This is a Phase II study following subjects proceeding with Treosulfan (36g/m2) preparative regimen followed by a related, unrelated, or partially matched family donor stem cell infusion, with post-transplant cyclophosphamide (PTCy) at 40mg/kg, tacrolimus and MMF for GVHD prophylaxis.
Patients 2-75 years of age
≤7 5 years of age: Karnofsky score ≥ 70% (≥ 16 years) or Lansky play score ≥ 50 (< 16 years) with appropriate organ criteria as below (in other inclusion criteria)
5/6 or 6/6 related donor, OR a 5-8/8 HLA-A, B, C, DRB1 allele match unrelated donor, OR a haplotype (at least 5/10) related donor
adequate liver (no decompensated liver failure, Child Pugh A, AST/ALT <5X ULN) and renal function (creatinine <2.0)
Pregnant or breastfeeding
Evidence of untreated/uncontrolled HIV infection
Untreated active serious infection
Active CNS malignancy