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

  • Associate Professor of Pediatrics, Pediatrics, Hematology-Oncology, Pediatrics 2017 - 2020
  • Assistant Professor in Medicine, Medicine, Hematology, Medicine 2014 - 2020
  • 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 Operations Director, Pediatric Hematology/Oncology
    Director, Pediatric Sickle Cell Program
    Director, Inherited Bone Marrow Failure Program
• Research
• 

  Selected Grants

  • PROTOCOL AG348-C-008 Pyruvate Kinase Deficiency Global Longitudinal Registry awarded by Agios Pharmaceuticals, Inc. 2018 - 2026
  • A Phase 2, Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occl awarded by Novartis Pharmaceuticals Corporation 2018 - 2025
  • A Phase 3b, Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial with an Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immun awarded by DOVA Pharmaceuticals 2021 - 2025
  • A phase II, open-label, non-controlled, intrapatient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naiive severe aplastic anemia or rec awarded by Novartis Pharmaceuticals Corporation 2017 - 2024
  • AG348-C-011 extension study of adult subjects with Pyruvate Kinase Deficiency awarded by Agios Pharmaceuticals, Inc. 2019 - 2024
  • Real-World Treatment Patterns and Outcomes in Patients with Hemophagocytic Lymphohistiocytosis (HLH) and other Clinical Conditions Treated with Emapalumab across Treatment Centers in the US (REAL-HLH Study) awarded by SOBI 2021 - 2023
  • Phase III study of AG-348 in not regulary transfused adult subjects with Pyruvate kinase deficiency awarded by Agios Pharmaceuticals, Inc. 2018 - 2023
  • A Phase 3 Study of Eltrombopag vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children awarded by Novartis Pharmaceuticals Corporation 2019 - 2022
  • 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 - 2021
  • Sickle Cell Data Collection Program in North Carolina awarded by NC Department of Health and Human Services 2021
  • Education and Mentoring to BRing Access to CarE SCD - EMBRACE SCD awarded by Carolinas HealthCare System 2017 - 2021
  • CSA - Pfizer 212511 B5201002 Phase 3 Randomized Multicenter Trial awarded by Pfizer, Inc. 2015 - 2021
  • Transfusion Medicine and Hematology awarded by National Institutes of Health 1975 - 2021
  • ITP-APPS (Immune ThrombocytoPenia Association of Platelet Parameters Study) awarded by Boston Children's Hospital 2019 - 2021
  • 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

    • Chonat, Satheesh, Stefan W. Eber, Susanne Holzhauer, Nina Kollmar, D Holmes Morton, Bertil Glader, Ellis J. Neufeld, et al. “Pyruvate kinase deficiency in children.” Pediatr Blood Cancer 68, no. 9 (September 2021): e29148. https://doi.org/10.1002/pbc.29148.
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    • Shimano, Kristin A., Anupama Narla, Melissa J. Rose, Nicholas J. Gloude, Steven W. Allen, Katie Bergstrom, Larisa Broglie, et al. “Diagnostic work-up for severe aplastic anemia in children: Consensus of the North American Pediatric Aplastic Anemia Consortium.” Am J Hematol, August 3, 2021. https://doi.org/10.1002/ajh.26310.
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    • Al-Samkari, Hanny, Kathryn Addonizio, Bertil Glader, D Holmes Morton, Satheesh Chonat, Alexis A. Thompson, Kevin H. M. Kuo, et al. “The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency.” Br J Haematol 192, no. 6 (March 2021): 1092–96. https://doi.org/10.1111/bjh.16724.
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    • Niss, Omar, Robert B. Lorsbach, Mikaela Berger, Satheesh Chonat, Morgan McLemore, David Buchbinder, Timothy McCavit, et al. “Congenital dyserythropoietic anemia type I: First report from the Congenital Dyserythropoietic Anemia Registry of North America (CDAR).” Blood Cells Mol Dis 87 (March 2021): 102534. https://doi.org/10.1016/j.bcmd.2020.102534.
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    • Rothman, Jennifer A., Jenny L. Stevens, Fabienne L. Gray, and Theodosia A. Kalfa. “How I approach hereditary hemolytic anemia and splenectomy.” Pediatr Blood Cancer 67, no. 11 (November 2020): e28337. https://doi.org/10.1002/pbc.28337.
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    • Rothman, Jennifer A. “Osteomyelitis in sickle cell disease: You know it when you see it?” Pediatr Blood Cancer 67, no. 10 (October 2020): e28585. https://doi.org/10.1002/pbc.28585.
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    • Hall, B. J., A. J. Reiter, B. R. Englum, J. A. Rothman, and H. E. Rice. “Long-term hematologic and clinical outcomes of splenectomy in children with hereditary spherocytosis and sickle cell disease.” Pediatric Blood and Cancer 67, no. 8 (August 1, 2020). https://doi.org/10.1002/pbc.28290.
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    • Al-Samkari, Hanny, Eduard J. van Beers, D Holmes Morton, Wilma Barcellini, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, et al. “Characterization of the severe phenotype of pyruvate kinase deficiency.” Am J Hematol, July 3, 2020. https://doi.org/10.1002/ajh.25926.
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    • Allen, T. M., L. M. Anderson, S. M. Brotkin, J. A. Rothman, and M. J. Bonner. “Computerized cognitive training in pediatric sickle cell disease: A randomized controlled pilot study.” Clinical Practice in Pediatric Psychology 8, no. 4 (January 1, 2020): 390–401. https://doi.org/10.1037/cpp0000313.
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    • Rogers, Zora R., Taizo A. Nakano, Timothy S. Olson, Alison A. Bertuch, Winfred Wang, Alfred Gillio, Thomas D. Coates, et al. “Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.” Haematologica 104, no. 10 (October 2019): 1974–83. https://doi.org/10.3324/haematol.2018.206540.
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    • Grace, Rachael F., Kristin A. Shimano, Rukhmi Bhat, Cindy Neunert, James B. Bussel, Robert J. Klaassen, Michele P. Lambert, et al. “Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes.” Am J Hematol 94, no. 7 (July 2019): 741–50. https://doi.org/10.1002/ajh.25479.
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    • Gallagher, Patrick G., Yelena Maksimova, Kimberly Lezon-Geyda, Peter E. Newburger, Desiree Medeiros, Robin D. Hanson, Jennifer Rothman, et al. “Aberrant splicing contributes to severe α-spectrin-linked congenital hemolytic anemia.” J Clin Invest 129, no. 7 (April 30, 2019): 2878–87. https://doi.org/10.1172/JCI127195.
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    • Beers, Eduard J. van, Stephanie van Straaten, D Holmes Morton, Wilma Barcellini, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, et al. “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): e51–53. https://doi.org/10.3324/haematol.2018.196295.
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    • Chonat, Satheesh, Mary Risinger, Haripriya Sakthivel, Omar Niss, Jennifer A. Rothman, Loan Hsieh, Stella T. Chou, et al. “Corrigendum: The Spectrum of SPTA1-Associated Hereditary Spherocytosis.” Front Physiol 10 (2019): 1331. https://doi.org/10.3389/fphys.2019.01331.
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    • Tan, Queenie K-G, Heidi Cope, Rebecca C. Spillmann, Nicholas Stong, Yong-Hui Jiang, Marie T. McDonald, Jennifer A. Rothman, et al. “Further evidence for the involvement of EFL1 in a Shwachman-Diamond-like syndrome and expansion of the phenotypic features.” Cold Spring Harb Mol Case Stud 4, no. 5 (October 2018). https://doi.org/10.1101/mcs.a003046.
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    • Grace, Rachael F., Jenny M. Despotovic, Carolyn M. Bennett, James B. Bussel, Michelle Neier, Cindy Neunert, Shelley E. Crary, et al. “Physician decision making in selection of second-line treatments in immune thrombocytopenia in children.” Am J Hematol 93, no. 7 (July 2018): 882–88. https://doi.org/10.1002/ajh.25110.
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    • Grace, Rachael F., Paola Bianchi, Eduard J. van Beers, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, Jenny M. Despotovic, et al. “Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.” Blood 131, no. 20 (May 17, 2018): 2183–92. https://doi.org/10.1182/blood-2017-10-810796.
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    • Borst, Alexandra J., Debra L. Sudan, Laura A. Wang, Michael J. Neuss, Jennifer A. Rothman, and Thomas L. Ortel. “Bleeding and thrombotic complications of pediatric liver transplant.” Pediatr Blood Cancer 65, no. 5 (May 2018): e26955. https://doi.org/10.1002/pbc.26955.
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    • Allen, Taryn M., Lindsay M. Anderson, Jennifer A. Rothman, and Melanie J. Bonner. “[Formula: see text]Executive functioning and health-related quality of life in pediatric sickle cell disease.” Child Neuropsychol 23, no. 8 (November 2017): 889–906. https://doi.org/10.1080/09297049.2016.1205011.
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    • Cancio, Maria I., Banu Aygun, David H. K. Chui, Jennifer A. Rothman, J Paul Scott, Jeremie H. Estepp, and Jane S. Hankins. “The clinical severity of hemoglobin S/Black (A γδβ)0 -thalassemia.” Pediatr Blood Cancer 64, no. 11 (November 2017). https://doi.org/10.1002/pbc.26596.
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    • Reiter, Audra, Henry Rice, and Jennifer Rothman. “LONG-TERM CLINICAL AND HEMATOLOGIC OUTCOMES OF SPLENECTOMY IN CHILDREN WITH CONGENITAL HEMOLYTIC ANEMIA.” Pediatric Blood & Cancer 63 (June 1, 2016): S57–S57.
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    • Estepp, Jeremie H., Chiara Melloni, Courtney D. Thornburg, Paweł Wiczling, Zora Rogers, Jennifer A. Rothman, Nancy S. Green, et al. “Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia.” J Clin Pharmacol 56, no. 3 (March 2016): 298–306. https://doi.org/10.1002/jcph.598.
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    • Wang, Hsin-Hsiao Scott, Katherine W. Herbst, Jennifer A. Rothman, Nirmish R. Shah, John S. Wiener, and Jonathan C. Routh. “Trends in Sickle Cell Disease-related Priapism in U.S. Children's Hospitals.” Urology 89 (March 2016): 118–22. https://doi.org/10.1016/j.urology.2015.11.023.
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    • Ware, Russell E., Barry R. Davis, William H. Schultz, R Clark Brown, Banu Aygun, Sharada Sarnaik, Isaac Odame, et al. “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 387, no. 10019 (February 13, 2016): 661–70. https://doi.org/10.1016/S0140-6736(15)01041-7.
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    • Luchtman-Jones, Lori, Sara Pressel, Lee Hilliard, R Clark Brown, Mary G. Smith, Alexis A. Thompson, Margaret T. Lee, et al. “Effects of hydroxyurea treatment for patients with hemoglobin SC disease.” Am J Hematol 91, no. 2 (February 2016): 238–42. https://doi.org/10.1002/ajh.24255.
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    • Englum, Brian R., Jennifer Rothman, Sarah Leonard, Audra Reiter, Courtney Thornburg, Mary Brindle, Nicola Wright, et al. “Hematologic outcomes after total splenectomy and partial splenectomy for congenital hemolytic anemia.” J Pediatr Surg 51, no. 1 (January 2016): 122–27. https://doi.org/10.1016/j.jpedsurg.2015.10.028.
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    • Wood, John C., Alan R. Cohen, Sara L. Pressel, Banu Aygun, Hamayun Imran, Lori Luchtman-Jones, Alexis A. Thompson, et al. “Organ iron accumulation in chronically transfused children with sickle cell anaemia: baseline results from the TWiTCH trial.” Br J Haematol 172, no. 1 (January 2016): 122–30. https://doi.org/10.1111/bjh.13791.
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    • Allewelt, Heather, Jessica Saricicek, Sachit Patel, and Jennifer Rothman. “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.
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    • Rice, Henry E., Brian R. Englum, Jennifer Rothman, Sarah Leonard, Audra Reiter, Courtney Thornburg, Mary Brindle, et al. “Clinical outcomes of splenectomy in children: report of the splenectomy in congenital hemolytic anemia registry.” Am J Hematol 90, no. 3 (March 2015): 187–92. https://doi.org/10.1002/ajh.23888.
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    • Mize, Lindsay, Shelly Burgett, Julia Xu, Jennifer Rothman, and Nirmish Shah. “The Use of Chronic Transfusions in Sickle Cell Disease for Non-Stroke Related Indications.” Blood 124, no. 21 (December 6, 2014).
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    • Zimmerman, Sarah P., Jennifer A. Rothman, Jessica L. Hansen, Molly M. Rusin, Matthew A. Bertone, and Harvey J. Hamrick. “Systemic loxoscelism in a nonendemic area: a diagnostic challenge for the unsuspecting physician.” Clin Pediatr (Phila) 53, no. 11 (October 2014): 1098–1100. https://doi.org/10.1177/0009922813512419.
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    • Leonard, Sarah, Jennifer Rothman, Nirmish Shah, Courtney Thornburg, and Henry Rice. “CURRENT PRACTICE PATTERNS FOR PARTIAL VERSUS TOTAL SPLENECTOMY IN PEDIATRIC PATIENTS WITH CONGENITAL HEMOLYTIC ANEMIA.” Pediatric Blood & Cancer 61 (May 1, 2014): S92–S92.
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    • Mouttalib, Sofia, Henry E. Rice, Denise Snyder, Justin S. Levens, Audra Reiter, Pauline Soler, Jennifer A. Rothman, and Courtney D. Thornburg. “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. https://doi.org/10.1002/pbc.24057.
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    • Shah, Nirmish, Daniel Landi, Radhika Shah, Jennifer Rothman, Laura M. De Castro, and Courtney D. Thornburg. “Complications of implantable venous access devices in patients with sickle cell disease.” Am J Hematol 87, no. 2 (February 2012): 224–26. https://doi.org/10.1002/ajh.22230.
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    • Rothman, Jennifer A., Rupali Das, David T. Teachey, Michele E. Paessler, and Kim E. Nichols. “Rapamycin does not control hemophagocytic lymphohistiocytosis in LCMV-infected perforin-deficient mice.” Pediatr Blood Cancer 57, no. 7 (December 15, 2011): 1239–43. https://doi.org/10.1002/pbc.23226.
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    • Mouttalib, Sofia, Denise Snyder, Justin Levens, Pauline Soler, Jennifer Rothman, Courtney Thornburg, and Henry Rice. “USE OF INTERNET-BASED REGISTRY FOR EVALUATION OF PARTIAL AND TOTAL SPLENECTOMY IN SICKLE CELL DISEASE.” Pediatric Blood & Cancer 56, no. 6 (June 1, 2011): 911–911.
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    • Talaat, Kawsar R., Jennifer A. Rothman, Jeffrey I. Cohen, Mariarita Santi, John K. Choi, Miguel Guzman, Robert Zimmerman, et al. “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): 1120–23. https://doi.org/10.1002/pbc.22185.
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    • Impens, A. J., J. Rothman, E. Schiopu, J. C. Cole, J. Dang, N. Gendrano, R. C. Rosen, and J. R. Seibold. “Sexual activity and functioning in female scleroderma patients.” Clinical and Experimental Rheumatology 27, no. 3 SUPPL. 54 (November 9, 2009).
    • Schiopu, Elena, Vivien M. Hsu, Ann J. Impens, Jennifer A. Rothman, Deborah A. McCloskey, Julianne E. Wilson, Kristine Phillips, and James R. Seibold. “Randomized placebo-controlled crossover trial of tadalafil in Raynaud's phenomenon secondary to systemic sclerosis.” J Rheumatol 36, no. 10 (October 2009): 2264–68. https://doi.org/10.3899/jrheum.090270.
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    • Impens, A. J., J. Rothman, E. Schiopu, J. C. Cole, J. Dang, N. Gendrano, R. C. Rosen, and J. R. Seibold. “Sexual activity and functioning in female scleroderma patients.” Clin Exp Rheumatol 27, no. 3 Suppl 54 (May 2009): 38–43.
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    • Nunez-Cruz, Selene, WC Janice Yeo, Jennifer Rothman, Priti Ojha, Hamid Bassiri, Marisa Juntilla, Dominique Davidson, André Veillette, Gary A. Koretzky, and Kim E. Nichols. “Differential requirement for the SAP-Fyn interaction during NK T cell development and function.” J Immunol 181, no. 4 (August 15, 2008): 2311–20. https://doi.org/10.4049/jimmunol.181.4.2311.
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    • Rao, Aarati, Jennifer Rothman, and Kim E. Nichols. “Genetic testing and tumor surveillance for children with cancer predisposition syndromes.” Curr Opin Pediatr 20, no. 1 (February 2008): 1–7. https://doi.org/10.1097/MOP.0b013e3282f4249a.
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    • Bassiri, Hamid, W. C. Janice Yeo, Jennifer Rothman, Gary A. Koretzky, and Kim E. Nichols. “X-linked lymphoproliferative disease (XLP): a model of impaired anti-viral, anti-tumor and humoral immune responses.” Immunol Res 42, no. 1–3 (2008): 145–59. https://doi.org/10.1007/s12026-008-8048-7.
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    • Chonat, Satheesh, Mary Risinger, Haripriya Sakthivel, Omar Niss, Jennifer A. Rothman, Loan Hsieh, Stella T. Chou, et al. “The Spectrum of SPTA1-Associated Hereditary Spherocytosis.” Frontiers in Physiology 10 (n.d.). https://doi.org/10.3389/fphys.2019.00815.
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  • Book Sections

    • Greiner, R. J., S. Peterson-Carmichael, J. A. Rothman, K. W. Gow, R. F. Tamburro, and R. Barfield. “Care of the oncology patient in the picu.” In Pediatric Critical Care Medicine: Volume 3: Gastroenterological, Endocrine, Renal, Hematologic, Oncologic and Immune Systems, 343–61, 2014. https://doi.org/10.1007/978-1-4471-6416-6_24.
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  • Conference Papers

    • Rothman, Jennifer, and Ashley Thrower. “COVID-19 AND SICKLE CELL DISEASE (SCD): MULTI-SYSTEM INFLAMMATORY SYNDROME IN CHILDREN WITH SCD.” In Pediatric Blood & Cancer, 68:S123–S123, 2021.
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    • Robles, Joanna, Marissa Just, Ashley Frazier, Karan Kumar, Andrew Yazman, Heather McLean, Jennifer Rothman, and Kristy Pahl. “VTE IN HOSPITALIZED PEDIATRIC PATIENTS: IMPLEMENTING A RISK ASSESSMENT TOOL.” In Pediatric Blood & Cancer, 67:S59–S59. WILEY, 2020.
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    • Addonizio, Kathryn, Hanny Al-Samkari, Bertil Glader, D Holmes Morton, Satheesh Chonat, Alexis A. Thompson, Kevin H. M. Kuo, et al. “Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency.” In Blood, 134:3515–3515. American Society of Hematology, 2019. https://doi.org/10.1182/blood-2019-125248.
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    • Al-Samkari, Hanny, Eduard J. van Beers, D Holmes Morton, Wilma Barcellini, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, et al. “Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency.” In Blood, 134:949–949. American Society of Hematology, 2019. https://doi.org/10.1182/blood-2019-125352.
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    • Boscoe, Audra N., Yan Yan, Elizabeth Hedgeman, Eduard J. van Beers, Hanny Al-Samkari, Wilma Barcellini, Stefan W. Eber, et al. “Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency.” In Blood, 134:2175–2175. American Society of Hematology, 2019. https://doi.org/10.1182/blood-2019-123069.
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    • Just, Marissa A., Joanna Robles, Karan R. Kumar, Andrew Yazman, Jennifer A. Rothman, and Kristy S. Pahl. “A Retrospective Review of Hospital-Acquired Venous Thromboembolism at a Large Pediatric Tertiary Care Center.” In Blood, 134:3471–3471. American Society of Hematology, 2019. https://doi.org/10.1182/blood-2019-129398.
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    • Nannes, Caleb, Jennifer Rothman, Addison Gross, and Susan Kreissman. “IMPROVING NUTRITIONAL SUPPORT FOR PEDIATRIC HEMATOLOGY & ONCOLOGY PATIENTS.” In Pediatric Blood & Cancer, Vol. 66. WILEY, 2019.
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    • Chonat, Satheesh, Mary Risinger, Haripriya Sakthivel, Omar Niss, Jennifer A. Rothman, Loan Hsieh, Stella T. Chou, et al. “The Spectrum of SPTA1-Associated Hereditary Spherocytosis.” In Front Physiol, 10:815, 2019. https://doi.org/10.3389/fphys.2019.00815.
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    • Van Beers, Eduard J., Kevin H. M. Kuo, D Holmes Morton, Wilma Barcellini, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, et al. “Health Related Quality of Life and Fatigue in Patients with Pyruvate Kinase Deficiency.” In Blood, 132:4807–4807. American Society of Hematology, 2018. https://doi.org/10.1182/blood-2018-99-113206.
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    • Grace, Rachael F., Robert J. Klaassen, Jennifer A. Rothman, Kerry Hege, Ellis J. Neufeld, Carolyn M. Bennett, Michele Lambert, et al. “Clinical Characteristics and Quality of Life of Children with ITP Starting Second Line Treatments: Data from the ITP Consortium of North America ICON1 Study.” In Blood, 128:249–249. American Society of Hematology, 2016. https://doi.org/10.1182/blood.v128.22.249.249.
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    • Lambert, Michele P., Rachael F. Grace, Cindy Neunert, Jenny M. Despotovic, Rukhmi Bhat, Yves D. Pastore, George R. Buchanan, et al. “Physician Factors Determining Treatment Decisions in Selecting Second Line Agents for Pediatric ITP.” In Blood, 128:1008–1008. American Society of Hematology, 2016. https://doi.org/10.1182/blood.v128.22.1008.1008.
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    • Leraas, Harold J., Jina Kim, Zhifei Sun, Uttara P. Nag, Brian D. Ezekian, Reed W. Kamyszek, Brian C. Gulack, et al. “Postoperative Venous Thromboembolism in Children Is Increased in Setting of Cancer or Infection.” In Blood, 128:2391–2391. American Society of Hematology, 2016. https://doi.org/10.1182/blood.v128.22.2391.2391.
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    • Ware, Russell E., Adam Lane, William H. Schultz, Hamayun Imran, Margaret T. Lee, Matthew M. Heeney, Clark Brown, et al. “Variation in Serial TCD Velocity Measurements in the TCD with Transfusions Changing to Hydroxyurea (TWiTCH) Trial.” In Blood, 128:1019–1019. American Society of Hematology, 2016. https://doi.org/10.1182/blood.v128.22.1019.1019.
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    • Bianchi, Paola, Elisa Fermo, Kimberly Lezon-Geyda, Patrick G. Gallagher, D Holmes Morton, Wilma Barcellini, Bertil Glader, et al. “Molecular Characterization of 140 Patients in the Pyruvate Kinase Deficiency (PKD) Natural History Study (NHS): Report of 20 New Variants.” In Blood, Vol. 126. AMER SOC HEMATOLOGY, 2015.
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    • Grace, Rachael F., D Holmes Morton, Wilma Barcellini, Stefan W. Eber, Jenny M. Depsotovic, Christine M. Knoll, Hassan M. Yaish, et al. “The Phenotypic Spectrum of Pyruvate Kinase Deficiency (PKD) from the PKD Natural History Study (NHS): Description of Four Severity Groups By Anemia Status.” In Blood, Vol. 126. AMER SOC HEMATOLOGY, 2015.
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    • Ware, Russell E., Barry R. Davis, William H. Schultz, Clark Brown, Banu Aygun, Sharada A. Sarnaik, Isaac Odame, 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.” In Blood, Vol. 126. AMER SOC HEMATOLOGY, 2015.
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    • Grace, R., W. Barcellini, S. Eber, J. Kunz, J. Despotovic, A. Thompson, D. H. Morton, et al. “CATEGORIZATION OF CLINICAL SEVERITY IN PYRUVATE KINASE DEFICIENCY (PKD) IN AN INTERNATIONAL, OBSERVATIONAL COHORT.” In Haematologica, 100:130–31. FERRATA STORTI FOUNDATION, 2015.
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    • Morton, D. H., C. Knoll, J. Rothman, B. Glader, E. Neufeld, W. Barcellini, S. Eber, et al. “THE CLINICAL FEATURES AND TREATMENT OF IRON OVERLOAD IN PYRUVATE KINASE DEFICIENCY (PKD): DATA FROM THE PKD NATURAL HISTORY STUDY (NHS).” In Haematologica, 100:588–588. FERRATA STORTI FOUNDATION, 2015.
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