Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases

OBJECTIVES:

Primary

- Determine the impact of the use of umbilical cord blood as a source of hematopoietic
stem cells for children with life-threatening oncologic, hematologic, or
genetic/metabolic disorders in need of a stem cell transplant.

- Compare the incidence of graft-versus-host disease in patients receiving cord blood
transplants in this study with historical data for unrelated donor stem cell
transplants.

- Compare the incidence of engraftment in patients receiving cord blood transplants in
this study with historical data for unrelated donor stem cell transplants.

OUTLINE:

- Preparative therapy: Patients are treated on 1 of 4 preparative therapy regimens.

- Regimen A: Patients undergo total body irradiation (TBI) two times daily on days
-7 to -4. Patients receive cyclophosphamide IV over 30-60 minutes on days -3 and
-2 and anti-thymocyte globulin (ATG) IV over at least 6 hours on days -3 to -1.

- Regimen B (patients who do not receive TBI): Patients receive oral busulfan 4
times daily on days -8 to -5, and ATG IV over at least 6 hours and melphalan IV
over 15-20 minutes on days -4 to -2.

- Regimen C (patients with Fanconi's anemia and related disorders): Patients undergo
TBI on day -6. Patients receive ATG IV over at least 6 hours and
methylprednisolone IV on days -5 to -1 and fludarabine IV over 30 minutes and
cyclophosphamide IV over 30-60 minutes on days -5 to -2.

- Regimen D: Patients receive oral or IV busulfan 4 times daily on days -9 to -5,
ATG IV over at least 6 hours on days -5 to -3, and cyclophosphamide IV over 30-60
minutes on days -5 to -2.

- Cord blood transplant: All patients undergo umbilical cord blood transplantation on day
0.

- Graft-versus-host disease prophylaxis: Patients receive oral or IV cyclosporine twice
daily beginning on day -1. Patients also receive methylprednisolone IV twice daily
beginning on day 5 and continuing until at least day 28.

PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study.

DISEASE CHARACTERISTICS:

- Diagnosis of malignant or non-malignant disease, including but not limited to any of
the following:

- Acute myeloid leukemia or acute lymphoblastic leukemia (ALL) with resistant
disease beyond first clinical remission (CR)

- ALL in first CR at high-risk because of 1 of the following factors:

- Hypoploidy

- Pseudodiploidy with translocations t(9;22), t(4;11), or t(8;14)

- Elevated WBC at diagnosis as follows:

- > 100,000/mm^3 for patients 6-12 months of age

- > 50,000/mm^3 for patients 10-20 years of age

- > 20,000/mm^3 for patients 21 years of age

- Burkitt's lymphoma/leukemia

- Chronic myelogenous leukemia in first chronic phase or beyond

- Juvenile myelomonocytic leukemia

- Advanced stage or relapsed lymphoma

- Advanced stage or relapsed solid tumors, including any of the following:

- Neuroblastoma

- Ewing's sarcoma

- Rhabdomyosarcoma

- Myelodysplastic syndromes, excluding patients with grade 3 or 4 myelofibrosis

- Familial erythrophagocytic histiocytosis

- Histiocytosis unresponsive to medical management

- Inborn errors of metabolism

- Langerhans cell histiocytosis unresponsive to medical management

- Immune deficiencies, including:

- Severe combined immune deficiency

- Wiskott-Aldrich

- Hemoglobinopathies, including sickle cell disease and thalassemia

- Severe aplastic anemia

- Fanconi's anemia

- Metabolic storage diseases

- Unrelated cord blood donor must be HLA-identical OR may be mismatched for 1, 2, or 3
HLA-loci (A, B, DR)

- No other existing HLA-identical related donor available at the time of
transplantation

PATIENT CHARACTERISTICS:

Age

- 21 and under

Performance status

- Not specified

Life expectancy

- Not specified

Hematopoietic

- See Disease Characteristics

Hepatic

- Not specified

Renal

- Not specified

PRIOR CONCURRENT THERAPY:

Biologic therapy

- Not specified

Chemotherapy

- Not specified

Endocrine therapy

- Not specified

Radiotherapy

- Not specified

Surgery

- Not specified