BOSTON — Thalidomide appears promising for the treatment of nephrogenic fibrosing dermopathy, according to the results of a small, open-label trial.
If larger trials show thalidomide to be more effective than placebo or other therapies, patients who have the intractable condition with few treatment options will need to weigh its benefits against associated risks, which include an increased incidence of fetal abnormalities if taken during pregnancy, Stevens-Johnson syndrome, hypotension, and peripheral neuropathy. Side effects of thalidomide include fatigue and drowsiness.
Jonathan Kay, M.D., and colleagues at Massachusetts General Hospital in Boston treated nine men and women with chronic renal failure and a diagnosis of nephrogenic fibrosing dermopathy (NFD) confirmed by biopsy. All patients in the off-label trial received 50 mg of thalidomide daily. Duration of therapy was from 18 days to more than 13 months.
Overall, “the majority of patients experienced improvement in the hardness and tethering of their skin; some also experienced improvement in their joint contractures,” Dr. Kay said at a meeting on rheumatology sponsored by Harvard Medical School.
NFD typically involves hyper-pigmentation and tethered, thickened skin, as shown on the lower legs of this patient. Courtesy Dr. Jonathan Kay