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.
| Shawna McIntyre, RN | |
| 412-692-5552 | |
| mcintyresm@upmc.edu |
| Paul Szabolcs, MD | |
| 412-692-5427 | |
| paul.szabolcs@chp.edu |
1 United States site
2 Months - 60 Years
Observational
All
Drug
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.
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