Nephrogenic Systemic Fibrosis and Treatment Availability

Nephrogenic Systemic Fibrosis (NSF) is a rare degenerative disease that affects patients with serious kidney problems. It causes a buildup of scar tissue — fibrosis — on the skin and on several vital internal organs, including the heart, lung and diaphragm. Its main symptom is the sudden appearance of skin lesions on the arms, legs, hands and feet, sometimes accompanied by swelling, pain and blisters. These lesions are painful and unsightly, looking a little like the skin of an orange — but more importantly, they can be crippling. Over time, the hardened skin can prevent patients from bending joints, robbing them of the ability to walk and perform simple tasks. Patients may be bound to a wheelchair within a few weeks. In addition, scar tissue can build up so far on internal organs that it interferes with their functions, eventually causing death. Scientists have strongly associated NSF with the use of a chemical called gadolinium — a dye injected to make blood vessels easier to see in magnetic resonance imaging and magnetic resonance angiography (MRI/MRA) tests.

Studies have found that more than 95% of American NSF patients have been directly exposed to gadolinium. In response, the U.S. Scientists are researching the dye and its connection to NSF, in addition to that, the FDA has ordered a “black box” warning on gandolinium labels. Preventive Measures Unfortunately

Treatment of NSF is still based around prevention. The FDA has issued guidelines three times since June of 2006, each strongly suggesting that doctors avoid using gadolinium in patients with moderate to end-stage kidney disease. For patients with severe kidney failure, it is suggested that medical professionals avoid using gandolinium for MRAs and MRIs; or in patients with any level of kidney dysfunction caused by liver problems. It is advised that the dye is discontinued for use in these types of patients unless absolutely necessary. Even then, they are asked to make sure they do not use more than the recommended dosage. And the FDA has asked doctors to consider sending these patients for dialysis as soon as possible after the MRI.

Experimental Treatments Currently, there is no consistently successful treatment for NSF. In fact, the disease is so new — it was first identified in 1997 — that doctors are still investigating several treatments to see which give patients the best long-term relief. Treatments that have helped some patients include:

* Improving kidney function is the treatment with the most consistent good results. This can mean medical treatment (including dialysis), a kidney transplant or both. While improved kidney function has actually reversed the course of NSF in some cases, it has had no effect in other cases.

* Oral steroids are pills that have improved the skin problems of some patients. They do not work for everyone, and may cause calcium deficiency, high blood sugar and ulcers, making them inappropriate for many people with some other health problems.

* Skin creams and ointments, such as vitamin D3 and forms of cortisone, have helped some people fight their skin symptoms.

* Physical therapy, especially swimming, and deep massage have helped some patients slow down NSF’s effect on their joints.

While several individuals have seen a decrease in symptoms when using thalidomide, cytoxin and pentoxifylline, the drugs have not had a widespread success that many were hoping for. Similarly, two types of blood treatments — plasmapheresis and extracorporeal photopheresis — and ultraviolet light therapy have been helpful for one or two patients. But in all of these cases, more research is needed.

In addition, a recent study from Wake Forest University Baptist Medical Center suggests that NSF could be stopped before it starts if doctors are able to administer drugs that inhibit a bodily enzyme called transglutaminase-2 (TG2). These researchers found high levels of TG2 in patients with NSF, which suggests that the enzyme helps activate the disease. If that is in fact the truth, medical professionals may be able to prescribe drugs inhibiting TG2 that will prevent NSF. NSF can be a fatal and the disease is currently incurable. The disease is found in patients with kidney disease and kidney failure. It is also completely preventable — if doctors work closely with patients and their loved ones to avoid using gadolinium in kidney patients. The FDA found that there were 215 individuals worldwide that suffered from NSF in December 2006; every single one whose medical history they could review had been exposed to gadolinium. Receiving answers after developing NSF from gandolinium injects for a MRI or MRA is important for individuals and their families.