Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3

N/AEnrolling By InvitationNCT06627543

Noha Osman44 enrolled

Overview

all cases confirmed diagnosis of Gaucher disease type 3 recieving enzyme replacement therapy at Assiut university hospital

evaluate cardiac involvement in a group of GD type 3 patients, evaluate pulmonary hypertension and valvular involvement, compare the findings of basic echo before the start of replacement therapy as well as with healthy controls, to assess the effects of enzyme replacement therpay on the cardiac function.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Gaucher Disease Type 3

Interventions

echoDIAGNOSTIC_TEST

echocardiographic

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: * Any sex * No age limit * Gaucher disease type 3 * Receiving enzyme replacement therapy Exclusion Criteria: * Age18 years old * Cases of gaucher type 1 and 2 * Other lipid storage disease

Locations (1)

Ahmed Mohammed Mohammed

Asyut, Egypt

Outcomes

Primary Outcomes

Evaluate Pulmonary Hypertension (PH) in Egyptian GD Type 3 Patients

To assess the presence and severity of pulmonary hypertension using echocardiography. Unit of Measure: mmHg (for systolic pulmonary artery pressure)

Time frame: 6 months

Evaluate Valvular Involvement in Egyptian GD Type 3 Patients

To assess the degree of valvular involvement, including stenosis or regurgitation, using echocardiography. Unit of Measure: Severity grading (mild, moderate, severe)

Time frame: 6 months

Data from ClinicalTrials.gov

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