Pome Disease

Pompe Disease-
Pompe disease ( glycogenosis type 2 , acid maltase deficiency disease ) is an inherited
disorder of glycogen metabolism caused by the absence of marked deficiency of the lyosomal
enzyme acid glucosidase.

Cardiovascular-  62%    bradycardia  21%     tachycardia 23%

Dermatologic-   82%     diaper dermatitis  36%    rash   54%    urticaria  21%

GI-  82%                       constipation 23%    diarrhea 62%  gastroenteritis  41%
                                     gastroesophageal reflux disease 26%  vomiting 49%

Haematologic/lymphatic - 44%      anemia  31%

Respiratory- 97%        cough 46%  phraryngitis 36%   pneumonia 46%  respiratory distress 31%
                                      tachypnea 23%          upper respiratory tract infection 44%

Special senses-          ear infection 33%    nasopharyngitis 23%   otitis media  44%
                                      rhinorrhea  28%

Miscellaneous -          bronchiolitis 23%   cather-related infection 28%    flushing  21%  
                                     general disorders and administration site conditions 97%
                                    infections and infestations  95%  injury ,
                                    poisioning and procedural complications 72%
                                    oral candidiasis  31%     oxygen saturation decreased 41%
                                    post procedural pain  26%    pyrexia   92%   vascular disorders 36%            

Special Precautions-
Cardiac effects- use caution when administering general anesthesia for placement of a central
venous catherter in an infantile- onset Pompe disease patients with cardiac hypertrophy.

Cardio-respiratory failure- acute cardio-respiratory failure requiring intubation and inotropic support has been reported after infusion with alglucerase alfa in infantile                 -onset Pomp disease with underlying cardiac myopathy, possibly associated with fluid overload with IV administration of alglucosidase alfa.

Infusion reactions- patients may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from infusion reactions, Therefore monitor thes patients more closely during administration                     of alglucerase alfa.

Concomittant illness-  patients with an acute underlying illness at the time of alglucosidase alfa infusion  appear at greater risk  for infusion reactions. Give              careful consideration to the patients clinical status  prior to administration of     alglucosidase alfa    

Hypersensitivity reactions- severe hypersensitivity reactions including anaphylactic reactions
have been reported during alglucosidase alfa  infusion. Becuase of potential  for severe
hypersensitivity reactions, make appropiate medical support available when administering
algucosidase alfa .

Pregnancy- there are no adequate and well controlled studies in pregnant woman. Because animal reproduction studies are not always predictive of human response,        only use this drug during pregnancy if clearly required

Lactation-excercise caution when administering alglucosidae alfa to a breast feeding woman.

Children- risks and benefits of algulcosidase  alfa have not been establihed in the  juvenile-onset  Pompe  disease  population

Elderly- Clinical studies did not include any subjects 65 years and older. It is not known whether thy respond differently than younger subjects.

Monitoring- evaluate liver enzymes prior to the initiation of aglucosidase alfa treatment and
periodically thereafter.  Execrise care in interpreting these tests because aspartate
aminotransferase and alanine aminotransferase levels may  be raised as a result of
muscle pathology with Pompe disease

Monitor vital signs at the end of each infusion increase

Indication-

Pompe disease

Dosage-
Recommended dosage- 20mg/kg/body weight administered every 2 weeks as an intraveous
infusion. The total volume is determined by the patients body weight and should be administrered over a period of 4 hours.

1. Inform patients and their caregivers that a registry for patients with Pompe disease has
    been established in order to better understand the variability and progression of Pompe
   disease and to continue to monitor and evaluate treatment

2. Encourage patients and their caregives to participate and advise them that participation
    may involve long-term follow -up  

Pregnancy-
There are no adequate and well controlled studies in pregnant woman. Because
animal reproduction studies are not always predictive of human response, only use this drug
during pregnancy if clearly required

Lactation-
Excercise caution when administering alglucosidae alfa to a breast feeding woman.

Children-
Risks and benefits of algulcosidase  alfa have not been establihed in the  juvenile-onset
 Pompe  disease  population

Elderly-
Clinical studies did not include any subjects 65 years and older. It is not known whether
thy respond differently than younger subjects.