MRA With Feraheme in HHT
| Status: | Completed |
|---|---|
| Conditions: | Cardiology |
| Therapuetic Areas: | Cardiology / Vascular Diseases |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 1/27/2019 |
| Start Date: | November 2016 |
| End Date: | November 2018 |
The Use of Ferumoxytol (Feraheme) for Whole Body Magnetic Resonance Angiography in Hereditary Hemorrhagic Telangiectasia
Magnetic resonance (MR) imaging is performed with contrast agents to highlight the blood
vessels and allow interpretation and diagnosis of blood vessel abnormalities. HHT (Hereditary
Hemorrhagic Telangiectasia) is a disease of blood vessels, and can suffer fatal bleeding if
abnormal blood vessels are not detected and treated early. Patients with HHT also require
many imaging studies through their lifetimes for surveillance of blood vessels. Many HHT
patients also have co-existing iron deficiency anemia from bleeding in their nose and
gastrointestinal tract, and receive daily iron therapy.
Ferumoxytol is an alternative MR contrast agent, which is FDA (Food and Drug Administration)
approved for the treatment of iron deficiency anemia. In addition, it is not associated with
the risks to the kidneys of the other agents. The use of ferumoxytol for MR imaging may
benefit the patients who do not currently receive imaging due to the contraindications of the
conventional contrast agents. It avoids the use of ionizing radiation. Also, the conventional
contrast agents are associated with risks. Iodinated contrast in CT is associated with
significant risks of kidney damage. Another imaging technique, MR, uses gadolinium based
contrast agents. Gadolinium, if used in patients with pre existing kidney dysfunction
(defined as GFR < 30ml/min) is associated with the development of another devastating disease
called nephrogenic systemic fibrosis. As HHT patients will require repeated scans throughout
their lifetimes, this study will provide them a safer alternative.
Ten patients from the HHT clinic in whom the use of ferumoxytol as an MR agent is clinically
indicated will be invited to participate in this study, which will determine if MR with
ferumoxytol is able to detect and characterize vascular malformations in HHT.
vessels and allow interpretation and diagnosis of blood vessel abnormalities. HHT (Hereditary
Hemorrhagic Telangiectasia) is a disease of blood vessels, and can suffer fatal bleeding if
abnormal blood vessels are not detected and treated early. Patients with HHT also require
many imaging studies through their lifetimes for surveillance of blood vessels. Many HHT
patients also have co-existing iron deficiency anemia from bleeding in their nose and
gastrointestinal tract, and receive daily iron therapy.
Ferumoxytol is an alternative MR contrast agent, which is FDA (Food and Drug Administration)
approved for the treatment of iron deficiency anemia. In addition, it is not associated with
the risks to the kidneys of the other agents. The use of ferumoxytol for MR imaging may
benefit the patients who do not currently receive imaging due to the contraindications of the
conventional contrast agents. It avoids the use of ionizing radiation. Also, the conventional
contrast agents are associated with risks. Iodinated contrast in CT is associated with
significant risks of kidney damage. Another imaging technique, MR, uses gadolinium based
contrast agents. Gadolinium, if used in patients with pre existing kidney dysfunction
(defined as GFR < 30ml/min) is associated with the development of another devastating disease
called nephrogenic systemic fibrosis. As HHT patients will require repeated scans throughout
their lifetimes, this study will provide them a safer alternative.
Ten patients from the HHT clinic in whom the use of ferumoxytol as an MR agent is clinically
indicated will be invited to participate in this study, which will determine if MR with
ferumoxytol is able to detect and characterize vascular malformations in HHT.
The safety of the use of gadolinium based contrast agents in MR is a concern for the FDA,
with risks of development of nephrogenic systemic fibrosis and the more recent discovery of
accumulation of gadolinium in the brain in patients who have received multiple prior MR
scans.
Hereditary hemorrhagic telangiectasia (HHT) manifests with multiple vascular malformations
(VMs) in the skin, mucous membranes and solid organs affecting the spine, brain, liver,
gastrointestinal tract, pancreas, and lungs. The disease has an autosomal dominant
inheritance and affects 1 in 5000 individuals.
Cerebral vascular malformations occur in 23 % of HHT patients, with a bleeding risk of 0.5%
per year. Pulmonary AVMs occur in 15-50% of HHT patients, with a complication rate of 50%
ranging from fatal hemoptysis or hemothorax to stroke or cerebral abscess. Liver vascular
malformations are present in 32-78% of HHT patients.
The rationale for screening for vascular malformations is detection of a treatable lesion
prior to the development of a fatal complication. The international guidelines currently
recommend different first line screening tests in each organ: MRI for cerebral VMs,
transthoracic echocardiography for pulmonary VMs, endoscopy for gastrointestinal VMs, Doppler
US or CT for liver VMs.
Contrast enhanced magnetic resonance angiography (CE-MRA) may play an important role in the
simultaneous whole body screening of vascular malformations. The advantages of CE-MRA include
visualization of the entire body vasculature in one examination, high spatial resolution
comparable to CT, no ionizing radiation and easy of multiplanar reconstructions.
Substituting a conventional gadolinium based contrast agent (GBCA) with an ultra small, super
paramagnetic iron oxide agent (USPIO) e.g ferumoxytol, will eliminate any potential risk of
developing nephrogenic systemic fibrosis. Since 2009, ferumoxytol ('Feraheme' Advanced
Magnetics, Cambridge, MA) has been FDA approved for the treatment of iron deficiency anemia
in adult patients with chronic kidney disease. The results of prior studies suggest that
ferumoxytol is comparable to standard GBCAs for CE- MRA. Our experience with use of
ferumoxytol to date suggests that it will be a superior agent for detection of vascular
malformations in a range of vascular territories and that it will be uniquely capable of
interrogating multiple territories in one sitting, due to its highly stable intravascular
residence time.
with risks of development of nephrogenic systemic fibrosis and the more recent discovery of
accumulation of gadolinium in the brain in patients who have received multiple prior MR
scans.
Hereditary hemorrhagic telangiectasia (HHT) manifests with multiple vascular malformations
(VMs) in the skin, mucous membranes and solid organs affecting the spine, brain, liver,
gastrointestinal tract, pancreas, and lungs. The disease has an autosomal dominant
inheritance and affects 1 in 5000 individuals.
Cerebral vascular malformations occur in 23 % of HHT patients, with a bleeding risk of 0.5%
per year. Pulmonary AVMs occur in 15-50% of HHT patients, with a complication rate of 50%
ranging from fatal hemoptysis or hemothorax to stroke or cerebral abscess. Liver vascular
malformations are present in 32-78% of HHT patients.
The rationale for screening for vascular malformations is detection of a treatable lesion
prior to the development of a fatal complication. The international guidelines currently
recommend different first line screening tests in each organ: MRI for cerebral VMs,
transthoracic echocardiography for pulmonary VMs, endoscopy for gastrointestinal VMs, Doppler
US or CT for liver VMs.
Contrast enhanced magnetic resonance angiography (CE-MRA) may play an important role in the
simultaneous whole body screening of vascular malformations. The advantages of CE-MRA include
visualization of the entire body vasculature in one examination, high spatial resolution
comparable to CT, no ionizing radiation and easy of multiplanar reconstructions.
Substituting a conventional gadolinium based contrast agent (GBCA) with an ultra small, super
paramagnetic iron oxide agent (USPIO) e.g ferumoxytol, will eliminate any potential risk of
developing nephrogenic systemic fibrosis. Since 2009, ferumoxytol ('Feraheme' Advanced
Magnetics, Cambridge, MA) has been FDA approved for the treatment of iron deficiency anemia
in adult patients with chronic kidney disease. The results of prior studies suggest that
ferumoxytol is comparable to standard GBCAs for CE- MRA. Our experience with use of
ferumoxytol to date suggests that it will be a superior agent for detection of vascular
malformations in a range of vascular territories and that it will be uniquely capable of
interrogating multiple territories in one sitting, due to its highly stable intravascular
residence time.
Inclusion Criteria:
- Definite diagnosis of HHT (clinically or genetically confirmed)
- Known or suspected AVMs in the brain, lung, and/or liver
- Use of ferumoxytol as an MR agent is clinically indicated
Exclusion Criteria:
- Age <18
- Unable to have MRI scan
- Prior adverse reaction to ferumoxytol
We found this trial at
1
site
UCLA UCLA's primary purpose as a public research university is the creation, dissemination, preservation and...
Click here to add this to my saved trials
