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@ARTICLE{Riedmeier:307581,
author = {M. Riedmeier and H. Frey and S. R. Antonini and G. F. L.
Canali and C. F. Classen and N. Domínguez-Pinilla and M.
Fassnacht and J. Finger and S. Fuchs$^*$ and M. Grönroos
and M. P. Halah and C. Härtel and D. Janus and A.
Jaspers-Bakker and R. R. de Krijger and T. Kutluk and M.
Mezoued and J. Munarin and M. van Noesel and N. Ö. Köse
and S. H. Pearce and T. Perwein and S. Puglisi and P.-G.
Schlegel and V. Binder-Blaser and G. Tuli and J. Walenciak
and B. Yalcin and V. Wiegering},
title = {{M}itotane dosage, plasma levels, and anthropometric
measurements in pediatric adrenocortical carcinoma.},
journal = {Endocrine oncology},
volume = {6},
number = {1},
issn = {2634-4793},
address = {[Bristol]},
publisher = {Bioscientifica Ltd.},
reportid = {DKFZ-2026-00076},
pages = {e240081},
year = {2026},
note = {#DKTKZFB9#},
abstract = {Mitotane is an effective treatment for advanced
adrenocortical carcinoma (ACC). Given the limited pediatric
data available, this study aims to evaluate the associations
between mitotane dosage, plasma drug levels, and
anthropometric measurements, as well as their potential
impact on dosage requirements to optimize therapeutic
outcomes in pediatric patients with ACC (pACC).A
retrospective, international, multicenter study was
conducted on pediatric ACC patients treated with mitotane
across 18 centers. Mitotane serum levels were obtained from
the Lysosafe Online® database or directly from the centers.
Data from the cohort with plasma levels within the target
range (≥14 mg/L; n = 319) were analyzed and compared to
those with levels outside this range (n = 320).Fifty
pediatric patients $(60\%$ female) diagnosed between 2004
and 2023 were included, with a median follow-up of 34.5
months and a 10-year overall survival of 33 months. The
median age at diagnosis was 8.6 years, with most tumors
$(84\%)$ hormone-secreting. Among 49 patients undergoing
surgery, 31 $(62\%)$ achieved R0 resection. The median
treatment duration was 18 months, with a median mitotane
dose of 87 mg/kg/day in patients within the target plasma
level range, showing no significant difference from those
outside the range. However, BMI was significantly associated
with doses of plasma levels in target range (P = 0.001), as
underweight (105.4 mg/kg/day) and healthy weight patients
(98.4 mg/kg/day) required higher doses than overweight/obese
patients (44.4 mg/kg/day). No significant differences in
daily dose levels (mg/kg/day and mg/m2/day) were observed
based on body weight.This study supports estimating mitotane
dosages in pediatric ACC, emphasizing the need for close
monitoring and frequent follow-ups at specialized centers
due to individualized dosing and a narrow therapeutic
window.},
keywords = {mitotane (Other) / mitotane dosage (Other) / mitotane
plasma level (Other) / pediatric adrenocortical carcinoma
(Other) / pediatric adrenocortical tumor (Other)},
cin = {BE01},
ddc = {610},
cid = {I:(DE-He78)BE01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41503252},
pmc = {pmc:PMC12771559},
doi = {10.1530/EO-24-0081},
url = {https://inrepo02.dkfz.de/record/307581},
}