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This study is an access and distribution protocol for unlicensed cryopreserved cord blood
units (CBUs) in pediatric and adult patients with hematologic malignancies and other
indications.

The primary purpose of the Research Database is to have a comprehensive source of
observational data that can be used to study HSC transplantation and cellular therapies.

A secondary purpose of the Research Database is to have a comprehensive source of data to
study marrow toxic injuries.

Objectives:

To learn more about what makes stem cell transplants and cellular therapies work well such
as:

- Determine how well recipients recover from their transplants or cellular therapy;

- Determine how recovery after a transplant or cellular therapy can be improved;

- Determine how a donor's or recipient's genetics impact recipient recovery after a
transplant or cellular therapy;

- Determine how access to transplant or cellular therapy for different groups of patients
can be improved;

- Determine how well donors recover from the collection procedures.

The investigators want to study if lower doses of chemotherapy will help babies with SCID to
achieve good immunity with less short and long-term risks of complications after
transplantation. This trial identifies babies with types of immune deficiencies that are most
likely to succeed with this approach and offers them transplant early in life before they get
severe infections or later if their infections are under control. It includes only patients
receiving unrelated or mismatched related donor transplants.

The study will test if patients receiving transplant using either a low dose busulfan or a
medium dose busulfan will have immune recovery of both T and B cells, measured by the ability
to respond to immunizations after transplant. The exact regimen depends on the subtype of
SCID the patient has. Donors used for transplant must be unrelated or half-matched related
(haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance
of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T
cells removed, using a newer, experimental approach of a well-established technology. Once
the stem cell transplant is completed, patients will be followed for 3 years. Approximately
9-18 months after the transplant, vaccinations will be administered, and a blood test
measuring whether your child's body has responded to the vaccine will be collected.

The purpose of this study is to determine the clinical benefit and characterize the safety
profile of tabelecleucel for the treatment of Epstein-Barr virus-associated post-transplant
lymphoproliferative disease (EBV+ PTLD) in the setting of (1) solid organ transplant (SOT)
after failure of rituximab and rituximab plus chemotherapy or (2) allogeneic hematopoietic
cell transplant (HCT) after failure of rituximab.

This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and
toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell
disease (SCD). Based on their age and entry criteria patients are stratified into two groups:
(1) children with severe SCD; and (2) adults with severe SCD.