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一名新生儿因罕见的纯合错义突变导致的Glanzmann血小板无力症。

Glanzmann Thrombasthenia in a Newborn Due to a Rare Homozygous Missense Mutation.

作者信息

Faraz Saima, Nikhat Fareeda, Hayel Suleiman Beshtawi Hiyam, Malik Sofia A, Yahya Hashim Kauthar

机构信息

Obstetrics and Gynecology, Latifa Women and Children Hospital, Dubai, ARE.

Obstetrics and Gynecology, Latifa Hospital, Dubai, ARE.

出版信息

Cureus. 2024 Dec 7;16(12):e75291. doi: 10.7759/cureus.75291. eCollection 2024 Dec.

DOI:10.7759/cureus.75291
PMID:39776701
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11706618/
Abstract

Glanzmann thrombasthenia (GT) is an autosomal recessive platelet functional bleeding disorder caused by mutations in the ITGA2B or ITGB3 genes, often presenting as mucocutaneous bleeding. GT typically presents in infancy, but this study reports a rare case of neonatal presentation in a female infant born to consanguineous parents. The mother, a 27-year-old woman with a family history of GT, presented at 36 weeks gestation for an elective cesarean due to a breech presentation. The newborn, delivered with an appearance, pulse, grimace, activity, and respiration (APGAR) score of 9, exhibited spontaneous bruising, gum bleeding, and hyperbilirubinemia, necessitating neonatal intensive care admission. An initial hematologic workup showed normal coagulation profiles, but platelet function was significantly impaired. Genetic analysis identified a homozygous ITGB3 mutation, p.Asp145Asn, with both parents confirmed as heterozygous carriers. Management included factor VIIa, platelet transfusions, fresh frozen plasma, and RBCs. This case underscores the critical need for early recognition of GT in neonates with severe bleeding, especially with family history and consanguinity, and highlights the implications of the p.Asp145Asn mutation in the severe neonatal presentation. Genetic counseling is recommended for the family given the 25% recurrence risk in future pregnancies, and prospective partner testing may aid in assessing recurrence risks for descendants.

摘要

Glanzmann血小板无力症(GT)是一种常染色体隐性血小板功能性出血障碍,由ITGA2B或ITGB3基因突变引起,常表现为黏膜皮肤出血。GT通常在婴儿期发病,但本研究报告了一例罕见的新生儿发病病例,患儿为近亲结婚父母所生的女婴。母亲是一名27岁有GT家族史的女性,因臀位在妊娠36周时择期剖宫产。新生儿出生时外观、脉搏、面部表情、活动和呼吸(APGAR)评分为9分,但出现了自发性瘀斑、牙龈出血和高胆红素血症,因此需要入住新生儿重症监护病房。初步血液学检查显示凝血指标正常,但血小板功能严重受损。基因分析确定了一个纯合的ITGB3突变,即p.Asp145Asn,父母双方均被确认为杂合携带者。治疗措施包括使用凝血因子VIIa、输注血小板、新鲜冰冻血浆和红细胞。该病例强调了对于有严重出血的新生儿,尤其是有家族史和近亲结婚情况时,早期识别GT的迫切需求,并突出了p.Asp145Asn突变在严重新生儿发病中的影响。鉴于未来妊娠有25%的复发风险,建议对该家庭进行遗传咨询,前瞻性伴侣检测可能有助于评估后代的复发风险。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c8f8/11706618/89c5c75f0067/cureus-0016-00000075291-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c8f8/11706618/89c5c75f0067/cureus-0016-00000075291-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c8f8/11706618/89c5c75f0067/cureus-0016-00000075291-i01.jpg

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