Treosulfan (Specialist drug)

Administration should be supervised by a physician experienced in conditioning treatment followed by allogeneic haematopoietic stem cell transplantation. In the malignant-disease regimen, fludarabine 30 mg/m2 BSA per day is given as a 0.5-hour intravenous infusion on five consecutive days (day -6, -5, -4, -3, -2) before stem cell infusion, total fludarabine dose 150 mg/m2. Adults with non-malignant disease (in combination with fludarabine, with or without thiotepa): treosulfan 14 g/m2 BSA per day as a two-hour intravenous infusion on three consecutive days (day -6, -5, -4) before stem cell infusion, total dose 42 g/m2; fludarabine 30 mg/m2 BSA per day over 0.5 hours on five consecutive days (day -7, -6, -5, -4, -3), total 150 mg/m2; treosulfan given before fludarabine on days -6, -5, -4 (FT 14 regimen); thiotepa 5 mg/kg twice a day as two intravenous infusions over 2 to 4 hours on day -2. Paediatric population older than 1 month (given with fludarabine, with thiotepa as the intensified FT 10-14 TT regimen or without thiotepa as the FT 10-14 regimen): treosulfan 10 to 14 g/m2 BSA per day as a two-hour intravenous infusion on three consecutive days (day -6, -5, -4) before stem cell infusion, total dose 30 to 42 g/m2, with the dose adapted to body surface area — BSA below 0.4 m2: 10.0 g/m2; BSA 0.4 to below 0.9 m2: 12.0 g/m2; BSA 0.9 m2 and above: 14.0 g/m2; plus fludarabine 30 mg/m2 BSA per day over 0.5 hours on five consecutive days (day -7 to -3), total 150 mg/m2, treosulfan given before fludarabine; thiotepa 5 mg/kg twice a day over 2 to 4 hours on day -2 in the intensified regimen. The paediatric dose is body-surface-area based, not per kg, so no per-kg paediatric dose is recorded. Safety and efficacy in children less than 1 month of age has not yet been established. Elderly: no dose adjustment is necessary in any subset of the elderly population.