Session: 721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster I
Hematology Disease Topics & Pathways:
Here we report on a matched-pair analysis of 26 patients treated with either a treosulfan- or melphalan-based sequential conditioning for haplo-HSCT using PTCY as GvHD prophylaxis in HR MDS/AML.
We retrospectively analyzed the outcome and toxicity profile of 26 patients undergoing sequential haplo-HSCT at our center between January 2009 and June 2019. Thirteen patients with HR AML/MDS and >54 years old who underwent sequential haplo-HSCT using treosulfan (3x10g/m2) for RIC were considered for potential matching with recipients (n=30) of a sequential melphalan-based RIC regimen. Matching criteria comprised (1) disease activity (blast yes or no), (2) disease status (relapse, refractory, high-risk cytogenetics), (3) HCT-CI and (4) age (+/- 5 years). Post-grafting immunosuppression consisted of cyclophosphamide, tacrolimus and MMF.
Thirteen patients undergoing treosulfan-haplo-HSCT were successfully pair-matched with thirteen recipients of melphalan-based haplo-HSCT, respectively ((1) p=1.0; (2) p=1.0; (3) p=1.0; (4) p=0,76). Median age of the entire cohort was 63 years (54-71). Each group consisted of two MDS patients and eleven AML patients. All recipients treated with treosulfan showed neutrophil engraftment with a median of 20 days while only 69% of the melphalan treated patients engrafted (median=19 days, p= 0.9). In the melphalan group one graft rejection occurred, three patients died in early aplasia. Acute GvHD °II-IV occurred in 23% and 44% of the patients treated with treosulfan or melphalan, respectively. Severe (°III-IV) non-hematologic regimen-related toxicities were seen in 2/13 pts of the treosulfan and 7/13 pts of the melphalan group, predominately affecting the GI-tract in both. NRM at day +100 was 0% and 31% (p=0.06) for the treosulfan and melphalan group, respectively. Thereby, infections made up for most NRM events. Conversely, CI of relapse at one year was 23% vs 0 % (p=0.004) for treosulfan vs melphalan. OS at 1-year (treosulfan group: 69% vs melphalan gropup: 62%) and PFS at 1-year (treosulfan group: 69% vs melphalan group: 62%) did not differ significantly between the groups (p=0.72)
Sequential haplo-HSCT using treosulfan and PTCY in older advanced MDS/AML patients is safe, resulting in lower NRM at the expense of higher relapse incidence compared to the melphalan-based sequential conditioning approach. Treosulfan-based RIC in haplo-HSCT though might be an alternative in older pts with low leukemic burden. However for disease control its intensity should be reconsidered and all available post-grafting mantainance strategies applied.
Disclosures: Fraccaroli: Medac: Other: Travel Grant. Buecklein: Celgene: Research Funding; Amgen: Consultancy; Pfizer: Consultancy; Gilead: Consultancy, Research Funding; Novartis: Research Funding.
See more of: Oral and Poster Abstracts