Efficacy results
leukopenia, GVHD, test.#Neutrophilic granulocytes ≤, neutropenia
NEUTROPENIA, LEUKOPENIA, DISEASE, EVENT, PRIMARY GRAFT FAILURE, SECONDARY, REGRESSION, GVHD
The incidence of freedom from TRM until Day +100 was 90.0% (95% CI: 78.2%, 96.7%) and 100.0% (95% CI: 93.0%, 100.0%) in the busulfan and treosulfan arm (difference of incidences –10.0% [95% CI: –21.8%, –2.0%]; Freedom from Transplantation (treatment)-related Mortality and Secondary Outcomes (FAS).*Adjusted for thiotepa and disease.†Stratified Cochran-Mantel-Haenszel test.‡Based on Kaplan-Meier estimates.§Adjusted for thiotepa and disease as factors using Cox regression model.||Only if this event occurred first.¶Based on Pepe-Mori test.#Neutrophilic granulocytes ≤ 0.5 g/L at least once between Day -7 and Day +28.∗∗First date with neutropenia until date of engraftment (patients at risk = patients with neutropenia and neutrophilic granulopoiesis).††Based on the Wilcoxon-Mann-Whitney test.‡‡Leukocytes ≤ 1 g/L at least once between Day -7 and Day +28.aFirst date with leukopenia until date of engraftment (patients at risk = patients with leukopenia and leukopoiesis).bRate of primary/secondary graft failure calculated as number of patients with graft failure by the number of patients at risk.-At risk for primary graft failure: Patients with HSCT.-At risk for secondary graft failure: Patients whose neutrophilic granulocytes engrafted after HSCT or were never below the required level.cAdjusted for thiotepa and disease as factors using Fine and Gray model.dPatients are at risk if they have an examination at the Day +28, Day +100, Month 12 or if they have survived day +30, +107, +379, respectively.eMissing values are excluded for odds ratio calculation and tests.CI, confidence interval; FAS, full analysis set; GVHD, Graft‑versus-host disease; N, total number of patients; NE, not estimated; OS, overall survival; Q1, first quartile; Q3, third quartile; TRM, transplantation related mortality.The Kaplan-Meier estimate of TRM at 12 months was 12.0% (95% CI: 5.6%, 24.8%) and 3.9% (95% CI: 1.0%, 14.8%) in the busulfan and treosulfan arm (HR: 0.29 [95% CI: 0.06, 1.41]). Estimate of TRM at 12 months in the disease subgroup HBPs was 7.7% for busulfan and 0% for treosulfan. After a median follow-up of 25 months (busulfan range: 11.7-63.3 months; treosulfan range: 10.7-60.9 months) the 12-month estimate of OS was 88.0% (95% CI: 75.2%, 94.4%) in the busulfan arm versus 96.1% (95% CI: 85.2%, 99.0%) in the treosulfan arm (HR: 0.29 [95% CI: 0.06, 1.41]; Fig. 