TCRαβ+/CD19+ Depleted Allogeneic Hematopoietic Stem Cell Transplantation for Malignant and Non-malignant Disorders

Phase 2

Terminated

Interventions: Drug: TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT)

Brief Summary: The purpose of this study is to investigate the safety and efficacy of TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT) for malignant and non-malignant disorders in children and adolescent/young adult patients using the CliniMACS® immunomagnetic selection device (Miltenyi Biotec).

Detailed Description: Acute graft versus host disease (GVHD) remains a significant cause of morbidity and mortality and is the biggest barrier to successful allogeneic hematopoietic cell transplantation (HSCT) outcomes. Improved methods of acute GVHD prevention are needed. TCRαβ+/CD19+ depletion of allogeneic hematopoietic stem cell products offers an opportunity to limit the risk of acute GVHD by removing TCRαβ+ T cells and CD19+ B cells which participate in acute GVHD initiation and perpetuation. The purpose of this study is to investigate the safety and efficacy of TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT) for malignant and non-malignant disorders in children and adolescent/young adult patients using the CliniMACS® immunomagnetic selection device (Miltenyi Biotec).

Recruitment & Eligibility

Inclusion Criteria

Any patient being treated at Cincinnati Children's Hospital requiring an allogeneic HSCT who lacks an HLA-genotypically matched related donor. Genotypically matched related donors are allowed when there is a clinical desire to avoid the use of GVHD prophylaxis medications.

Exclusion Criteria

Prior allogeneic transplant with active acute or chronic GVHD, or life-threatening infection. Patients with a prior history of allogenic transplant without active GVHD or life-threatening infection can be considered.

Arm && Interventions

Group	Intervention	Description
TCRαβ+/CD19+ depleted HSCT	TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT)	-

Primary Outcome Measures

Name	Time	Method
Incidence of Infusion-related Reactions	100 days	Number of patients who experienced infusion reactions including rash, fever, difficulty breathing, and blood pressure abnormalities at the time of infusion of stem cells.

Secondary Outcome Measures

Name	Time	Method
Engraftment and Sustained Donor Chimerism	28 days and 1 year	Initial neutrophil engraftment prior to day +28 was determined by monitoring for neutrophil count recovery post-transplant and performing blood tests to confirm presence of donor cells. Sustained donor chimerism at 1 year post transplant was determined again by performing blood tests to confirm presence of donor cells.
Number of Participants With Acute GVHD	100 days	Patients were monitored for symptoms of acute graft versus host disease including rash, diarrhea, and increased bilirubin using the Modified Glucksberg Criteria.
Number of Participants With Chronic GVHD	1 year	Patients were monitored for symptoms of chronic graft versus host disease using the NIH Consensus Criteria.
GVHD-free Survival	1 year	GVHD-free survival was determined based on the presence or not of acute or chronic GVHD at 1 year.