Everolimus treatment of a newborn with rhabdomyoma causing severe arrhythmia

Öztunç, Funda; Atik, Sezen Ugan; Güneş, Aslı Okbay

doi:10.1017/s1047951114002261

Cited by 28 publications

(12 citation statements)

References 12 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…One study classified as “other” was excluded as it was a notifying article about the planned randomized study of everolimus in CRs treatment [ 23 ]. Overall, 30 reports were included in the systematic review [ 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 47 , 48 , 49 , 50 , 51 , 52 , 53 ]. Three of the included articles partially duplicated the case reports [ 24 , 34 , 53 ].…”

Section: Resultsmentioning

confidence: 99%

Treatment of Cardiac Rhabdomyomas with mTOR Inhibitors in Children with Tuberous Sclerosis Complex—A Systematic Review

Sugalska

Tomik

Jóźwiak

et al. 2021

IJERPH

View full text Add to dashboard Cite

Background: Cardiac rhabdomyomas (CRs) are the earliest sign of tuberous sclerosis complex (TSC). Most of them spontaneously regress after birth. However, multiple and/or large tumors may result in heart failure or cardiac arrhythmia. Recently, the attempts to treat CRs with mTOR inhibitors (mTORi) have been undertaken. We reviewed the current data regarding the effectiveness and safety of mTORi in the treatment of CRs in children with TSC. Methods: The review was conducted according to the PRISMA guidelines. Medline, Embase, Cochrane library, and ClinicalTrial.gov databases were searched for original, full-text articles reporting the use of mTORi (everolimus or sirolimus) in the treatment of CRs in children with TSC. Results: Thirty articles describing 41 patients were identified (mostly case reports, no randomized or large cohort studies). Thirty-three children (80.5%) had symptomatic CRs and mTORi therapy resulted in clinical improvement in 30 of them (90.9%). CRs size reduction was reported in 95.1%. Some CRs regrew after mTORi withdrawal but usually without clinical symptoms recurrence. The observed side effects were mostly mild. Conclusions: mTORi may be considered as a temporary and safe treatment for symptomatic CRs in children with TSC, especially in high-risk or inoperable tumors. However, high-quality, randomized trials are still lacking.

show abstract

Section: Resultsmentioning

confidence: 99%

Treatment of Cardiac Rhabdomyomas with mTOR Inhibitors in Children with Tuberous Sclerosis Complex—A Systematic Review

Sugalska

Tomik

Jóźwiak

et al. 2021

IJERPH

View full text Add to dashboard Cite

show abstract

“…In recent years, there have been reports of severe arrhythmias that require urgent treatment. 8,9,29 We believe mTOR inhibitors may be an alternative for these patients. In this study, after sirolimus treatment, cardiac rhabdomyoma disappeared in 1 patient with severe outflow tract obstruction at birth and regressed in 2 patients whose cardiac rhabdomyoma relapsed after surgery.…”

Section: Discussionmentioning

confidence: 97%

Sirolimus Can Increase the Disappearance Rate of Cardiac Rhabdomyomas Associated with Tuberous Sclerosis: A Prospective Cohort and Self-Controlled Case Series Study

Chen

Wang

Zhang

et al. 2021

The Journal of Pediatrics

View full text Add to dashboard Cite

Objectives To investigate the efficacy and safety of sirolimus in the treatment of cardiac rhabdomyomas associated with tuberous sclerosis complex and the specific benefits in different subgroups. Study designThe study was a prospective cohort and self-controlled case series study. Based on the prevalence of cardiac rhabdomyoma at different ages, we estimated the natural tumor disappearance rate. The subgroup analysis was done by Cox regression. Self-controlled case series method was used to assess the magnitude and duration of the drug effect. Adverse events were described.Results A total of 217 patients were included in the cohort study. Tumor disappearance rate was higher in younger age groups (hazard ratio = 0.99, P = .027) and female patients (hazard ratio = 2.08, P = .015). The age-adjusted incidence ratio showed that the disappearance of rhabdomyomas between 3 and 6 months was more related to sirolimus. Adverse events were observed 60 times in 42 of 217 children, mainly stomatitis.Conclusions Sirolimus can increase the disappearance rate of cardiac rhabdomyoma in the tuberous sclerosis complex population. Efficacy varies by sex and age: female and younger patients have higher tumor disappearance rate. Sirolimus is well-tolerated.

show abstract

“…Having examined the series of published cases using mammalian target of rapamycin inhibitors for haemodynamically significant rhabdomyomas, these agents are clearly efficacious with all cases showing reduction in size or full resolution of cardiac rhabdomyoma. [10][11][12][13][14][15][16][17][18][19][20][21][22][23][26][27][28][29][30]32,33 However, these drugs are not without risk, and standardised dosing and treatment duration regimens remain to be defined. From the published case reports to date, there is no general consensus on dose of mammalian target of rapamycin inhibitors (variations from 0.1 mg OD to 0.25 mg BD twice per week to 0.5 mg BD twice per week).…”

Section: Discussionmentioning

confidence: 99%

Literature review of international mammalian target of rapamycin inhibitor use in the non-surgical management of haemodynamically significant cardiac rhabdomyomas

Cleary

McMahon

2020

Cardiol Young

View full text Add to dashboard Cite

AbstractCardiac rhabdomyomas represent the most common primary paediatric cardiac tumour and typically regresses over time in the majority of patients. Among those who are symptomatic, surgical resection or catheterisation procedures have traditionally proven effective. More recently, those invasive or challenging tumours have been successfully treated with mammalian target of rapamycin inhibitors, typically everolimus and sirolimus. This review outlines the current medical literature of the state-of-the-art medical treatment of these tumours. We specifically focus on dosing regimens, duration of therapy, and side-effect profiles of mammalian target of rapamycin inhibitors among this population. Although the majority of cases responded to mammalian target of rapamycin inhibition, standardised guidelines for dosing and duration of treatment remain to be defined.

show abstract

Everolimus treatment of a newborn with rhabdomyoma causing severe arrhythmia

Cited by 28 publications

References 12 publications

Treatment of Cardiac Rhabdomyomas with mTOR Inhibitors in Children with Tuberous Sclerosis Complex—A Systematic Review

Treatment of Cardiac Rhabdomyomas with mTOR Inhibitors in Children with Tuberous Sclerosis Complex—A Systematic Review

Sirolimus Can Increase the Disappearance Rate of Cardiac Rhabdomyomas Associated with Tuberous Sclerosis: A Prospective Cohort and Self-Controlled Case Series Study

Literature review of international mammalian target of rapamycin inhibitor use in the non-surgical management of haemodynamically significant cardiac rhabdomyomas

Contact Info

Product

Resources

About