A Prospective Outcomes Study of Pediatric and Adult Patients with Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation with a Reduced-Intensity Conditioning Regimen (PRO-RIC)
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.
• 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.