Literature DB >> 28658866

Unusual Presentation of Atypical Infantile Pompe Disease in the Newborn Period with Left Ventricular Hypertrophy.

Sanjay Kumar1, Amit Kumar2.   

Abstract

Pompe disease, also known as glycogen storage disease Type II, is a lysosomal storage disorder caused by α-glucosidase deficiency. In general, the clinical spectrum varies with respect to the age of onset, residual enzyme activity and organ involvement. Infantile onset disease has two subtypes: classical and non-classical (atypical). This case report describes the case of a newborn who presented with generalized hypotonia and elevated serum enzyme levels of aspartate aminotransferase 93 IU/L, lactate dehydrogenase 888 IU/L and creatine kinase 670 μg/L. The electrocardiogram showed short PR interval with large QRS complexes with echocardiography suggesting evidence of left ventricular hypertrophy with infiltration in its walls. On the basis of the clinical signs and laboratory results, dried blood spots from the baby were tested to determine the acid α-glucosidase (GAA) activity, and the result confirmed that the GAA activity was only 1.42 units, normal range 5.5 to 29.6 units, leading to a diagnosis of Pompe disease (atypical infantile). Recognizing this disease and initiating enzyme replacement therapy in infants at the earliest can improve the quality of life of patients.

Entities:  

Keywords:  Enzyme replacement therapy; Lysosomes; Non-classical (atypical)

Year:  2017        PMID: 28658866      PMCID: PMC5483768          DOI: 10.7860/JCDR/2017/20756.9849

Source DB:  PubMed          Journal:  J Clin Diagn Res        ISSN: 0973-709X


  5 in total

1.  Identification of two subtypes of infantile acid maltase deficiency.

Authors:  A E Slonim; L Bulone; S Ritz; T Goldberg; A Chen; F Martiniuk
Journal:  J Pediatr       Date:  2000-08       Impact factor: 4.406

Review 2.  Phenotype variations in early onset Pompe disease: diagnosis and treatment results with Myozyme.

Authors:  Samuel Ignacio Pascual Pascual
Journal:  Adv Exp Med Biol       Date:  2009       Impact factor: 2.622

Review 3.  Pompe disease in infants and children.

Authors:  Priya Sunil Kishnani; R Rodney Howell
Journal:  J Pediatr       Date:  2004-05       Impact factor: 4.406

4.  Pompe disease diagnosis and management guideline.

Authors:  Priya S Kishnani; Robert D Steiner; Deeksha Bali; Kenneth Berger; Barry J Byrne; Laura E Case; Laura Case; John F Crowley; Steven Downs; R Rodney Howell; Richard M Kravitz; Joanne Mackey; Deborah Marsden; Anna Maria Martins; David S Millington; Marc Nicolino; Gwen O'Grady; Marc C Patterson; David M Rapoport; Alfred Slonim; Carolyn T Spencer; Cynthia J Tifft; Michael S Watson
Journal:  Genet Med       Date:  2006-05       Impact factor: 8.822

5.  Clinical features of Pompe disease.

Authors:  Fiore Manganelli; Lucia Ruggiero
Journal:  Acta Myol       Date:  2013-10
  5 in total
  1 in total

1.  A Newborn with Infantile-Onset Pompe Disease Improving after Administration of Enzyme Replacement Therapy: Case Report.

Authors:  Meltem Bor; Ozkan Ilhan; Evren Gumus; Solmaz Ozkan; Meryem Karaca
Journal:  J Pediatr Intensive Care       Date:  2020-07-15
  1 in total

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