Unrelated Donor Transplant for 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