Scholars@Duke

• Background
• 

  Education, Training, & Certifications

  • Pediatric Hem-Onc Fellowship, Pediatrics, Children's Hospital of Philadelphia 2006 - 2009
  • Pediatric Residency, Pediatrics, Children's Hospital of Philadelphia 2003 - 2006
  • M.D., University of Medicine and Dentistry of New Jersey 2003
• 

  Duke Appointment History

  • Assistant Professor of Pediatrics, Pediatrics, Hematology-Oncology, Pediatrics 2011 - 2017
  • Medical Instructor in the Department of Pediatrics, Pediatrics, Hematology-Oncology, Pediatrics 2009 - 2011
• 

  Leadership & Clinical Positions at Duke

  • Program Director, Pediatric Fellowship Program
    Clinical Director, Pediatric Hematology/Oncology
    Director, Pediatric Sickle Cell Program
    Director, Inherited Bone Marrow Failure Program
• Research
• 

  Selected Grants

  • PKD Registry AG348-C-008 awarded by Agios Pharmaceuticals, Inc. 2018 - 2026
  • SEG101B221 (Phase 2) awarded by Novartis Pharmaceuticals Corporation 2018 - 2025
  • Eltrombopag for SAA awarded by Novartis Pharmaceuticals Corporation 2017 - 2024
  • Phase III study of AG-348 in not regulary transfused adult subjects with Pyruvate kinase deficiency awarded by Agios Pharmaceuticals, Inc. 2018 - 2023
  • An integrated and diverse genomic medicine program for undiagnosed diseases awarded by National Institutes of Health 2014 - 2022
  • Transfusion Medicine and Hematology awarded by National Institutes of Health 1975 - 2021
  • AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE SAFETY OF RIVIPANSEL (GMI-1070) IN THE TREATMENT OF ONE OR MORE VASOOCCLUSIVE CRISES IN HOSPITALIZED SUBJECTS WITH SICKLE CELL DISEASE. awarded by Pfizer, Inc. 2016 - 2019
  • CSA - Pfizer 212511 B5201002 Phase 3 Randomized Multicenter Trial awarded by Pfizer, Inc. 2015 - 2019
  • Pediatric Hydroxyurea Phase III Clinical Trial (Baby Hug): Follow-Up Observational Study II awarded by National Institutes of Health 2017 - 2018
  • ICON 1 awarded by Boston Children's Hospital 2013 - 2017
  • PKD Natural Study awarded by Boston Children's Hospital 2014 - 2017
  • TCD With Transfusions Changing to Hydroxyurea (TWITCH) Closeout awarded by Cincinnati Children's Hospital Medical Center 2014 - 2015
• Publications & Artistic Works
• 

  Selected Publications

  • Academic Articles

    • van Beers, EJ, van Straaten, S, Morton, DH, Barcellini, W, Eber, SW, Glader, B, Yaish, HM, Chonat, S, Kwiatkowski, JL, Rothman, JA, Sharma, M, Neufeld, EJ, Sheth, S, Despotovic, JM, Kollmar, N, Pospíšilová, D, Knoll, CM, Kuo, K, Pastore, YD, Thompson, AA, Newburger, PE, Ravindranath, Y, Wang, WC, Wlodarski, MW, Wang, H, Holzhauer, S, Breakey, VR, Verhovsek, M, Kunz, J, McNaull, MA, Rose, MJ, Bradeen, HA, Addonizio, K, Li, A, Al-Sayegh, H, London, WB, and Grace, RF. "Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study." Haematologica 104, no. 2 (February 2019): e51-e53. (Letter)  Full Text
    • Tan, QK-G, Cope, H, Spillmann, RC, Stong, N, Jiang, Y-H, McDonald, MT, Rothman, JA, Butler, MW, Frush, DP, Lachman, RS, Lee, B, Bacino, CA, Bonner, MJ, McCall, CM, Pendse, AA, Walley, N, Undiagnosed Diseases Network, , Shashi, V, and Pena, LDM. "Further evidence for the involvement of EFL1 in a Shwachman-Diamond-like syndrome and expansion of the phenotypic features." Cold Spring Harbor Molecular Case Studies 4, no. 5 (October 2018).  Full Text
    • Grace, RF, Despotovic, JM, Bennett, CM, Bussel, JB, Neier, M, Neunert, C, Crary, SE, Pastore, YD, Klaassen, RJ, Rothman, JA, Hege, K, Breakey, VR, Rose, MJ, Shimano, KA, Buchanan, GR, Geddis, A, Haley, KM, Lorenzana, A, Thompson, A, Jeng, M, Neufeld, EJ, Brown, T, Forbes, PW, and Lambert, MP. "Physician decision making in selection of second-line treatments in immune thrombocytopenia in children." American Journal of Hematology 93, no. 7 (July 2018): 882-888.  Full Text
    • Grace, RF, Bianchi, P, van Beers, EJ, Eber, SW, Glader, B, Yaish, HM, Despotovic, JM, Rothman, JA, Sharma, M, McNaull, MM, Fermo, E, Lezon-Geyda, K, Morton, DH, Neufeld, EJ, Chonat, S, Kollmar, N, Knoll, CM, Kuo, K, Kwiatkowski, JL, Pospíšilová, D, Pastore, YD, Thompson, AA, Newburger, PE, Ravindranath, Y, Wang, WC, Wlodarski, MW, Wang, H, Holzhauer, S, Breakey, VR, Kunz, J, Sheth, S, Rose, MJ, Bradeen, HA, Neu, N, Guo, D, Al-Sayegh, H, London, WB, Gallagher, PG, Zanella, A, and Barcellini, W. "Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study." Blood 131, no. 20 (May 2018): 2183-2192.  Full Text
    • Allen, TM, Anderson, LM, Rothman, JA, and Bonner, MJ. "[Formula: see text]Executive functioning and health-related quality of life in pediatric sickle cell disease." Child neuropsychology : a journal on normal and abnormal development in childhood and adolescence 23, no. 8 (November 2017): 889-906.  Full Text
    • Cancio, MI, Aygun, B, Chui, DHK, Rothman, JA, Scott, JP, Estepp, JH, and Hankins, JS. "The clinical severity of hemoglobin S/Black (A γδβ)0 -thalassemia." Pediatric Blood & Cancer 64, no. 11 (November 2017).  Full Text
    • Reiter, A, Rice, H, and Rothman, J. "LONG-TERM CLINICAL AND HEMATOLOGIC OUTCOMES OF SPLENECTOMY IN CHILDREN WITH CONGENITAL HEMOLYTIC ANEMIA." PEDIATRIC BLOOD & CANCER 63 (June 2016): S57-S57.  Link to Item
    • Estepp, JH, Melloni, C, Thornburg, CD, Wiczling, P, Rogers, Z, Rothman, JA, Green, NS, Liem, R, Brandow, AM, Crary, SE, Howard, TH, Morris, MH, Lewandowski, A, Garg, U, Jusko, WJ, Neville, KA, and Best Pharmaceuticals for Children Act-Pediatric Trials Network Administrative Core Committee, . "Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia." Journal of clinical pharmacology 56, no. 3 (March 2016): 298-306.  Full Text
    • Wang, H-HS, Herbst, KW, Rothman, JA, Shah, NR, Wiener, JS, and Routh, JC. "Trends in Sickle Cell Disease-related Priapism in U.S. Children's Hospitals." Urology 89 (March 2016): 118-122.  Full Text
    • Luchtman-Jones, L, Pressel, S, Hilliard, L, Brown, RC, Smith, MG, Thompson, AA, Lee, MT, Rothman, J, Rogers, ZR, Owen, W, Imran, H, Thornburg, C, Kwiatkowski, JL, Aygun, B, Nelson, S, Roberts, C, Gauger, C, Piccone, C, Kalfa, T, Alvarez, O, Hassell, K, Davis, BR, and Ware, RE. "Effects of hydroxyurea treatment for patients with hemoglobin SC disease." American Journal of Hematology 91, no. 2 (February 2016): 238-242.  Full Text
    • Ware, RE, Davis, BR, Schultz, WH, Brown, RC, Aygun, B, Sarnaik, S, Odame, I, Fuh, B, George, A, Owen, W, Luchtman-Jones, L, Rogers, ZR, Hilliard, L, Gauger, C, Piccone, C, Lee, MT, Kwiatkowski, JL, Jackson, S, Miller, ST, Roberts, C, Heeney, MM, Kalfa, TA, Nelson, S, Imran, H, Nottage, K, Alvarez, O, Rhodes, M, Thompson, AA, Rothman, JA, Helton, KJ, Roberts, D, Coleman, J, Bonner, MJ, Kutlar, A, Patel, N, Wood, J, Piller, L, Wei, P, Luden, J, Mortier, NA, Stuber, SE, Luban, NLC, Cohen, AR, Pressel, S, and Adams, RJ. "Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial." Lancet (London, England) 387, no. 10019 (February 2016): 661-670.  Full Text  Open Access Copy
    • Englum, BR, Rothman, J, Leonard, S, Reiter, A, Thornburg, C, Brindle, M, Wright, N, Heeney, MM, Jason Smithers, C, Brown, RL, Kalfa, T, Langer, JC, Cada, M, Oldham, KT, Scott, JP, St Peter, SD, Sharma, M, Davidoff, AM, Nottage, K, Bernabe, K, Wilson, DB, Dutta, S, Glader, B, Crary, SE, Dassinger, MS, Dunbar, L, Islam, S, Kumar, M, Rescorla, F, Bruch, S, Campbell, A, Austin, M, Sidonio, R, Blakely, ML, Rice, HE, and Splenectomy in Congenital Hemolytic Anemia Consortium, . "Hematologic outcomes after total splenectomy and partial splenectomy for congenital hemolytic anemia." Journal of Pediatric Surgery 51, no. 1 (January 2016): 122-127.  Full Text
    • Wood, JC, Cohen, AR, Pressel, SL, Aygun, B, Imran, H, Luchtman-Jones, L, Thompson, AA, Fuh, B, Schultz, WH, Davis, BR, Ware, RE, and TWiTCH Investigators, . "Organ iron accumulation in chronically transfused children with sickle cell anaemia: baseline results from the TWiTCH trial." British Journal of Haematology 172, no. 1 (January 2016): 122-130.  Full Text
    • Allewelt, H, Saricicek, J, Patel, S, and Rothman, J. "BARRIERS TO IMPROVING ANALGESIA FOR VASO-OCCLUSIVE PAIN CRISES IN PEDIATRIC PATIENTS WITH SICKLE CELL DISEASE." Pediatric Blood & Cancer 62 (June 1, 2015): 40-40.  Link to Item
    • Rice, HE, Englum, BR, Rothman, J, Leonard, S, Reiter, A, Thornburg, C, Brindle, M, Wright, N, Heeney, MM, Smithers, C, Brown, RL, Kalfa, T, Langer, JC, Cada, M, Oldham, KT, Scott, JP, St Peter, S, Sharma, M, Davidoff, AM, Nottage, K, Bernabe, K, Wilson, DB, Dutta, S, Glader, B, Crary, SE, Dassinger, MS, Dunbar, L, Islam, S, Kumar, M, Rescorla, F, Bruch, S, Campbell, A, Austin, M, Sidonio, R, Blakely, ML, and Splenectomy in Congenital Hemolytic Anemia (SICHA) Consortium, . "Clinical outcomes of splenectomy in children: report of the splenectomy in congenital hemolytic anemia registry." American Journal of Hematology 90, no. 3 (March 2015): 187-192.  Full Text
    • Mize, L, Burgett, S, Xu, J, Rothman, J, and Shah, N. "The Use of Chronic Transfusions in Sickle Cell Disease for Non-Stroke Related Indications." BLOOD 124, no. 21 (December 6, 2014).  Link to Item
    • Zimmerman, SP, Rothman, JA, Hansen, JL, Rusin, MM, Bertone, MA, and Hamrick, HJ. "Systemic loxoscelism in a nonendemic area: a diagnostic challenge for the unsuspecting physician." Clin Pediatr (Phila) 53, no. 11 (October 2014): 1098-1100.  Full Text  Link to Item
    • Leonard, S, Rothman, J, Shah, N, Thornburg, C, and Rice, H. "CURRENT PRACTICE PATTERNS FOR PARTIAL VERSUS TOTAL SPLENECTOMY IN PEDIATRIC PATIENTS WITH CONGENITAL HEMOLYTIC ANEMIA." PEDIATRIC BLOOD & CANCER 61 (May 2014): S92-S92.  Link to Item
    • Mouttalib, S, Rice, HE, Snyder, D, Levens, JS, Reiter, A, Soler, P, Rothman, JA, and Thornburg, CD. "Evaluation of partial and total splenectomy in children with sickle cell disease using an Internet-based registry." Pediatr Blood Cancer 59, no. 1 (July 15, 2012): 100-104.  Full Text  Link to Item
    • Shah, N, Landi, D, Shah, R, Rothman, J, De Castro, LM, and Thornburg, CD. "Complications of implantable venous access devices in patients with sickle cell disease." American Journal of Hematology 87, no. 2 (February 2012): 224-226.  Full Text
    • Rothman, JA, Das, R, Teachey, DT, Paessler, ME, and Nichols, KE. "Rapamycin does not control hemophagocytic lymphohistiocytosis in LCMV-infected perforin-deficient mice." Pediatr Blood Cancer 57, no. 7 (December 15, 2011): 1239-1243.  Full Text  Link to Item
    • Mouttalib, S, Snyder, D, Levens, J, Soler, P, Rothman, J, Thornburg, C, and Rice, H. "USE OF INTERNET-BASED REGISTRY FOR EVALUATION OF PARTIAL AND TOTAL SPLENECTOMY IN SICKLE CELL DISEASE." PEDIATRIC BLOOD & CANCER 56, no. 6 (June 2011): 911-911.  Link to Item
    • Talaat, KR, Rothman, JA, Cohen, JI, Santi, M, Choi, JK, Guzman, M, Zimmerman, R, Nallasamy, S, Brucker, A, Quezado, M, Pittaluga, S, Patronas, NJ, Klion, AD, and Nichols, KE. "Lymphocytic vasculitis involving the central nervous system occurs in patients with X-linked lymphoproliferative disease in the absence of Epstein-Barr virus infection." Pediatr Blood Cancer 53, no. 6 (December 2009): 1120-1123.  Full Text  Link to Item
    • Schiopu, E, Hsu, VM, Impens, AJ, Rothman, JA, McCloskey, DA, Wilson, JE, Phillips, K, and Seibold, JR. "Randomized placebo-controlled crossover trial of tadalafil in Raynaud's phenomenon secondary to systemic sclerosis." J Rheumatol 36, no. 10 (October 2009): 2264-2268.  Full Text  Link to Item
    • Impens, AJ, Rothman, J, Schiopu, E, Cole, JC, Dang, J, Gendrano, N, Rosen, RC, and Seibold, JR. "Sexual activity and functioning in female scleroderma patients." Clin Exp Rheumatol 27, no. 3 Suppl 54 (May 2009): 38-43.  Link to Item
    • Impens, AJ, Rothman, J, Schiopu, E, Cole, JC, Dang, J, Gendrano, N, Rosen, RC, and Seibold, JR. "Sexual activity and functioning in female scleroderma patients." Clinical and Experimental Rheumatology 27, no. 3 SUPPL. 54 (2009): S38-S43.
    • Nunez-Cruz, S, Yeo, WCJ, Rothman, J, Ojha, P, Bassiri, H, Juntilla, M, Davidson, D, Veillette, A, Koretzky, GA, and Nichols, KE. "Differential requirement for the SAP-Fyn interaction during NK T cell development and function." Journal of Immunology (Baltimore, Md. : 1950) 181, no. 4 (August 2008): 2311-2320.  Full Text
    • Rao, A, Rothman, J, and Nichols, KE. "Genetic testing and tumor surveillance for children with cancer predisposition syndromes." Curr Opin Pediatr 20, no. 1 (February 2008): 1-7. (Review)  Full Text  Link to Item
    • Bassiri, H, Janice Yeo, WC, Rothman, J, Koretzky, GA, and Nichols, KE. "X-linked lymphoproliferative disease (XLP): a model of impaired anti-viral, anti-tumor and humoral immune responses." Immunol Res 42, no. 1-3 (2008): 145-159. (Review)  Full Text  Link to Item

  • Book Sections

    • Greiner, RJ, Peterson-Carmichael, S, Rothman, JA, Gow, KW, Tamburro, RF, and Barfield, R. "Care of the oncology patient in the picu." In Pediatric Critical Care Medicine: Volume 3: Gastroenterological, Endocrine, Renal, Hematologic, Oncologic and Immune Systems, 343-361. January 1, 2014.  Full Text

  • Conference Papers

    • Borst, AJ, Sudan, DL, Wang, LA, Neuss, MJ, Rothman, JA, and Ortel, TL. "Bleeding and thrombotic complications of pediatric liver transplant." January 19, 2018.  Full Text
    • Grace, RF, Klaassen, RJ, Rothman, JA, Hege, K, Neufeld, EJ, Bennett, CM, Lambert, M, Breakey, VR, Bussel, JB, Buchanan, GR, Rose, MJ, Geddis, A, Shimano, K, Haley, KM, Lorenzana, A, Neunert, C, Thompson, AA, Pastore, YD, Jeng, M, Crary, SE, Neier, M, Brown, T, Forbes, P, and Despotovic, JM. "Clinical Characteristics and Quality of Life of Children with ITP Starting Second Line Treatments: Data from the ITP Consortium of North America ICON1 Study." 58th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH). San Diego, CA. December 3, 2016 - December 6, 2016.: AMER SOC HEMATOLOGY, December 2, 2016.  Link to Item
    • Lambert, MP, Grace, RF, Neunert, C, Despotovic, JM, Bhat, R, Pastore, YD, Buchanan, GR, Klaassen, RJ, Bennett, CM, Rothman, JA, Breakey, VR, Bussel, JB, Rose, MJ, Geddis, A, Shimano, K, Hege, K, Haley, KM, Lorenzana, A, Jeng, M, Crary, SE, Neier, M, Brown, T, Forbes, P, and Neufeld, EJ. "Physician Factors Determining Treatment Decisions in Selecting Second Line Agents for Pediatric ITP." 58th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH). San Diego, CA. December 3, 2016 - December 6, 2016.: AMER SOC HEMATOLOGY, December 2, 2016.  Link to Item
    • Leraas, HJ, Kim, J, Sun, Z, Nag, UP, Ezekian, BD, Kamyszek, RW, Gulack, BC, Rice, HE, Adibe, OO, Borst, AJ, Rothman, JA, and Tracy, ET. "Postoperative Venous Thromboembolism in Children Is Increased in Setting of Cancer or Infection." 58th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH). San Diego, CA. December 3, 2016 - December 6, 2016.: AMER SOC HEMATOLOGY, December 2, 2016.  Link to Item
    • Ware, RE, Lane, A, Schultz, WH, Imran, H, Lee, MT, Heeney, MM, Brown, C, Luchtman-Jones, L, Webb, J, Jackson, S, Aygun, B, Miller, ST, Rothman, JA, Thompson, AA, Luden, J, Davis, BR, and Adams, RJ. "Variation in Serial TCD Velocity Measurements in the TCD with Transfusions Changing to Hydroxyurea (TWiTCH) Trial." 58th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH). San Diego, CA. December 3, 2016 - December 6, 2016.: AMER SOC HEMATOLOGY, December 2, 2016.  Link to Item
    • Bianchi, P, Fermo, E, Lezon-Geyda, K, Gallagher, PG, Morton, DH, Barcellini, W, Glader, B, Eber, SW, Despotovic, JM, Knoll, CM, Yaish, HM, Newburger, PF, Rothman, J, Thompson, AA, Ravindranath, Y, Nottage, KA, Wang, H, van Beers, EJ, Kunz, J, Wlodarski, MW, Sharma, M, Rose, MJ, Pastore, YD, Giardina, PJ, Rhodes, M, Kuo, K, Guo, D, London, WB, and Grace, RF. "Molecular Characterization of 140 Patients in the Pyruvate Kinase Deficiency (PKD) Natural History Study (NHS): Report of 20 New Variants." December 3, 2015.  Link to Item
    • Chonat, S, Risinger, M, Dagaonkar, N, Maghathe, T, Rothman, J, Connor, J, Kalinyak, K, Begtrup, AH, Zhang, K, and Kalfa, TA. "The Spectrum of Alpha-Spectrin Associated Hereditary Spherocytosis." December 3, 2015.  Link to Item
    • Grace, RF, Morton, DH, Barcellini, W, Eber, SW, Depsotovic, JM, Knoll, CM, Yaish, HM, Newburger, PE, Rothman, J, Thompson, AA, Ravindranath, Y, Kunz, J, van Beers, EJ, Wlodarski, MW, Nottage, KA, Wang, H, Sharma, M, Rose, MJ, Pastore, YD, Giardina, PJ, Rhodes, M, Kuo, K, Breakey, VR, Neufeld, EJ, Guo, D, London, WB, Ealcone, J, and Glader, B. "The Phenotypic Spectrum of Pyruvate Kinase Deficiency (PKD) from the PKD Natural History Study (NHS): Description of Four Severity Groups By Anemia Status." December 3, 2015.  Link to Item
    • Ware, RE, Davis, BR, Schultz, WH, Brown, C, Aygun, B, Sarnaik, SA, Odame, I, Fuh, B, George, A, Owen, W, Luchtman-Jones, L, Rogers, ZR, Hilliard, L, Gauger, C, Piccone, CM, Lee, MT, Kwiatkowski, J, Jackson, S, Miller, ST, Roberts, CW, Heeney, MM, Kalfa, TA, Nelson, SC, Imran, H, Nottage, KA, Alvarez, OA, Rhodes, M, Thompson, AA, Rothman, J, Helton, KJ, Roberts, D, Coleman, J, Bonner, MJ, Kutlar, A, Patel, N, Wood, JC, Piller, L, Wei, P, Luden, J, Mortier, NA, Stuber, S, Luban, NLC, and Cohen, AR et al. "TCD with Transfusions Changing to Hydroxyurea (TWiTCH): Hydroxyurea Therapy As an Alternative to Transfusions for Primary Stroke Prevention in Children with Sickle Cell Anemia." December 3, 2015.  Link to Item
    • Grace, R, Barcellini, W, Eber, S, Kunz, J, Despotovic, J, Thompson, A, Morton, DH, Glader, B, Yaish, H, Knoll, C, Rothman, J, Newburger, P, Nottage, K, Wang, H, Guo, D, London, W, Merica, E, and Neufeld, E. "CATEGORIZATION OF CLINICAL SEVERITY IN PYRUVATE KINASE DEFICIENCY (PKD) IN AN INTERNATIONAL, OBSERVATIONAL COHORT." June 2015.  Link to Item
    • Morton, DH, Knoll, C, Rothman, J, Glader, B, Neufeld, E, Barcellini, W, Eber, S, Yaish, H, Despotovic, J, Thompson, A, Usmani, N, Nottage, K, Wang, H, Kunz, J, Guo, D, London, W, Conner, K, Merica, E, and Grace, R. "THE CLINICAL FEATURES AND TREATMENT OF IRON OVERLOAD IN PYRUVATE KINASE DEFICIENCY (PKD): DATA FROM THE PKD NATURAL HISTORY STUDY (NHS)." June 2015.  Link to Item
    • Willen, S, Shah, N, Thornburg, C, and Rothman, J. "Timing of the Initiation of Hydroxyurea and Hematologic Outcomes in Patients with Sickle Cell Disease (SCD)." November 16, 2012.  Link to Item
    • Rothman, J, Burgett, S, Ware, RE, and Thornburg, C. "Hydroxyurea Reduces Conversion From Conditional to Abnormal TCD Velocities In Children with Sickle Cell Anemia (SCA)." November 19, 2010.  Link to Item
    • Shah, N, Landi, D, Shah, R, Rothman, J, and Thornburg, C. "Complications of Implantable Venous Access Devices In Patients with Sickle Cell Disease." November 19, 2010.  Link to Item

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