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血红蛋白巴氏水肿胎儿综合征存活患者的结局与发病情况:20年回顾性研究

Outcomes and morbidities of patients who survive haemoglobin Bart's hydrops fetalis syndrome: 20-year retrospective review.

作者信息

Chan W Yk, Leung A Wk, Luk C W, Li R Ch, Ling A Sc, Ha S Y

机构信息

Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, Pokfulam, Hong Kong.

Department of Paediatrics and Adolescent Medicine, Prince of Wales Hospital, Shatin, Hong Kong.

出版信息

Hong Kong Med J. 2018 Apr;24(2):107-118. doi: 10.12809/hkmj176336. Epub 2018 Apr 6.

Abstract

INTRODUCTION

Haemoglobin Bart's hydrops fetalis syndrome was once considered a fatal condition. However, advances over the past two decades have enabled survival of affected patients. Data relating to their morbidities and outcomes will help medical specialists formulate a management plan and parental counselling.

METHODS

All babies with the syndrome who survived beyond the neonatal period and were subsequently managed long-term in eight public hospitals in Hong Kong from 1 January 1996 to 31 December 2015 were included. Patient and parent characteristics, antenatal care, reasons for continuation of pregnancy, intrauterine interventions, perinatal course, presence of congenital malformations, stem-cell transplantation details, and long-term neurodevelopmental outcomes were reviewed.

RESULTS

A total of nine patients were identified, of whom five were female and four male. The median follow-up duration was 7 years. All were Chinese and were homozygous for the Southeast Asian α-thalassaemia deletion. Five of the nine mothers received antenatal care at a public hospital and opted to continue the pregnancy after antenatal diagnosis and counselling. Despite intrauterine transfusions, all babies were born with respiratory depression and required intubation and mechanical ventilation during the neonatal period. Hypospadias was identified in all four male infants. Growth retardation, global developmental delay, and residual neurological deficits were noted in two-thirds of the patients. Haematopoietic stem-cell transplantation was performed in two patients, who became transfusion-independent.

CONCLUSIONS

Survival of patients with Bart's hydrops fetalis syndrome is possible but not without short- and long-term complications; local epidemiology is comparable to that documented for an international registry. Detailed antenatal counselling of parents with a non-judgemental attitude and cautious optimism are imperative.

摘要

引言

血红蛋白巴氏水肿胎儿综合征曾被认为是一种致命疾病。然而,过去二十年的进展使受影响的患者能够存活下来。有关其发病率和预后的数据将有助于医学专家制定管理计划并为家长提供咨询。

方法

纳入1996年1月1日至2015年12月31日期间在香港八家公立医院存活至新生儿期后并接受长期治疗的所有该综合征婴儿。回顾了患者及家长的特征、产前护理、继续妊娠的原因、宫内干预、围产期过程、先天性畸形的存在、干细胞移植细节以及长期神经发育结局。

结果

共确定了9名患者,其中5名女性,4名男性。中位随访时间为7年。所有患者均为中国人,且均为东南亚α地中海贫血缺失纯合子。9名母亲中有5名在公立医院接受了产前护理,并在产前诊断和咨询后选择继续妊娠。尽管进行了宫内输血,但所有婴儿出生时均有呼吸抑制,在新生儿期需要插管和机械通气。所有4名男婴均被诊断为尿道下裂。三分之二的患者存在生长发育迟缓、全面发育迟缓以及残留神经功能缺损。两名患者接受了造血干细胞移植,术后不再依赖输血。

结论

血红蛋白巴氏水肿胎儿综合征患者有可能存活,但会伴有短期和长期并发症;当地的流行病学情况与国际登记处记录的情况相当。必须以无偏见的态度并持谨慎乐观的态度为家长提供详细的产前咨询。

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