Department of Pediatric Hematology/Oncology, Eskişehir Osmangazi University Faculty of Medicine, Ekişehir, Turkey.
Department of Pediatric Neurology, Eskişehir Osmangazi University Faculty of Medicine, Ekişehir, Turkey.
Pediatr Blood Cancer. 2024 Jul;71(7):e30773. doi: 10.1002/pbc.30773. Epub 2024 Apr 21.
The complementation Q group (FANCQ) subtype of Fanconi anemia (FA) caused by the ERCC4/XPF mutation is very rare. Two siblings, aged 13 and 10 with Fanconi phenotypic features, presented with right hemiparesis and focal-onset seizures. In both cases, cranial magnetic resonance imaging (MRI) showed mass-like lesions accompanied by peripheral edema and calcification. In one case, oral steroid treatment and surgical excision were performed, while in the other case, the cranial lesion regressed just with steroid treatment and without surgery. Both siblings remained wheelchair-bound due to neurological dysfunction. One case died due to hepatocellular carcinoma. ERCC4/XPF gene mutation was detected in both siblings.
FA 是一种罕见的常染色体隐性遗传性疾病,由 ERCC4/XPF 基因突变导致的 Q 组(FANCQ)亚型更为罕见。我们报道了 2 例 FA 患儿,均为同胞兄妹,年龄分别为 13 岁和 10 岁,均表现为范可尼贫血表型特征,伴有右侧偏瘫和局灶性癫痫发作。头颅磁共振成像(MRI)显示,2 例患儿均存在肿块样病变,伴有周围水肿和钙化。其中 1 例接受了口服皮质类固醇治疗和手术切除,另 1 例仅接受皮质类固醇治疗,未行手术,颅内病变消退。由于神经功能障碍,2 例患儿均遗留有轮椅依赖。其中 1 例患儿因肝细胞癌死亡。2 例患儿均检测到 ERCC4/XPF 基因突变。