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  • Open Access

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

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Pediatric Rheumatology20086 (Suppl 1) :P267

  • Published:


  • Neuropathy
  • Cyclophosphamide
  • Vasculitis
  • Thalidomide
  • Acute Stroke


Polyarteritis nodosa (PAN) is a necrotizing vasculitis seldom reported on childhood, with a severe prognosis.


We report two sisters (16 and 8 years old) with PAN refractory to different drugs who had a persistent improvement over 5-years of thalidomide.

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.

No side effects have been observed.


Both our patients refractory to steroids and immunosuppressants had a significant improvement when thalidomide was introduced. No flares have been observed during a 5-year follow-up. Although the exact mechanism of action is not fully understood, thalidomide mainly prescribed in connective diseases, could be considered an alternative therapy in severe PAN.

Authors’ Affiliations

Department of Pediatrics, Pediatric Rheumatology, Messina, Italy
Department of iomedicine, Division of Rheumatology, Transition Unit, Florence, Italy


  1. Ting TV, Hashkes PJ: Update on childhood vasculitides. Curr Opin Rheumatol. 2004, 16 (5): 560-5. 10.1097/01.bor.0000130287.62007.9f.View ArticlePubMedGoogle Scholar


© Vitale et al; licensee BioMed Central Ltd. 2008

This article is published under license to BioMed Central Ltd.