Refractory and relapsed paediatric ACC in the MET studies – A challenging situation necessitating novel diagnostic and therapeutic concepts

Kuhlen, Michaela; Kunstreich, Marina; Lessel, Lienhard; Wudy, Stefan A.; Holterhus, Paul-Martin; Vokuhl, Christian; Juettner, Eva; Roecken, Christoph; Seitz, Guido; Slavetinsky, Christoph; Fuchs, Jörg; Schewe, Denis M.; Claus, Rainer; Johann, Pascal D.; Frühwald, Michael C.; Vorwerk, Peter; Redlich, Antje

doi:10.1016/j.ejcped.2023.100015

Cited by 2 publications

(1 citation statement)

References 34 publications

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“…Up to 50% of them relapse after complete tumor resection [3][4][5][6][7][8][9]. We have previously reported that only a small proportion of these patients can be rescued by salvage therapy [10].…”

Section: Introductionmentioning

confidence: 99%

Locally Advanced Adrenocortical Carcinoma in Children and Adolescents—Enigmatic and Challenging Cases

Kuhlen,

Mier,

Kunstreich

et al. 2023

Cancers

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Background: Locally advanced tumors account for approximately 50% of children and adolescents with adrenocortical carcinoma (ACC), and of these, up to 50% relapse. We explored the five-item microscopic score and the pS-GRAS score for guiding management. Methods: Data from children and adolescents with COG stage II and III ACC registered in the MET studies were included. The five-item and pS-GRAS score were retrospectively calculated. Results: By December 2021, 55 patients with stage II and III (stage II n = 18, stage III n = 37) had been reported. Median age was 4.3 years [0.1–17.8], median duration of follow-up 6.0 years [0–16.7]. 3-year event-free survival (EFS) rate was 76.5% and 49.8% (p = 0.088), respectively. In stage II tumors, neither the five-item score (p = 0.872) nor pS-GRAS grouping (p = 0.218) had any effect as prognostic factors. In stage III patients, EFS was impaired in tumors with unfavorable histology according to the five-item score (100% vs. 30.8%, p = 0.018). No difference was observed for pS-GRAS groups (p = 0.798). Conclusions: In patients with COG stage III, but not stage II, the five-item score affected EFS. Further studies are needed to identify patients at risk in COG stage II.

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Section: Introductionmentioning

confidence: 99%

Locally Advanced Adrenocortical Carcinoma in Children and Adolescents—Enigmatic and Challenging Cases

Kuhlen,

Mier,

Kunstreich

et al. 2023

Cancers

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The Endocrine Phenotype Induced by Pediatric Adrenocortical Tumors Is Age- and Sex-Dependent

Kunstreich,

Dunstheimer,

Mier

et al. 2024

The Journal of Clinical Endocrinology &Amp; Metabolism

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Context Adrenocortical carcinomas are very rare malignancies in childhood associated with poor outcome in advanced disease. Most adrenocortical tumours (ACT) are functional causing signs and symptoms of adrenal hormone excess. In most studies, endocrine manifestations were reported 4-6 months prior to diagnosis. Objective We seeked to extend our knowledge on endocrine manifestations with regard to age and sex to facilitate early diagnosis. Design/Settings/Patients We retrospectively analysed features of adrenal hormone excess in children and adolescents with ACT registered with the GPOH-MET studies between 1997 and 2022. Stage of puberty was defined as `prepubertal` in females <8 years of age and males <9 years. Results By December 2022, 155 patients (110 female, 45 male) with data on endocrine manifestations had been reported. Median age at ACT diagnosis was 4.2 years [0.1-17.8], median interval from first symptoms 4.2 months [0-90.7]. In 63 females of prepubertal age pubarche (68.3%), clitoral hypertrophy (49.2%), and weight gain (31.7%) were most frequently reported, in 47 pubertal female excessive pubic hair (46.8%), acne (36.2%), and hypertension (36.2%). Leading symptoms in 34 males of prepubertal age were pubarche (55.9%), penile growth (47.1%), and acne (32.4%) and in 11 pubertal males, weight gain (45.5%), hypertension (36.4%), excessive pubic hair (27.3%), and cushingoid appearance (27.3%). In pubertal patients, symptoms of androgen excess were mainly unrecognized as part of pubertal development while symptoms of Cushing’s syndrome were more frequently apparent. Conclusions The endocrine phenotype induced by paediatric ACT is age- and sex-dependent.

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Refractory and relapsed paediatric ACC in the MET studies – A challenging situation necessitating novel diagnostic and therapeutic concepts

Cited by 2 publications

References 34 publications

Locally Advanced Adrenocortical Carcinoma in Children and Adolescents—Enigmatic and Challenging Cases

Locally Advanced Adrenocortical Carcinoma in Children and Adolescents—Enigmatic and Challenging Cases

The Endocrine Phenotype Induced by Pediatric Adrenocortical Tumors Is Age- and Sex-Dependent

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