Cystic Fibrosis Clinical Trial
Official title:
The Utility of Point Shear-wave Elastography to Assess for Hepatic & Pancreatic Fibrosis in Pediatric Cystic Fibrosis Patients
Diagnosis of hepatic fibrosis is challenging as specific tests for detection of fibrosis in pediatric Cystic Fibrosis associated liver disease (CFALD) have not been developed and existing investigations do not correlate well with presence or severity of disease. Using a Liver biopsy it is difficult to diagnose this condition because of the patchy nature of the disease. Investigators intend to identify hepatic and pancreatic fibrosis in Cystic Fibrosis patients using Elastography and correlate this with their biochemical markers as well as histological findings of patients who have undergone liver biopsy for diagnosis of CFALD.
Liver disease is increasingly common in cystic fibrosis (CF). As new therapeutic options
emerge, life expectancy increases and common hepatobiliary manifestations impact on quality
of life and survival of CF patients. Hepatobiliary abnormalities in CF vary in nature and
range from defects attributable to the underlying CFTR gene defect to those related to
systemic disease and malnutrition. Today complications of liver disease represent the third
most frequent cause of disease-related death in patients with CF.
Cystic fibrosis-associated liver disease (CFALD) belongs to the group of common symptoms of
this disease; however, due to the lack of specific and sensitive CFALD diagnostic markers,
the epidemiological data may be incomplete. According to various sources, the prevalence rate
of CFALD, diagnosed on the basis of clinical, biochemical and imaging (ultrasonography)
tests, is 2-37% in children and young adults.
Cirrhosis is a final, irreversible stage of liver damage that leads to the failure of the
organ. While liver biopsy is considered the gold standard to assess for Hepatic Fibrosis; it
is invasive and potentially life threatening. The prognosis and management of chronic liver
disease depends on the extent and progression of liver fibrosis, which constitutes the most
important predictor of disease outcome.
The gold standard for diagnosis and staging of liver fibrosis has been liver biopsy. In
addition to being an invasive procedure with potential complications of bleeding and severe
pain, sampling error is an intrinsic problem due to the small sample size in a heterogeneous
process.7,8 Inter-observer variability also limits diagnostic consistency.9-11 The
development of several blood markers such as platelets, hyaluronic acid, type IV collagen,
aminotransferase/platelet ratio index (APRI) and algorithm based serum models (Fibro Index,
FIB-4, and Fibro Test) have been used but are affected by factors unrelated to the liver.
In the last decade, methods to noninvasively quantify liver fibrosis have been developed. The
first available method was transient elastography (TE).It is a single-element ultrasound
transducer operating at 5 MHz built on the axis of a piston like vibrator. By pushing a
button, low-frequency (50 Hz) transient vibrations are transmitted, and the generated elastic
shear waves propagate through underlying tissues. Pulse-echo ultrasound acquisitions are used
to follow the propagation of the shear wave and to measure its velocity .
Several studies have demonstrated a high accuracy of TE in identifying significant fibrosis
(F> 2) and cirrhosis (F= 4) in patients. It is a novel diagnostic tool that offers a rapid,
non-invasive method for monitoring HF. The device measures liver stiffness by transmitting a
vibration to determine the velocity of an elastic shear wave propagated through liver tissue.
Studies have documented utility of transient elastography in cystic fibrosis patients to
assess hepatic fibrosis and secondary complications.
Newer modalities like Shear wave elastography techniques have been implemented in
conventional real-time ultrasound systems, and several studies have shown their accuracy in
the assessment of liver fibrosis. Shear wave elastography relies on the generation of shear
waves determined by the displacement of tissues induced by the force of a focused ultrasound
beam or by external pressure. The shear waves are lateral waves, with a motion perpendicular
to the direction of the force that has generated them. They travel slowly (between 1 and 10
m/s) and are rapidly attenuated by tissue. The propagation velocity of the shear waves
correlates with the elasticity of tissue .
Compared with TE, these techniques have the advantage of B-mode image guidance; thus, they
can allow the user to choose the best acoustic window for correctly performing an examination
in real time.
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