The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.
CD3/CD19 depletion of mismatched donor grafts in the setting of reduced intensity, immune-ablative conditioning for patients with sickle cell disease and other transfusion-dependent anemias should sufficiently achieve engraftment while decreasing the incidence of treatment-related toxicities and achieving an acceptable incidence of graft versus host disease. Utilizing mismatched unrelated volunteer donors and haploidentical related donors will increase the number of patients able to undergo hematopoietic stem cell transplant (HSCT) for these diseases. Additionally, the institutional availability of virus-specific, donor-derived cytotoxic T lymphocytes should address complicated viral infections refractory to standard anti-viral therapy. The purpose is to: * To provide alternate donor transplantation from cryopreserved stem cell grafts that are fully characterized for safety and potency to patients with severe sickle cell disease, beta-thalassemia major, or Diamond-Blackfan anemia who do not have matched sibling donor, matched unrelated donor or cord blood donor options. * To utilize a reduced-intensity conditioning regimen to achieve minimal treatment-related morbidity and mortality while attaining sustained donor engraftment and donor chimerism \>20% in order to rescue disease phenotype, specifically in SCD patients. * To utilize ex-vivo T-cell depletion methods to prevent graft-versus-host disease in the setting of mismatched donor transplantation. * To utilize additional donor cell products to ensure sufficient immune reconstitution in the immediate post-transplant period, to improve mixed chimerism or provide non-specific anti-viral activity in patients with virus reactivation in the post-transplant period. * To utilize calcineurin inhibitor-free regimen in an effort to minimize/prevent central nervous system toxicity
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Negative selection for CD3+/CD19+ cells will be performed on the CliniMACS® depletion device.
Negative selection for CD45RA will be performed on the CliniMACS® depletion device.
Sickle Cell Disease Conditioning
Sickle Cell Disease Conditioning
Sickle Cell Disease Conditioning
Sickle Cell Disease Conditioning
Sickle Cell Disease Conditioning
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
RECRUITINGGraft rejection
How frequent, if any, graft rejection occurs
Time frame: Day -30 through study completion, an average of 2 years
Post Transplant treatment related mortality
Number of deaths that occurred from treatment
Time frame: By day 100
Acute Graft versus host disease
The number of patients who develop acute graft versus host disease (GVHD)post transplant
Time frame: Day 0 through study completion, an average of 2 years
Chronic Graft versus host disease
The number of patients who develop chronic graft versus host disease (GVHD) post transplant
Time frame: Day 0 through study completion, an average of 2 years
Post Transplant treatment related mortality
Number of deaths that occurred from treatment
Time frame: Day 180
Post Transplant treatment related mortality
Number of deaths that occurred from treatment
Time frame: 1 year
Neutrophil recovery
≥ 0.5 x 103/μL neutrophils for three consecutive days tested on different days.
Time frame: Day 0 through study completion, an average of 2 years
Donor Cell Engraftment
≥ 5% donor cells on day +42 and ≥ 10% donor cells on day +100. We will record if subjects have attained robust donor cell engraftment (\> 50% donor chimerism at 180 days).
Time frame: From Day 0, Day 42, Day 100 and Day 180. Further testing can be done if clinically indicated up to 2 years post transplant
Neurological complications
To evaluate the incidence of neurological complications
Time frame: Day 0 through study completion, an average of 2 years
Immune reconstitution
The pace of systemic immune reconstitution
Time frame: Day 0 through study completion, an average of 2 years
Cytomegalovirus (CMV) infection
Incidence of CMV infection by Polymerase chain reaction (PCR) as clinically indicated
Time frame: Day 0 through study completion, an average of 2 years
Donor Lymphocyte Infusions response
Evaluate for delayed immune reconstitution, mixed chimerism or viral reactivation
Time frame: Day 0 through study completion, an average of 2 years
Response to donor-derived virus-specific cytotoxic T-cell therapy
Activation or reactivation of Cytomegalovirus (CMV), Epstein-Barr Virus (EBV) or adenovirus testing by PCR
Time frame: Day 0 through study completion, an average of 2 years
Sickle Cell disease phenotype recurrence
The incidence of Sickle Cell recurrence as clinical evidence of vaso occlusive crisis, detection of HgbS\>25% and acute chest syndrome.
Time frame: Day 0 through study completion, an average of 2 years
Recurrence of transfusion-dependence
Chronic transfusion therapy defined as \> 8 packed red blood cell transfusions per year in the year prior to enrollment and/or evidence of red blood cell alloimmunization.
Time frame: Day 0 through study completion, an average of 2 years
Organ toxicity
Incidence of Grade 3-4
Time frame: Day 0 through study completion, an average of 2 years
Long-term complications-Sterility, endocrinopathy, and secondary malignancy
Incidence of long term complications
Time frame: Day 0 through study completion, an average of 2 years
Pediatric Quality of Life Inventory
Measures Pain/Hurt ,Pain Impact,Pain Management/Control ,Worry ,Emotions ,Treatment , Communication
Time frame: Baseline through study completion, an average of 2 years
Platelet Recovery
Platelet count of ≥ 20,000/μL without platelet transfusion in the previous 7 days.
Time frame: Day 0 through study completion, an average of 2 years
Adult Sickle Cell Quality of Life Measurement System (ASCQ)
Patient reported outcome measurement system that assesses the physical, social and emotional impact of Sickle Cell Disease.
Time frame: Baseline through study completion, an average of 2 years
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