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

Egyéb feltételek

• 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