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Successful treatment of multiple angiomyolipomas with sirolimus in a child

1 Department of Paediatrics, Children's Hospital at the London Health Sciences Centre, London, Ontario, Canada
2 Department of Paediatrics, Children's Hospital at the London Health Sciences Centre; Department of Pathology and Laboratory Medicine; Department of Medicine, University of Western Ontario, London, Ontario, Canada

Correspondence Address:
G Filler,
Department of Paediatrics, Children's Hospital of Western Ontario, 800 Commissioners Road East, London, Ontario, N6A 5W9
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Source of Support: None, Conflict of Interest: None

Tuberous sclerosis complex frequently results in the formation of renal angiomyolipomas (AMLs). Sirolimus (SIR) or everolimus can be used to treat large AMLs, although this treatment has rarely been described in children, particularly for multiple renal AMLs. A 15-year-old girl presented with bilateral severe chronic flank pain coinciding with increased renal size and hundreds of renal AMLs. We opted to treat her with SIR over the course of 3.5 years. Following her treatment, her renal size had substantially decreased and the AMLs had shrunk. The patient's pain subsided as well. Our case, which has never been described in published literature, demonstrates that a child with multiple renal AMLs can be treated with SIR, and suggests that this treatment may be a viable option for preventing the development of secondary morbidities such as chronic pain.

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Indian Journal of Nephrology
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Online since 20th Sept '07