RecruitingNCT00006150

Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)


Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Enrollment

600 participants

Start Date

Aug 10, 2000

Study Type

OBSERVATIONAL

Conditions

Summary

The Hyper IgE Syndromes (HIES) are primary immunodeficiencies resulting in eczema and recurrent skin and lung infections. Autosomal dominant Hyper IgE syndrome (AD-HIIES; Job's syndrome) is caused by STAT3 mutations, and is a multi-system disorder with skeletal, vascular, and connective tissue manifestations. Understanding how STAT3 mutations cause these diverse clinical manifestations is critical to our complete understanding of bone metabolism, bronchiectasis, dental maturation, and atherosclerosis. Bi-allelic mutations in DOCK8 cause a combined immunodeficiency previously described as autosomal-recessive Hyper IgE syndrome. These individuals suffer from extensive viral infections as well as have a high incidence of malignancy and mortality. The pathogenesis of this disease and long-term natural history is being investigated. Therefore, we seek to enroll patients and families with a confirmed or suspected diagnosis of HIES syndrome for extensive phenotypic and genotypic study as well as disease management. Patients will be carefully examined by a multidisciplinary team and followed longitudinally. Through these studies we hope to better characterize the clinical presentation of STAT3-mutated HIES, DOCK8 deficiency and other causes of the hyper IgE phenotype, and to be able to identify further genetic etiologies, as well as understand the pathogenesis of HIES. We seek to enroll 300 patients and 300 relatives.


Eligibility

Min Age: 1 MonthMax Age: 120 Years

Inclusion Criteria7

  • Patients may be included in this study who:
  • Were referred to the NIH with a diagnosis or a suspicion of Hyper IgE syndrome.
  • Are patients referred for other immune syndromes that demonstrate some of the characteristics of HIES.
  • \>=1 month for affected subjects
  • Aged \>=2 years for unaffected subjects
  • For unaffected subjects, are able to understand and have the willingness to sign a written informed consent document.
  • Unaffected biological relatives of HIES patients are also eligible to enroll in a separate relative cohort.

Exclusion Criteria2

  • Coronary CTA will not be performed on any patient younger than 30 years or with contraindication to IV contrast media. This includes patients with 1) creatinine value of \>1.3 mg/dL, 2) history of multiple myeloma, 3) Use of metformin-containing products less than 24 hours prior to contrast media, and 4) history of significant allergic reaction to CT contrast agents despite the use of premedication.
  • Subjects with a medical, psychiatric, or social condition which, in the opinion of the investigator, would place undue burden on the subject, NIH resources, or increase risk of participation, may be excluded.

Locations(1)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

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NCT00006150


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