysplastic syndromes (MDS), but whether cytoreductive pretreatment and molecular “downstaging” according to the IPSS-M improves outcomes remains unclear. We retrospectively analyzed 128 consecutive adults with MDS who underwent ASCT grouped as frontline transplantation (<i>n</i> = 87) or pretreated before transplant (<i>n</i> = 41). Median bone marrow blasts at diagnosis were 12% vs. 10%. IPSS-M was calculated at diagnosis and immediately before transplant using cytogenetic and next-generation sequencing data. IPSS-M improved in 26% of frontline and 34% of pretreated patients, was unchanged in 41% and 34%, and worsened in 30% and 32%, respectively. After a median follow-up of 17.3 months, overall survival (OS), relapse-free survival (RFS) and graft-versus-host disease relapse-free survival (GRFS) were superior with frontline transplantation (median OS 112.6 vs 14.0 months, <i>p</i> = 0.03, median RFS 61.0 vs 8.9 months, <i>p</i> = 0.007 and median GRFS 13.3 vs 5.3 months, <i>p</i> = 0.004). However, in a landmark analysis starting at the time of transplantation, the difference in OS was no longer statistically significant. Non-relapse mortality was significantly higher after pretreatment (<i>p</i> = 0.018). Pretransplant cytoreduction did not improve post-transplant outcomes despite modest IPSS-M improvements, supporting molecular-risk–guided timing and early donor identification rather than treatment aimed at IPSS-M downstaging.</p>

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IPSS-M downstaging before transplantation does not improve the prognosis of patients with myelodysplastic neoplasms

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月31日

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  • T. Richardson,
  • D. Schütte,
  • P. Gödel,
  • C. von dem Bongart,
  • C. Burkhard-Meier,
  • E. Lorsy,
  • K. Kreuzer,
  • L. Frenzel,
  • M. Hallek,
  • U. Holtick,
  • C. Scheid

摘要

Allogeneic hematopoietic cell transplantation (ASCT) is the only curative option for patients with myelodysplastic syndromes (MDS), but whether cytoreductive pretreatment and molecular “downstaging” according to the IPSS-M improves outcomes remains unclear. We retrospectively analyzed 128 consecutive adults with MDS who underwent ASCT grouped as frontline transplantation (n = 87) or pretreated before transplant (n = 41). Median bone marrow blasts at diagnosis were 12% vs. 10%. IPSS-M was calculated at diagnosis and immediately before transplant using cytogenetic and next-generation sequencing data. IPSS-M improved in 26% of frontline and 34% of pretreated patients, was unchanged in 41% and 34%, and worsened in 30% and 32%, respectively. After a median follow-up of 17.3 months, overall survival (OS), relapse-free survival (RFS) and graft-versus-host disease relapse-free survival (GRFS) were superior with frontline transplantation (median OS 112.6 vs 14.0 months, p = 0.03, median RFS 61.0 vs 8.9 months, p = 0.007 and median GRFS 13.3 vs 5.3 months, p = 0.004). However, in a landmark analysis starting at the time of transplantation, the difference in OS was no longer statistically significant. Non-relapse mortality was significantly higher after pretreatment (p = 0.018). Pretransplant cytoreduction did not improve post-transplant outcomes despite modest IPSS-M improvements, supporting molecular-risk–guided timing and early donor identification rather than treatment aimed at IPSS-M downstaging.