Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Inclusion 47

• Minimum age: 0.17 years
• Maximum age: 60 years
• Study condition: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
• Patient, parent, or legal guardian must have given written informed consent.
• Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
• Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to the following:
• A. Primary Immunodeficiency Syndromes
• 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)
• Hyper IgM (XHIM) syndrome
• IPEX syndrome
• Chediak-Higashi Syndrome
• Autoimmune Lymphoproliferative Syndrome (ALPS)
• Hemophagocytic Lymphohistiocytosis (HLH) syndromes
• Lymphocyte Signaling defects
• B. Congenital Bone Marrow Failure Syndromes
• Congenital Amegakaryocytic Thrombocytopenia (CAMT)
• Osteopetrosis
• C. Inherited Metabolic Disorders (IMD)
• 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)
• Other inherited metabolic disorders
• Alpha Mannosidosis
• Gaucher Disease
• Other inheritable metabolic diseases where HSCT may be beneficial
• D. Hereditary Anemias
• Thalassemia major
• Sickle cell disease (SCD)
• Diamond Blackfan Anemia (DBA)
• E. Inflammatory Conditions
• Crohn's Disease or Inflammatory Bowel Disease
• IPEX or IPEX-like Syndromes
• Rheumatoid Arthritis
• Other inflammatory conditions where HSCT may be beneficial
• Subjects receive either umbilical cord blood, bone marrow, or peripheral blood stem cell transplant with an alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen, according to clinical practice at UPMC Children's Hospital of Pittsburgh.
• There are no exclusion criteria.