A new publication entitled Efficacy of Sirolimus in Patients Requiring Tracheostomy for Life-Threatening Lymphatic Malformation of the Head and Neck: A Report From the European Reference Network has just been published in Frontiers in Pediatrics. It is co-authored by members of the Vascular Anomalies Working Group (VASCA WG) and presents the results of a multicenter retrospective study conducted by the group.
Read the full article here
Thirteen patients treated in the multidisciplinary vascular anomaly centers of the VASCA WG with sirolimus (after not responding to standard therapies) were analyzed. All patients had required a tracheostomy due to a life-threatening lymphatic malformation of the head and neck region that led to compression of the airway. The efficacy of the treatment was defined as achieving removal of the tracheostomy, and the safety of sirolimus, defined as the occurrence of adverse events (AEs).
The results were promising: “Under sirolimus therapy, tracheostomy could be reversed in 8/13 (62%) patients, a further 2/13 (15%) patients improved markedly, and removal of the tracheostomy was planned at the time of writing, while 3/13 (23%) patients showed insufficient or absent response to sirolimus, rendering tracheostomy reversal not feasible. The median duration of sirolimus treatment until removal of tracheostomy was 18 months (range, 8 months to 5.6 years). Adverse events of sirolimus therapy were common [10/13 (77%) patients], yet the majority of these were mild [9/10 (90%) patients] and only one severe adverse event was recorded, with ulceration and necrosis at a catheter insertion site.”
We congratulate the VASCA WG for this important publication that gives hope to patients with life-threatening lymphatic malformations of the head and neck requiring a tracheostomy. We look forward to future research in this domain in order to better understand this drug and the factors that lead to a positive treatment response in patients with these types of vascular anomalies.
Reference: Annegret Holm, Maroeska te Loo, Leo Schultze Kool, Päivi Salminen, Veronica Celis, Eulalia Baselga, Sophie Duignan, Veronika Dvorakova, Alan D. Irvine, Laurence M. Boon, Miikka Vikkula, Nader Ghaffarpour, Charlotte M. Niemeyer, Jochen Rössler and Friedrich G. Kapp (2021). Efficacy of sirolimus in patients requiring tracheostomy for life-threatening lymphatic malformation of the head and neck: a report from the European Reference Network. Frontiers in Pediatrics.