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- Title
Treosulfan-Versus Melphalan-Based Reduced Intensity Conditioning in HLA-Haploidentical Transplantation for Patients ≥ 50 Years with Advanced MDS/AML.
- Authors
Fraccaroli, Alessia; Stauffer, Elena; Haebe, Sarah; Prevalsek, Dusan; Weiss, Lena; Dorman, Klara; Drolle, Heidrun; von Bergwelt-Baildon, Michael; Stemmler, Hans-Joachim; Herold, Tobias; Tischer, Johanna
- Abstract
Simple Summary: Relapse and treatment-related side effects pose significant challenges for older patients with high-risk blood cancers (myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML)) undergoing hematopoietic stem cell transplants from HLA-haploidentical donors. We compared two preparative treatment regimens to identify the most tolerable and effective approach: one using fludarabine, cyclophosphamide, and melphalan, and the other using fludarabine, cyclophosphamide, and treosulfan. Our goal was to determine which treatment offers better survival rates and fewer side effects. We found that both regimens resulted in similar survival outcomes. However, the melphalan regimen was associated with fewer relapses but more treatment-related deaths, while the treosulfan regimen had fewer side effects but exhibited poorer disease control. These findings suggest that treosulfan may be safer, though higher doses might improve disease control, providing valuable insights for future treatments. Relapse and regimen-related toxicities remain major challenges in achieving long-term survival, particularly among older patients with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Previous studies have demonstrated the feasibility of treosulfan-based conditioning, noting stable engraftment and low non-relapse mortality (NRM) in patients undergoing HLA-matched allo-HSCT. However, data on treosulfan-based conditioning in the HLA-haploidentical transplantation (HaploT) setting are limited. We retrospectively compared conditioning with fludarabine–cyclophosphamide (FC)–melphalan (110 mg/m2) and FC-treosulfan (30 g/m2) prior to HaploT using post-transplantation cyclophosphamide (PTCy) in patients with high-risk MDS/AML patients ≥ 50 years, transplanted from 2009–2021 at our institution (n = 80). After balancing patient characteristics by a matched-pair analysis, we identified twenty-one matched pairs. Two-year OS and LFS were similar among the groups (OS 66% and LFS 66%, p = 0.8 and p = 0.57). However, FC-melphalan was associated with a significantly lower probability of relapse compared to FC-treosulfan (0% vs. 24%, p = 0.006), counterbalanced by a higher NRM (33% vs. 10%, p = 0.05). Time to engraftment and incidences of acute and chronic graft-versus-host disease (GvHD) did not differ significantly. In conclusion, HaploT using FC-treosulfan in combination with PTCy in patients aged ≥50 years with MDS/AML appears safe and effective, particularly in advanced disease stages. We confirm the favorable extramedullary toxicity profile, allowing for potential dose intensification to enhance antileukemic activity.
- Subjects
GERMANY; THERAPEUTIC use of antineoplastic agents; MYELODYSPLASTIC syndromes; HEMATOPOIETIC stem cell transplantation; GRAFT versus host disease; STATISTICAL significance; SCIENTIFIC observation; KRUSKAL-Wallis Test; FISHER exact test; AGE distribution; TREATMENT effectiveness; RETROSPECTIVE studies; CHI-squared test; DESCRIPTIVE statistics; MELPHALAN; KAPLAN-Meier estimator; MEDICAL records; ACQUISITION of data; CANCER patient psychology; CONFIDENCE intervals; DATA analysis software; OVERALL survival
- Publication
Cancers, 2024, Vol 16, Issue 16, p2859
- ISSN
2072-6694
- Publication type
Article
- DOI
10.3390/cancers16162859