Neoplasms

All Conditions

Symptoms and General Pathology

Nutritional and Metabolic Diseases

Blood and Lymph Conditions

Immune System Diseases

Rare Diseases

Carcinoma

Neoplasm Metastasis

Metabolic Diseases

Syndrome

Recurrence

Leukemia

Hematologic Neoplasms

Anemia

Graft vs Host Disease

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Failure Disorders

Pancytopenia

Thymoma

Thymus Neoplasms

Hematologic Diseases

Anemia, Refractory

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Bone Marrow Diseases

Precancerous Conditions

Thoracic Neoplasms

Lymphatic Diseases

Homologous Wasting Disease

Sideroblastic Anemia Pyridoxine-refractory Autosomal Recessive

Antineoplastic Agents

Antirheumatic Agents

All Drugs and Chemicals

Anti-Infective Agents

Dermatologic Agents

Anti-Inflammatory Agents

Antiemetics

Neuroprotective Agents

Gastrointestinal Agents

Cyclophosphamide

Melphalan

Cyclosporine

Cyclosporins

Prednisolone

Busulfan

Methylprednisolone

Methylprednisolone Acetate

Methylprednisolone Hemisuccinate

Prednisolone acetate

Prednisolone hemisuccinate

Prednisolone phosphate

Antilymphocyte Serum

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

Enzyme Inhibitors

Clotrimazole

Miconazole

Antifungal Agents

Calcineurin Inhibitors

Glucocorticoids

Hormones

Hormone Antagonists

Protective Agents

anti-thymocyte globulin

busulfan

cyclophosphamide

cyclosporine

melphalan

methylprednisolone

umbilical cord blood transplantation

radiation therapy

Barbara Jean Bambach, MD

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.

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.

Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases

A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Adults and Children With Bone Marrow Failure Syndromes or Inherited Metabolic or Hematologic Diseases

National Cancer Institute (NCI)

Roswell Park Cancer Institute

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases	NCT00003662	Graft Versus Ho... Leukemia Myelodysplastic... Thymic Carcinom...	anti-thymocyte ... busulfan cyclophosphamid... cyclosporine melphalan methylprednisol... umbilical cord ... radiation thera...	-	Roswell Park Cancer Institute
Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases	NCT00003662	Graft Versus Ho... Leukemia Myelodysplastic... Thymic Carcinom...	anti-thymocyte ... busulfan cyclophosphamid... cyclosporine melphalan methylprednisol... umbilical cord ... radiation thera...	-	Roswell Park Cancer Institute

Spots Global Cancer Trial Database for noninvasive thymoma and thymic carcinoma