Efficacy and safety of thalidomide in two sisters with severe refractory polyarteritis nodosa (PAN)

In the older, the presenting symptoms were fever and migrant arthralgias. At onset, she was 6 year old. Laboratory work-up showed increased ESR, CRP, PTL count, and anaemia. Over time she developed ischemic colitis and a transient cerebral ischemic attack. Cerebral Angio MRI revealed multiple periventricular hyperintensities prompting the diagnosis of PAN. Steroids, cyclophosphamide, methotrexate, and azathioprine were unsuccessful, and the disease was complicated by neuropathy and ischemic lesions of several digits. Thalidomide was started in December 2003; since then, the disease is stable and laboratory work up normal. First clinical features of the younger sister occurred at three years of age. She had an acute stroke. Following hypertension, renal disease and neuropathy, PAN was diagnosed. Steroids, azathioprine and cyclophosphamide were given with scarce benefit. After another ischemic cerebrovascular event, thalidomide was effective in controlling the disease activity.