Thalassemia Clinical Trial
Official title:
A Pilot Study of Reduced Intensity Conditioning in Pediatric Patients <21 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood Transplantation
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients < 21 years receiving cord blood transplantation for non-malignant disorders.
Myeloablative doses of chemotherapy and/or radiation therapy are employed with the primary
purpose of eradicating malignant cells. Additionally, these regimens exert varying degree of
immunosuppression/immunoablation that aids in reducing the likelihood of rejection by host
hematopoietic cells. However, myeloablative /immunoablative regimens have also been
associated with significant regimen related toxicity (RRT) and regimen related mortality
(RRM) that may cause death in up to 20% of patients and significantly higher rate of severe
organ dysfunction or failure. While most of these RRT occur typically in the first 100 days
[ e.g. VOD (veno occlusive disease), pulmonary or intracranial hemorrhage, multiorgan
failure (MOF)], there are significant long term toxicities of TBI and/or chemotherapy
including growth impairment, gonadal dysfunction/failure, hypothyroidism, cataracts,
neurocognitive impairment, and second malignancies.
The primary objective is to determine the feasibility of attaining acceptable rates of donor
cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning
(RIC) regimens in pediatric patients < 21 years receiving cord blood transplantation for
non-malignant disorders.
The secondary objectives are:
- To describe the pace of neutrophil and platelet recovery
- To evaluate the pace of immune reconstitution.
- To determine the treatment related mortality, overall survival and disease free
survival by days 100 and 180 post-transplant
- To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) and chronic
extensive GVHD
- To describe the incidence of grade 3-4 organ toxicity
- To evaluate long-term complications, such as sterility, endocrinopathy, and growth
failure
- To evaluate the incidence of late graft failures at 2 years post-transplant
;
Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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