Challenging Identification of a Novel PiISF and the Rare PiMmaltonZ α1-Antitrypsin Deficiency Variants in Two Patients

α1-Antitrypsin (AAT) deficiency is associated with an increased risk for lung and liver disease. Identification of AAT deficiency as the underlying cause of these diseases is important in correct patient management. AAT deficiency is commonly diagnosed by demonstrating low concentrations of AAT foll...

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Vydáno v:American journal of clinical pathology Ročník 141; číslo 5; s. 742 - 746
Hlavní autoři: Suh-Lailam, Brenda B., Procter, Melinda, Krautscheid, Patti, Haas, Jason, Kumar, Shiva, Mao, Rong, Grenache, David G.
Médium: Journal Article
Jazyk:angličtina
Vydáno: England 01.05.2014
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ISSN:0002-9173, 1943-7722, 1943-7722
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Abstract α1-Antitrypsin (AAT) deficiency is associated with an increased risk for lung and liver disease. Identification of AAT deficiency as the underlying cause of these diseases is important in correct patient management. AAT deficiency is commonly diagnosed by demonstrating low concentrations of AAT followed by genotype and/or phenotype testing. However, this algorithm may miss novel AAT phenotypes. We report two cases of AAT deficiency in two patients: a case of the novel phenotype PiISF, misclassified as PiII by phenotyping, and a case of the rare phenotype PiMmaltonZ misclassified as PiM2Z. These cases highlight the importance of understanding the limitations of a commonly used diagnostic algorithm, use of further gene sequencing in applicable cases, and the potential for underdiagnosis of AAT deficiency in patients with chronic obstructive pulmonary disease.
AbstractList α1-Antitrypsin (AAT) deficiency is associated with an increased risk for lung and liver disease. Identification of AAT deficiency as the underlying cause of these diseases is important in correct patient management. AAT deficiency is commonly diagnosed by demonstrating low concentrations of AAT followed by genotype and/or phenotype testing. However, this algorithm may miss novel AAT phenotypes. We report two cases of AAT deficiency in two patients: a case of the novel phenotype PiISF, misclassified as PiII by phenotyping, and a case of the rare phenotype PiMmaltonZ misclassified as PiM2Z. These cases highlight the importance of understanding the limitations of a commonly used diagnostic algorithm, use of further gene sequencing in applicable cases, and the potential for underdiagnosis of AAT deficiency in patients with chronic obstructive pulmonary disease.
α1-Antitrypsin (AAT) deficiency is associated with an increased risk for lung and liver disease. Identification of AAT deficiency as the underlying cause of these diseases is important in correct patient management.OBJECTIVESα1-Antitrypsin (AAT) deficiency is associated with an increased risk for lung and liver disease. Identification of AAT deficiency as the underlying cause of these diseases is important in correct patient management.AAT deficiency is commonly diagnosed by demonstrating low concentrations of AAT followed by genotype and/or phenotype testing. However, this algorithm may miss novel AAT phenotypes.METHODSAAT deficiency is commonly diagnosed by demonstrating low concentrations of AAT followed by genotype and/or phenotype testing. However, this algorithm may miss novel AAT phenotypes.We report two cases of AAT deficiency in two patients: a case of the novel phenotype PiISF, misclassified as PiII by phenotyping, and a case of the rare phenotype PiMmaltonZ misclassified as PiM2Z.RESULTSWe report two cases of AAT deficiency in two patients: a case of the novel phenotype PiISF, misclassified as PiII by phenotyping, and a case of the rare phenotype PiMmaltonZ misclassified as PiM2Z.These cases highlight the importance of understanding the limitations of a commonly used diagnostic algorithm, use of further gene sequencing in applicable cases, and the potential for underdiagnosis of AAT deficiency in patients with chronic obstructive pulmonary disease.CONCLUSIONSThese cases highlight the importance of understanding the limitations of a commonly used diagnostic algorithm, use of further gene sequencing in applicable cases, and the potential for underdiagnosis of AAT deficiency in patients with chronic obstructive pulmonary disease.
Author Suh-Lailam, Brenda B.
Procter, Melinda
Haas, Jason
Krautscheid, Patti
Kumar, Shiva
Grenache, David G.
Mao, Rong
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Issue 5
Keywords α1-Antitrypsin deficiency
Mmalton
Genotype
Phenotype
AAT
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  ident: key 20180602031927_b6-1410742
  article-title: A review of alpha1-antitrypsin deficiency
  publication-title: Am J Respir Crit Care Med
  doi: 10.1164/rccm.201108-1428CI
– volume: 33
  start-page: 354
  year: 1981
  ident: key 20180602031927_b12-1410742
  article-title: New variants of alpha 1-antitrypsin: comparison of Pi typing techniques
  publication-title: Am J Hum Genet
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Snippet α1-Antitrypsin (AAT) deficiency is associated with an increased risk for lung and liver disease. Identification of AAT deficiency as the underlying cause of...
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StartPage 742
SubjectTerms Adult
Algorithms
alpha 1-Antitrypsin - genetics
alpha 1-Antitrypsin Deficiency - diagnosis
alpha 1-Antitrypsin Deficiency - genetics
Female
Genotype
Humans
Male
Middle Aged
Phenotype
Pulmonary Disease, Chronic Obstructive - diagnosis
Pulmonary Disease, Chronic Obstructive - genetics
Risk
Title Challenging Identification of a Novel PiISF and the Rare PiMmaltonZ α1-Antitrypsin Deficiency Variants in Two Patients
URI https://www.ncbi.nlm.nih.gov/pubmed/24713750
https://www.proquest.com/docview/1514438385
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