Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: Current status and critical needs Journal Article
| Authors: | Griffith, L. M.; Cowan, M. J.; Kohn, D. B.; Notarangelo, L. D.; Puck, J. M.; Schultz, K. R.; Buckley, R. H.; Eapen, M.; Kamani, N. R.; O'Reilly, R. J.; Parkman, R.; Roifman, C. M.; Sullivan, K. E.; Filipovich, A. H.; Fleisher, T. A.; Shearer, W. T. |
| Article Title: | Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: Current status and critical needs |
| Abstract: | Allogeneic hematopoietic cell transplantation (HCT) has been used for 40 years to ameliorate or cure primary immune deficiency (PID) diseases, including severe combined immunodeficiency (SCID) and non-SCID PID. There is a critical need for evaluation of the North American experience of different HCT approaches for these diseases to identify best practices and plan future investigative clinical trials. Our survey of incidence and prevalence of PID in North American practice sites indicates that such studies are feasible. A conference of experts in HCT treatment of PID has recommended (1) a comprehensive cross-sectional and retrospective analysis of HCT survivors with SCID; (2) a prospective study of patients with SCID receiving HCT, with comparable baseline and follow-up testing across participating centers; (3) a pilot study of newborn screening for SCID to identify affected infants before compromise by infection; and (4) studies of the natural history of disease in patients who do or do not receive HCT for the non-SCID diseases of Wiskott-Aldrich syndrome and chronic granulomatous disease. To accomplish these goals, collaboration by a consortium of institutions in North America is proposed. Participation of immunologists and HCT physicians having interest in PID and experts in laboratory methods, clinical outcomes assessment, databases, and analysis will be required for the success of these studies. © 2008 American Academy of Allergy, Asthma & Immunology. |
| Keywords: | disease-free survival; survival rate; transplantation, homologous; clinical trial; follow up; follow-up studies; clinical practice; quality of life; morbidity; prevalence; genotype; health survey; hematopoietic stem cell transplantation; infant, newborn; education; pilot projects; long term care; allogeneic hematopoietic stem cell transplantation; immune deficiency; databases, factual; newborn screening; severe combined immunodeficiency; immunologic deficiency syndromes; multicenter studies as topic; north america; allogeneic hematopoietic cell transplantation; wiskott aldrich syndrome; primary immune deficiency |
| Journal Title: | Journal of Allergy and Clinical Immunology |
| Volume: | 122 |
| Issue: | 6 |
| ISSN: | 0091-6749 |
| Publisher: | Mosby Elsevier |
| Date Published: | 2008-12-01 |
| Start Page: | 1087 |
| End Page: | 1096 |
| Language: | English |
| DOI: | 10.1016/j.jaci.2008.09.045 |
| PUBMED: | 18992926 |
| PROVIDER: | scopus |
| PMCID: | PMC3357108 |
| DOI/URL: | |
| Notes: | --- - "Cited By (since 1996): 16" - "Export Date: 17 November 2011" - "CODEN: JACIB" - "Source: Scopus" |
