INCLUSION CRITERIA:
RECIPIENTS:
Patients with Hb SS, SC, or Sb-thalo at high risk for disease related mortality or morbidity as defined by having one or more of the following:
- a previous neurologic event (either symptomatic or found by imaging alone),
- more than 2 painful crises per year for the last 2 years each requiring hospitalization,
- a previous acute chest syndrome,
- evidence of renal damage
- red cell alloimmunization,
- stage I or II sickle chest, (Stage I patients have normal oxygen saturation but 80% of predicted normal pulmonary function tests. Stage II patients also have normal oxygen saturation but 60% of predicted normal pulmonary function tests.)
- osteonecrosis of multiple joints
- pulmonary hypertension as measured by a TRV jet of greater than 2.5m/s
and
- having failed hydroxyurea, as defined by a failure to achieve a hematologic response and/or clinical response where clinical/hematologic response is defined as a significant decrease in the number of crises experienced after a 6 month trial or a 2-3 fold increase in the hemoglobin F level unless has renal insufficiency preventing hydroxyurea use, or indication for transplant is or includes a neurologic event.
or
Patients with transfusion dependent Diamond Blackfan anemia
or
Patients with Thalassemia with Class II or III Iron overload
Ages 18- 65
6/6 HLA matched family donor available
Ability to comprehend and willing to sign an informed consent
Negative Serum B-HCG
INCLUSION CRITERIA:
DONOR
6/6 HLA identical family donor
Age greater than or equal to 18
Fit to receive G-CSF and give peripheral blood stem cells (normal blood counts, normotensive and no history of stroke)
Ability to comprehend and willing to sign an informed consent; assent obtained from minors
EXCLUSION CRITERIA:
RECIPIENT
(any of the following would exclude the subject from participating)
Age less than 18 years or greater than 65 years
ECOG performance status of 3 or more.
Diffusion capacity of carbon monoxide (DLCO) less than 60% predicted.
Left ventricular ejection fraction: less than 40%.
Transaminases greater than 5x upper limit of normal
Psychiatric disorder or mental deficiency severe enough as to make compliance with the BMT treatment unlikely, and making informed consent impossible.
Major anticipated illness or organ failure incompatible with survival from PBSC transplant.
Pregnant or lactating
EXCLUSION CRITERIA:
DONOR:
(any of the following would exclude the donor from participating)
Age less than 18
Pregnant or lactating
Donor unfit to receive G-CSF and undergo apheresis. (Uncontrolled hypertension, history of congestive heart failure or unstable angina, thrombocytopenia)
HIV positive
Hemoglobin SS, SC, or Sbeta thal0, or beta thalassemia intermedia
Abnormal Red Cell Adenosine Deaminase level
Warren Grant Magnuson Clinical Center (CC)