Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Criteria the registry did not label

• Inclusion:
• A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x 10e7 cells/kg, or double unit grafts, each cord blood unit delivering at least 2 x 10e7 cells/kg OR an 8 of 8 or 7 of 8 HLA allele level matched unrelated donor bone marrow or peripheral blood progenitor graft.
• Adequate organ function as measured by:
  • Creatinine ≤ 2.0 mg/dL and creatinine clearance ≥ 50 mL/min/1.73 m2.
  • Hepatic transaminases (ALT/AST) ≤ 4 x upper limit of normal (ULN).
  • Adequate cardiac function by echocardiogram or radionuclide scan (shortening fraction \> 26% or ejection fraction \> 40% or \> 80% of normal value for age).
  • Pulmonary evaluation testing demonstrating CVC or FEV1/FVC of ≥ 50% of predicted for age and/or resting pulse oximeter ≥ 92% on room air or clearance by the pediatric or adult pulmonologist. For adult patients DLCO (corrected for hemoglobin) should be ≥ 50% of predicted if the DLCO can be obtained.
• Written informed consent and/or assent according to FDA guidelines.
• Negative pregnancy test if pubertal and/or menstruating.
• HIV negative.
• A non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to:
  • Primary Immunodeficiency syndromes including but not limited to:
  • Severe Combined Immune Deficiency (SCID) with NK cell activity
  • Omenn Syndrome
  • Bare Lymphocyte Syndrome (BLS)
  • Combined Immune Deficiency (CID) syndromes
  • Combined Variable Immune Deficiency (CVID) syndrome
  • Wiskott-Aldrich Syndrome
  • Leukocyte adhesion deficiency
  • Chronic granulomatous disease (CGD)
  • X-linked Hyper IgM (XHIM) syndrome
  • IPEX syndrome
  • Chediak - Higashi Syndrome
  • Autoimmune Lymphoproliferative Syndrome (ALPS)
  • Hemophagocytic Lymphohistiocytosis (HLH) syndromes
  • Lymphocyte Signaling defects
  • Other primary immune defects where hematopoietic stem cell transplantation may be beneficial
  • Congenital bone marrow failure syndromes including but not limited to:
  • Dyskeratosis Congenita (DC)
  • Congenital Amegakaryocytic Thrombocytopenia (CAMT)
  • Osteopetrosis
  • Inherited Metabolic Disorders (IMD) including but not limited to:
  • Mucopolysaccharidoses
  • Hurler syndrome (MPS I)
  • Hunter syndrome (MPS II)
  • Leukodystrophies
  • Krabbe Disease, also known as globoid cell leukodystrophy
  • Metachromatic leukodystrophy (MLD)
  • X-linked adrenoleukodystrophy (ALD)
  • Hereditary diffuse leukoencephalopathy with spheroids (HDLS)
  • Other inherited metabolic disorders
  • alpha mannosidosis
  • Gaucher Disease
  • Other inheritable metabolic diseases where hematopoietic stem cell transplantation may be beneficial.
  • Hereditary anemias
  • Thalassemia major
  • Sickle cell disease (SCD) - patients with sickle disease must have one or more of the following:
  • Overt or silent stroke
  • Pain crises ≥ 2 episodes per year for past year
  • One or more episodes of acute chest syndrome
  • Osteonecrosis involving ≥ 1 joints
  • Priapism
  • Diamond Blackfan Anemia (DBA)
  • Other congenital transfusion dependent anemias
  • Inflammatory Conditions
  • Crohn's Disease/Inflammatory Bowel Disease
  • Exclusion:
• Allogeneic hematopoietic stem cell transplant within the previous 6 months.
• Any active malignancy or MDS.
• Severe acquired aplastic anemia.
• Uncontrolled bacterial, viral or fungal infection (currently taking medication and with progression of clinical symptoms).
• Pregnancy or nursing mother.
• Poorly controlled pulmonary hypertension.
• Any condition that precludes serial follow-up.