Research News: New Publication on thalidomide therapy in patients with severe arteriovenous malformations

A new paper titled Case report study of thalidomide therapy in 18 patients with severe arteriovenous malformations, co-authored by Prof. Laurence Boon (member of the VASCA-WG) and Prof. Miikka Vikkula (Chair of the VASCA-WG), was recently published in the Nature Cardiovascular Research Journal.

Arteriovenous malformations (AVMs) are fast-flow lesions that may be destructive and are the most difficult-to-treat vascular anomalies. Embolization followed by surgical resection is commonly used; however, complete resection is rarely possible and partial resection often leads to dramatic worsening.

This article reports the results from a prospective experimental observational study testing the effects of the angiogenesis inhibitor thalidomide on 18 patients with a severely symptomatic AVM that is refractory to conventional therapies.

Main findings:

“Thalidomide was rapidly effective, within 1–5 months, in relieving chronic pain (VAS reduced from 6–8 to 0–3). It also reduced bleeding in extensive stage III and stage IV AVMs for which conventional therapies had failed. We did not observe any wound healing problems. Ulcerations healed within 1–6 months of thalidomide treatment and did not recur while patients were taking the medication. In three patients (patients 4, 5 and 12) receiving thalidomide therapy, the AVM reduced in size and vascularization on arteriography. One patient had no clinical symptoms or blush seen on arteriography after 8 years of follow-up”.

The findings suggest that thalidomide is effective in the management of chronic pain, bleeding and ulceration of extensive AVMs that are resistant to conventional therapy.

Read the full article here

Congratulations Prof. Boon and Prof. Vikkula on this excellent research!


Boon, L.M., Dekeuleneer, V., Coulie, J. et al. Case report study of thalidomide therapy in 18 patients with severe arteriovenous malformations. Nat Cardiovasc Res 1, 562–567 (2022).

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