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Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation

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 d...

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Autores principales: El-Chemaly, Souheil, O’Brien, Kevin J., Nathan, Steven D., Weinhouse, Gerald L., Goldberg, Hilary J., Connors, Jean M., Cui, Ye, Astor, Todd L., Camp, Philip C., Rosas, Ivan O., Lemma, Merte, Speransky, Vladislav, Merideth, Melissa A., Gahl, William A., Gochuico, Bernadette R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5856338/
https://www.ncbi.nlm.nih.gov/pubmed/29547626
http://dx.doi.org/10.1371/journal.pone.0194193
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author El-Chemaly, Souheil
O’Brien, Kevin J.
Nathan, Steven D.
Weinhouse, Gerald L.
Goldberg, Hilary J.
Connors, Jean M.
Cui, Ye
Astor, Todd L.
Camp, Philip C.
Rosas, Ivan O.
Lemma, Merte
Speransky, Vladislav
Merideth, Melissa A.
Gahl, William A.
Gochuico, Bernadette R.
author_facet El-Chemaly, Souheil
O’Brien, Kevin J.
Nathan, Steven D.
Weinhouse, Gerald L.
Goldberg, Hilary J.
Connors, Jean M.
Cui, Ye
Astor, Todd L.
Camp, Philip C.
Rosas, Ivan O.
Lemma, Merte
Speransky, Vladislav
Merideth, Melissa A.
Gahl, William A.
Gochuico, Bernadette R.
author_sort El-Chemaly, Souheil
collection PubMed
description 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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spelling pubmed-58563382018-03-28 Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation El-Chemaly, Souheil O’Brien, Kevin J. Nathan, Steven D. Weinhouse, Gerald L. Goldberg, Hilary J. Connors, Jean M. Cui, Ye Astor, Todd L. Camp, Philip C. Rosas, Ivan O. Lemma, Merte Speransky, Vladislav Merideth, Melissa A. Gahl, William A. Gochuico, Bernadette R. PLoS One Research Article 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. Public Library of Science 2018-03-16 /pmc/articles/PMC5856338/ /pubmed/29547626 http://dx.doi.org/10.1371/journal.pone.0194193 Text en https://creativecommons.org/publicdomain/zero/1.0/ This is an open access article, free of all copyright, and may be freely reproduced, distributed, transmitted, modified, built upon, or otherwise used by anyone for any lawful purpose. The work is made available under the Creative Commons CC0 (https://creativecommons.org/publicdomain/zero/1.0/) public domain dedication.
spellingShingle Research Article
El-Chemaly, Souheil
O’Brien, Kevin J.
Nathan, Steven D.
Weinhouse, Gerald L.
Goldberg, Hilary J.
Connors, Jean M.
Cui, Ye
Astor, Todd L.
Camp, Philip C.
Rosas, Ivan O.
Lemma, Merte
Speransky, Vladislav
Merideth, Melissa A.
Gahl, William A.
Gochuico, Bernadette R.
Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation
title Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation
title_full Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation
title_fullStr Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation
title_full_unstemmed Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation
title_short Clinical management and outcomes of patients with Hermansky-Pudlak syndrome pulmonary fibrosis evaluated for lung transplantation
title_sort clinical management and outcomes of patients with hermansky-pudlak syndrome pulmonary fibrosis evaluated for lung transplantation
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5856338/
https://www.ncbi.nlm.nih.gov/pubmed/29547626
http://dx.doi.org/10.1371/journal.pone.0194193
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