CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

This is an interventional treatment trial for Bone Marrow Failure Syndrome focused on measuring Unrelated donor transplant, Haploidentical donor transplant, Peripheral blood stem cell transplantation, CD34+ selection, Non-malignant disease, Bone marrow failure syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndrome, Acquired Immunodeficiency Syndrome, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia Read More

Inclusion Criteria:

• General Eligibility (All Patients)

  • Patient must be < or = 40 years of age. Patients with sickle cell anemia must be at least 2 years of age.
  • Patient or the patient's legally authorized guardian must be fully informed about their illness and the investigational nature of the study protocol (including foreseeable risks and possible side effects) and must sign an informed consent in accordance with the institutional policies approved by the U.S. Department of Health and Human Services.
  • Approval for the use of this treatment protocol by the individual institution's Human Rights Committee must be obtained, in accordance with the institutional assurance policies of the U. S. Department of Health and Human Services.

  • Human leukocyte antigen (HLA) typing will be performed by high-resolution molecular DNA typing for HLA Class I A, B, and C and HLA Class II DRB1 and DQB1 alleles.

    • Unrelated donor: An 8/10, 9/10 or 10/10 matched unrelated adult donor (MUD) will be required for study entry.
    • Related Donor: A 5/10, 6/10, 7/10, 8/10, 9/10 or 10/10 matched (or partially matched) family donor will be required for study entry.
  • Non-malignant Disorders per protocol.
  • Hemoglobinopathies per protocol.
  • Requirement for CD34+ stem cell selection for a second infusion of stem cells following an allogeneic stem cell transplant from a related or unrelated adult donor.

• Additional eligibility for patients with non-malignant disorders receiving myeloablative conditioning

  • Adequate renal function as determined by the institutional normal range.
  • Adequate liver function per protocol.
  • Adequate cardiac function defined by radionucleotide angiogram or echocardiogram.
  • Adequate pulmonary function by pulmonary function test. For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry >94% on room air.

• Additional eligibility for patients with non-malignant disorders receiving reduced intensity conditioning

  • Adequate renal function as determined by the institutional normal range.
  • Adequate liver function per protocol.
  • Adequate cardiac function per protocol.
  • Adequate pulmonary function per protocol.

Exclusion Criteria:

• Patients with documented uncontrolled infection at the time of study entry are not eligible.

• Pregnancy/Breast-Feeding Females who are pregnant or breast feeding at the time of study entry are not eligible.

  -- The following additional exclusion criteria for patients with sickle cell anemia the following exclusion criteria also apply

• Patients with bridging fibrosis or cirrhosis of the liver.
• Uncontrolled bacterial, viral or fungal infection in the past month.
• Seropositivity for HIV.
• Patients who have received prior hematocrit (HCT) within three months of enrollment for reduced intensity regimen and within six months for myeloablative regimen/reduced toxicity regimens.