Systemic, local, and sclerotherapy drugs: What do we know about drug prescribing in vascular anomalies?

Abstract

Off-label drug prescribing, frequent in the treatment of vascular anomalies (VA), relies on the quality of the literature reporting drug efficacy and safety. Our objective is to review the level of evidence (LOE) surrounding drug use in VA, which is more prevalent in pediatric care. A list of drugs used in VA was created with a literature review in July 2020. For each drug listed, the article displaying the highest LOE was determined and then compared between efficacy/safety data, routes of administration, pharmacological categories and a subset of VA. The influence of research quality on study results was also explored. The median LOE for the 74 drugs identified poor methodological quality, with a predominance of retrospective studies or case reports. Drug safety is currently inadequately reported. This is alarming as many treatments display significant safety concerns. Also, current literature displays major publication bias that probably leads to overestimation of drug efficacy in VA.

Defining vascular anomaly phenotypes in children based on a systematic literature search: A critical step in developing a single severity score for interventional clinical trials

“…Our study has potential limitations. First, studies showing drug efficacy are more likely to get published, 14 thereby underrepresenting phenotypes in non‐sirolimus responders. Furthermore, some VA with poor sirolimus response (e.g., arterio‐venous malformations) were probably underrepresented.…”

“…This precludes evidence-based practices as reflected by the low level of evidence surrounding medical therapies in this field. 8,14 In systemic treatments (e.g., sirolimus), the absence of a previously validated scoring system is circumvented by combining different treatment response data: generic quality-of-life scores, as well as treatment-response assessment using oncology-based radiologic and clinical response scoring systems. [15][16][17] Of note, none of these scores or their combination has ever been validated in patients with VA, creating an unmet need for a VA-specific score to be generated and subsequently vali-dated.…”

“…Nevertheless, for the most severe and complex VAs requiring specialized multidisciplinary care, no severity scores exist. This precludes evidence‐based practices as reflected by the low level of evidence surrounding medical therapies in this field 8,14 . In systemic treatments (e.g., sirolimus), the absence of a previously validated scoring system is circumvented by combining different treatment response data: generic quality‐of‐life scores, as well as treatment‐response assessment using oncology‐based radiologic and clinical response scoring systems 15–17 .…”

Evaluating the Non-conventional Achalasia Treatment Modalities

“…Ethanolamine oleate (EO) is a substance resulting from the synthetic mixture of ethanolamine and oleic acid. EO acts as a sclerosing agent that produces local inflammatory response and, subsequently, tissue fibrosis (28). This sclerosing agent is generally used to treat vascular lesions and varices (28).…”

“…EO acts as a sclerosing agent that produces local inflammatory response and, subsequently, tissue fibrosis (28). This sclerosing agent is generally used to treat vascular lesions and varices (28). It is assumed that the EO injection in the lower esophageal sphincter may induce excitatory neuron injury, provoking a predominance of inhibitory activity and reduced sphincter pressure (29).…”