干细胞研究与治疗杂志

抽象的

Survival Benefit of Intravenous Busulfan (120 mg/M2) and Fludarabine Myeloablative Regimen for Treatment of Myeloid Malignancies

Niparuck P, Pukiat S, Puavilai T, Ativitavas T, Chantrathammachart P, Boonyawat K, Wacharapomin P, Angchaisuksiri P, Chuncharunee S, Jootar S, Atichartakarn V and Ungkanont A

Background: Intravenous fludarabine and busulfan 130 mg/m2 (Flu/Bu) conditioning regimen has induced the long term survival with a low treatment related mortality rate. However, there have been few reports on long term survival of patients undergoing allo-HSCT with intravenous Flu/Bu regimen. Therefore, we conducted a retrospective study of 42 patients diagnosed with myeloid malignancies received allo-HSCT with intravenous fludarabine and busulfan (120 mg/m2) regimen between 2006 and 2015 at Ramathibodi hospital. The aim of our study was to observe the long term survival and the complication after transplantation.
Findings: Thirty- four, three and five patients were AML, MDS and CML-CP respectively. With a median followup of 95 months, 1- year EFS and 8- year EFS were 82 and 70% respectively. Overall survival (OS) rate at 1 and 8 years were equally observed in 88%. Patient younger than age 45 years had significantly longer OS than patients aged 45 years and older (96 vs 70%, p=0.019). Eight- year OS in AML, MDS and CML were 88, 67 and 100% respectively. Acute and chronic graft versus host disease were found in 29 and 46.3% of 41 evaluable patients respectively. Whereas the rates of sinusoidal obstruction syndrome, sepsis, CMV reactivation, cyclosporine and tacrolimus induced thrombotic microangiopathy were 2, 10, 12, 5 and 2% respectively. Non- relapse mortality rate at day +100, 1 and 8 year were only 9.5, 13.8 and 13.8% respectively. There was no neurological toxicity, severe mucositis, secondary malignancy or therapy related MDS syndrome in this study.
Conclusion: Allogeneic hematopoietic stem cell transplantation with intravenous fludarabine and busulfan at the dose of 120 mg/m2 was well tolerated and demonstrated impressive treatment outcomes in young adult patients diagnosed with myeloid malignancies.