Alpha‐1‐antitrypsin deficiency associated with panniculitis treated with plasma exchange therapy

Oliveira, Priscila De; Paz‐Melgar, Libania; Takahashi, Marcelo Straus; Nico, Marcello Menta Simonsen; Rivitti, Evandro A.; Mendrone, Alfredo; Tatsui, Nelson Hidekazu

doi:10.1111/j.1365-4632.2004.02054.x

Cited by 11 publications

(14 citation statements)

References 25 publications

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“…In the same context, de Oliveira et al (São Paulo, Brazil) published in 2004 the case of a 23-year-old patient with extensive, ulcerative relapsing panniculitis, and AAT serum levels of 22 mg/dL and phenotype ZZ [73]. The patient was treated with 16 sessions of plasma exchange therapy, with cutaneous lesions clinically controlled after five sessions.…”

Section: Resultsmentioning

confidence: 99%

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

Blanco

Lara

Serres

2011

Orphanet J Rare Dis

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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 null 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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Section: Resultsmentioning

confidence: 99%

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

Blanco

Lara

Serres

2011

Orphanet J Rare Dis

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“…Two Spanish sisters with severe ZZ AAT deficiency and fibromyalgia (FM) experienced a rapid, progressive, and constant control of their FM symptoms during AAT replacement therapy [127]. Another report shows that ZZ AAT deficiency 21-years old woman with septal panniculitis poorly responded to dapsone and doxycycline treatment, but was successfully treated with intravenous infusion of AAT [128]. Recently Chowdhury et al have described 33-year-old woman with rapidly progressing panniculitis and extensive skin necrosis severe caused by ZZ AAT deficiency.…”

Section: -Antitrypsin Replacement Therapymentioning

confidence: 99%

New Insights into the Biology of 1-Antitrypsin and its Role in Chronic Obstructive Pulmonary Disease

Janciauskiene¹,

Stevens²,

Blanco³

2007

CRMR

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1-antitrypsin, an acute phase protein, is the prototypic member of the serpin super family and a major inhibitor of serine proteases. As an acute phase protein, 1-antitrypsin is thought to play an important role in limiting host tissue injury at sites of inflammation. The clinical importance of 1-antitrypsin is highlighted in individuals with inherited 1-antitrypsin deficiency who exhibit an increased susceptibility to develop chronic inflammatory conditions including chronic obstructive pulmonary disease, systemic vasculitis and necrotizing panniculitis. There is now an increasing evidence that 1-antitrypsin may also exhibit biological activity independent of its protease inhibitor function. Thus, conformationally modified and degraded forms of 1-antitrypsin, which lack antiprotease activity, demonstrate specific biological effects in vitro and in vivo and highlight the potentially broader modulatory role of 1-antitrypsin in inflammatory diseases.In this review we discuss the biological properties of 1-antitrypsin and its role in chronic obstructive pulmonary diseases. A more comprehensive understanding of the biology of native 1-antitrypsin and its modified forms may have a significant impact on our understanding and ultimately treatment of disease pathologies arising from both natural point mutations and from post-synthetically modified byproducts of 1-antitrypsin.

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“…Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder usually associated with hepatic and pulmonary manifestations. [ 1 2 ] In rare and severe situations, a panniculitis may be present. [ 1 2 ] It may present before the onset of other systemic manifestations giving a clue to an earlier diagnosis and adequate monitoring.…”

mentioning

confidence: 99%

“…[ 1 2 ] In rare and severe situations, a panniculitis may be present. [ 1 2 ] It may present before the onset of other systemic manifestations giving a clue to an earlier diagnosis and adequate monitoring. [ 3 ]…”

mentioning

confidence: 99%

Cutaneous manifestation of Alpha-1 antitrypsin deficiency: A case of panniculitis

et al. 2018

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Alpha‐1‐antitrypsin deficiency associated with panniculitis treated with plasma exchange therapy

Abstract: Plasma exchange therapy represents an alternative treatment which restores serum and tissue alpha-1-antitrypsin levels. This method is proposed because of its clinical benefits and greater availability.

Cited by 11 publications

References 25 publications

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

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

New Insights into the Biology of 1-Antitrypsin and its Role in Chronic Obstructive Pulmonary Disease

Cutaneous manifestation of Alpha-1 antitrypsin deficiency: A case of panniculitis

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