How we approach the use of sirolimus and new agents: Medical therapy to treat vascular anomalies

Shimano, Kristin A.; Eng, Whitney; Adams, Denise M.

doi:10.1002/pbc.29603

Cited by 13 publications

(9 citation statements)

References 37 publications

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“…It is unknown whether the rate of serious infection can decrease with the presence of a low sirolimus dose. Clinically, high sirolimus doses may be needed in the initial treatment of patients with complicated VAs and patients with severe conditions (e.g., KMP), whereas low sirolimus doses can be used in patients who have already achieved a favorable response but need prolonged maintenance therapy [ 33 ].…”

Section: Discussionmentioning

confidence: 99%

Oral antibiotic prophylaxis for infection in patients with vascular anomalies receiving sirolimus treatment: a multicenter retrospective study

Qiu

Gong

et al. 2023

Orphanet J Rare Dis

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Objectives Patients with vascular anomalies (VAs) who receive oral sirolimus may be at high risk of infectious complications. Antibiotic prophylaxis with trimethoprim-sulfamethoxazole (TMP-SMZ) has been advocated. However, there have been few evidence-based analyses on this topic. This study assessed the effect of prophylactic TMP-SMZ on the incidence of infections in VA patients receiving sirolimus monotherapy. Methods A retrospective, multicenter chart review was performed on all VA patients receiving sirolimus treatment from August, 2013 to January, 2021. Results Before January 2017, 112 patients were treated with sirolimus without antibiotic prophylaxis. In the subsequent period, 195 patients were treated with TMP-SMZ for at least 12 months during sirolimus therapy. The percentage of patients with at least one serious infection during the initial 12 months of sirolimus treatment did not differ between the groups (difference, 1.1%; 95% CI − 7.0–8.0%). We observed no difference in the incidence of individual infection or total adverse events between the groups. The rate of sirolimus discontinuation due to adverse events did not differ significantly between groups. Conclusions We demonstrated that prophylactic TMP-SMZ does not decrease the incidence of infection or improve tolerance in VA patients receiving sirolimus monotherapy.

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Section: Discussionmentioning

confidence: 99%

Oral antibiotic prophylaxis for infection in patients with vascular anomalies receiving sirolimus treatment: a multicenter retrospective study

Qiu

Gong

et al. 2023

Orphanet J Rare Dis

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“…Following this study, several clinical trials and case series have demonstrated the efficacy of sirolimus in treating certain types of VAs, especially VAs with lymphatic components 10–13 . As a result, sirolimus has become a central component of treatment strategies for VAs 14,15 …”

Section: Introductionmentioning

confidence: 89%

“…[10][11][12][13] As a result, sirolimus has become a central component of treatment strategies for VAs. 14,15 Many VAs are driven by post-zygotic, somatic variants of genes in the mTOR pathway, especially PIK3CA and TEK. 3 The mTOR pathway controls cell proliferation, adhesion, migration, metabolism, and survival.…”

Section: Introductionmentioning

confidence: 99%

Infectious complications of vascular anomalies treated with sirolimus: A systematic review

Kalbfell,

Cohen‐Cutler,

Grisham

et al. 2023

Pediatric Blood & Cancer

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Background and objectivesInitially developed as immunosuppressive agents, mammalian target of rapamycin (mTOR) inhibitors are currently used widely in the management of vascular malformations and tumors. The incidence of infectious complications in the vascular anomalies (VA) population is not well defined. The goal of this systematic review was to better define the types and severity of reported infectious complications in patients with VAs treated with mTOR inhibition.MethodsThis was a systematic review conducted following PRISMA guidelines evaluating all research articles focused on infectious complications in patients with VAs treated with sirolimus or everolimus. Thirty articles including 1182 total patients and 316 infections (in 291 unique patients) were ultimately included.ResultsThe majority of infections were viral upper respiratory (n = 137, 54%), followed by pneumonia (n = 53, 20%), and cutaneous infections (n = 20, 8%). There were six total infection‐related fatalities, which all occurred in patients younger than 2 years. Two cases of Pneumocystis jirovecii pneumonia (PJP) were reported. These were infants with kaposiform hemangioendothelioma (KHE) who were also treated with steroids and did not receive PJP prophylaxis. Almost one‐third (n = 96, 32%) of infectious complications were graded 3–4 according to Common Terminology Criteria for Adverse Events (CTCAE) criteria. Details of patient age, subtype of VA, and timing of infection were lacking from many reports.ConclusionsMost infectious complications reported in patients with VA on mTOR inhibitors were viral respiratory infections and non‐severe. Bacteremia, infectious fatalities, and PJP are exceedingly rare. Future studies are needed to clarify the spectrum of infectious risks in VA patients and to provide guidance for infection prevention.

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“…The questions of how genetics can inform diagnosis are addressed, as well as now the expanding area of therapeutics 9 . With these advancements in the understanding of genetics, a growing list of targeted therapies are in clinical trials and hold promise for our vascular anomalies patients in the future 10 …”

Section: What Are the Gaps In Knowledge?mentioning

confidence: 99%

“…9 With these advancements in the understanding of genetics, a growing list of targeted therapies are in clinical trials and hold promise for our vascular anomalies patients in the future. 10 Operationally, how does one care for these patients? This has been a question whether a provider is at a small practice, a medium sized program or a large academic center.…”

Section: What Are the Gaps In Knowledge?mentioning

confidence: 99%

Introduction: The changing role of the pediatric hematologist/oncologist in the care of people with vascular anomalies

Snyder

Takemoto

2022

Pediatric Blood & Cancer

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How we approach the use of sirolimus and new agents: Medical therapy to treat vascular anomalies

Cited by 13 publications

References 37 publications

Oral antibiotic prophylaxis for infection in patients with vascular anomalies receiving sirolimus treatment: a multicenter retrospective study

Oral antibiotic prophylaxis for infection in patients with vascular anomalies receiving sirolimus treatment: a multicenter retrospective study

Infectious complications of vascular anomalies treated with sirolimus: A systematic review

Introduction: The changing role of the pediatric hematologist/oncologist in the care of people with vascular anomalies

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