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Type II Glycogen Storage Disease (Pompe Disease)

Updated : September 3, 2022





Background

Epidemiology

Anatomy

Pathophysiology

Etiology

Genetics

Prognostic Factors

Clinical History

Physical Examination

Age group

Associated comorbidity

Associated activity

Acuity of presentation

Differential Diagnoses

Laboratory Studies

Imaging Studies

Procedures

Histologic Findings

Staging

Treatment Paradigm

by Stage

by Modality

Chemotherapy

Radiation Therapy

Surgical Interventions

Hormone Therapy

Immunotherapy

Hyperthermia

Photodynamic Therapy

Stem Cell Transplant

Targeted Therapy

Palliative Care

Medication

 

avalglucosidase alfa 

Indicated for late-onset Pompe disease:


≥30kg: 20mg/kg intravenous every two weeks



Dose Adjustments

Hypersensitivity reactions
Severe hypersensitivity (including anaphylaxis) or severe infusion-associated reaction (IAR): Immediately discontinue and implement appropriate medical treatment

Mild-to-moderate
• Consider temporarily holding or slowing the infusion rate and initiating appropriate medical treatment as follows
• If symptoms persist, wait at least 30 minutes for symptoms to resolve before stopping infusion for the day
• If symptoms subside, resume infusion for 30 minutes by decreasing the rate to 50% at which the reaction occurred, and subsequently increase the infusion rate by 50% for 15-30 minutes; if symptoms do not recur, increase infusion rate to rate at which the reaction occurred and consider continuing to increase in a stepwise manner
• This information pertains to managing mild-to-moderate and severe hypersensitivity or severe infusion-associated reactions (IAR) to a medication administered through an infusion
• In case of a severe hypersensitivity reaction (including anaphylaxis) or severe IAR, the infusion should be immediately discontinued, and appropriate medical treatment should be initiated

In mild-to-moderate reactions, the infusion rate should be temporarily held or slowed down, and appropriate medical treatment should be initiated. If symptoms persist, the infusion should be stopped for the day and resumed the next day after a waiting period of at least 30 minutes. If symptoms subside, the infusion rate should be gradually increased stepwise, starting with a 50% reduction in the rate at which the reaction occurred. If symptoms do not recur, the infusion rate can be gradually increased until it reaches the rate at which the reaction occurred

 

avalglucosidase alfa 

Indicated for late-onset Pompe disease
:


<30kg: 40mg/kg intravenous every two weeks
≥30kg: 20mg/kg intravenous every 2 weeks



Dose Adjustments

Hypersensitivity reactions
In case of severe hypersensitivity or severe infusion-associated reaction, the infusion should be immediately discontinued, and appropriate medical treatment should be initiated. Consider temporarily holding or slowing the infusion rate and initiating appropriate medical treatment for mild to moderate reactions. If symptoms persist, wait for at least 30 minutes for symptoms to resolve before discontinuing the infusion for the day. If symptoms subside, the infusion rate can be resumed, decreased by 50%, and then gradually increased over time to monitor for the recurrence of symptoms

 

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References

Type II Glycogen Storage Disease (Pompe Disease)

Updated : September 3, 2022




avalglucosidase alfa 

Indicated for late-onset Pompe disease:


≥30kg: 20mg/kg intravenous every two weeks



Dose Adjustments

Hypersensitivity reactions
Severe hypersensitivity (including anaphylaxis) or severe infusion-associated reaction (IAR): Immediately discontinue and implement appropriate medical treatment

Mild-to-moderate
• Consider temporarily holding or slowing the infusion rate and initiating appropriate medical treatment as follows
• If symptoms persist, wait at least 30 minutes for symptoms to resolve before stopping infusion for the day
• If symptoms subside, resume infusion for 30 minutes by decreasing the rate to 50% at which the reaction occurred, and subsequently increase the infusion rate by 50% for 15-30 minutes; if symptoms do not recur, increase infusion rate to rate at which the reaction occurred and consider continuing to increase in a stepwise manner
• This information pertains to managing mild-to-moderate and severe hypersensitivity or severe infusion-associated reactions (IAR) to a medication administered through an infusion
• In case of a severe hypersensitivity reaction (including anaphylaxis) or severe IAR, the infusion should be immediately discontinued, and appropriate medical treatment should be initiated

In mild-to-moderate reactions, the infusion rate should be temporarily held or slowed down, and appropriate medical treatment should be initiated. If symptoms persist, the infusion should be stopped for the day and resumed the next day after a waiting period of at least 30 minutes. If symptoms subside, the infusion rate should be gradually increased stepwise, starting with a 50% reduction in the rate at which the reaction occurred. If symptoms do not recur, the infusion rate can be gradually increased until it reaches the rate at which the reaction occurred

avalglucosidase alfa 

Indicated for late-onset Pompe disease
:


<30kg: 40mg/kg intravenous every two weeks
≥30kg: 20mg/kg intravenous every 2 weeks



Dose Adjustments

Hypersensitivity reactions
In case of severe hypersensitivity or severe infusion-associated reaction, the infusion should be immediately discontinued, and appropriate medical treatment should be initiated. Consider temporarily holding or slowing the infusion rate and initiating appropriate medical treatment for mild to moderate reactions. If symptoms persist, wait for at least 30 minutes for symptoms to resolve before discontinuing the infusion for the day. If symptoms subside, the infusion rate can be resumed, decreased by 50%, and then gradually increased over time to monitor for the recurrence of symptoms