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Bone Marrow Transplantation in Treating Children With Sickle Cell Disease
This study is currently recruiting patients.
Sponsored by: | Fred Hutchinson Cancer Research Center |
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Information provided by: | Office of Rare Diseases (ORD) |
Purpose
RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ damage. Bone marrow transplantation, is a procedure in which the soft, sponge-like tissue in the center of bones producing white blood cells, red blood cells, and platelets is replaced by bone marrow from a another person. Bone marrow transplantation may be an effective treatment in relieving the symptoms of sickle cell disease.
PURPOSE: Phase I/II trial to study the effectiveness of bone marrow transplantation in treating children who have sickle cell disease.
Condition | Treatment or Intervention | Phase |
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Sickle Cell Anemia |
Drug: cyclosporine Drug: fludarabine Drug: mycophenolate mofetil Procedure: Bone Marrow Transplantation |
Phase I Phase II |
MedlinePlus related topics: Sickle Cell Anemia
Genetics Home Reference related topics: sickle cell anemia
Study Type: Interventional
Study Design: Treatment
Official Title: Phase I/II Study of Induction of Stable Mixed Chimerism after Bone Marrow Transplantation from HLA-Identical Donors in Children With Sickle Cell Disease
Expected Total Enrollment: 50
Study start: December 1999
PROTOCOL OUTLINE: This is a multicenter study. Patients undergo total body irradiation on day 0, followed by allogeneic bone marrow transfusion. Patients also receive fludarabine IV daily and cyclosporine IV twice a day on days -1 to 1. Patients then receive oral cyclosporine on days 1-90, and oral mycophenolate mofetil twice a day on days 0-27. Patients are followed for 100 days, monthly for 6 months and then annually for 2 years.
Eligibility
Ages Eligible for Study: up to 16 Years, Genders Eligible for Study: Both
Criteria
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of sickle cell anemia with clinically severe disease manifestations defined by: Recurrent painful events (at least 2 painful events in past year) which cannot be explained by other causes Pain lasts at least 4 hours Requires treatment with parenteral narcotics, equianalgesic dose of oral narcotics, or parenteral nonsteroidal antiinflammatory drugs Acute chest syndrome (ACS) with at least 2 episodes within past 2 years that required hospitalization, oxygen, and RBC transfusion Any combination of painful events and ACS episodes that total 2 events within the past year Abnormal cerebral MRI, abnormal angiography (MR or conventional), and abnormal neuropsychologic testing performance
No stage III or IV sickle cell lung disease
Genotypically HLA identical sibling donor available
--Prior/Concurrent Therapy--
No prior transfusions with greater than 5 units RBC
--Patient Characteristics--
Performance status: Karnofsky 70-100%
Hepatic:
Renal: Glomerular filtration rate at least 30% predicted for age
Neurologic:
Other:
Location and Contact Information
More Information
U.S. National Library of Medicine, Contact NLM Customer Service | ||||||||||||||
National Institutes of Health, Department of Health & Human Services | ||||||||||||||
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