Protocol for Investigation of Metabolic Disease in Acutely Ill Neonates and Young Children

Department of Chemical Pathology
The Chinese University of Hong Kong
Faculty of Medicine/
Prince of Wales Hospital
Shatin, N.T.

Appendix 1 Test Profiles in Selected Metabolic Disorders


                               Plasma/ Blood                Urine           

                            Glu   NH3    AA     pH  Ketones  Sug   AA   GLC   

Disease Group and Disease                      Na/K                               

1. Urea Cycle Defects                                                             

Carbamoylphosphate                 +      +                         +           
synthase (CPS 1)                                                                  
 deficiency                                                                       

Ornithine                          +      +                         +           
carbamoyltransferase                                                              
 deficiency                                                                       

Citrullinuria or                   +      D                         D           
citrullinaemia                                                                    
 Arginosuccinate synthase                                                         
deficiency                                                                        

Argininosuccinic                   +      D                         D           
aciduria, ASA-uria                                                                
 ASA-lyase deficiency                                                             

Hyperornithinaemia                 +      +                         +           

Lysinuric protein                  +                                +           
intolernace (LPI) with                                                            
 dibasic aminoaciduria                                                            

                               Plasma/ Blood                Urine           

                            Glu   NH3    AA     pH   Ketones  Sug   AA   GLC   

Disease Group and Disease                      Na/K                               

2. Amino Acid Defects                                                             

Maple syrup urine disease  (dec)          D     +       +           +     D    
(MSUD),                                                                           
 Branched chain                                                                   
ketoacidosis                                                                      

Non-ketotic                               +                         +           
hyperglycinaemia (NKHG)                                                           

Tyrosinaemia Type 1                       +                         +     D    
 (Fumarylacetoacetase                                                             
deficiency)                                                                       

Pyroglutamic aciduria                           +                         D    
(5-Oxoprolinuria)                                                                 

                               Plasma/ Blood                Urine           

                            Glu   NH3    AA     pH   Ketones  Sug   AA   GLC   

Disease Group and Disease                      Na/K                               

3. Organic acidurias                                                              

Isovaleric acidaemia               +      +     +       +           +     D    
("Sweaty foot                                                                     
 syndrome")                                                                       

-Methylcrotonyl-CoA                +      +     +       +           +     D    
carboxylase                                                                       
 deficiency                                                                       

-Hydroxy--methylglutaric   dec                  +       +                 D    
acidura                                                                           

-Ketothiolase deficiency           +      +     +       +                 D    

2-Methyl-3-hydroxy-butyric                                                        
 aciduria                                                                         

Methylmonic acidurias      dec     +      +     +       +           +     D    

Propionic acidaemia.       dec     +      +     +       +           +     D    
Propionyl-CoA                                                                     
 carboxylase deficiency                                                           

Glutaric aciduria.         dec                  +                         D    
Glutaryl-CoA                                                                      
 dehydrogenase deficiency                                                         

                               Plasma/ Blood                Urine           

                            Glu   NH3    AA     pH   Ketones  Sug   AA   GLC   

Disease Group and Disease                      Na/K                               

4. Endocrine disorders                                                            

Congenital adrenal                             NaK dec                               
hyperplasia                                                                       
 21-hydroxylase                                                                   
deficiency                                                                        

                               Plasma/ Blood                Urine           

                            Glu   NH3    AA     pH   Ketones  Sug   AA   GLC   

Disease Group and Disease                      Na/K                               

5. Carbohydrate disorders                                                         

Galactosaemia;            (dec)           +     +            U-Gal  +     +    
galactose-1-phosphate                                                             
 uridyl transferase                                                               
deficiency                                                                        

Hereditary fructose                       +     +            U-Fru  +     +    
intolerance                                                                       
 Fructose-1-PO4aldolase                                                           
deficiency                                                                        

Fructose-1,6               dec            +     +                   +  Lactate 
diphosphatase deficiency                                                          

Phosphoenolpyruvate        dec            +     +                   +  Lactate 
carboxy kinase                                                                    
 deficiency                                                                       

Pyruvate carboxylase       dec                  +                      Lactate 
deficiency                                                                        

Pyruvate dehydrogenase    (dec)                 +                      Lactate 
deficiency                                                                        

Glycogen storage disease   dec                  +       +              Lactate 
Type 1:                                                                           
 Glucose-1-phosphatose                                                            
deficiency                                                                        

Glycogen storage disease                                                          
Type 2:                                                                           

a-1,4-glucosidase                                                                 
deficiency                                                                        



Key:

(dec) Possible hypoglycaemia
dec Definite hypoglycaemia
+ Positive finding
+ Not positive in all cases
NaKdec decrease in Na & K
D Diagnostically significant
Lactate Lactate present in urine (by gas chromatography)

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