Research output: Contribution to journal › Article › Academic › peer-review
Pasireotide treatment for severe congenital hyperinsulinism due to a homozygous ABCC8 mutation. / Mooij, Christiaan F; Tacke, Carline E; van Albada, Mirjam E et al.
In: Annals of pediatric endocrinology & metabolism, Vol. 26, No. 4, 12.2021, p. 278-283.Research output: Contribution to journal › Article › Academic › peer-review
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TY - JOUR
T1 - Pasireotide treatment for severe congenital hyperinsulinism due to a homozygous ABCC8 mutation
AU - Mooij, Christiaan F
AU - Tacke, Carline E
AU - van Albada, Mirjam E
AU - Barthlen, Winfried
AU - Bikker, Hennie
AU - Mohnike, Klaus
AU - Oomen, Matthijs W N
AU - van Trotsenburg, A S Paul
AU - Zwaveling-Soonawala, Nitash
PY - 2021/12
Y1 - 2021/12
N2 - 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.
AB - 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.
U2 - 10.6065/apem.2142010.005
DO - 10.6065/apem.2142010.005
M3 - Article
C2 - 33971706
VL - 26
SP - 278
EP - 283
JO - Annals of pediatric endocrinology & metabolism
JF - Annals of pediatric endocrinology & metabolism
SN - 2287-1012
IS - 4
ER -
ID: 21049881