High-Dose Thiotepa-Cyclophosphamide (TT/Cy) Results in Superior Outcomes Compared with Total Body Irradiation-Cyclophosphamide (TBI/Cy) in Patients with Acute Myeloid Leukemia (AML), Advanced Chronic Myeloid Leukemia (CML) and High-Grade Myelodysplasia (MDS) Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (HCT)

Abstract

disease (GvHD) prophylaxis regimen. Survival analysis was calculated by Kaplan-Meier analysis and log-rank test was used to compare variables. Forty-three patients (21 of male) were enrolled; 22, 19 and 2 patients were diagnosed as ALL, AML and MDS, respectively. Twenty-three patients received TBI-regimen, and 26 patients received mother derived stem cell products. Most of the donors had 5/10 or 6/10 HLA-matched. The median CD34+ cell dose was 9.24 (2.06-21.8) cells £10 6 /kg. One patient died from disseminated adenoviral infection prior to neutrophil engraftment. For 42 evaluable patients, the median time to neutrophil and platelet engraftment was 15 (12-26) days and 22 (12-100) days, respectively. At the median follow-up time of 18.5 (0.6-74.2) months, the overall survival rates of TBI-regimen and Thio-regimen were 63.68 and 61.98%, respectively (p=0.69), while the event-free survival rates of TBI-regimen and Thio-regimen were 68.82 and 66.67%, respectively (p=0.61). Viral infections, CMV, BKV and adenovirus, were the most common infectious complications and were comparable in both groups. Moreover, rates of acute and chronic GVHD in both groups were not significantly different. Relapse was the most common cause of death in both regimens while non-relapse mortality rates of both regimens were approximately 17%. Haplo-identical SCT in high-risk pediatric hematologic malignancies using Thio-regimen had comparable clinical outcomes and complications as those using TBI-regimen.

A familialSAMD9variant present in pediatric myelodysplastic syndrome

“…This treatment plan prevented her disease from progressing to AML and bridged her to stem cell transplant as the next phase of therapy. She received a haploidentical bone marrow stem cell transplant from her father after fludarabine, thiotepa, and cyclophosphamide conditioning regimen (Hyder et al 2019). She did not receive a busulfan-based conditioning regimen based on the increased risk of mortality seen in patients with Gilbert's disease and opted for a non-total-body irradiation-based regimen because of the concern for hepatotoxicity in the setting of Gilbert's disease (McDonald et al 2016).…”