Unrelated Donor Stem Cell Transplant for Patients With Malignant and Non-Malignant Disorders
Inclusion Criteria:
- Adequate renal function defined as: serum creatinine 2.0 x normal, or creatinine
clearance or radioisotope GFR > 40 ml/min/m2 or > 40 ml/min/1.73 m2 or an equivalent
GFR as determined by the institutional normal range.
- Adequate liver function defined as: total bilirubin < 2.5 x normal; or SGOT (AST) or
SGPT (ALT) < 5.0 x normal.
- Adequate cardiac function defined as: shortening fraction of > 25% by echocardiogram,
or ejection fraction of > 40% by radionuclide angiogram or echocardiogram.
- Adequate pulmonary function defined as: DLCO > 35% by pulmonary function test. For
children who are uncooperative, no evidence of dyspnea at rest, no exercise
intolerance, and a pulse oximetry > 94% in room air.
- Diseases:
- CML (CP, AP or BC)
- AML/MDS/JCML
- ALL
- Lymphoma (Hodgkin's and non-Hodgkin's)
- Non-malignant disorders
- Bone Marrow Failure Syndromes: Patients with the following diagnoses are eligible:
- Severe Aplastic Anemia:
- Fanconi Anemia
- Severe Congenital Neutropenia (Kostmann's Syndrome)
- Amegakaryocytic Thrombocytopenia
- Diamond-Blackfan Anemia
- Infantile Osteopetrosis
- Schwachman-Diamond Syndrome
- Dyskeratosis Congenita
- Other bone marrow failure syndromes at discretion of Principal Investigator
- Immunodeficiencies:
- SCIDS, all subtypes
- Combined Immunodeficiency Syndrome
- Wiskott-Aldrich syndrome
- Chronic Granulomatous Disease
- Chediak-Higashi Syndrome
- Leukocyte Adhesion Deficiency
- Other immunodeficiencies at discretion of Principal Investigator
- Inborn Errors of Metabolism (IEOM):
- Transplant is recommended for the following disorders: Hurler syndrome
(L-iduronidase deficiency, MPS-I), Maroteaux-Lamy syndrome
(galactosamine-4-sulfatase deficiency, MP VI), Sly syndrome (glucuronidase
deficiency, MPS-VII), Globoid cell Leukodystrophy
(galactocerebrosidasedeficiency), Metachromatic leukodystrophy (arylsulfatase A
deficiency), Childhood-onset X-linked adrenoleukodystrophy (X-ALD), Fucosidosis
(fucosidase deficiency), Mannosidosis, Aspartylglucosaminuria, Niemann-Pick
Disease Type B (acid sphingomyelinase deficiency), Gaucher disease
(glucocerebrosidase deficiency) Type I (non neuropathic), Other diagnoses may be
considered at the discretion of the Principal Investigator
- For X-ALD patients greater than 5 years of age, IQ > 80 is required. For other
patients greater than 5 years of age, IQ > 70 is required.
- For patients less than 5 years of age, the developmental quotient or clinical
neurodevelopmental examination should demonstrate potential for stabilization at
a level of functioning where continuous life support (e.g. mechanical
ventilation) would not be predicted to be required in the year following
transplantation.
- Histiocytosis:
- Hemophagocytic Lymphohistiocytosis (HLH)
- Familial Erythrophagocytic Lymphohistiocytosis
- Langerhans Cell Histiocytosis
- Malignant Histiocytosis
- Other Malignant and non-malignant diseases: Other malignant and non-malignant
diseases not listed above may be eligible if deemed appropriate by the Principal
Investigator.
Exclusion Criteria:
- Women who are pregnant and/or breast feeding are ineligible