[Show abstract][Hide abstract] ABSTRACT: Unlabelled:
Several lines of evidence support the involvement of inflammatory and immunologic abnormalities in chronic fatigue syndrome (CFS). Since recent studies have shown that α-1 antitrypsin (AAT) possesses anti-inflammatory properties, the potential therapeutic effect of AAT treatment on CFS has been investigated.
A 49-year-old woman diagnosed with CFS was treated with intravenous infusions of a human plasma-derived AAT concentrate (60 mg/kg body weight weekly for 8 consecutive weeks). The patient's monocyte elastase, a regulator of inflammatory processes, was 1170 U/mg. At completion of treatment, improvement in maximal workload was observed (54.0-71.7% of predicted). Additionally, amelioration in working memory (scores: 83-94) and perceptual organization (scores: 75-83) were detected on the Wechsler Adult Intelligence Scale-III test. Monocyte elastase decreased to a normal range (<150 U/mg). Improvement in functional capacity allowed the patient to work in part-time employment.
These findings suggest a possible role for AAT in the treatment of CFS.
[Show abstract][Hide abstract] ABSTRACT: To assess clinical effect of a human plasma-derived alpha-1 antitrypsin (AAT) concentrate in reducing pain severity of patients with fibromyalgia (FM).
Thirteen subjects with FM completed a randomized, double-blind, placebo-controlled, crossover study which consisted of 9 weeks trial of AAT or placebo with a washout period of 6 weeks. Primary efficacy endpoint was change on pain severity score, assessed by a daily visual analogue scale (VAS) for pain. Other outcome measures included a tender point score, the Fibromyalgia Impact Questionnaire, (FIQ), the Medical Outcomes Study Short Form 36 (SF-36), the Health Assessment Questionnaire Disability Index (HAQ-DI), the Hospital Anxiety and Depression Scale (HADS) and tiredness score evaluated by VAS.
No statistically significant differences were observed in either pain severity or other secondary outcome measures in either of the treatment groups, or between treatment groups in either of the treatment periods. No carryover or order of intervention effect was observed from one treatment to the other. Both investigational interventions were generally well tolerated, and vital signs during the drug infusions were within the respective normal ranges.
Treatment with a human plasma-derived AAT concentrate did not demonstrate significant improvement over placebo on reducing pain severity and other symptoms of FM. Further research should examine other FM subpopulations and drug doses.
No preview · Article · Sep 2012 · Musculoskeletal Care
[Show abstract][Hide abstract] ABSTRACT: Aim. In the perspective of postmarketing surveillance of human alpha-1 antitrypsin (AAT) products for intravenous administration, clinical studies following long-term augmentation therapy in significant cohorts of AAT-deficient subjects are needed. The main objective of this study was to assess tolerance of Trypsone®, a lyophilized and sterile preparation of AAT highly purified from human plasma, as chronic replacement therapy in AAT-deficient subjects with associated pulmonary emphysema. Furthermore, the safety of the study drug was evaluated as a secondary objective. Methods. This was a multicenter, prospective, non-controlled, observational clinical study with 23 individuals. Tolerance to the drug exposure was assessed by monitoring vital signs and considering adverse events (AEs) which took place during the AAT infusions. For safety evaluation, all AEs occurring to any participating subject were considered; from the first study drug exposure to the end of the prospective period of observation, regardless whether the event occurred during the infusions. Results. No clinically significant changes of vital signs were reported as AEs during the administration of the study drug. Only one, among 555 infusions, had an AE possibly related to the study drug. This AE consisted in a vasovagal reaction with arterial hypotension and dizziness which was classified as non-serious, of moderate intensity and expected. Regarding other AEs not occurring during the infusions, a total of 5 serious AEs were reported in 4 subjects but none of them were related to the study drug. Conclusion. Trypsone® represents a viable and safe treatment option for augmentation therapy with in AAT deficient individuals.
No preview · Article · Jun 2010 · Minerva pneumologica