Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation.
Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation.
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DOI：
10.1371/journal.pone.0194193
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发表时间：
2018
期刊：
影响因子：
3.7
通讯作者：
Gochuico BR
中科院分区：
文献类型：
--
作者：
El-Chemaly S;O'Brien KJ;Nathan SD;Weinhouse GL;Goldberg HJ;Connors JM;Cui Y;Astor TL;Camp PC Jr;Rosas IO;Lemma M;Speransky V;Merideth MA;Gahl WA;Gochuico BR
肺纤维化是Hermansky-Pudlak综合征(HPS)的一种进行性、致命性表现。一些晚期HPS肺纤维化患者接受了肺移植，尽管他们有疾病相关的出血倾向；另一些患者在等待捐献器官的过程中死亡。本研究的目的是确定一组接受肺移植评估的晚期HPS肺纤维化患者的临床处理和结果。在国立卫生研究院临床中心和两个区域肺移植中心之一对6名HPS-1肺纤维化患者进行了评估。他们移植前的中位年龄为41.5岁。六名患者中有三名在没有接受肺移植的情况下死亡。其中一名患者与终末期肺纤维化有关，并在获得供体器官之前死亡，另外两名因先前输血产品致敏的患者的供体器官尚未确定。六名患者中有三名接受了双侧肺移植；他们没有过度出血的病史。1例围手术期应用去氨加压素，1例术中输注血小板，1例术中应用体外膜氧合联合凝血酶原复合体、血小板输注和去氨加压素。一名移植受者经历了对脉冲类固醇有反应的急性排斥反应。这三个肺移植受者在移植后6年内没有发现慢性肺移植功能障碍或HPS肺纤维化复发的证据。综上所述，肺移植和体外膜肺氧合是治疗HPS肺纤维化的可行选择。HPS患者的同种异体免疫是肺移植的一个重要和潜在的可预防的障碍；应对有肺纤维化风险的HPS患者实施限制同种免疫的干预措施，以优化他们未来肺移植的候选条件。
Pulmonary fibrosis is a progressive, fatal manifestation of Hermansky-Pudlak syndrome (HPS). Some patients with advanced HPS pulmonary fibrosis undergo lung transplantation despite their disease-associated bleeding tendency; others die while awaiting donor organs. The objective of this study is to determine the clinical management and outcomes of a cohort with advanced HPS pulmonary fibrosis who were evaluated for lung transplantation. Six patients with HPS-1 pulmonary fibrosis were evaluated at the National Institutes of Health Clinical Center and one of two regional lung transplant centers. Their median age was 41.5 years pre-transplant. Three of six patients died without receiving a lung transplant. One of these was referred with end-stage pulmonary fibrosis and died before a donor organ became available, and donor organs were not identified for two other patients sensitized from prior blood product transfusions. Three of six patients received bilateral lung transplants; they did not have a history of excessive bleeding. One patient received peri-operative desmopressin, one was transfused with intra-operative platelets, and one received extracorporeal membrane oxygenation and intra-operative prothrombin complex concentrate, platelet transfusion, and desmopressin. One transplant recipient experienced acute rejection that responded to pulsed steroids. No evidence of chronic lung allograft dysfunction or recurrence of HPS pulmonary fibrosis was detected up to 6 years post-transplant in these three lung transplant recipients. In conclusion, lung transplantation and extracorporeal membrane oxygenation are viable options for patients with HPS pulmonary fibrosis. Alloimmunization in HPS patients is an important and potentially preventable barrier to lung transplantation; interventions to limit alloimmunization should be implemented in HPS patients at risk of pulmonary fibrosis to optimize their candidacy for future lung transplants.
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影响因子：
3.8
作者：
O'Brien, Kevin;Troendle, James;Gochuico, Bernadette R.;Markello, Thomas C.;Salas, Jose;Cardona, Hilda;Yao, Jianhua;Bernardini, Isa;Hess, Richard;Gahl, William A.
通讯作者：
Gahl, William A.
影响因子：
3.1
作者：
Kistler KD;Nalysnyk L;Rotella P;Esser D
通讯作者：
Esser D
影响因子：
8.8
作者：
Tinckam, K. J.;Keshavjee, S.;Singer, L. G.
通讯作者：
Singer, L. G.
影响因子：
3.2
作者：
Cordova, A;Barrios, NJ;Santiago-Borrero, PJ
通讯作者：
Santiago-Borrero, PJ
影响因子：
0.5
作者：
de Montpreville, Vincent Thomas;Mussot, Sacha;Dartevelle, Philippe
通讯作者：
Dartevelle, Philippe