近日，美国杜克大学Joseph W. Turek团队研究了先天性心脏病的部分心脏移植的疗效。2025年8月27日出版的《美国医学会杂志》发表了这项成果。

部分心脏移植，或活体瓣膜置换术，通过提供一种具有生长能力的可行选择，有可能推进对不可修复的瓣膜疾病的外科治疗。该研究旨在描述先天性心脏瓣膜病患者部分心脏移植的早期经验，并评估其可行性、安全性和有效性。

2022年4月至2024年12月，研究组招募在美国一个大容量儿童心脏手术和移植中心接受部分心脏移植的前19名患者的病例系列。没有患者被排除或丢失随访。利用供体心脏半月瓣进行部分心脏移植。维持性免疫抑制包括他克莫司单药治疗，谷水平目标为4至8 ng/mL。主要疗效的定义是移植的瓣膜环和小叶随时间的生长。次要结局包括瓣膜功能障碍和免疫抑制相关并发症。

在19名患有不可修复的先天性心脏瓣膜功能障碍的参与者中，53%为男性，47%为女性。移植时的中位年龄为97天。中位随访时间为26周。3例患者接受了两个半月瓣的部分心脏移植，7例患者在肺位行活体肺动脉瓣置换术，2例在主动脉位行活体同种异体主动脉瓣移植，7例在肺位行活体同种异体主动脉瓣移植。9例患者构成部分心脏移植接受者的初始队列，他们的环直径和瓣膜小叶长度纵向分析生长情况。所有瓣膜功能良好，并显示出沿适当的z分数增长。主动脉瓣和肺动脉瓣的中位直径分别由7毫米和9毫米增加到14毫米和17毫米。小叶长度同样从0.5 mm（主动脉瓣）和0.49 mm（肺动脉瓣）的中位数分别增加到1 mm（主动脉瓣）和0.675 mm（肺动脉瓣）。一名患者需要与植入瓣膜无关的再次手术。未见与免疫抑制相关的明显并发症。

研究结果表明，部分心脏移植是可行、安全、有效的。所有移植的瓣膜都显示出基于环和小叶长度测量的生长。仔细的跟踪和监测对于支持这种新技术的持续扩展至关重要。

附：英文原文

Title: Partial Heart Transplant for Congenital Heart Disease

Author: Douglas M. Overbey, Berk Aykut, John A. Kucera, Cathlyn K. Medina, Neeta J. Sethi, Piers C. A. Barker, Erin V. Shea, Joseph W. Turek

Issue&Volume: 2025-08-27

Abstract:

Importance  Partial heart transplant, or living valve replacement, has the potential to advance surgical management of irreparable valvular disease by providing a viable option with capacity for growth.

Objectives  To describe the early experience and assess the feasibility, safety, and efficacy of partial heart transplant in patients with congenital heart valve disease.

Design, Setting, and Participants  Case series of the first 19 patients to undergo partial heart transplant at a single high-volume pediatric cardiac surgery and transplant center in the US between April 2022 and December 2024. No patients were excluded or lost to follow-up.

Exposures  Partial heart transplant using semilunar valves from donor hearts. Maintenance immunosuppression consisted of tacrolimus monotherapy with a trough level goal of 4 to 8 ng/mL.

Main Outcomes and Measures  Efficacy was defined as growth of the transplanted valve annulus and leaflets over time. Secondary outcomes included valve dysfunction and complications related to immunosuppression.

Results  Among the 19 participants with irreparable congenital heart valve dysfunction, 53% were male and 47% female. The median age at the time of transplant was 97 days. The median follow-up was 26 weeks. Three patients received partial heart transplant of both semilunar valves, 7 underwent living pulmonary valve replacement in the pulmonary position, 2 had a living aortic valve allograft in the aortic position, and 7 had a living aortic valve allograft in the pulmonary position. Nine patients constituting the initial cohort of partial heart transplant recipients had their annular diameter and valve leaflet length longitudinally analyzed for growth. All valves functioned well and demonstrated growth along appropriate z scores. Annular diameter increased from medians of 7 mm (aortic valve) and 9 mm (pulmonary valve) to 14 mm (aortic valve) and 17 mm (pulmonary valve), respectively. Leaflet length similarly increased from medians of 0.5 mm (aortic valve) and 0.49 mm (pulmonary valve) to 1 mm (aortic valve) and 0.675 mm (pulmonary valve), respectively. One patient required reoperation unrelated to the implanted valve. No significant complications related to immunosuppression were observed.

Conclusions and Relevance  Partial heart transplant appears feasible, safe, and efficacious. All transplanted valves demonstrated growth based on annular and leaflet length measurements. Careful follow-up and monitoring are crucial to support the continued expansion of this novel technique.

DOI: 10.1001/jama.2025.13580

Source: https://jamanetwork.com/journals/jama/fullarticle/2838086