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ZENODO
Dataset . 2025
Data sources: ZENODO
ZENODO
Dataset . 2025
Data sources: Datacite
ZENODO
Dataset . 2025
Data sources: Datacite
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Reduced relapse rate and improved GVHD/relapse free survival (GRFS) with pharmacokinetics-guided busulfan conditioning regimen for allogeneic stem cell transplantation in adult patients with myeloid hematologic malignancies

Authors: Mariotti, Jacopo; de Philippis, Chiara; De Gregori, Simona; Giordano, Laura; Tentori, Cristina Astrid; Taurino, Daniela; sarina, barbara; +4 Authors

Reduced relapse rate and improved GVHD/relapse free survival (GRFS) with pharmacokinetics-guided busulfan conditioning regimen for allogeneic stem cell transplantation in adult patients with myeloid hematologic malignancies

Abstract

This record contains raw data related to article "Reduced relapse rate and improved GVHD/relapse free survival (GRFS) with pharmacokinetics-guided busulfan conditioning regimen for allogeneic stem cell transplantation in adult patients with myeloid hematologic malignancies" Abstract The introduction of pharmacokinetic (PK)-guided busulfan in the conditioning regimen has shown beneficial effects, particularly in reducing toxicity, for patients undergoing allogeneic stem cell transplantation (Allo-SCT). However, its impact on relapse rate has been partially investigated and whether this translates into improved graft-versus-host-disease (GVHD)/relapse progression free survival (GRFS) has not yet been reported. We prospectively analyzed 90 patients receiving thiotepa-busulfan-fludarabine (TBF) conditioning regimen with PK-guided busulfan (PK-TBF), targeting an AUC of either 20,000 or 16,000 𝜇mol*min, and compared their outcomes with a historical cohort (n=64) treated with fixed busulfan dose (nPK-TBF). Platelet engraftment occurred significantly earlier in the PK-TBF group: median 20 vs 28 days (p=0.013). The cumulative incidence of relapse (CIR) and grade II-IV acute GVHD were significantly lower in the PK-TBF relative to nPK-TBF cohort: 20% vs 31% (p=0.056) and 13% vs 30% (p=0.009), respectively. Consequently, overall survival (OS) and GRFS were significantly improved with PK-TBF relative to nPK-TBF: at 3-years, 61% vs 47% (p=0.025) and 49% vs 34% (p=0.019), respectively. By multivariable analysis, PK-TBF remained an independent predictor of relapse, OS, and GRFS. Outcomes did not differ between PK-TBF AUC targets of 20,000 and 16,000 𝜇mol*min with respect to GVHD or non-relapse mortality (NRM); however, the higher AUC (20,000 𝜇mol*min) cohort was associated with a significant reduction in CIR (at 3 years: 0% vs. 25%, p=0.035). In conclusion, PK guided TBF significantly improves outcome of patients receiving Allo-SCT and higher busulfan exposures may enhance disease control without increasing toxicity.

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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
0
Average
Average
Average