Oral H, Guven D C, Özdemir D Ateş, Usubütün A, Gonc N, Arik Z
Department of Internal Medicine, Hacettepe University Faculty of Medicine, Ankara, Turkey.
Department of Internal Medicine, Division of Oncology, Hacettepe University Faculty of Medicine, Ankara, Turkey.
Acta Endocrinol (Buchar). 2022 Oct-Dec;18(4):508-511. doi: 10.4183/aeb.2022.508.
Proprotein convertase 1/3 (PC 1/3) deficiency is a rare, autosomal recessive disorder caused by mutations in the PCSK1 gene. The disease is characterized by early-onset chronic diarrhea/malabsorption, followed by severe obesity and hormonal deficiencies such as hypocortisolism, hypothyroidism, diabetes insipidus, hypogonadism, growth deficiency, and diabetes mellitus. Ewing's sarcoma is a rare tumor, usually of small dimensions of neuroectodermal origin that is difficult to distinguish pathologically from a primitive neuroectodermal tumor. A 22-year-old female patient with PC 1/3 deficiency was admitted to our clinic with recurrent urinary tract infections. Magnetic resonance imaging (MRI) revealed an 11x12 cm pelvic mass displacing the uterus. A core-needle biopsy was performed on the pelvic mass. As a result of the pathological evaluation, ıt was diagnosed with pelvic Ewing's sarcoma. The patient was started on the VAC-IE chemotherapy protocol. We report a case of pelvic Ewing's sarcoma in a patient with PC 1/3 deficiency. Further research is needed to assess malignancy risk in metabolic disorders including very rare disorders like PC 1/3 deficiency.
前蛋白转化酶1/3(PC 1/3)缺乏症是一种罕见的常染色体隐性疾病,由PCSK1基因突变引起。该疾病的特征是早发性慢性腹泻/吸收不良,随后出现严重肥胖和激素缺乏,如皮质醇减少症、甲状腺功能减退、尿崩症、性腺功能减退、生长发育迟缓以及糖尿病。尤因肉瘤是一种罕见肿瘤,通常体积较小,起源于神经外胚层,在病理上难以与原始神经外胚层肿瘤区分开来。一名患有PC 1/3缺乏症的22岁女性患者因反复尿路感染入住我院。磁共振成像(MRI)显示盆腔有一个11×12厘米的肿块,使子宫移位。对盆腔肿块进行了粗针活检。病理评估结果显示,该患者被诊断为盆腔尤因肉瘤。患者开始接受VAC-IE化疗方案治疗。我们报告了一例患有PC 1/3缺乏症的患者发生盆腔尤因肉瘤的病例。需要进一步研究来评估包括PC 1/3缺乏症等非常罕见疾病在内的代谢紊乱中的恶性肿瘤风险。