Skip to main content

Table 2 Registry endpoints

From: Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry

Primary endpoints

 

Safety of idursulfase

 • Occurrence of infusion-related reactions and other adverse events (including serious adverse events)

 • Immunogenicity, as determined by time to first positive antibody response (antibody level and isotype), antibody titre, isotype, and neutralizing antibodies

Effectiveness of idursulfase

 • Urinary glycosaminoglycan levels

 • Growth parameters (height, weight, head circumference)

 • Distance walked in the 6-min walk test

 • Left ventricular mass index (calculated by echocardiography)

 • Pulmonary function (measured by forced expiratory volume in 1 s and forced vital capacity)

 • Liver and spleen size (as estimated by palpation)

 • Prevalence of cardiac- and pulmonary-related hospitalizations

 • Deaths (including age at death and cause of death)

 

Secondary endpoints

 

 • Evaluation of the natural history of MPS II (based on the following signs and symptoms: hepatosplenomegaly, central nervous system involvement, skeletal involvement, and signs and symptoms of cardiac, pulmonary, and ear, nose, and throat involvement)

 • Evaluation of dosing of idursulfase (prescribed dose, administered dose, total infusion time, missed infusions, and reasons for missed infusions)

 • Scoring in five domains in the patient- and parent-reported versions of the HS-FOCUS questionnaire

 
  1. HS-FOCUS Hunter Syndrome Functional Outcomes for Clinical Understanding Scale, MPS II mucopolysaccharidosis type II