Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO (THALLO)
Thalassemia
About this trial
This is an interventional treatment trial for Thalassemia focused on measuring transfusion-dependent, homozygous b0/+-thalassemia, severe variants of b0/+-thalassemia, transfusion, iron chelating agents, severe, transfusion-dependent homozygous
Eligibility Criteria
Inclusion Criteria:
Patients with documented diagnosis of severe (transfusion-dependent) homozygous b0/+-thalassemia or severe variants of b0/+-thalassemia requiring chronic transfusion therapy and iron chelating agents, who fulfill the following conditions:
- Patient does not have an HLA genotype-identical donor available and has a 5/6 or 6/6 matched unrelated donor, or a 5/6 matched related donor available.
- Must be between 1 and 16 yrs of age (all Pesaro risk groups).
- Patients older than 17 yrs of age must be in Pesaro Risk Class 2 or lower (see Appendix B).
- Women of childbearing potential must have a negative pregnancy test.
- Documentation of compliance with iron chelation, absence or presence of hepatomegaly, and presence or absence of hepatic fibrosis prior to transplant (criteria for the Pesaro Risk Classification). This information will be obtained by history, physical exam and interpretation of liver biopsy results.
- Documentation of awareness of alternative treatment options.
Exclusion Criteria:
- Biopsy-proven chronic active hepatitis or fibrosis with portal bridging.
- Has previous history of malignancies.
- Creatinine clearance < 35 mL/min/1.73 M2.
- Severe cardiac dysfunction defined as shortening fraction < 25%.
- HIV infection.
- Inadequate intellectual capacity to give informed consent (in the case of minors, this criteria must be fulfilled by the legal guardian).
- Be pregnant, lactating or unwilling to use appropriate birth control.
Sites / Locations
- Texas Children's Hospital
Arms of the Study
Arm 1
Experimental
Bone Marrow or Stem Cell Infusion
Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor.