Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders
Trial Parameters
Brief Summary
This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TBI); or, fludarabine/busulfan for patients unable to receive further radiation). followed by a post-transplant graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF).
Eligibility Criteria
-Inclusion Criteria: * Age: ≤ 60 years of age * Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70 * Consent: Voluntary written consent (adult or legally authorized representative; or parental/guardian) * Adequate Organ Function: * Renal: Creatinine \<2x upper limit of normal. Patients above this limit must have creatinine clearance ≥ 40 ml/min/1.73m2 as determined by an age-appropriate method, such as cystatin C GFR. * Hepatic: Bilirubin, AST, alkaline phosphatase \<4 times the upper limit of institutional normal * Pulmonary: Diffusion capacity of oxygen, corrected for hemoglobin, \> 50% of predicted. For pediatric patients not able to undergo PFTs or diffusion testing: O2 sat of \>95% on room air * Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \> 45%. For children not able to cooperate with MUGA or echocardiography, such should be clearly stated in the physician's documentation * HIV Status: HIV