Organ donation from patients with a rare disease is often safe: the italian guidelines.
guidelines
organ donors
rare disease
risk assessment
Journal
Clinical transplantation
ISSN: 1399-0012
Titre abrégé: Clin Transplant
Pays: Denmark
ID NLM: 8710240
Informations de publication
Date de publication:
09 2022
09 2022
Historique:
revised:
19
05
2022
received:
09
02
2022
accepted:
04
07
2022
pubmed:
31
7
2022
medline:
21
9
2022
entrez:
30
7
2022
Statut:
ppublish
Résumé
Although a disease is defined as rare when it has a prevalence of less than 1:2000, the overall prevalence of rare diseases in the population is greater than 1%. Among potential organ donors, a similar frequency is observed. To date, guidelines have not been established, and operational decisions have been made empirically, case- by-case, based on the experience and expertise of clinicians. For this reason, the Italian Superior Health Council (CSS) has appointed a working Group to address "patients with a rare disease as potential organ donors," with the aim of devising recommendations for the management of transplant cases in which the donors have a rare disease. This group evaluated 493 diseases (10% of all rare diseases, including over 95% of patients with a rare disease) to deliver a technical report dealing with the suitability of organ donation and transplantation, with a focus on the organs most frequently used, including kidney, liver, heart, lung, and pancreas. This work has made it clear that a rare disease "per se" does not contraindicate organ donation at all. Indeed, in donors affected by a rare disease, almost 80% of the organs are suitable for transplantation, approximately 7% are unsuitable, and approximately 14% are suitable as non-standard with an acceptable risk.
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e14769Informations de copyright
© 2022 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
Références
Solid Organ Donor Suitability Assessment Protocol, defined by the Italian law: CONFERENZA STATO-REGIONI DEL 24.01.2018: Accordo, ai sensi dell'articolo 4 del decreto legislativo 28 agosto 1997, n. 281, tra il Governo, le Regioni e le Province autonome di Trento e Bolzano sul documento recante “Protocollo per la valutazione di idoneità del donatore di organi solidi”. Repertorio Atti n: 17/CSR del 24/01/2018
United States Food and Drug Administration. Orphan Drug Act. Public Law No. 97-414 96 Stat. 2049. 1983.
European Parliament and the Council of the European Union. Decision No 1295/1999/EC of the European Parliament and of the Council of 29 April 1999 adopting a programme of Community action on rare diseases within the framework for action in the field of public health (1999 to 2003). 1999. Accessed August 26, 2021. http://eur-lex.europa.eu/legal-content/EN/TXT/?qid=1395750802170&uri=CELEX:31999D1295
Richter T, Nestler-Parr S, Babela R, Khan ZM, Tesoro T, Molsen E. Rare disease terminology and definitions-a systematic global review: report of the ISPOR Rare Disease Special Interest Group. Value Health. 2015;18:906-914.
Moliner AM, Waligóra J. The European Union policy in the field of rare diseases. Public Health Genomics. 2013;16:268-277.
European Organisation for Rare Diseases. Rare diseases: understanding this public health priority. Eurordis. 2005.
Mazzucato M, Facchin P, Salamanca E, Angin C, Rath A. Orphacodes’ use for the codification of rare diseases: results of the testing activity carried out within the rd-action framework. 9th European Conference on Rare Diseases & Orphan Products (ECRD Vienna 2018). Orphanet J Rare Dis. 2018;13(Suppl 2):167.
Koboldt DC, Steinberg KM, Larson DE, Wilson RK, Mardis ER. The next-generation sequencing revolution and its impact on genomics. Cell. 2013;155:27-38.
Baldovino S, Moliner AM, Taruscio D, Daina E, Roccatello D. Rare diseases in europe: from a wide to a local perspective. Isr Med Assoc J. 2016;18(6):359-363. 14.
Report EURORDIS 2005. European Conference on Rare Diseases. 2005. Luxembourg. Accessed August 26, 2021. https://www.eurordis.org/IMG/pdf/EN-ECRDtotal-2.pdf
Jimenez-Sanchez G, Childs B, Valle D. (2014). The effect of Mendelian disease on human health. In: Beaudet AL, Vogelstein B, Kinzler KW, eds. The online metabolic and molecular bases of inherited disease (Vol. 1). The McGraw-Hill Companies; 2001.
Peritore D, Trapani S, La Rocca V, et al. Rare disease patients as potential organ donors. Transplant Proc. 2020;52(5):1522-1524.
European Committee on Organ Transplantation. Guide to the quality and safety of organs for transplantation. EDQM 7th ed. 2018.
Herden U, Grabhorn E, Santer R, et al. Surgical aspects of liver transplantation and domino liver transplantation in maple syrup urine disease: analysis of 15 donor-recipient pairs. Liver Transpl. 2019;25:889-900.
Orphanet Report Series. Prevalence of rare diseases: bibliographic data. January 2019. Accessed August 26, 2021. http://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf
Maldonado EM, Taha F, Rahman J, Rahman S. Systems biology approaches toward understanding primary mitochondrial diseases. Front Genet. 2019;10:19.
Rahman J, Rahman S. Mitochondrial medicine in the omics era. Lancet. 2018;391(10139):2560-2574.