Laura Battaglia, Corrado Ini', Manuela Lo Bianco, Roberta Leonardi, Eleonora Ini', Pietro Valerio Foti, Stefano Palmucci, Marco Fichera, Martino Ruggieri, Antonio Basile
{"title":"1例鲁宾斯坦-泰比综合征患者的毛瘤:诊断和治疗线索。","authors":"Laura Battaglia, Corrado Ini', Manuela Lo Bianco, Roberta Leonardi, Eleonora Ini', Pietro Valerio Foti, Stefano Palmucci, Marco Fichera, Martino Ruggieri, Antonio Basile","doi":"10.1177/26330040251330316","DOIUrl":null,"url":null,"abstract":"<p><p>Pilomatricoma is a rare benign neoplasm originating from hair cortex cells and typically manifests in children as a slow-growing bluish-red, superficial and firm mass. Multiple pilomatricomas can be associated with genetic mutations and syndromic disorders, most commonly with Rubinstein-Taybi syndrome, Gardner syndrome, myotonic dystrophy, Turner syndrome, and Sotos syndrome. Ultrasound examination allows this tumor to be characterized, to assess the involvement of deeper structures and to plan treatment. Pilomatricoma shows some distinctive ultrasonographic features that aid in its diagnosis and it may be seen on ultrasound as an ovoid complex mass. Complications and malignant transformation of pilomatricomas have been described as a possible tumor evolution, and surgical resection is recommended. We present a rare case of a 17-year-old female patient with intellectual disability and microcephaly, and with the evidence of multiple pilomatricomas in the head-neck region on ultrasound examination. The syndromic features of the patient and genetic tests led to a diagnosis of Rubinstein-Taybi syndrome. We also focused on the association between pilomatricomas and genetic mutations in patients with Rubinstein-Taybi syndrome.</p>","PeriodicalId":75218,"journal":{"name":"Therapeutic advances in rare disease","volume":"6 ","pages":"26330040251330316"},"PeriodicalIF":0.0000,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033402/pdf/","citationCount":"0","resultStr":"{\"title\":\"Pilomatricomas in a patient with Rubinstein-Taybi syndrome: diagnostic and therapeutic clues.\",\"authors\":\"Laura Battaglia, Corrado Ini', Manuela Lo Bianco, Roberta Leonardi, Eleonora Ini', Pietro Valerio Foti, Stefano Palmucci, Marco Fichera, Martino Ruggieri, Antonio Basile\",\"doi\":\"10.1177/26330040251330316\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Pilomatricoma is a rare benign neoplasm originating from hair cortex cells and typically manifests in children as a slow-growing bluish-red, superficial and firm mass. Multiple pilomatricomas can be associated with genetic mutations and syndromic disorders, most commonly with Rubinstein-Taybi syndrome, Gardner syndrome, myotonic dystrophy, Turner syndrome, and Sotos syndrome. Ultrasound examination allows this tumor to be characterized, to assess the involvement of deeper structures and to plan treatment. Pilomatricoma shows some distinctive ultrasonographic features that aid in its diagnosis and it may be seen on ultrasound as an ovoid complex mass. Complications and malignant transformation of pilomatricomas have been described as a possible tumor evolution, and surgical resection is recommended. We present a rare case of a 17-year-old female patient with intellectual disability and microcephaly, and with the evidence of multiple pilomatricomas in the head-neck region on ultrasound examination. The syndromic features of the patient and genetic tests led to a diagnosis of Rubinstein-Taybi syndrome. We also focused on the association between pilomatricomas and genetic mutations in patients with Rubinstein-Taybi syndrome.</p>\",\"PeriodicalId\":75218,\"journal\":{\"name\":\"Therapeutic advances in rare disease\",\"volume\":\"6 \",\"pages\":\"26330040251330316\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-04-12\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12033402/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Therapeutic advances in rare disease\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1177/26330040251330316\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Therapeutic advances in rare disease","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1177/26330040251330316","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/1 0:00:00","PubModel":"eCollection","JCR":"","JCRName":"","Score":null,"Total":0}
Pilomatricomas in a patient with Rubinstein-Taybi syndrome: diagnostic and therapeutic clues.
Pilomatricoma is a rare benign neoplasm originating from hair cortex cells and typically manifests in children as a slow-growing bluish-red, superficial and firm mass. Multiple pilomatricomas can be associated with genetic mutations and syndromic disorders, most commonly with Rubinstein-Taybi syndrome, Gardner syndrome, myotonic dystrophy, Turner syndrome, and Sotos syndrome. Ultrasound examination allows this tumor to be characterized, to assess the involvement of deeper structures and to plan treatment. Pilomatricoma shows some distinctive ultrasonographic features that aid in its diagnosis and it may be seen on ultrasound as an ovoid complex mass. Complications and malignant transformation of pilomatricomas have been described as a possible tumor evolution, and surgical resection is recommended. We present a rare case of a 17-year-old female patient with intellectual disability and microcephaly, and with the evidence of multiple pilomatricomas in the head-neck region on ultrasound examination. The syndromic features of the patient and genetic tests led to a diagnosis of Rubinstein-Taybi syndrome. We also focused on the association between pilomatricomas and genetic mutations in patients with Rubinstein-Taybi syndrome.