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家族性眼皮肤白化病伴无色素性/色素减退性黑素瘤。

Amelanotic/hypopigmented melanoma in a sibship with oculocutaneous albinism.

机构信息

The University of Queensland Diamantina Institute, The University of Queensland, Dermatology Research Centre, Brisbane, Queensland, Australia.

Department of Dermatology, Princess Alexandra Hospital, Brisbane, Queensland, Australia.

出版信息

J Dermatol. 2022 Nov;49(11):1183-1187. doi: 10.1111/1346-8138.16528. Epub 2022 Jul 27.

Abstract

Oculocutaneous albinism (OCA) is a rare condition characterized by hypopigmentation. A female proband and her sister, both with primary amelanotic/hypopigmented melanoma, underwent three-dimensional total-body photography and dermoscopy. Both sisters had exome sequencing along with their brother, who had OCA but no history of melanoma. Imaging analysis was consistent with OCA in terms of individual typology angle scores, degree of sun damage, and high naevus counts. Exome data filtered for variants in known OCA and melanoma/naevi susceptibility genes (n = 98) found all siblings were compound heterozygous for TYR mutations (Arg402Ter and Val275Phe), previously reported as causative OCA variants. A rare missense variant in PARP1 (p.Pro377Ser) was solely present in the melanoma-unaffected brother, which is noteworthy as this was previously reported as potentially protective in a familial melanoma pedigree positive for CDKN2A mutations. Evaluation and confirmation of functional impact in larger cohorts could personalize melanoma screening in OCA.

摘要

眼皮肤白化病(OCA)是一种罕见的以色素减退为特征的疾病。一位女性先证者及其妹妹均患有原发性无色素/色素减退性黑色素瘤,接受了三维全身摄影和皮肤镜检查。先证者及其妹妹和弟弟一起进行了外显子组测序,弟弟患有 OCA,但没有黑色素瘤病史。就个体类型角评分、太阳损伤程度和高痣计数而言,成像分析与 OCA 一致。针对已知 OCA 和黑色素瘤/痣易感性基因(n=98)的外显子数据筛选发现,所有兄弟姐妹均为 TYR 突变(Arg402Ter 和 Val275Phe)的复合杂合子,这些突变此前被报道为 OCA 的致病变体。PARP1 中的一个罕见错义变体(p.Pro377Ser)仅存在于未患黑色素瘤的弟弟中,这值得注意,因为此前在一个 CDKN2A 突变阳性的家族性黑色素瘤家系中,该变体被报道为具有潜在保护作用。在更大的队列中评估和确认其功能影响可能会使 OCA 患者的黑色素瘤筛查个体化。

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