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| 2. |
- Mercuri, E., et al.
(författare)
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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- 2020
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
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Tidskriftsartikel (refereegranskat)abstract
- Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p <= 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
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| 4. |
- Diswall, Mette, 1979, et al.
(författare)
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Glycolipid studies in small intestine and pancreas of alpha1,3-galactosyltransferase knockout miniature swine: alpha1,3GALT-KO animals lack alphaGAL antigens and contain novel blood group H compounds.
- 2008
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Ingår i: Transplantation proceedings. - : Elsevier BV. - 0041-1345. ; 40:2, s. 543-6
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Tidskriftsartikel (refereegranskat)abstract
- BACKGROUND: To avoid hyperacute rejection of xeno-organs, alpha1,3-galactosyltransferase knock-out (GalT-KO) pigs have been produced. However, Galalpha1,3Gal (Gal) determinant elimination may expose cryptic carbohydrate antigens and/or generate new antigens that might interfere with the human immune response. METHODS: Glycolipids isolated from small intestine and pancreas of two GalT-KO and one wild-type (WT) pig were tested for immune reactivity with antibodies on thin-layer chromatograms after separation by high-performance liquid chromatography, and selected fractions were analysed by proton NMR spectroscopy. RESULTS: Immunostaining using purified human anti-Gal Abs revealed that tissues from WT animals express large amounts of Gal-antigens whereas GalT-KO tissues lacked these antigens. Proton NMR spectroscopy on small intestine fractions revealed both linear and branched nona- and decaglycosylceramides, respectively, with terminal Gal-epitopes. In corresponding GalT-KO fractions, Gal-epitopes seemed to be replaced by terminal alpha1,2fucoses. Two novel branched blood group H compounds was found in the GalT-KO intestine. CONCLUSIONS: The structural complexity of alphaGal-terminating antigens in the WT organs is very high. Knockout of alpha1,3GalT by gene-targeting results in elimination of Gal-determinants. In addition structurally novel alpha1,2fucose-terminated blood group H compounds were identified in the GalT-KO tissue. These compounds are not expected to be recognized by the human immune system.
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| 5. |
- Van Dellen, D., et al.
(författare)
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Pre-emptive live donor kidney transplantation-moving barriers to opportunities: An ethical, legal and psychological aspects of organ transplantation view
- 2021
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Ingår i: World Journal of Transplantation. - 2220-3230. ; 11:4, s. 88-98
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Tidskriftsartikel (refereegranskat)abstract
- Live donor kidney transplantation (LDKT) is the optimal treatment modality for end stage renal disease (ESRD), enhancing patient and graft survival. Pre-emptive LDKT, prior to requirement for renal replacement therapy (RRT), provides further advantages, due to uraemia and dialysis avoidance. There are a number of potential barriers and opportunities to promoting pre-emptive LDKT. Significant infrastructure is needed to deliver robust programmes, which varies based on socio-economic standards. National frameworks can impact on national prioritisation of pre-emptive LDKT and supporting education programmes. Focus on other programme's components, including deceased kidney transplantation and RRT, can also hamper uptake. LDKT programmes are designed to provide maximal benefit to the recipient, which is specifically true for pre-emptive transplantation. Health care providers need to be educated to maximize early LDKT referral. Equitable access for varying population groups, without socioeconomic bias, also requires prioritisation. Cultural barriers, including religious influence, also need consideration in developing successful outcomes. In addition, the benefit of pre-emptive LDKT needs to be emphasised, and opportunities provided to potential donors, to ensure timely and safe work-up processes. Recipient education and preparation for pre-emptive LDKT needs to ensure increased uptake. Awareness of the benefits of pre-emptive transplantation require prioritisation for this population group. We recommend an approach where patients approaching ESRD are referred early to pre-transplant clinics facilitating early discussion regarding pre-emptive LDKT and potential donors for LDKT are prioritized for work-up to ensure success. Education regarding preemptive LDKT should be the norm for patients approaching ESRD, appropriate for the patient's cultural needs and physical status. Pre-emptive transplantation maximize benefit to potential recipients, with the potential to occur within successful service delivery. To fully embrace preemptive transplantation as the norm, investment in infrastructure, increased awareness, and donor and recipient support is required. 2021 © adno.no.All right reserved.
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| 7. |
- Courtney, Aisling E, et al.
(författare)
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Living Donor Kidney Transplantation in Older Individuals: An Ethical Legal and Psychological Aspects of Transplantation (ELPAT) View.
- 2023
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Ingår i: Transplant international : official journal of the European Society for Organ Transplantation. - 1432-2277. ; 36
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Tidskriftsartikel (refereegranskat)abstract
- Living donor transplantation is the optimal treatment for suitable patients with end-stage kidney disease. There are particular advantages for older individuals in terms of elective surgery, timely transplantation, and early graft function. Yet, despite the superiority of living donor transplantation especially for this cohort, older patients are significantly less likely to access this treatment modality than younger age groups. However, given the changing population demographic in recent decades, there are increasing numbers of older but otherwise healthy individuals with kidney disease who could benefit from living donor transplantation. The complex reasons for this inequity of access are explored, including conscious and unconscious age-related bias by healthcare professionals, concerns relating to older living donors, ethical anxieties related to younger adults donating to aging patients, unwillingness of potential older recipients to consider living donation, and the relevant legislation. There is a legal and moral duty to consider the inequity of access to living donor transplantation, recognising both the potential disparity between chronological and physiological age in older patients, and benefits of this treatment for individuals as well as society.
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| 8. |
- Diswall, Mette, 1979, et al.
(författare)
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Studies on glycolipid antigens in small intestine and pancreas from alpha1,3-galactosyltransferase knockout miniature swine.
- 2007
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Ingår i: Transplantation. - : Ovid Technologies (Wolters Kluwer Health). - 0041-1337. ; 84:10, s. 1348-56
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Tidskriftsartikel (refereegranskat)abstract
- BACKGROUND: To avoid hyperacute rejection of xeno-organs, alpha1,3-galactosyltransferase knockout (GalT-KO) pigs have been produced. Galalpha1,3Gal determinant elimination may expose cryptic carbohydrate antigens and/or generate new antigens. This is the first biochemical study of carbohydrate antigens in GalT-KO pig organs. METHODS: Neutral and acidic glycolipids were isolated from small intestine and pancreas of two GalT-KO and one wild-type (WT) pig. Glycolipid immune reactivity was tested on thin-layer chromatograms. Small intestine neutral glycolipids were separated by high-performance liquid chromatography and selected fractions were analyzed by proton nuclear magnetic resonance spectroscopy. Total gangliosides were quantified on thin-layer chromatograms and in microtiter wells. RESULTS: Using Galalpha1,3nLc4 glycolipid reference, total Galalpha1,3Gal glycolipid antigens in the WT animal was estimated at about 30 microg (small intestine) and 3 microg (pancreas) per gram of dry tissue. Galalpha1,3Gal determinants were not detected in GalT-KO tissues at a detection limit of less than 0.25% (small intestine) and 0.5% (pancreas) of the WT tissues. Isoglobotriaosylceramide (iGb3) was absent but trace amounts of Fuc-iGb3 was found in both GalT-KO and WT pig small intestine. Blood group H type 2 core saccharide compounds were increased in GalT-KO pancreas. Total amount of gangliosides was decreased in GalT-KO tissues. The alpha1,3-galactosyltransferase acceptor, N-acetyllactosamine determinant, was not increased in GalT-KO tissues. Human serum antibodies reacted with WT organ Galalpha1,3Gal antigens and gangliosides, of which the ganglioside reactivity remained in GalT-KO tissues. CONCLUSIONS: Knockout of porcine alpha1,3-galactosyltransferase gene results in elimination of Galalpha1,3Gal-terminated glycolipid compounds. GalT-KO genetic modification did not produce new compensatory glycolipid compounds reactive with human serum antibodies.
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| 9. |
- Dor, Frank J M F, et al.
(författare)
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New classification of ELPAT for living organ donation.
- 2011
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Ingår i: Transplantation. - 1534-6080. ; 91:9, s. 935-8
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Tidskriftsartikel (refereegranskat)abstract
- In the literature, varying terminology for living organ donation can be found. However, there seems to be a need for a new classification to avoid confusion. Therefore, we assessed existing terminology in the light of current living organ donation practices and suggest a more straightforward classification. We propose to concentrate on the degree of specificity with which donors identify intended recipients and to subsequently verify whether the donation to these recipients occurs directly or indirectly. According to this approach, one could distinguish between "specified" and "unspecified" donation. Within specified donation, a distinction can be made between "direct" and "indirect" donation.
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| 10. |
- Lennerling, Annette, 1963, et al.
(författare)
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Living organ donation practices in Europe - results from an online survey.
- 2013
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Ingår i: Transplant international : official journal of the European Society for Organ Transplantation. - : Frontiers Media SA. - 1432-2277. ; 26:2, s. 145-53
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Tidskriftsartikel (refereegranskat)abstract
- In Europe, living organ donation (LOD) is increasingly accepted as a valuable solution to overcome the organ shortage. However, considerable differences exist between European countries regarding frequency, practices and acceptance of donor-recipient relations. As a response, the Coordination Action project 'Living Organ Donation in Europe' (www.eulod.eu), funded by the Seventh Framework Programme of the European Commission, was initiated. Transplant professionals from 331 European kidney and liver transplant centres were invited to complete an online survey on living kidney donation (LKD) and living liver donation (LLD). In total, 113 kidney transplant centres from 40 countries and 39 liver transplant centres from 24 countries responded. 96.5% and 71.8% performed LKD and LLD respectively. The content of the medical screening of donors was similar, but criteria for donor acceptance varied. Few absolute contraindications for donation existed. The reimbursement policies diverged and the majority of the donors did not get reimbursed for their income loss during recovery. Large discrepancies were found between geographical European regions (the Eastern, the Mediterranean and the North-Western). As a result of this survey we suggest several recommendations to improve quality and safety of LOD in Europe.
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