Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases
- Conditions
- Graft Versus Host DiseaseLeukemiaMyelodysplastic SyndromesThymic Carcinoma
- Registration Number
- NCT00003662
- Lead Sponsor
- Roswell Park Cancer Institute
- Brief Summary
RATIONALE: Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy or radiation therapy.
PURPOSE: Phase II trial to study the effectiveness of umbilical cord blood transplantation in treating patients who have hematologic cancer or other hematologic or metabolic diseases.
- Detailed Description
OBJECTIVES: I. Determine the rates of durable engraftment in patients with severe aplastic anemia, myelodysplastic syndrome, inborn errors of metabolism, or inherited hematopoietic disorders refractory to medical management, who are undergoing high dose chemoradiotherapy followed by unrelated cord blood (UCB) transplantation. II. Determine the incidence and severity of acute and chronic graft-versus-host disease in these patients. III. Monitor overall and event-free survival of these patients. IV. Evaluate rate and quality of immunologic reconstitution of these patients. V. Determine whether nucleated cell or progenitor cell content of the graft is predictive of engraftment.
OUTLINE: This is a multicenter study. Patients are stratified according to low vs high weight. Patients with severe aplastic anemia, myelodysplastic syndrome, or bone marrow failure receive cyclophosphamide IV over 1 hour on days -6 to -3 or melphalan IV over 20 minutes on days -4 to -2, antithymocyte globulin (ATG) IV over 4 hours or methylprednisolone IV over 1 hour twice a day on days -3 to -1, and total lymphoid irradiation on day -1. On day 0, patients receive umbilical cord blood (UCB) infusion. Patients with inborn errors of metabolism or inherited hematopoietic disorders receive oral busulfan every 6 hours on days -9 to -6, cyclophosphamide IV over 1 hour on days -5 to -2 or melphalan IV over 20 minutes on days -4 to -2, and ATG IV over 4 hours or methylprednisolone IV over 1 hour on days -3 to -1. On day 0, patients receive UCB infusion. Patients with Fanconi's anemia receive ATG IV over 4 hours or methylprednisolone IV over 1 hour on days -6 to -1, cyclophosphamide IV over 1 hour on days -5 to -2, thoracoabdominal irradiation on day -1, and then the UCB infusion on day 0. Patients also receive cyclosporine and methylprednisolone beginning on day -2 and continuing as necessary as graft-versus-host disease prophylaxis. Patients are followed indefinitely for survival and late toxicity.
PROJECTED ACCRUAL: A total of 4-90 patients will be accrued for this study within 5 years.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 90
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (15)
Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
St. Christopher's Hospital for Children
🇺🇸Philadelphia, Pennsylvania, United States
University of Florida Health Science Center
🇺🇸Gainesville, Florida, United States
Division of Pediatric Surgery
🇺🇸Jacksonville, Florida, United States
Lineberger Comprehensive Cancer Center, UNC
🇺🇸Chapel Hill, North Carolina, United States
Children's Hospital of New Orleans
🇺🇸New Orleans, Louisiana, United States
Cardinal Glennon Children's Hospital
🇺🇸Saint Louis, Missouri, United States
New York Blood Center
🇺🇸New York, New York, United States
H. Lee Moffitt Cancer Center and Research Institute
🇺🇸Tampa, Florida, United States
Rush-Presbyterian-St. Luke's Medical Center
🇺🇸Chicago, Illinois, United States
University of Chicago Cancer Research Center
🇺🇸Chicago, Illinois, United States
Duke Comprehensive Cancer Center
🇺🇸Durham, North Carolina, United States
Roswell Park Cancer Institute
🇺🇸Buffalo, New York, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
University of South Carolina School of Medicine
🇺🇸Columbia, South Carolina, United States