Phelan Mary C
Cytogenetics Laboratory, Molecular Pathology Laboratory Network, 250 East Broadway, Maryville, TN 37804, USA.
Orphanet J Rare Dis. 2008 May 27;3:14. doi: 10.1186/1750-1172-3-14.
The deletion 22q13.3 syndrome (deletion 22q13 syndrome or Phelan-McDermid syndrome) is a chromosome microdeletion syndrome characterized by neonatal hypotonia, global developmental delay, normal to accelerated growth, absent to severely delayed speech, and minor dysmorphic features. The deletion occurs with equal frequency in males and females and has been reported in mosaic and non-mosaic forms. Due to lack of clinical recognition and often insufficient laboratory testing, the syndrome is under-diagnosed and its true incidence remains unknown. Common physical traits include long eye lashes, large or unusual ears, relatively large hands, dysplastic toenails, full brow, dolicocephaly, full cheeks, bulbous nose, and pointed chin. Behavior is autistic-like with decreased perception of pain and habitual chewing or mouthing. The loss of 22q13.3 can result from simple deletion, translocation, ring chromosome formation and less common structural changes affecting the long arm of chromosome 22, specifically the region containing the SHANK3 gene. The diagnosis of deletion 22q13 syndrome should be considered in all cases of hypotonia of unknown etiology and in individuals with absent speech. Although the deletion can sometimes be detected by high resolution chromosome analysis, fluorescence in situ hybridization (FISH) or array comparative genomic hybridization (CGH) is recommended for confirmation. Differential diagnosis includes syndromes associated with hypotonia, developmental delay, speech delay and/or autistic-like affect (Prader-Willi, Angelman, Williams, Smith-Magenis, Fragile X, Sotos, FG, trichorhinophalangeal and velocardiofacial syndromes, autism spectrum disorders, cerebral palsy). Genetic counseling is recommended and parental laboratory studies should be considered to identify cryptic rearrangements and detect parental mosaicism. Prenatal diagnosis should be offered for future pregnancies in those families with inherited rearrangements. Individuals with deletion 22q13 should have routine examinations by the primary care physician as well as genetic evaluations with referral to specialists if neurological, gastrointestinal, renal, or other systemic problems are suspected. Affected individuals benefit from early intervention programs, intense occupational and communication therapies, adaptive exercise and sport programs, and other therapies to strengthen their muscles and increase their communication skills. No apparent life-threatening organic abnormalities accompany the diagnosis of deletion 22q13.
22q13.3缺失综合征(22q13缺失综合征或费兰 - 麦克德米德综合征)是一种染色体微缺失综合征,其特征为新生儿肌张力减退、全面发育迟缓、生长正常至加速、言语缺失至严重延迟以及轻微的畸形特征。该缺失在男性和女性中出现的频率相等,并且已报道有嵌合型和非嵌合型。由于缺乏临床认识且实验室检测往往不足,该综合征诊断不足,其真实发病率仍然未知。常见的身体特征包括长睫毛、大耳朵或形状异常的耳朵、相对较大的手、发育异常的脚趾甲、饱满的眉毛、长头型、丰满的脸颊、球根状鼻子和尖下巴。行为表现类似自闭症,对疼痛的感知降低,有习惯性咀嚼或用口部动作。22q13.3的缺失可能由简单缺失、易位、环状染色体形成以及影响22号染色体长臂(特别是包含SHANK3基因的区域)的较罕见结构变化导致。对于所有病因不明的肌张力减退病例以及言语缺失的个体,均应考虑22q13缺失综合征的诊断。虽然有时通过高分辨率染色体分析可以检测到该缺失,但建议采用荧光原位杂交(FISH)或阵列比较基因组杂交(CGH)进行确诊。鉴别诊断包括与肌张力减退、发育迟缓、言语延迟和/或类似自闭症影响相关的综合征(普拉德 - 威利综合征、天使综合征、威廉姆斯综合征、史密斯 - 马吉尼斯综合征、脆性X综合征、索托斯综合征、FG综合征、毛发鼻指综合征和腭心面综合征、自闭症谱系障碍、脑瘫)。建议进行遗传咨询,应考虑对父母进行实验室研究以识别隐匿性重排并检测父母的嵌合现象。对于那些有遗传重排的家庭,应提供未来妊娠的产前诊断。患有22q13缺失的个体应由初级保健医生进行常规检查,如果怀疑有神经、胃肠、肾脏或其他全身性问题,还应转诊至专科医生进行遗传评估。受影响的个体受益于早期干预项目、强化职业和沟通治疗、适应性运动和体育项目以及其他增强肌肉力量和提高沟通技能的治疗。22q13缺失的诊断并不伴有明显的危及生命的器质性异常。