Hershfield M S, Chaffee S, Sorensen R U
Department of Medicine, Duke University Medical Center, Durham, North Carolina 27710.
Pediatr Res. 1993 Jan;33(1 Suppl):S42-7; discussion S47-8. doi: 10.1203/00006450-199305001-00236.
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, these patients have entered a trial of gene therapy, but continue to receive enzyme replacement.
在过去6年中，29例腺苷脱氨酶（ADA）缺乏所致联合免疫缺陷患者接受了聚乙二醇（PEG）修饰的牛ADA（PEG-ADA）治疗。我们与北美、欧洲及澳大利亚的免疫学家和临床医生合作，监测了这些患者的血浆ADA活性、治疗的代谢效应以及抗PEG-ADA抗体的演变情况，他们则监测了免疫功能和治疗的临床反应。本文总结了PEG-ADA治疗的现状并给出了使用建议。文中展示了3例患者在接受PEG-ADA治疗期间特异性免疫功能的恢复情况，这3例患者分别代表了恢复较早、较晚的情况，这些患者已进入基因治疗试验，但仍继续接受酶替代治疗。
