Unrelated Donor Transplant for Malignant and Non-Malignant Disorders

This is an interventional treatment trial for Leukemia focused on measuring Allogeneic Stem Cell Transplant, Unrelated donor, Cord blood donor Read More

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