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Abstract：
Mulibrey-Nanism (Muscle-liver-brain-eye Nanism = dwarfism; MUL) is a rare genetic syndrome. The underlying TRIM37 mutation predisposes these children to develop tumors frequently. In the largest published series of MUL, 8% patients were reported to develop Wilms tumor (WT). The published literature lacks data regarding the best treatment protocol and outcome of this cohort of children with WT and MUL. We report here a 2-year-old boy with WT and MUL and present a review of literature on WT in MUL.
Our patient had associated cardiac problems of atrial septal defect, atrial flutter and an episode of sudden cardiac arrest. We managed him successfully with chemotherapy, surgery and multi-speciality care. He is alive and in remission at follow-up of 6 months.
A total of 14 cases (including present case) of WT have been reported in MUL and treatment details were available for six cases. They were managed primarily with surgery, chemotherapy with/without radiotherapy, and all achieved remission. The outcome data is available only for two cases, one has been followed up till 15 years post treatment for WT and other is our patient.
Our patient had associated cardiac problems of atrial septal defect, atrial flutter and an episode of sudden cardiac arrest. We managed him successfully with chemotherapy, surgery and multi-speciality care. He is alive and in remission at follow-up of 6 months.
A total of 14 cases (including present case) of WT have been reported in MUL and treatment details were available for six cases. They were managed primarily with surgery, chemotherapy with/without radiotherapy, and all achieved remission. The outcome data is available only for two cases, one has been followed up till 15 years post treatment for WT and other is our patient.
摘要：
背景：Mulibrey-Nanism（肌-肝-脑-眼Nanism=侏儒症；MUL）是一种罕见的遗传综合征。潜在的TRIM37突变使这些儿童经常发生肿瘤。在MUL出版的最大系列中，据报道8%的患者发展为Wilms肿瘤(WT)。已发表的文献缺乏有关WT和MUL儿童队列的最佳治疗方案和结果的数据。我们在这里报告了一个患有WT和MUL的2岁男孩，并对MUL中WT的文献进行了综述。
方法：我们的患者有房间隔缺损的相关心脏问题，房扑和心脏骤停的发作。我们用化疗成功控制了他，手术和多专业护理。他还活着，并在6个月的随访中缓解。
结论：在MUL中报告了总共14例WT（包括目前的病例），并且有6例的治疗细节。他们主要通过手术来管理，有/无放疗的化疗，都实现了缓解。结果数据仅适用于两种情况，其中一位在WT治疗后随访至15年，另一位是我们的患者。
方法：我们的患者有房间隔缺损的相关心脏问题，房扑和心脏骤停的发作。我们用化疗成功控制了他，手术和多专业护理。他还活着，并在6个月的随访中缓解。
结论：在MUL中报告了总共14例WT（包括目前的病例），并且有6例的治疗细节。他们主要通过手术来管理，有/无放疗的化疗，都实现了缓解。结果数据仅适用于两种情况，其中一位在WT治疗后随访至15年，另一位是我们的患者。
