Hostname: page-component-586b7cd67f-dlnhk Total loading time: 0 Render date: 2024-11-28T04:17:28.914Z Has data issue: false hasContentIssue false

Rapid regression of large cardiac rhabdomyomas in neonates after sirolimus therapy

Published online by Cambridge University Press:  13 December 2017

M. David Weiland*
Affiliation:
Department of Pediatrics, Division of Pediatric Cardiology, Duke University Hospital and Health Center, Durham, NC, United States of America
Kristin Bonello
Affiliation:
Department of Pediatrics, Duke University Hospital and Health System, Durham, NC, United States of America
Kevin D. Hill
Affiliation:
Department of Pediatrics, Division of Pediatric Cardiology, Duke University Hospital and Health Center, Durham, NC, United States of America
*
Correspondence to: M. D. Weiland, MD, Department of Pediatrics, Division of Pediatric Cardiology, Duke University Health System, DUMC Box 3090, Durham, NC 27710, United States of America. Tel: +1 919 684 8111; Fax: +919 681 7892; E-mail: [email protected]

Abstract

Cardiac rhabdomyomas are the most common tumours in children and are typically seen in association with the tuberous sclerosis complex. Although benign and often associated with spontaneous regression, in rare circumstances surgical resection is indicated to relieve obstruction or other mass-related effects. Recent clinical trials have demonstrated the benefits of mammalian target of rapamycin inhibitors for the treatment of other tumour sub-types associated with tuberous sclerosis. Here we report rapid regression of several massive cardiac rhadomyomas in two neonates with the use of the mammalian target of rapamycin inhibitor sirolimus.

Type
Brief Report
Copyright
© Cambridge University Press 2017 

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

1. Beghetti, M, Gow, RM, Haney, I, Mawson, J, Williams, WG, Freedom, RM. Pediatric primary benign cardiac tumors: a 15-year review. Am Heart J 1997; 134: 11071114.Google Scholar
2. Crino, PB, Nathanson, KL, Henske, EP. The tuberous sclerosis complex. N Engl J Med 2006; 355: 13451356.Google Scholar
3. Bissler, JJ, McCormack, FX, Young, LR, et al. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med 2008; 358: 140151.Google Scholar
4. Franz, DN, Belousova, E, Sparagana, S, et al. Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial. Lancet 2013; 381: 125132.Google Scholar
5. Krueger, DA, Care, MM, Holland, K, et al. Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. N Engl J Med 2010; 363: 18011811.Google Scholar
6. Schubert, M, Venkataramanan, R, Holt, DW, et al. Pharmacokinetics of sirolimus and tacrolimus in pediatric transplant patients. Am J Transplant 2004; 4: 767773.Google Scholar
7. Tiberio, D, Franz, DN, Phillips, JR. Regression of a cardiac rhabdomyoma in a patient receiving everolimus. Pediatrics 2011; 127: e1335e1337.Google Scholar
8. Breathnach, C, Pears, J, Franklin, O, Webb, D, McMahon, CJ. Rapid regression of left ventricular outflow tract rhabdomyoma after sirolimus therapy. Pediatrics 2014; 134: e1199e1202.Google Scholar
9. Demir, HA, Ekici, F, Yazal Erdem, A, Emir, S, Tunc, B. Everolimus: a challenging drug in the treatment of multifocal inoperable cardiac rhabdomyoma. Pediatrics 2012; 130: e243e247.Google Scholar
10. Hoshal, SG, Samuel, BP, Schneider, JR, Mammen, L, Vettukattil, JJ. Regression of massive cardiac rhabdomyoma on everolimus therapy. Pediatr Int 2016; 58: 397399.Google Scholar
11. MacKeigan, JP, Krueger, DA. Differentiating the mTOR inhibitors everolimus and sirolimus in the treatment of tuberous sclerosis complex. Neuro Oncol 2015; 17: 15501559.Google Scholar