MT2017-17:T Cell Receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
∙ For FA patients:
• Diagnosis of Fanconi anemia
‣ Age \<65 years of age
• Has one of the following risk factors:
‣ Severe aplastic anemia (SAA)
⁃ Myelodysplastic features
⁃ High risk genotype
⁃ Immunodeficiency associated with history of recurrent infections
• Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients \<16 years of age
‣ Adequate pulmonary, cardiac and liver function
⁃ Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care
∙ For TBD patients:
∙ • Diagnosis of TBD
• Age \<70 years of age
• Has one of the following risk factors:
• Severe aplastic anemia (SAA)
• Myelodysplastic features
• Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score
• ≥ 50% for patients \<16 years of age
• Adequate pulmonary, cardiac and liver function
• Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care