Mucopolysaccharidosis I

Updated: June 1, 2023

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

laronidase 

0.58 mg/kg intravenously (3 to 4-hour infusion) once weekly; maximum total volume of 100 mL for patients under 20 kg and 250 mL for those above 20 kg.
As tolerated, increase the infusion rate by 10 mcg/kg/hr every 15 minutes for the first hour, up to 200 mcg/kg/hr

idursulfase

Administer dose of 0.5 mg/kg intravenously every week and infuse over 1 to 3 hours every week

laronidase 

<5 years: Safety and efficacy not established
≥5 years:
<20kg- Every week, 0.58 mg/kg intravenous (3 to 4-hour infusion); total volume 100 mL
≥20kg- 0.58 mg/kg IV (3 to 4-hour infusion) weekly; total volume 250 mL
Starting at ten mcg/kg/h, the infusion rate may be increased by 15 mcg/kg/h as needed throughout the first hour, up to 200 mcg/kg/h.

idursulfase

For <5 years old: Safety and efficacy not established
For ≥5 years old:
Administer dose of 0.5 mg/kg intravenously every week and infuse over 1 to 3 hours

Future Trends