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KMID : 1036920210260040278
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Annals of Pediatric Endocrinology & Metabolism
2021 Volume.26 No. 4 p.278 ~ p.283
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Pasireotide treatment for severe congenital hyperinsulinism due to a homozygous ABCC8 mutation
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Mooij Christiaan F.
Tacke Carline E.
van Albada Mirjam E.
Barthlen Winfried
Bikker Hennie
Mohnike Klaus
Oomen Matthijs W. N.
van Trotsenburg A. S. Paul
Zwaveling-Soonawala Nitash
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Abstract
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ABCC8 and KCJN11 mutations cause the most severe diazoxide-resistant forms of congenital hyperinsulinism (CHI). Somatostatin analogues are considered as secondline treatment in diazoxide-unresponsive cases. Current treatment protocols include the first-generation somatostatin analogue octreotide, although pasireotide, a second-generation somatostatin analogue, might be more effective in reducing insulin secretion. Herein we report the first off-label use of pasireotide in a boy with a severe therapy-resistant form of CHI due to a homozygous ABCC8 mutation. After partial pancreatectomy, hyperinsulinism persisted; in an attempt to prevent further surgery, off-label treatment with pasireotide was initiated. Short-acting pasireotide treatment caused high blood glucose level shortly after injection. Long-acting pasireotide treatment resulted in more stable glycemic control. No side effects (e.g., central adrenal insufficiency) were noticed during a 2-month treatment period. Because of recurrent hypoglycemia despite a rather high carbohydrate intake, the boy underwent near-total pancreatectomy at the age of 11 months. In conclusion, pasireotide treatment slightly improved glycemic control without side effects in a boy with severe CHI. However, the effect of pasireotide was not sufficient to prevent near-total pancreatectomy in this case of severe CHI.
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KEYWORD
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Congenital hyperinsulinism, Pasireotide, Somatostatin analogue, Pancreatectomy
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