Reduced‐toxicity conditioning with fludarabine, once‐daily intravenous busulfan, and antithymocyte globulins prior to allogeneic stem cell transplantation: Results of a multicenter prospective phase 2 trial. Issue 4 (3rd October 2014)

• Record Type: Journal Article
• Authors: Mohty, Mohamad; Malard, Florent; Blaise, Didier; Milpied, Noel; Furst, Sabine; Tabrizi, Resa; Guillaume, Thierry; Vigouroux, Stéphane; El‐Cheikh, Jean; Delaunay, Jacques; Le Gouill, Steven; Moreau, Philippe; Labopin, Myriam; Chevallier, Patrice
• Is Part Of: Cancer. Volume 121:Issue 4(2015)
• Page Start: 562
• Page End: 569
• Publication Date: 2014-10-03
• Subjects: allogeneic stem cell transplantation -- reduced‐intensity conditioning -- submyeloablative conditioning -- intravenous busulfan -- prospective trial; Cancer -- Periodicals; Cancer -- Cytopathology -- Periodicals; 616.99405
• DOI: 10.1002/cncr.29087
• Languages: English
• ISSNs: 0008-543X

Abstract

Abstract : BACKGROUND: The optimal intensity of myeloablation delivered as part of a reduced‐intensity/toxicity conditioning (RIC/RTC) regimen to decrease the recurrence rate, without increasing nonrecurrence mortality (NRM), remains to be established. METHODS: The current phase 2, prospective, multicenter trial aimed to assess the efficacy and safety of an RIC/RTC regimen based on busulfan at a dose of 130 mg/m 2 /day intravenously for 3 days, fludarabine at a dose of 30 mg/m 2 /day for 5 days, and antithymocyte globulins at a dose of 2.5 mg/kg/day for 2 days. A total of 80 patients (median age, 53 years; range, 25‐64 years) with hematological malignancies were included. RESULTS: With a median follow‐up of 21 months (range, 12‐36.5 months), the Kaplan‐Meier estimates of overall and disease‐free survival at 2 years were 62% (95% confidence interval [95% CI], 51%‐73%) and 50% (95% CI, 33%‐57%), respectively. The cumulative incidences of grade 2 to 4 acute graft‐versus‐host disease (GVHD) and chronic GVHD (all grades) were 29% (95% CI, 19%‐39%) and 35% (95% CI, 24%‐46%), respectively. At 2 years, the cumulative incidence of recurrence/disease progression and NRM were 44% (95% CI, 31%‐56%) and 11% (95% CI, 6%‐19%), respectively. Patient age, diagnosis, donor type, sex, presence of comorbidities, and the Hematopoietic cell transplantation‐specific comorbidities index did not appear to have any statistically significant impact on NRM, recurrence/disease progression, disease‐free survival, or overall survival. CONCLUSIONS: The RIC/RTC regimen used in the current study appeared to be safe, with a low NRM rate at 2 years noted among high‐risk patients, and efficient disease control, warranting prospective phase 3 trials. Cancer 2015;121:562–569. © 2014 American Cancer Society . Abstract : The reduced toxicity regimen based on a busulfan dose of 130 mg/m 2 /day intravenously for 3 days, fludarabine at a dose of 30 mg/m 2 /day for 5 days, and antithymocyte globulins at a dose of 2.5 mg/kg/day for 2 days was evaluated in a prospective phase 2 trial. This regimen appeared to be safe, with a low nonrecurrence mortality rate at 2 years in high‐risk patients, and efficient disease control, thereby warranting prospective phase 3 trials.