Stem-cell transplantation for inherited immunodeficiency disorders.
Journal Article (Journal Article;Review)
For patients with well-characterized, rapidly fatal, nonmalignant immunodeficiency disorders, such as SCID, the decision to proceed with allogeneic SCT is clear-cut. For patients with many other disorders, this decision can be extremely difficult. Disorders such as LAD or CGD have a variable natural history. Each patient must be considered individually, with the risk for SCT-related morbidity and mortality carefully weighed against that of the underlying disease. Significant advances during the past 10 years have made SCT a much safer procedure. Use of nonmyeloablative conditioning regimens as a means of reducing toxicity of high-dose chemotherapy and irradiation hold great promise. Highly immunosuppressive, nonchemotherapeutic agents that inhibit graft rejection or GVHD by blocking the critical costimulatory component of the T-cell receptor-antigen interaction are beginning to emerge and may be ideal for SCT of nonmalignant diseases. Therefore, the risk-benefit equation must be reassessed each year as the severity of patients' disorders is better defined and techniques of SCT improve.
Full Text
Duke Authors
Cited Authors
- Horwitz, ME
Published Date
- December 2000
Published In
Volume / Issue
- 47 / 6
Start / End Page
- 1371 - 1387
PubMed ID
- 11131001
International Standard Serial Number (ISSN)
- 0031-3955
Digital Object Identifier (DOI)
- 10.1016/s0031-3955(05)70276-5
Language
- eng
Conference Location
- United States