MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases

MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases

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.

Inclusion Criteria:

• 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)
• absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction ≥ 40%
• DLCO FEV1, FVC ≥ 40% predicted, and absence of O2 requirement

Exclusion Criteria:

• Pregnant or breastfeeding
• Evidence of untreated/uncontrolled HIV infection
• Untreated active serious infection
• Active CNS malignancy
• CML in blast crisis not in a complete remission by abnormal blast count.
• Less than 3 months since prior myeloablative transplant