Rationale and design of the Nephrotic Syndrome Study Network (NEPTUNE) Match in glomerular diseases: designing the right trial for the right patient, today
Glomerular diseases are classified using a descriptive taxonomy that is not reflective of the heterogeneous underlying molecular drivers. This limits not only diagnostic and therapeutic patient management, but also impacts clinical trials evaluating targeted interventions. The Nephrotic Syndrome Stu...
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Veröffentlicht in: | Kidney international 2024-02, Vol.105 (2), p.218-230 |
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creator | Trachtman, Howard Desmond, Hailey Williams, Amanda L. Mariani, Laura H. Eddy, Sean Ju, Wenjun Barisoni, Laura Ascani, Heather K. Uhlmann, Wendy R. Holzman, Lawrence B. Sedor, John R. Subramanian, Lalita Manieri, Tina Roberts, Scott J. Gipson, Debbie S. Kretzler, Matthias Massengill, Susan Lo, Layla Sedor, John Sharma, Silpa Vento, Suzanne Canetta, Pietro Gbadegesin, Rasheed Smith, Maurice Wang, Chia-shi Yun, Emily Adler, Sharon Amarah, Amatur Williams, Miahje Lieske, John Hogan, Marie Fervenza, Fernando Alston, Cheryl Reidy, Kim Zhdanova, Olga Pehrson, Laura Jane Miranda, Melanie Dave, Shiktij Batla, Sadaf Ling, Paul Dylewski, James Rogers, Nathan Fornoni, Alessia Williams, A. Ni, Roxy Rheault, Michelle Kowalski, Amy Derebail, Vimal Froment, Anne Meyers, Kevin Kallem, Krishna Edwards, Aliya Sharma, Samin Sambandam, Kamalanathan Brown, Elizabeth Jefferson, Ashley Tuttle, Katherine Manahan, Linda Kuykendall, Kelli Dharnidharka, Vikas Salmon, Eloise Trachtman, Howard Alter, Gabrielle Arbit, Michael Eddy, Sean Larkina, Maria Scherr, Rebecca Troost, Jonathan Williams, Amanda Zhai, Yan Kincaid, Colleen Li, Shengqian Li, Shannon Gadegbeku, Crystal Barisoni, Laura Zee, Jarcy Bagnasco, Serena Bu, Lihong Caltharp, Shelley Cassol, Clarissa Demeke, Dawit Hassler, Jared Herlitz, Leal Hewitt, Stephen Hodgin, Jeff Holanda, Danni Kambham, Neeraja Lemley, Kevin Messias, Nidia Mikhailov, Alexei Moreno, Vanessa Najafian, Behzad Rosenberg, Avi Sekulik, Miroslav Thomas, David Wu, Ming Yamashita, Michifumi Yin, Hong Zuo . Cochairs, Yiqin Nast, Cynthia |
description | Glomerular diseases are classified using a descriptive taxonomy that is not reflective of the heterogeneous underlying molecular drivers. This limits not only diagnostic and therapeutic patient management, but also impacts clinical trials evaluating targeted interventions. The Nephrotic Syndrome Study Network (NEPTUNE) is poised to address these challenges. The study has enrolled >850 pediatric and adult patients with proteinuric glomerular diseases who have contributed to deep clinical, histologic, genetic, and molecular profiles linked to long-term outcomes. The NEPTUNE Knowledge Network, comprising combined, multiscalar data sets, captures each participant’s molecular disease processes at the time of kidney biopsy. In this editorial, we describe the design and implementation of NEPTUNE Match, which bridges a basic science discovery pipeline with targeted clinical trials. Noninvasive biomarkers have been developed for real-time pathway analyses. A Molecular Nephrology Board reviews the pathway maps together with clinical, laboratory, and histopathologic data assembled for each patient to compile a Match report that estimates the fit between the specific molecular disease pathway(s) identified in an individual patient and proposed clinical trials. The NEPTUNE Match report is communicated using established protocols to the patient and the attending nephrologist for use in their selection of available clinical trials. NEPTUNE Match represents the first application of precision medicine in nephrology with the aim of developing targeted therapies and providing the right medication for each patient with primary glomerular disease. |
doi_str_mv | 10.1016/j.kint.2023.11.018 |
format | Article |
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Cochairs, Yiqin ; Nast, Cynthia</creator><creatorcontrib>Trachtman, Howard ; Desmond, Hailey ; Williams, Amanda L. ; Mariani, Laura H. ; Eddy, Sean ; Ju, Wenjun ; Barisoni, Laura ; Ascani, Heather K. ; Uhlmann, Wendy R. ; Holzman, Lawrence B. ; Sedor, John R. ; Subramanian, Lalita ; Manieri, Tina ; Roberts, Scott J. ; Gipson, Debbie S. ; Kretzler, Matthias ; Massengill, Susan ; Lo, Layla ; Sedor, John ; Sharma, Silpa ; Vento, Suzanne ; Canetta, Pietro ; Gbadegesin, Rasheed ; Smith, Maurice ; Wang, Chia-shi ; Yun, Emily ; Adler, Sharon ; Amarah, Amatur ; Williams, Miahje ; Lieske, John ; Hogan, Marie ; Fervenza, Fernando ; Alston, Cheryl ; Reidy, Kim ; Zhdanova, Olga ; Pehrson, Laura Jane ; Miranda, Melanie ; Dave, Shiktij ; Batla, Sadaf ; Ling, Paul ; Dylewski, James ; Rogers, Nathan ; Fornoni, Alessia ; Williams, A. ; Ni, Roxy ; Rheault, Michelle ; Kowalski, Amy ; Derebail, Vimal ; Froment, Anne ; Meyers, Kevin ; Kallem, Krishna ; Edwards, Aliya ; Sharma, Samin ; Sambandam, Kamalanathan ; Brown, Elizabeth ; Jefferson, Ashley ; Tuttle, Katherine ; Manahan, Linda ; Kuykendall, Kelli ; Dharnidharka, Vikas ; Salmon, Eloise ; Trachtman, Howard ; Alter, Gabrielle ; Arbit, Michael ; Eddy, Sean ; Larkina, Maria ; Scherr, Rebecca ; Troost, Jonathan ; Williams, Amanda ; Zhai, Yan ; Kincaid, Colleen ; Li, Shengqian ; Li, Shannon ; Gadegbeku, Crystal ; Barisoni, Laura ; Zee, Jarcy ; Bagnasco, Serena ; Bu, Lihong ; Caltharp, Shelley ; Cassol, Clarissa ; Demeke, Dawit ; Hassler, Jared ; Herlitz, Leal ; Hewitt, Stephen ; Hodgin, Jeff ; Holanda, Danni ; Kambham, Neeraja ; Lemley, Kevin ; Messias, Nidia ; Mikhailov, Alexei ; Moreno, Vanessa ; Najafian, Behzad ; Rosenberg, Avi ; Sekulik, Miroslav ; Thomas, David ; Wu, Ming ; Yamashita, Michifumi ; Yin, Hong ; Zuo . Cochairs, Yiqin ; Nast, Cynthia ; NEPTUNE investigators</creatorcontrib><description>Glomerular diseases are classified using a descriptive taxonomy that is not reflective of the heterogeneous underlying molecular drivers. This limits not only diagnostic and therapeutic patient management, but also impacts clinical trials evaluating targeted interventions. The Nephrotic Syndrome Study Network (NEPTUNE) is poised to address these challenges. The study has enrolled >850 pediatric and adult patients with proteinuric glomerular diseases who have contributed to deep clinical, histologic, genetic, and molecular profiles linked to long-term outcomes. The NEPTUNE Knowledge Network, comprising combined, multiscalar data sets, captures each participant’s molecular disease processes at the time of kidney biopsy. In this editorial, we describe the design and implementation of NEPTUNE Match, which bridges a basic science discovery pipeline with targeted clinical trials. Noninvasive biomarkers have been developed for real-time pathway analyses. A Molecular Nephrology Board reviews the pathway maps together with clinical, laboratory, and histopathologic data assembled for each patient to compile a Match report that estimates the fit between the specific molecular disease pathway(s) identified in an individual patient and proposed clinical trials. The NEPTUNE Match report is communicated using established protocols to the patient and the attending nephrologist for use in their selection of available clinical trials. NEPTUNE Match represents the first application of precision medicine in nephrology with the aim of developing targeted therapies and providing the right medication for each patient with primary glomerular disease.</description><identifier>ISSN: 0085-2538</identifier><identifier>ISSN: 1523-1755</identifier><identifier>EISSN: 1523-1755</identifier><identifier>DOI: 10.1016/j.kint.2023.11.018</identifier><identifier>PMID: 38245210</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Adult ; Biomarkers ; Child ; clinical trials ; Clinical Trials as Topic ; disease pathway ; Humans ; Kidney Diseases ; Kidney Glomerulus - pathology ; Nephrotic Syndrome - diagnosis ; Nephrotic Syndrome - genetics ; Nephrotic Syndrome - therapy ; patient communication</subject><ispartof>Kidney international, 2024-02, Vol.105 (2), p.218-230</ispartof><rights>2023 International Society of Nephrology</rights><rights>Copyright © 2023 International Society of Nephrology. 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Cochairs, Yiqin</creatorcontrib><creatorcontrib>Nast, Cynthia</creatorcontrib><creatorcontrib>NEPTUNE investigators</creatorcontrib><title>Rationale and design of the Nephrotic Syndrome Study Network (NEPTUNE) Match in glomerular diseases: designing the right trial for the right patient, today</title><title>Kidney international</title><addtitle>Kidney Int</addtitle><description>Glomerular diseases are classified using a descriptive taxonomy that is not reflective of the heterogeneous underlying molecular drivers. This limits not only diagnostic and therapeutic patient management, but also impacts clinical trials evaluating targeted interventions. The Nephrotic Syndrome Study Network (NEPTUNE) is poised to address these challenges. The study has enrolled >850 pediatric and adult patients with proteinuric glomerular diseases who have contributed to deep clinical, histologic, genetic, and molecular profiles linked to long-term outcomes. The NEPTUNE Knowledge Network, comprising combined, multiscalar data sets, captures each participant’s molecular disease processes at the time of kidney biopsy. In this editorial, we describe the design and implementation of NEPTUNE Match, which bridges a basic science discovery pipeline with targeted clinical trials. Noninvasive biomarkers have been developed for real-time pathway analyses. A Molecular Nephrology Board reviews the pathway maps together with clinical, laboratory, and histopathologic data assembled for each patient to compile a Match report that estimates the fit between the specific molecular disease pathway(s) identified in an individual patient and proposed clinical trials. The NEPTUNE Match report is communicated using established protocols to the patient and the attending nephrologist for use in their selection of available clinical trials. NEPTUNE Match represents the first application of precision medicine in nephrology with the aim of developing targeted therapies and providing the right medication for each patient with primary glomerular disease.</description><subject>Adult</subject><subject>Biomarkers</subject><subject>Child</subject><subject>clinical trials</subject><subject>Clinical Trials as Topic</subject><subject>disease pathway</subject><subject>Humans</subject><subject>Kidney Diseases</subject><subject>Kidney Glomerulus - pathology</subject><subject>Nephrotic Syndrome - diagnosis</subject><subject>Nephrotic Syndrome - genetics</subject><subject>Nephrotic Syndrome - therapy</subject><subject>patient communication</subject><issn>0085-2538</issn><issn>1523-1755</issn><issn>1523-1755</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kd1uEzEQhS0EoqHwAlwgX7YSu7W96_1BSBWqwo9UAqLtteXYs7tON3awvUV5Fl4Wh6RVueHKGs-ZM0fzIfSakpwSWp2t8ltjY84IK3JKc0KbJ2hGOSsyWnP-FM0IaXjGeNEcoRchrEiq24I8R0dFw0rOKJmh3z9kNM7KEbC0GmsIprfYdTgOgBewGbyLRuGrrdXerQFfxUlvUyP-cv4Wnyzm369vFvNT_FVGNWBjcT8mmZ9G6bE2AWSA8O5ga2z_19abfog4eiNH3Dn_6G-TwoCNb3F0Wm5fomedHAO8OrzH6Obj_Pric3b57dOXiw-XmSp5FTOoJFdtyYnWBQFFoFF6qUu95G3dLUmlm6bgumuVBF4RTUlHyoZpWtVN12lVF8fofO-7mZZr0Col8HIUG2_W0m-Fk0b827FmEL27E5SSllSsSg4nBwfvfk4QoliboGAcpQU3BcHaHZGqrIskZXup8i4ED93DHkrEDqtYiR1WscOaNoiENQ29eZzwYeSeYxK83wsg3enOgBdBpUsq0MaDikI78z__P2aEt8A</recordid><startdate>202402</startdate><enddate>202402</enddate><creator>Trachtman, Howard</creator><creator>Desmond, Hailey</creator><creator>Williams, Amanda L.</creator><creator>Mariani, Laura H.</creator><creator>Eddy, Sean</creator><creator>Ju, Wenjun</creator><creator>Barisoni, Laura</creator><creator>Ascani, Heather K.</creator><creator>Uhlmann, Wendy R.</creator><creator>Holzman, Lawrence B.</creator><creator>Sedor, John R.</creator><creator>Subramanian, Lalita</creator><creator>Manieri, Tina</creator><creator>Roberts, Scott J.</creator><creator>Gipson, Debbie S.</creator><creator>Kretzler, Matthias</creator><creator>Massengill, Susan</creator><creator>Lo, Layla</creator><creator>Sedor, John</creator><creator>Sharma, Silpa</creator><creator>Vento, Suzanne</creator><creator>Canetta, Pietro</creator><creator>Gbadegesin, Rasheed</creator><creator>Smith, Maurice</creator><creator>Wang, Chia-shi</creator><creator>Yun, Emily</creator><creator>Adler, Sharon</creator><creator>Amarah, Amatur</creator><creator>Williams, Miahje</creator><creator>Lieske, John</creator><creator>Hogan, Marie</creator><creator>Fervenza, Fernando</creator><creator>Alston, Cheryl</creator><creator>Reidy, Kim</creator><creator>Zhdanova, Olga</creator><creator>Pehrson, Laura Jane</creator><creator>Miranda, Melanie</creator><creator>Dave, Shiktij</creator><creator>Batla, Sadaf</creator><creator>Ling, Paul</creator><creator>Dylewski, James</creator><creator>Rogers, Nathan</creator><creator>Fornoni, Alessia</creator><creator>Williams, A.</creator><creator>Ni, Roxy</creator><creator>Rheault, Michelle</creator><creator>Kowalski, Amy</creator><creator>Derebail, Vimal</creator><creator>Froment, Anne</creator><creator>Meyers, Kevin</creator><creator>Kallem, Krishna</creator><creator>Edwards, Aliya</creator><creator>Sharma, Samin</creator><creator>Sambandam, Kamalanathan</creator><creator>Brown, Elizabeth</creator><creator>Jefferson, Ashley</creator><creator>Tuttle, Katherine</creator><creator>Manahan, Linda</creator><creator>Kuykendall, Kelli</creator><creator>Dharnidharka, Vikas</creator><creator>Salmon, Eloise</creator><creator>Trachtman, Howard</creator><creator>Alter, Gabrielle</creator><creator>Arbit, Michael</creator><creator>Eddy, Sean</creator><creator>Larkina, Maria</creator><creator>Scherr, Rebecca</creator><creator>Troost, Jonathan</creator><creator>Williams, Amanda</creator><creator>Zhai, Yan</creator><creator>Kincaid, Colleen</creator><creator>Li, Shengqian</creator><creator>Li, Shannon</creator><creator>Gadegbeku, Crystal</creator><creator>Barisoni, Laura</creator><creator>Zee, Jarcy</creator><creator>Bagnasco, Serena</creator><creator>Bu, Lihong</creator><creator>Caltharp, Shelley</creator><creator>Cassol, Clarissa</creator><creator>Demeke, Dawit</creator><creator>Hassler, Jared</creator><creator>Herlitz, Leal</creator><creator>Hewitt, Stephen</creator><creator>Hodgin, Jeff</creator><creator>Holanda, Danni</creator><creator>Kambham, Neeraja</creator><creator>Lemley, Kevin</creator><creator>Messias, Nidia</creator><creator>Mikhailov, Alexei</creator><creator>Moreno, Vanessa</creator><creator>Najafian, Behzad</creator><creator>Rosenberg, Avi</creator><creator>Sekulik, Miroslav</creator><creator>Thomas, David</creator><creator>Wu, Ming</creator><creator>Yamashita, Michifumi</creator><creator>Yin, Hong</creator><creator>Zuo . Cochairs, Yiqin</creator><creator>Nast, Cynthia</creator><general>Elsevier Inc</general><scope>6I.</scope><scope>AAFTH</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0003-4064-0582</orcidid></search><sort><creationdate>202402</creationdate><title>Rationale and design of the Nephrotic Syndrome Study Network (NEPTUNE) Match in glomerular diseases: designing the right trial for the right patient, today</title><author>Trachtman, Howard ; Desmond, Hailey ; Williams, Amanda L. ; Mariani, Laura H. ; Eddy, Sean ; Ju, Wenjun ; Barisoni, Laura ; Ascani, Heather K. ; Uhlmann, Wendy R. ; Holzman, Lawrence B. ; Sedor, John R. ; Subramanian, Lalita ; Manieri, Tina ; Roberts, Scott J. ; Gipson, Debbie S. ; Kretzler, Matthias ; Massengill, Susan ; Lo, Layla ; Sedor, John ; Sharma, Silpa ; Vento, Suzanne ; Canetta, Pietro ; Gbadegesin, Rasheed ; Smith, Maurice ; Wang, Chia-shi ; Yun, Emily ; Adler, Sharon ; Amarah, Amatur ; Williams, Miahje ; Lieske, John ; Hogan, Marie ; Fervenza, Fernando ; Alston, Cheryl ; Reidy, Kim ; Zhdanova, Olga ; Pehrson, Laura Jane ; Miranda, Melanie ; Dave, Shiktij ; Batla, Sadaf ; Ling, Paul ; Dylewski, James ; Rogers, Nathan ; Fornoni, Alessia ; Williams, A. ; Ni, Roxy ; Rheault, Michelle ; Kowalski, Amy ; Derebail, Vimal ; Froment, Anne ; Meyers, Kevin ; Kallem, Krishna ; Edwards, Aliya ; Sharma, Samin ; Sambandam, Kamalanathan ; Brown, Elizabeth ; Jefferson, Ashley ; Tuttle, Katherine ; Manahan, Linda ; Kuykendall, Kelli ; Dharnidharka, Vikas ; Salmon, Eloise ; Trachtman, Howard ; Alter, Gabrielle ; Arbit, Michael ; Eddy, Sean ; Larkina, Maria ; Scherr, Rebecca ; Troost, Jonathan ; Williams, Amanda ; Zhai, Yan ; Kincaid, Colleen ; Li, Shengqian ; Li, Shannon ; Gadegbeku, Crystal ; Barisoni, Laura ; Zee, Jarcy ; Bagnasco, Serena ; Bu, Lihong ; Caltharp, Shelley ; Cassol, Clarissa ; Demeke, Dawit ; Hassler, Jared ; Herlitz, Leal ; Hewitt, Stephen ; Hodgin, Jeff ; Holanda, Danni ; Kambham, Neeraja ; Lemley, Kevin ; Messias, Nidia ; Mikhailov, Alexei ; Moreno, Vanessa ; Najafian, Behzad ; Rosenberg, Avi ; Sekulik, Miroslav ; Thomas, David ; Wu, Ming ; Yamashita, Michifumi ; Yin, Hong ; Zuo . 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Cochairs, Yiqin</creatorcontrib><creatorcontrib>Nast, Cynthia</creatorcontrib><creatorcontrib>NEPTUNE investigators</creatorcontrib><collection>ScienceDirect Open Access Titles</collection><collection>Elsevier:ScienceDirect:Open Access</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Kidney international</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Trachtman, Howard</au><au>Desmond, Hailey</au><au>Williams, Amanda L.</au><au>Mariani, Laura H.</au><au>Eddy, Sean</au><au>Ju, Wenjun</au><au>Barisoni, Laura</au><au>Ascani, Heather K.</au><au>Uhlmann, Wendy R.</au><au>Holzman, Lawrence B.</au><au>Sedor, John R.</au><au>Subramanian, Lalita</au><au>Manieri, Tina</au><au>Roberts, Scott J.</au><au>Gipson, Debbie S.</au><au>Kretzler, Matthias</au><au>Massengill, Susan</au><au>Lo, Layla</au><au>Sedor, John</au><au>Sharma, Silpa</au><au>Vento, Suzanne</au><au>Canetta, Pietro</au><au>Gbadegesin, Rasheed</au><au>Smith, Maurice</au><au>Wang, Chia-shi</au><au>Yun, Emily</au><au>Adler, Sharon</au><au>Amarah, Amatur</au><au>Williams, Miahje</au><au>Lieske, John</au><au>Hogan, Marie</au><au>Fervenza, Fernando</au><au>Alston, Cheryl</au><au>Reidy, Kim</au><au>Zhdanova, Olga</au><au>Pehrson, Laura Jane</au><au>Miranda, Melanie</au><au>Dave, Shiktij</au><au>Batla, Sadaf</au><au>Ling, Paul</au><au>Dylewski, James</au><au>Rogers, Nathan</au><au>Fornoni, Alessia</au><au>Williams, A.</au><au>Ni, Roxy</au><au>Rheault, Michelle</au><au>Kowalski, Amy</au><au>Derebail, Vimal</au><au>Froment, Anne</au><au>Meyers, Kevin</au><au>Kallem, Krishna</au><au>Edwards, Aliya</au><au>Sharma, Samin</au><au>Sambandam, Kamalanathan</au><au>Brown, Elizabeth</au><au>Jefferson, Ashley</au><au>Tuttle, Katherine</au><au>Manahan, Linda</au><au>Kuykendall, Kelli</au><au>Dharnidharka, Vikas</au><au>Salmon, Eloise</au><au>Trachtman, Howard</au><au>Alter, Gabrielle</au><au>Arbit, Michael</au><au>Eddy, Sean</au><au>Larkina, Maria</au><au>Scherr, Rebecca</au><au>Troost, Jonathan</au><au>Williams, Amanda</au><au>Zhai, Yan</au><au>Kincaid, Colleen</au><au>Li, Shengqian</au><au>Li, Shannon</au><au>Gadegbeku, Crystal</au><au>Barisoni, Laura</au><au>Zee, Jarcy</au><au>Bagnasco, Serena</au><au>Bu, Lihong</au><au>Caltharp, Shelley</au><au>Cassol, Clarissa</au><au>Demeke, Dawit</au><au>Hassler, Jared</au><au>Herlitz, Leal</au><au>Hewitt, Stephen</au><au>Hodgin, Jeff</au><au>Holanda, Danni</au><au>Kambham, Neeraja</au><au>Lemley, Kevin</au><au>Messias, Nidia</au><au>Mikhailov, Alexei</au><au>Moreno, Vanessa</au><au>Najafian, Behzad</au><au>Rosenberg, Avi</au><au>Sekulik, Miroslav</au><au>Thomas, David</au><au>Wu, Ming</au><au>Yamashita, Michifumi</au><au>Yin, Hong</au><au>Zuo . Cochairs, Yiqin</au><au>Nast, Cynthia</au><aucorp>NEPTUNE investigators</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Rationale and design of the Nephrotic Syndrome Study Network (NEPTUNE) Match in glomerular diseases: designing the right trial for the right patient, today</atitle><jtitle>Kidney international</jtitle><addtitle>Kidney Int</addtitle><date>2024-02</date><risdate>2024</risdate><volume>105</volume><issue>2</issue><spage>218</spage><epage>230</epage><pages>218-230</pages><issn>0085-2538</issn><issn>1523-1755</issn><eissn>1523-1755</eissn><abstract>Glomerular diseases are classified using a descriptive taxonomy that is not reflective of the heterogeneous underlying molecular drivers. This limits not only diagnostic and therapeutic patient management, but also impacts clinical trials evaluating targeted interventions. The Nephrotic Syndrome Study Network (NEPTUNE) is poised to address these challenges. The study has enrolled >850 pediatric and adult patients with proteinuric glomerular diseases who have contributed to deep clinical, histologic, genetic, and molecular profiles linked to long-term outcomes. The NEPTUNE Knowledge Network, comprising combined, multiscalar data sets, captures each participant’s molecular disease processes at the time of kidney biopsy. In this editorial, we describe the design and implementation of NEPTUNE Match, which bridges a basic science discovery pipeline with targeted clinical trials. Noninvasive biomarkers have been developed for real-time pathway analyses. A Molecular Nephrology Board reviews the pathway maps together with clinical, laboratory, and histopathologic data assembled for each patient to compile a Match report that estimates the fit between the specific molecular disease pathway(s) identified in an individual patient and proposed clinical trials. The NEPTUNE Match report is communicated using established protocols to the patient and the attending nephrologist for use in their selection of available clinical trials. NEPTUNE Match represents the first application of precision medicine in nephrology with the aim of developing targeted therapies and providing the right medication for each patient with primary glomerular disease.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>38245210</pmid><doi>10.1016/j.kint.2023.11.018</doi><tpages>13</tpages><orcidid>https://orcid.org/0000-0003-4064-0582</orcidid><oa>free_for_read</oa></addata></record> |
fulltext | fulltext |
identifier | ISSN: 0085-2538 |
ispartof | Kidney international, 2024-02, Vol.105 (2), p.218-230 |
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language | eng |
recordid | cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_11090626 |
source | MEDLINE; Alma/SFX Local Collection |
subjects | Adult Biomarkers Child clinical trials Clinical Trials as Topic disease pathway Humans Kidney Diseases Kidney Glomerulus - pathology Nephrotic Syndrome - diagnosis Nephrotic Syndrome - genetics Nephrotic Syndrome - therapy patient communication |
title | Rationale and design of the Nephrotic Syndrome Study Network (NEPTUNE) Match in glomerular diseases: designing the right trial for the right patient, today |
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