Ogbuji Chukwunonye O, Ortega Lucio E, Ward Haven, Ugochukwu Nzubechukwu, Donthula Rakesh, Alapati Srilatha
Pediatrics, Texas Tech University Health Sciences Center, Amarillo, USA.
Pediatric Cardiology, Rady Children's Hospital, San Diego, USA.
Cureus. 2023 Sep 21;15(9):e45746. doi: 10.7759/cureus.45746. eCollection 2023 Sep.
1p36 deletion syndrome is a common terminal chromosomal deletion syndrome in humans. It is caused by the deletion of genetic material from a specific region in the short arm of chromosome 1. Symptoms range from seizure disorders, abnormalities of tone, visual and auditory disturbances. Cardiac abnormalities like left ventricular non-compaction (LVNC) and dilated cardiomyopathies (DCM) are commonly associated with this syndrome. This case report presents a 15-month-old female with dilated cardiomyopathy associated with 1p36 deletion syndrome, who has been followed from birth. Cardiac function was normal at birth with an ejection fraction of 65%. At three weeks of age, the patient presented with severe tachypnea, cyanosis, poor weight gain, and diaphoresis with feeding. Echocardiogram showed an ejection fraction of 22%. The patient was diagnosed with Modified Ross Heart Failure Class III. The patient was admitted to the cardiovascular intensive care unit where diuretics, phosphodiesterase inhibitors, and ionotropic agents were used to manage the heart failure. The patient relapsed two months later following a severe adenovirus infection. She was readmitted and heart failure medications were optimized. This patient has maintained a steady growth, meeting most milestones with no further relapse. The heterogeneity of 1p36 deletion syndrome presentation poses a diagnostic challenge for most clinicians. Cardiac involvements are very common and infants presenting with signs and symptoms of heart failure need to be screened for chromosomal abnormalities when other causes have been ruled out.
1p36缺失综合征是人类常见的末端染色体缺失综合征。它是由1号染色体短臂特定区域的遗传物质缺失引起的。症状包括癫痫障碍、肌张力异常、视觉和听觉障碍。心脏异常如左心室心肌致密化不全(LVNC)和扩张型心肌病(DCM)通常与该综合征相关。本病例报告介绍了一名15个月大患有与1p36缺失综合征相关的扩张型心肌病的女性,自出生起就接受随访。出生时心脏功能正常,射血分数为65%。三周大时,患者出现严重呼吸急促、紫绀、体重增加不佳以及喂食时出汗。超声心动图显示射血分数为22%。患者被诊断为改良罗斯心力衰竭III级。患者被收入心血管重症监护病房,使用利尿剂、磷酸二酯酶抑制剂和正性肌力药物来治疗心力衰竭。两个月后,患者在严重腺病毒感染后复发。她再次入院,心力衰竭药物治疗得到优化。该患者保持稳定生长,达到了大多数发育里程碑,未再复发。1p36缺失综合征表现的异质性给大多数临床医生带来了诊断挑战。心脏受累非常常见,当排除其他病因后,出现心力衰竭体征和症状的婴儿需要进行染色体异常筛查。