Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

The hypothesis of this study is that children with severe primary immunodeficiencies will benefit from early stem cell transplantation utilizing a reduced intensity conditioning regimen. This regimen is associated with a low risk of complications and will lead to correction of the underlying immunological defects.

Inclusion Criteria:

• Must show one of the following diseases:

  • SCIDS
  • Hyper-IgM
  • Wiskott-Aldrich Syndrome
  • Chediak-Higashi and Griscelli Syndromes
  • X-Linked Lymphoproliferative Diseases
  • IPEX Syndrome
  • NEMO Syndrome
  • other severe immunodeficiency diseases not stated above at the discretion of the Principal Investigator

• Informed Consent

• Adequate Renal Function

• Adequate Liver Function

• Adequate Cardiac Function

• Adequate Pulmonary Function

• Adequate Performance Statue

• Adequate Venous Access

Exclusion Criteria:

• Patient/Family has not signed informed consent
• Patient does not have a clear diagnosis of a severe immunodeficiency disease
• A suitable donor for the patient cannot be found
• Patient is HIV positive
• Patient has active Hepatitis B
• Patient is pregnant
• Patient is considered unsuitable for transplant at the discretion of the Principal Investigators or the medical director