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Efficacy of alpha1-antitrypsin augmentation therapy in conditions other than pulmonary emphysema.

Blanco I, Lara B, de Serres F - Orphanet J Rare Dis (2011)

Bottom Line: However, the compassionate use of augmentation therapy in recent years has proven outstanding efficacy in small cohorts of patients suffering from uncommon AAT deficiency-related diseases other than pulmonary emphysema, such as fibromyalgia, systemic vasculitis, relapsing panniculitis and bronchial asthma.Moreover, a series of preclinical studies provide evidence of the efficacy of AAT augmentation therapy in several infectious diseases, diabetes mellitus and organ transplant rejection.These facts have generated an expanding number of medical applications and patents with claims for other indications of AAT besides pulmonary emphysema.

View Article: PubMed Central - HTML - PubMed

Affiliation: Biomedical Research Office (OIB-FICYT), Rosal, 7. 33009 Oviedo, Principality of Asturias, Spain. ignablanco@yahoo.com

ABSTRACT
Up to now alpha 1-antitrypsin (AAT) augmentation therapy has been approved only for commercial use in selected adults with severe AAT deficiency-related pulmonary emphysema (i.e. PI*ZZ genotypes as well as combinations of Z, rare and alleles expressing AAT serum concentrations <11 μmol/L). However, the compassionate use of augmentation therapy in recent years has proven outstanding efficacy in small cohorts of patients suffering from uncommon AAT deficiency-related diseases other than pulmonary emphysema, such as fibromyalgia, systemic vasculitis, relapsing panniculitis and bronchial asthma. Moreover, a series of preclinical studies provide evidence of the efficacy of AAT augmentation therapy in several infectious diseases, diabetes mellitus and organ transplant rejection. These facts have generated an expanding number of medical applications and patents with claims for other indications of AAT besides pulmonary emphysema. The aim of the present study is to compile and analyze both clinical and histological features of the aforementioned published case studies and reports where AAT augmentation therapy was used for conditions other than pulmonary emphysema. Particularly, our research refers to ten case reports and two clinical trials on AAT augmentation therapy in patients with both AAT deficiency and, at least, one of the following diseases: fibromyalgia, vasculitis, panniculitis and bronchial asthma. In all the cases, AAT was successfully applied whereas previous maximal conventional therapies had failed. In conclusion, laboratory studies in animals and humans as well as larger clinical trials should be, thus, performed in order to determine both the strong clinical efficacy and security of AAT in the treatment of conditions other than pulmonary emphysema.

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Effects of a 6-month trial with alpha-1-antitrypsin augmentation therapy and placebo in a Fibromyalgia and Chronic Fatigue Syndrome patient with intermediate MZ alpha-1-antitrypsin deficiency. FIQ: Fibromyalgia Impact Questionnaire. HAQ: Health Assessment Questionnaire.
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Figure 4: Effects of a 6-month trial with alpha-1-antitrypsin augmentation therapy and placebo in a Fibromyalgia and Chronic Fatigue Syndrome patient with intermediate MZ alpha-1-antitrypsin deficiency. FIQ: Fibromyalgia Impact Questionnaire. HAQ: Health Assessment Questionnaire.

Mentions: Case 3 was reported in 2010 [69]. In 2004 a patient with both AAT deficiency (MZ) and severe fibromyalgia/CFS (Chronic Fatigue Syndrome) participated in a trial with AAT-intravenous augmentation therapy after the failure of conventional therapies. Since then, the patient has experienced a very good clinical response (Figure 4).


Efficacy of alpha1-antitrypsin augmentation therapy in conditions other than pulmonary emphysema.

Blanco I, Lara B, de Serres F - Orphanet J Rare Dis (2011)

Effects of a 6-month trial with alpha-1-antitrypsin augmentation therapy and placebo in a Fibromyalgia and Chronic Fatigue Syndrome patient with intermediate MZ alpha-1-antitrypsin deficiency. FIQ: Fibromyalgia Impact Questionnaire. HAQ: Health Assessment Questionnaire.
© Copyright Policy - open-access
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC3094201&req=5

Figure 4: Effects of a 6-month trial with alpha-1-antitrypsin augmentation therapy and placebo in a Fibromyalgia and Chronic Fatigue Syndrome patient with intermediate MZ alpha-1-antitrypsin deficiency. FIQ: Fibromyalgia Impact Questionnaire. HAQ: Health Assessment Questionnaire.
Mentions: Case 3 was reported in 2010 [69]. In 2004 a patient with both AAT deficiency (MZ) and severe fibromyalgia/CFS (Chronic Fatigue Syndrome) participated in a trial with AAT-intravenous augmentation therapy after the failure of conventional therapies. Since then, the patient has experienced a very good clinical response (Figure 4).

Bottom Line: However, the compassionate use of augmentation therapy in recent years has proven outstanding efficacy in small cohorts of patients suffering from uncommon AAT deficiency-related diseases other than pulmonary emphysema, such as fibromyalgia, systemic vasculitis, relapsing panniculitis and bronchial asthma.Moreover, a series of preclinical studies provide evidence of the efficacy of AAT augmentation therapy in several infectious diseases, diabetes mellitus and organ transplant rejection.These facts have generated an expanding number of medical applications and patents with claims for other indications of AAT besides pulmonary emphysema.

View Article: PubMed Central - HTML - PubMed

Affiliation: Biomedical Research Office (OIB-FICYT), Rosal, 7. 33009 Oviedo, Principality of Asturias, Spain. ignablanco@yahoo.com

ABSTRACT
Up to now alpha 1-antitrypsin (AAT) augmentation therapy has been approved only for commercial use in selected adults with severe AAT deficiency-related pulmonary emphysema (i.e. PI*ZZ genotypes as well as combinations of Z, rare and alleles expressing AAT serum concentrations <11 μmol/L). However, the compassionate use of augmentation therapy in recent years has proven outstanding efficacy in small cohorts of patients suffering from uncommon AAT deficiency-related diseases other than pulmonary emphysema, such as fibromyalgia, systemic vasculitis, relapsing panniculitis and bronchial asthma. Moreover, a series of preclinical studies provide evidence of the efficacy of AAT augmentation therapy in several infectious diseases, diabetes mellitus and organ transplant rejection. These facts have generated an expanding number of medical applications and patents with claims for other indications of AAT besides pulmonary emphysema. The aim of the present study is to compile and analyze both clinical and histological features of the aforementioned published case studies and reports where AAT augmentation therapy was used for conditions other than pulmonary emphysema. Particularly, our research refers to ten case reports and two clinical trials on AAT augmentation therapy in patients with both AAT deficiency and, at least, one of the following diseases: fibromyalgia, vasculitis, panniculitis and bronchial asthma. In all the cases, AAT was successfully applied whereas previous maximal conventional therapies had failed. In conclusion, laboratory studies in animals and humans as well as larger clinical trials should be, thus, performed in order to determine both the strong clinical efficacy and security of AAT in the treatment of conditions other than pulmonary emphysema.

Show MeSH
Related in: MedlinePlus