A Prospective Outcomes Study of Pediatric and Adult Patients With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation With a Reduced-Intensity Conditioning Regimen (PRO-RIC)
Overview
- Phase
- N/A
- Intervention
- Not specified
- Conditions
- Primary Immunodeficiency (PID)
- Sponsor
- Paul Szabolcs
- Enrollment
- 50
- Locations
- 2
- Primary Endpoint
- incidence of acute graft versus host disease (GVHD)
- Status
- Recruiting
- Last Updated
- 3 months ago
Overview
Brief Summary
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.
Detailed Description
Hematopoietic stem cell transplantation (HSCT) from a healthy donor can cure or alleviate a broad spectrum of non-malignant disorders (NMD). Although reduced-intensity conditioning (RIC) regimens promise decreased treatment-related morbidity and mortality, graft failure and infections are limiting the use of RIC in chemotherapy-naive patients. Dr. Szabolcs have completed several trials to evaluate a novel RIC regimen of alemtuzumab, hydroxyurea, fludarabine, melphalan, and thiotepa. The last trial at UPMC Children's Hospital of Pittsburgh of a highly effective and biologically rational chemotherapy-based RIC regimen paired with simple alemtuzumab dosing strata was tested and resulted in outstanding survival and remarkably low rates of graft failure. The favorable outcome described may serve as a toxicity and efficacy reference for emerging gene therapy strategies as well. This prospective collection of clinical data will allow the investigators to further assess engraftment, GVHD, immunosuppressant use and overall survival in this patient population.
Investigators
Paul Szabolcs
Chief, BMT-CT at CHP of UPMC and Professor of Pediatrics and Immunology, University of Pittsburgh
University of Pittsburgh
Eligibility Criteria
Inclusion Criteria
- •Patient, parent, or legal guardian must have given written informed consent.
- •Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
- •Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to the following:
- •A. Primary Immunodeficiency Syndromes
- •Severe Combined Immune Deficiency (SCID) with NK cell activity
- •Omenn Syndrome
- •Bare Lymphocyte Syndrome (BLS)
- •Combined Immune Deficiency (CID) syndromes
- •Combined Variable Immune Deficiency (CVID) syndrome
- •Wiskott-Aldrich Syndrome
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
incidence of acute graft versus host disease (GVHD)
Time Frame: up to 5 years
grades 3-4, chronic extensive GVHD
overall survival after HSCT
Time Frame: up to 5 years
review of the existing medical records to check on the participant's survival status
Secondary Outcomes
- Describe probability to discontinue systemic immunosuppression medications(by 6, 9, and 12 months post-HSCT)
- Describe the tempo of immune reconstitution(over the first year post transplant)
- Describe degree of engraftment, based upon chimerism data(up to 5 years)
- Describe the use of donor leukocyte infusion (DLI)(up to 5 years)