ADPedKD: A Global Online Platform on the Management of Children With ADPKD

Stéphanie de Rechter; Detlef Bockenhauer; Lisa M. Guay-Woodford; Isaac Liu; Andrew J. Mallett; Neveen A. Soliman; Lucimary C. Sylvestre; Franz Schaefer; Max C. Liebau; Djalila Mekahli; P. Adamczyk; N. Akinci; H. Alpay; C. Ardelean; N. Ayasreh; Z. Aydin; A. Bael; V. Baudouin; U. S. Bayrakci; A. Bensman; H. Bialkevich; A. Biebuyck; O. Boyer; O. Bjanid; A. Bryłka; S. Çalışkan; A. Cambier; A. Camelio; V. Carbone; M. Charbit; B. Chiodini; A. Chirita; N. Çiçek; R. Cerkauskiene; L. Collard; M. Conceiçao; I. Constantinescu; A. Couderc; B. Crapella; M. Cvetkovic; B. Dima; F. Diomeda; M. Docx; N. Dolan; C. Dossier; D. Drozdz; J. Drube; O. Dunand; P. Dusan; L. A. Eid; F. Emma; M. Espino Hernandez; M. Fila; M. Furlano; M. Gafencu; M. S. Ghuysen; M. Giani; M. Giordano; I. Girisgen; N. Godefroid; A. Godron-Dubrasquet; I. Gojkovic; E. Gonzalez; I. Gökçe; J. W. Groothoff; S. Guarino; A. Guffens; P. Hansen; J. Harambat; S. Haumann; G. He; L. Heidet; R. Helmy; F. Hemery; N. Hooman; B. llanas; A. Jankauskiene; P. Janssens; S. Karamaria; I. Kazyra; J. Koenig; S. Krid; P. Krug; V. Kwon; A. la Manna; V. Leroy; M. Litwin; J. Lombet; G. Longo; A. C. Lungu; A. Mallawaarachchi; A. Marin; P. Marzuillo; L. Massella; A. Mastrangelo; H. McCarthy; M. Miklaszewska; A. Moczulska; G. Montini; A. Morawiec-Knysak; D. Morin; L. Murer; I. Negru; F. Nobili; L. Obrycki; H. Otoukesh; S. Özcan; L. Pape; S. Papizh; P. Parvex; M. Pawlak-Bratkowska; L. Prikhodina; A. Prytula; C. Quinlan; A. Raes; B. Ranchin; N. Ranguelov; R. Repeckiene; C. Ronit; R. Salomon; R. Santagelo; S. K. Saygılı; S. Schaefer; M. Schreuder; T. Schurmans; T. Seeman; N. Segers; M. Sinha; E. Snauwaert; B. Spasojevic; S. Stabouli; C. Stoica; R. Stroescu; E. Szczepanik; M. Szczepańska; K. Taranta-Janusz; A. Teixeira; J. Thumfart; M. Tkaczyk; R. Torra; D. Torres; N. Tram; B. Utsch; J. Vande Walle; R. Vieux; R. Vitkevic; A. Wilhelm-Bals; E. Wühl; Z. Y. Yildirim; S. Yüksel; K. Zachwieja

doi:https://doi.org/10.1016/j.ekir.2019.05.015

ADPedKD: A Global Online Platform on the Management of Children With ADPKD

Stéphanie de Rechter, Detlef Bockenhauer, Lisa M. Guay-Woodford, Isaac Liu, Andrew J. Mallett, Neveen A. Soliman, Lucimary C. Sylvestre, Franz Schaefer, Max C. Liebau, Djalila Mekahli, P. Adamczyk, N. Akinci, H. Alpay, C. Ardelean, N. Ayasreh, Z. Aydin, A. Bael, V. Baudouin, U. S. Bayrakci, A. BensmanH. Bialkevich, A. Biebuyck, O. Boyer, O. Bjanid, A. Bryłka, S. Çalışkan, A. Cambier, A. Camelio, V. Carbone, M. Charbit, B. Chiodini, A. Chirita, N. Çiçek, R. Cerkauskiene, L. Collard, M. Conceiçao, I. Constantinescu, A. Couderc, B. Crapella, M. Cvetkovic, B. Dima, F. Diomeda, M. Docx, N. Dolan, C. Dossier, D. Drozdz, J. Drube, O. Dunand, P. Dusan, L. A. Eid, F. Emma, M. Espino Hernandez, M. Fila, M. Furlano, M. Gafencu, M. S. Ghuysen, M. Giani, M. Giordano, I. Girisgen, N. Godefroid, A. Godron-Dubrasquet, I. Gojkovic, E. Gonzalez, I. Gökçe, J. W. Groothoff, S. Guarino, A. Guffens, P. Hansen, J. Harambat, S. Haumann, G. He, L. Heidet, R. Helmy, F. Hemery, N. Hooman, B. llanas, A. Jankauskiene, P. Janssens, S. Karamaria, I. Kazyra, J. Koenig, S. Krid, P. Krug, V. Kwon, A. la Manna, V. Leroy, M. Litwin, J. Lombet, G. Longo, A. C. Lungu, A. Mallawaarachchi, A. Marin, P. Marzuillo, L. Massella, A. Mastrangelo, H. McCarthy, M. Miklaszewska, A. Moczulska, G. Montini, A. Morawiec-Knysak, D. Morin, L. Murer, I. Negru, F. Nobili, L. Obrycki, H. Otoukesh, S. Özcan, L. Pape, S. Papizh, P. Parvex, M. Pawlak-Bratkowska, L. Prikhodina, A. Prytula, C. Quinlan, A. Raes, B. Ranchin, N. Ranguelov, R. Repeckiene, C. Ronit, R. Salomon, R. Santagelo, S. K. Saygılı, S. Schaefer, M. Schreuder, T. Schurmans, T. Seeman, N. Segers, M. Sinha, E. Snauwaert, B. Spasojevic, S. Stabouli, C. Stoica, R. Stroescu, E. Szczepanik, M. Szczepańska, K. Taranta-Janusz, A. Teixeira, J. Thumfart, M. Tkaczyk, R. Torra, D. Torres, N. Tram, B. Utsch, J. Vande Walle, R. Vieux, R. Vitkevic, A. Wilhelm-Bals, E. Wühl, Z. Y. Yildirim, S. Yüksel, K. Zachwieja

Research output: Contribution to journal › Article › Academic › peer-review

15 Citations (Scopus)

Abstract

Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure. For several decades, ADPKD was regarded as an adult-onset disease. In the past decade, it has become more widely appreciated that the disease course begins in childhood. However, evidence-based guidelines on how to manage and approach children diagnosed with or at risk of ADPKD are lacking. Also, scoring systems to stratify patients into risk categories have been established only for adults. Overall, there are insufficient data on the clinical course during childhood. We therefore initiated the global ADPedKD project to establish a large international pediatric ADPKD cohort for deep characterization. Methods: Global ADPedKD is an international multicenter observational study focusing on childhood-diagnosed ADPKD. This collaborative project is based on interoperable Web-based databases, comprising 7 regional and independent but uniformly organized chapters, namely Africa, Asia, Australia, Europe, North America, South America, and the United Kingdom. In the database, a detailed basic data questionnaire, including genetics, is used in combination with data entry from follow-up visits, to provide both retrospective and prospective longitudinal data on clinical, radiologic, and laboratory findings, as well as therapeutic interventions. Discussion: The global ADPedKD initiative aims to characterize in detail the most extensive international pediatric ADPKD cohort reported to date, providing evidence for the development of unified diagnostic, follow-up, and treatment recommendations regarding modifiable disease factors. Moreover, this registry will serve as a platform for the development of clinical and/or biochemical markers predicting the risk of early and progressive disease.

Original language	English
Pages (from-to)	1271-1284
Journal	Kidney International Reports
Volume	4
Issue number	9
DOIs	https://doi.org/10.1016/j.ekir.2019.05.015
Publication status	Published - 1 Sept 2019

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de Rechter, S., Bockenhauer, D., Guay-Woodford, L. M., Liu, I., Mallett, A. J., Soliman, N. A., Sylvestre, L. C., Schaefer, F., Liebau, M. C., Mekahli, D., Adamczyk, P., Akinci, N., Alpay, H., Ardelean, C., Ayasreh, N., Aydin, Z., Bael, A., Baudouin, V., Bayrakci, U. S., ... Zachwieja, K. (2019). ADPedKD: A Global Online Platform on the Management of Children With ADPKD. Kidney International Reports, 4(9), 1271-1284. https://doi.org/10.1016/j.ekir.2019.05.015

@article{c5ae8362abf14ad8aa6f02fd8852bab1,

title = "ADPedKD: A Global Online Platform on the Management of Children With ADPKD",

abstract = "Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure. For several decades, ADPKD was regarded as an adult-onset disease. In the past decade, it has become more widely appreciated that the disease course begins in childhood. However, evidence-based guidelines on how to manage and approach children diagnosed with or at risk of ADPKD are lacking. Also, scoring systems to stratify patients into risk categories have been established only for adults. Overall, there are insufficient data on the clinical course during childhood. We therefore initiated the global ADPedKD project to establish a large international pediatric ADPKD cohort for deep characterization. Methods: Global ADPedKD is an international multicenter observational study focusing on childhood-diagnosed ADPKD. This collaborative project is based on interoperable Web-based databases, comprising 7 regional and independent but uniformly organized chapters, namely Africa, Asia, Australia, Europe, North America, South America, and the United Kingdom. In the database, a detailed basic data questionnaire, including genetics, is used in combination with data entry from follow-up visits, to provide both retrospective and prospective longitudinal data on clinical, radiologic, and laboratory findings, as well as therapeutic interventions. Discussion: The global ADPedKD initiative aims to characterize in detail the most extensive international pediatric ADPKD cohort reported to date, providing evidence for the development of unified diagnostic, follow-up, and treatment recommendations regarding modifiable disease factors. Moreover, this registry will serve as a platform for the development of clinical and/or biochemical markers predicting the risk of early and progressive disease.",

author = "{de Rechter}, St{\'e}phanie and Detlef Bockenhauer and Guay-Woodford, {Lisa M.} and Isaac Liu and Mallett, {Andrew J.} and Soliman, {Neveen A.} and Sylvestre, {Lucimary C.} and Franz Schaefer and Liebau, {Max C.} and Djalila Mekahli and P. Adamczyk and N. Akinci and H. Alpay and C. Ardelean and N. Ayasreh and Z. Aydin and A. Bael and V. Baudouin and Bayrakci, {U. S.} and A. Bensman and H. Bialkevich and A. Biebuyck and O. Boyer and O. Bjanid and A. Bry{\l}ka and S. {\c C}alı{\c s}kan and A. Cambier and A. Camelio and V. Carbone and M. Charbit and B. Chiodini and A. Chirita and N. {\c C}i{\c c}ek and R. Cerkauskiene and L. Collard and M. Concei{\c c}ao and I. Constantinescu and A. Couderc and B. Crapella and M. Cvetkovic and B. Dima and F. Diomeda and M. Docx and N. Dolan and C. Dossier and D. Drozdz and J. Drube and O. Dunand and P. Dusan and Eid, {L. A.} and F. Emma and {Espino Hernandez}, M. and M. Fila and M. Furlano and M. Gafencu and Ghuysen, {M. S.} and M. Giani and M. Giordano and I. Girisgen and N. Godefroid and A. Godron-Dubrasquet and I. Gojkovic and E. Gonzalez and I. G{\"o}k{\c c}e and Groothoff, {J. W.} and S. Guarino and A. Guffens and P. Hansen and J. Harambat and S. Haumann and G. He and L. Heidet and R. Helmy and F. Hemery and N. Hooman and B. llanas and A. Jankauskiene and P. Janssens and S. Karamaria and I. Kazyra and J. Koenig and S. Krid and P. Krug and V. Kwon and {la Manna}, A. and V. Leroy and M. Litwin and J. Lombet and G. Longo and Lungu, {A. C.} and A. Mallawaarachchi and A. Marin and P. Marzuillo and L. Massella and A. Mastrangelo and H. McCarthy and M. Miklaszewska and A. Moczulska and G. Montini and A. Morawiec-Knysak and D. Morin and L. Murer and I. Negru and F. Nobili and L. Obrycki and H. Otoukesh and S. {\"O}zcan and L. Pape and S. Papizh and P. Parvex and M. Pawlak-Bratkowska and L. Prikhodina and A. Prytula and C. Quinlan and A. Raes and B. Ranchin and N. Ranguelov and R. Repeckiene and C. Ronit and R. Salomon and R. Santagelo and Saygılı, {S. K.} and S. Schaefer and M. Schreuder and T. Schurmans and T. Seeman and N. Segers and M. Sinha and E. Snauwaert and B. Spasojevic and S. Stabouli and C. Stoica and R. Stroescu and E. Szczepanik and M. Szczepa{\'n}ska and K. Taranta-Janusz and A. Teixeira and J. Thumfart and M. Tkaczyk and R. Torra and D. Torres and N. Tram and B. Utsch and {Vande Walle}, J. and R. Vieux and R. Vitkevic and A. Wilhelm-Bals and E. W{\"u}hl and Yildirim, {Z. Y.} and S. Y{\"u}ksel and K. Zachwieja",

year = "2019",

month = sep,

day = "1",

doi = "https://doi.org/10.1016/j.ekir.2019.05.015",

language = "English",

volume = "4",

pages = "1271--1284",

journal = "Kidney International Reports",

issn = "2468-0249",

publisher = "Elsevier Inc.",

number = "9",

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de Rechter, S, Bockenhauer, D, Guay-Woodford, LM, Liu, I, Mallett, AJ, Soliman, NA, Sylvestre, LC, Schaefer, F, Liebau, MC, Mekahli, D, Adamczyk, P, Akinci, N, Alpay, H, Ardelean, C, Ayasreh, N, Aydin, Z, Bael, A, Baudouin, V, Bayrakci, US, Bensman, A, Bialkevich, H, Biebuyck, A, Boyer, O, Bjanid, O, Bryłka, A, Çalışkan, S, Cambier, A, Camelio, A, Carbone, V, Charbit, M, Chiodini, B, Chirita, A, Çiçek, N, Cerkauskiene, R, Collard, L, Conceiçao, M, Constantinescu, I, Couderc, A, Crapella, B, Cvetkovic, M, Dima, B, Diomeda, F, Docx, M, Dolan, N, Dossier, C, Drozdz, D, Drube, J, Dunand, O, Dusan, P, Eid, LA, Emma, F, Espino Hernandez, M, Fila, M, Furlano, M, Gafencu, M, Ghuysen, MS, Giani, M, Giordano, M, Girisgen, I, Godefroid, N, Godron-Dubrasquet, A, Gojkovic, I, Gonzalez, E, Gökçe, I, Groothoff, JW, Guarino, S, Guffens, A, Hansen, P, Harambat, J, Haumann, S, He, G, Heidet, L, Helmy, R, Hemery, F, Hooman, N, llanas, B, Jankauskiene, A, Janssens, P, Karamaria, S, Kazyra, I, Koenig, J, Krid, S, Krug, P, Kwon, V, la Manna, A, Leroy, V, Litwin, M, Lombet, J, Longo, G, Lungu, AC, Mallawaarachchi, A, Marin, A, Marzuillo, P, Massella, L, Mastrangelo, A, McCarthy, H, Miklaszewska, M, Moczulska, A, Montini, G, Morawiec-Knysak, A, Morin, D, Murer, L, Negru, I, Nobili, F, Obrycki, L, Otoukesh, H, Özcan, S, Pape, L, Papizh, S, Parvex, P, Pawlak-Bratkowska, M, Prikhodina, L, Prytula, A, Quinlan, C, Raes, A, Ranchin, B, Ranguelov, N, Repeckiene, R, Ronit, C, Salomon, R, Santagelo, R, Saygılı, SK, Schaefer, S, Schreuder, M, Schurmans, T, Seeman, T, Segers, N, Sinha, M, Snauwaert, E, Spasojevic, B, Stabouli, S, Stoica, C, Stroescu, R, Szczepanik, E, Szczepańska, M, Taranta-Janusz, K, Teixeira, A, Thumfart, J, Tkaczyk, M, Torra, R, Torres, D, Tram, N, Utsch, B, Vande Walle, J, Vieux, R, Vitkevic, R, Wilhelm-Bals, A, Wühl, E, Yildirim, ZY, Yüksel, S & Zachwieja, K 2019, 'ADPedKD: A Global Online Platform on the Management of Children With ADPKD', Kidney International Reports, vol. 4, no. 9, pp. 1271-1284. https://doi.org/10.1016/j.ekir.2019.05.015

TY - JOUR

T1 - ADPedKD: A Global Online Platform on the Management of Children With ADPKD

AU - de Rechter, Stéphanie

AU - Bockenhauer, Detlef

AU - Guay-Woodford, Lisa M.

AU - Liu, Isaac

AU - Mallett, Andrew J.

AU - Soliman, Neveen A.

AU - Sylvestre, Lucimary C.

AU - Schaefer, Franz

AU - Liebau, Max C.

AU - Mekahli, Djalila

AU - Adamczyk, P.

AU - Akinci, N.

AU - Alpay, H.

AU - Ardelean, C.

AU - Ayasreh, N.

AU - Aydin, Z.

AU - Bael, A.

AU - Baudouin, V.

AU - Bayrakci, U. S.

AU - Bensman, A.

AU - Bialkevich, H.

AU - Biebuyck, A.

AU - Boyer, O.

AU - Bjanid, O.

AU - Bryłka, A.

AU - Çalışkan, S.

AU - Cambier, A.

AU - Camelio, A.

AU - Carbone, V.

AU - Charbit, M.

AU - Chiodini, B.

AU - Chirita, A.

AU - Çiçek, N.

AU - Cerkauskiene, R.

AU - Collard, L.

AU - Conceiçao, M.

AU - Constantinescu, I.

AU - Couderc, A.

AU - Crapella, B.

AU - Cvetkovic, M.

AU - Dima, B.

AU - Diomeda, F.

AU - Docx, M.

AU - Dolan, N.

AU - Dossier, C.

AU - Drozdz, D.

AU - Drube, J.

AU - Dunand, O.

AU - Dusan, P.

AU - Eid, L. A.

AU - Emma, F.

AU - Espino Hernandez, M.

AU - Fila, M.

AU - Furlano, M.

AU - Gafencu, M.

AU - Ghuysen, M. S.

AU - Giani, M.

AU - Giordano, M.

AU - Girisgen, I.

AU - Godefroid, N.

AU - Godron-Dubrasquet, A.

AU - Gojkovic, I.

AU - Gonzalez, E.

AU - Gökçe, I.

AU - Groothoff, J. W.

AU - Guarino, S.

AU - Guffens, A.

AU - Hansen, P.

AU - Harambat, J.

AU - Haumann, S.

AU - He, G.

AU - Heidet, L.

AU - Helmy, R.

AU - Hemery, F.

AU - Hooman, N.

AU - llanas, B.

AU - Jankauskiene, A.

AU - Janssens, P.

AU - Karamaria, S.

AU - Kazyra, I.

AU - Koenig, J.

AU - Krid, S.

AU - Krug, P.

AU - Kwon, V.

AU - la Manna, A.

AU - Leroy, V.

AU - Litwin, M.

AU - Lombet, J.

AU - Longo, G.

AU - Lungu, A. C.

AU - Mallawaarachchi, A.

AU - Marin, A.

AU - Marzuillo, P.

AU - Massella, L.

AU - Mastrangelo, A.

AU - McCarthy, H.

AU - Miklaszewska, M.

AU - Moczulska, A.

AU - Montini, G.

AU - Morawiec-Knysak, A.

AU - Morin, D.

AU - Murer, L.

AU - Negru, I.

AU - Nobili, F.

AU - Obrycki, L.

AU - Otoukesh, H.

AU - Özcan, S.

AU - Pape, L.

AU - Papizh, S.

AU - Parvex, P.

AU - Pawlak-Bratkowska, M.

AU - Prikhodina, L.

AU - Prytula, A.

AU - Quinlan, C.

AU - Raes, A.

AU - Ranchin, B.

AU - Ranguelov, N.

AU - Repeckiene, R.

AU - Ronit, C.

AU - Salomon, R.

AU - Santagelo, R.

AU - Saygılı, S. K.

AU - Schaefer, S.

AU - Schreuder, M.

AU - Schurmans, T.

AU - Seeman, T.

AU - Segers, N.

AU - Sinha, M.

AU - Snauwaert, E.

AU - Spasojevic, B.

AU - Stabouli, S.

AU - Stoica, C.

AU - Stroescu, R.

AU - Szczepanik, E.

AU - Szczepańska, M.

AU - Taranta-Janusz, K.

AU - Teixeira, A.

AU - Thumfart, J.

AU - Tkaczyk, M.

AU - Torra, R.

AU - Torres, D.

AU - Tram, N.

AU - Utsch, B.

AU - Vande Walle, J.

AU - Vieux, R.

AU - Vitkevic, R.

AU - Wilhelm-Bals, A.

AU - Wühl, E.

AU - Yildirim, Z. Y.

AU - Yüksel, S.

AU - Zachwieja, K.

PY - 2019/9/1

Y1 - 2019/9/1

N2 - Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure. For several decades, ADPKD was regarded as an adult-onset disease. In the past decade, it has become more widely appreciated that the disease course begins in childhood. However, evidence-based guidelines on how to manage and approach children diagnosed with or at risk of ADPKD are lacking. Also, scoring systems to stratify patients into risk categories have been established only for adults. Overall, there are insufficient data on the clinical course during childhood. We therefore initiated the global ADPedKD project to establish a large international pediatric ADPKD cohort for deep characterization. Methods: Global ADPedKD is an international multicenter observational study focusing on childhood-diagnosed ADPKD. This collaborative project is based on interoperable Web-based databases, comprising 7 regional and independent but uniformly organized chapters, namely Africa, Asia, Australia, Europe, North America, South America, and the United Kingdom. In the database, a detailed basic data questionnaire, including genetics, is used in combination with data entry from follow-up visits, to provide both retrospective and prospective longitudinal data on clinical, radiologic, and laboratory findings, as well as therapeutic interventions. Discussion: The global ADPedKD initiative aims to characterize in detail the most extensive international pediatric ADPKD cohort reported to date, providing evidence for the development of unified diagnostic, follow-up, and treatment recommendations regarding modifiable disease factors. Moreover, this registry will serve as a platform for the development of clinical and/or biochemical markers predicting the risk of early and progressive disease.

AB - Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure. For several decades, ADPKD was regarded as an adult-onset disease. In the past decade, it has become more widely appreciated that the disease course begins in childhood. However, evidence-based guidelines on how to manage and approach children diagnosed with or at risk of ADPKD are lacking. Also, scoring systems to stratify patients into risk categories have been established only for adults. Overall, there are insufficient data on the clinical course during childhood. We therefore initiated the global ADPedKD project to establish a large international pediatric ADPKD cohort for deep characterization. Methods: Global ADPedKD is an international multicenter observational study focusing on childhood-diagnosed ADPKD. This collaborative project is based on interoperable Web-based databases, comprising 7 regional and independent but uniformly organized chapters, namely Africa, Asia, Australia, Europe, North America, South America, and the United Kingdom. In the database, a detailed basic data questionnaire, including genetics, is used in combination with data entry from follow-up visits, to provide both retrospective and prospective longitudinal data on clinical, radiologic, and laboratory findings, as well as therapeutic interventions. Discussion: The global ADPedKD initiative aims to characterize in detail the most extensive international pediatric ADPKD cohort reported to date, providing evidence for the development of unified diagnostic, follow-up, and treatment recommendations regarding modifiable disease factors. Moreover, this registry will serve as a platform for the development of clinical and/or biochemical markers predicting the risk of early and progressive disease.

UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85071336264&origin=inward

UR - https://www.ncbi.nlm.nih.gov/pubmed/31517146

U2 - https://doi.org/10.1016/j.ekir.2019.05.015

DO - https://doi.org/10.1016/j.ekir.2019.05.015

M3 - Article

C2 - 31517146

SN - 2468-0249

VL - 4

SP - 1271

EP - 1284

JO - Kidney International Reports

JF - Kidney International Reports

IS - 9

ER -

ADPedKD: A Global Online Platform on the Management of Children With ADPKD

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