Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Masonic Cancer Center, University of Minnesota
Age
0–25
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Sickle Cell Disease (SCD)
\* If diagnosis of SCD must meet one or more of the following disease characteristics:
* Stroke, CNS hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral MRI or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
* Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
* Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
* Impaired neuropsychological function and abnormal cerebral MRI scan
* Stage I or II sickle lung disease,
* Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate \[GFR\] 30-50% of the predicted normal value)
* Bilateral proliferative retinopathy and major visual impairment in at least one eye
* Osteonecrosis of multiple joints with documented destructive changes
* Requirement for chronic transfusions
* RBC alloimmunization
Transfusion Dependent Alpha- or Beta-Thalassemia
Other Non-Malignant Hematologic Disorders:
Transfusion dependent or involve other potential life-threatening cytopenias, including but not limited to Paroxysmal Nocturnal Hemoglobinuria, Glanzmann's Thrombasthenia, Severe Congenital Neutropenia and Shwachman-Diamond Syndrome
cALD
* Diagnosis of ALD by abnormal plasma very long chain fatty acid (VLCFA) profile or ABCD1 gene mutation
* Cerebral disease on MRI
* Absence of a Major Functional Disability (cortical blindness, loss of communication, wheelchair dependence) on the ALD Neurologic Function Scale
Other inherited metabolic disorders:
Any other inherited metabolic disorder for which alloHCT is indicated and for whom, in the opinion of the treating physician, the patient's best treatment option is with a haploidentical donor following non-myeloablatve conditioning.
