Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Stanford University
With
University of Minnesota
University of Alabama at Birmingham
Age
1–25
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Patients with sickle cell disease (SCD) 1-25 years of age with an HLA-identical, but unrelated, donor or 1 human leukocyte antigen (HLA) allele mismatched bone marrow or up to 2 HLA antigen mismatched umbilical cord blood (UCB) donor with one or more of the following:
* Stroke, central nervous system (CNS) hemorrhage or a neurologic event lasting longer than 24 hours.
* 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/or abnormal cerebral MRI scan or abnormal transcranial Doppler (TCD).
* 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 but with RBC alloimmunization \>2 antibodies during long term transfusion therapy.
* Failure of hydroxyurea (HU) therapy.
Patients aged 0-21 years with transfusion dependent alpha- or beta-thalassemia who have an HLA-identical or 1 HLA allele mismatched bone marrow or up to 2 HLA mismatched UCB donor.
You may not be if
Patients with one or more of the following:
* Karnofsky or Lansky performance score \<70 (See Appendices I and II).
* Stage III-IV lung disease (Appendix III).
* GFR\<30% predicted normal values.
* Pregnant or lactating females.
* Active serious infection whereby patient has been on intravenous antibiotics for one week prior to study entry.
* Any patient with AIDS or HIV seropositivity.
* Any patient with invasive aspergillus infection within one year of study entry.
* Psychologically incapable of undergoing bone marrow transplant (BMT) with associated strict isolation or documented history of medical non-compliance.
Clareo Health | Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
