Cubiró Xavier, Rozas-Muñoz Eduardo, Castel Pau, Roé Crespo Esther, Garcia-Melendo Cristina, Puig Lluis, Baselga Eulalia
Department of Dermatology, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain.
Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Pediatr Dermatol. 2020 Sep;37(5):833-838. doi: 10.1111/pde.14252. Epub 2020 Jul 1.
Diffuse capillary malformation with overgrowth (DCMO) has been well described. However, capillary malformation with undergrowth (CMU) has been less reported in the literature.
We sought to describe the clinical features and determine associated somatic mutations in patients with CMU.
We searched our multidisciplinary vascular anomalies clinic database for patients with CMU. Girth and length limb measurements were performed. In case of discrepancies in length, long leg radiograph studies were obtained. Whole-exome sequencing of blood and involved tissue DNA was carried out.
We included six patients with CM and soft-tissue and bone undergrowth. CMs were patchy, reticulated, segmental, poorly demarcated, pink-red stains affecting the lower limb (five patients) or the whole hemibody (one patient). In five patients, the stain was diffuse, affecting more than one anatomic region. Prominent superficial veins were observed in three patients. Five patients presented with lower limb girth discrepancy; in three of them, there was also lower limb length discrepancy. In the remaining patient, only lower limb length discrepancy was found. Whole-exome sequencing from DNA tissue/blood detected previously described pathogenic somatic mutations on DDR2 (c.314G > A; p.Arg105His), GRHL2 (c.791A > G; p.Glu264Gly), and PIK3CA (c.2740G > A; p.Gly914Arg) genes.
We propose the term "diffuse capillary malformation with undergrowth" for extensive reticular CMs associated with proportionate undergrowth. All our patients had a favorable outcome, and no genotype-phenotype association was found.
弥漫性毛细血管畸形伴过度生长(DCMO)已有详细描述。然而,毛细血管畸形伴生长不足(CMU)在文献中的报道较少。
我们旨在描述CMU患者的临床特征并确定相关的体细胞突变。
我们在多学科血管异常诊所数据库中搜索CMU患者。进行肢体周长和长度测量。若长度存在差异,则进行长腿X线片检查。对血液和受累组织的DNA进行全外显子组测序。
我们纳入了6例伴有软组织和骨骼生长不足的CM患者。CM表现为斑片状、网状、节段性,边界不清,为影响下肢(5例患者)或整个半侧身体(1例患者)的粉红色至红色斑。5例患者的斑为弥漫性,累及不止一个解剖区域。3例患者可见明显的浅表静脉。5例患者存在下肢周长差异;其中3例还存在下肢长度差异。在其余1例患者中,仅发现下肢长度差异。从DNA组织/血液中进行的全外显子组测序检测到DDR2(c.314G>A;p.Arg105His)、GRHL2(c.791A>G;p.Glu264Gly)和PIK3CA(c.2740G>A;p.Gly914Arg)基因上先前描述的致病性体细胞突变。
我们提出“弥漫性毛细血管畸形伴生长不足”这一术语,用于描述与成比例生长不足相关的广泛性网状CM。我们所有患者的预后良好,未发现基因型与表型之间的关联。