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| ENZYME REPLACEMENT THERAPY WITH POLYETHYLENE GLYCOL-ADENOSINE DEAMINASE IN ADENOSINE-DEAMINASE DEFICIENCY - OVERVIEW AND CASE-REPORTS OF 3 PATIENTS, INCLUDING 2 NOW RECEIVING GENE-THERAPY |
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| Author(s): HERSHFIELD MS, CHAFFEE S, SORENSEN RU |
| Source: PEDIATRIC RESEARCH Volume: 33 Issue: 1 Pages: S42-S48 Supplement: Suppl. S Published: JAN 1993 |
| Times Cited: 64 References: 19 |
| Abstract: During the past 6 y, 29 adenosine deaminase (ADA)-deficient patients with combined immunodeficiency have been treated with polyethylene glycol (PEG)-modified bovine ADA (PEG-ADA). We have monitored plasma ADA activity, metabolic effects of treatment, and the evolution of antibody to PEG-ADA in these patients, in collaboration with immunologists and clinicians in North America, Europe, and Australia, who have monitored immune function and clinical response to treatment. This article summarizes the current status of PEG-ADA therapy and provides recommendations for its use. Recovery of specific immune function during treatment with PEG-ADA is illustrated for three patients, who represent early, delayed, and late onset of immunodeficiency disease. Two of these patients have entered a trial of gene therapy, but continue to receive enzyme replacement. |
| Document Type: Proceedings Paper |
| Language: English |
| Reprint Address: HERSHFIELD, MS (reprint author), DUKE UNIV, MED CTR, DEPT MED, BOX 3049, ROOM 418, SANDS BLDG, DURHAM, NC 27710 USA |
Addresses:
1. DUKE UNIV, MED CTR, DEPT PEDIAT, DURHAM, NC 27710 USA
2. LOUISIANA STATE UNIV, MED CTR, DEPT PEDIAT, DIV ALLERGY & IMMUNOL, NEW ORLEANS, LA 70112 USA |
| Publisher: WILLIAMS & WILKINS, 351 WEST CAMDEN ST, BALTIMORE, MD 21201-2436 |
| Subject Category: Pediatrics |
| IDS Number: KF285 |
| ISSN: 0031-3998 |
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