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February 25 2021

Research News: VASCA Retrospective Study on Severe adverse events during sirolimus “off-label” therapy for Vascular Anomalies


A new article entitled Severe adverse events during sirolimus “off-label” therapy for vascular anomalies has just been published in the journal Pediatric Blood & Cancer. It is co-authored by Vascular Anomalies Working Group (VASCA WG) members Dr. Jochen Rössler, Dr. Andrea Diociaiuti, Dr. Maya El Hachem and Dr. Friedrich Kapp.

Access the article here

Sirolimus, also known as rapamycin, is an immunosuppressive agent used in patients after organ transplantation. For over a decade now, it has been used in an “off-label” setting (i.e. when an approved medication is used for an unapproved indication/age group/dosage) for the treatment of vascular anomalies and has shown to provide some benefit to certain patients, especially those with lymphatic and venous malformations. This retrospective survey (from September 2019-April 2020) gathered data from 113 patients taking sirolimus from 7 healthcare providers (including 2 HCP full members of VASCERN) and, from this population, 17 severe adverse effects (SAEs) were reported in 14 patients. The nature of the SAEs were described and they highlight the importance of a close follow-up in these patients as well as the need for
further clinical studies to establish the safety profile of sirolimus, the
incidence of SAEs in different patient populations, and the optimal
antibiotic prophylaxis.

Conclusion: Most SAEs are observed in the first year of sirolimus therapy; however, SAEs can also occur after a longer treatment period. SAEs are potentially life threatening, especially in early infancy. Presence of other risk factors, that is, underlying vascular anomaly or immune status, may contribute to the risk of SAEs. Sirolimus is an important therapeutic option for vascular anomalies, but patients and physicians need to be aware that adequate monitoring is necessary, especially in patients with complex lymphatic anomalies that are overrepresented in our cohort of SAEs.”

We congratulate the authors on this important publication, which we hope will inspire further studies on the use of sirolimus in patients with vascular anomalies in order to improve and provide more personalized therapy for patients with this rare and heterogeneous group of disorders.

Reference: Rössler J, Baselga E, Davila V, Celis V, Diociaiuti A, El Hachem M, Mestre S, Haeberli D, Prokop A, Hanke C, Loichinger W, Quéré I, Baumgartner I, Niemeyer CM, Kapp FG. Severe adverse events during sirolimus “off-label” therapy for vascular anomalies. Pediatr Blood Cancer. 2021 Feb 13:e28936. doi: 10.1002/pbc.28936. Epub ahead of print. PMID: 33580918.

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