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Christine M. Knoll, MD
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Journal Articles

Inhibit Clinical Trials Platform to Prevent and Eradicate Inhibitors: Feasibility Survey of Current Prophylaxis and Immune Tolerance Practices

Margaret V. Ragni, MD MPH, Frederico Xavier, MD MS, Craig D. Seaman, Suchitra Acharya, MD, Catherine E. McGuinn, MD, Eric J. Werner, MD, Courtney Elizabeth Lawrence, MDMS, Allison P. Wheeler, MD, Ulrike Reiss, MD, Irmel Ayala, MD, Erin Cockrell, DO, Cristina Tarango, MD, Amy Dunn, Roshni Kulkarni, MD, Sanjay P Ahuja, MD, Meera B. Chitlur, MD, Steven W. Pipe, MD, Lynn M Malec, MD, Vilmarie Rodriguez, MD, Shelley Crary, MD MS, Deborah Brown, MD, Rosa Diaz, MD, Maria Velez, MD, Cindy A. Leissinger, MD, Shannon L Carpenter, MD, Christine M. Knoll, MD, Michael Wang, MD, Guy Young, MD, Courtney D Thornburg, MD, Joseph L Lasky, III, MD, Tiffany Lin Lucas, MD, Nina Hwang, MD, Deborah Vehec, MSN, RN, Dana Ivanco, Tamara L. Haller, BS, Marnie Bertolet, PhD, Maria M Brooks, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 14–15.
https://doi.org/10.1182/blood-2020-136814
Published: 2020
Journal Articles

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Hanny Al-Samkari, MD, Eduard J. van Beers, MD PhD, D Holmes Morton, MD, Wilma Barcellini, MD, Stefan W. Eber, MD, Bertil Glader, MD PhD, Hassan M. Yaish, MD, Satheesh Chonat, MD, Kevin H. M. Kuo, MDMSc,FRCPC, Nina Kollmar, MD, Jenny M. Despotovic, DO, MS, Dagmar Pospisilova, Prof MD PhD, Christine M. Knoll, MD, Janet L. Kwiatkowski, MD MSCE, Yves D. Pastore, MD, Alexis A. Thompson, MD MPH, Marcin W. Wlodarski, MD PhD, Yaddanapudi Ravindranath, MBBS, Jennifer A. Rothman, MD, Heng Wang, MD PhD, Suzanne Holzhauer, MD MSc, Vicky R. Breakey, BSc,MDFRCPC, Madeleine M. Verhovsek, MD FRCPC, Joachim B. Kunz, MD, Sujit Sheth, MD, Mukta Sharma, MD MPH, Melissa J. Rose, DO, Heather Bradeen, Melissa A. McNaull, MD, Kathryn Addonizio, BS, Hasan Al-Sayegh, Wendy B. London, PhD, Rachael F. Grace, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 949.
https://doi.org/10.1182/blood-2019-125352
Published: 2019
Journal Articles

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

Audra N. Boscoe, PhD, Yan Yan, MS, Elizabeth Hedgeman, PhD, Eduard J. van Beers, MD PhD, Hanny Al-Samkari, MD, Wilma Barcellini, MD, Stefan W. Eber, MD, Bertil Glader, MD PhD, Hassan M. Yaish, MD, Satheesh Chonat, MD, Jennifer A. Rothman, MD, Mukta Sharma, MD MPH, Nina Kollmar, MD, Christine M. Knoll, MD, Kevin H. M. Kuo, MDMSc,FRCPC, Janet L. Kwiatkowski, MD MSCE, Yves D. Pastore, MD, Alexis A Thompson, MD MPH, Yaddanapudi Ravindranath, MBBS, Ellis J. Neufeld, MD, Heng Wang, MD PhD, Susanne Holzhauer, MD MSc, Vicky R. Breakey, BSc,MDFRCPC, Madeleine M. Verhovsek, MD FRCPC, Joachim B. Kunz, MD, Sujit Sheth, MD, Melissa A. McNaull, MD, Melissa J. Rose, DO, Dagmar Pospisilova, MD PhD, Jenny M. Despotovic, DO, Rachael F. Grace, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2175.
https://doi.org/10.1182/blood-2019-123069
Published: 2019
Journal Articles

Assessing Efficacy of Provider and Family Education, Electronic Health Record Documentation Tools, and Information Technology to Increase Use of Hydroxyurea in Children with Sickle Cell Anemia

Kyle Hickman, MD, Steve McCalley, BSN, RN-BC, Vinay Vaidya, MD, Esteban Gomez, MD, Christine M. Knoll, MD, Joanna L. Gendreau, DO, Lori Wagner, MSN, FNP-C, Meenakshi Goyal-Khemka, MD, Brooke Cook, BSN, RN, Kelli Fischbeck, BSN, RN, Sanjay J. Shah, MBBS
Journal: Blood
Blood (2019) 134 (Supplement_1): 2288.
https://doi.org/10.1182/blood-2019-127770
Published: 2019
Journal Articles

Use of a Electronic Sickle Cell Dashboard to Improve Annual Transcranial Doppler Ultrasound (TCD) Completion Rates

Joanna L. Gendreau, DO, Kelli Fischbeck, BSN, RN, Brooke Cook, BSN, RN, Steve McCalley, BSN, RN-BC, Lori Wagner, MSN, FNP-C, Vinay Vaidya, MD, Meenakshi Goyal-Khemka, MD, Esteban Gomez, MD, Christine M. Knoll, MD, Sanjay J. Shah, MBBS
Journal: Blood
Blood (2019) 134 (Supplement_1): 3384.
https://doi.org/10.1182/blood-2019-128846
Published: 2019
Journal Articles

Pklr Intron Splicing-Associated Mutations and Alternate Diagnoses Are Common in Pyruvate Kinase Deficient Patients with Single or No Pklr Coding Mutations

Kimberly Lezon-Geyda, PhD, Melissa J. Rose, DO, Melissa A. McNaull, MD, Christine M. Knoll, MD, Hassan M. Yaish, MD, Yves D. Pastore, MD, Elisa Fermi, Bertil Glader, MD, Paola Bianchi, BSc, PhD, Rachael F. Grace, MD, Patrick G. Gallagher, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3607.
https://doi.org/10.1182/blood-2018-99-117805
Published: 2018
Journal Articles

Leucine for the Treatment of Transfusion Dependence in Patients with Diamond Blackfan Anemia

Adrianna Vlachos, MD, Evangelia Atsidaftos, MA, MPH, Ellen Muir, RN, MSN, Zora R. Rogers, MD, Mohammad Lufti Lababidi, MD, Waseem Alhushki, MD, Jason E. Farrar, MD, Bertil Glader, MD, Barbara Gruner, MD, Helge Hartung, MD, Christine M. Knoll, MD, Grzegorz Nalepa, MDPhD, Anupama Narla, MD, Arun Ranjan Panigrahi, MD, Colin A. Sieff, MB, BCh, Kelly J. Walkovich, MD, Jeffrey M Lipton, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 755.
https://doi.org/10.1182/blood-2018-99-113570
Published: 2018
Journal Articles

Risk Factors and Outcomes of Deep Vein Thrombosis in Pediatric Osteomyelitis

Maria Ahmad, MBBS, Christine M. Knoll, MD, Sanjay J. Shah, MBBS, Lucia Mirea, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 5054.
https://doi.org/10.1182/blood-2018-99-111411
Published: 2018
Journal Articles

Health Related Quality of Life and Fatigue in Patients with Pyruvate Kinase Deficiency

Eduard J. Van Beers, MD PhD, Kevin H. M. Kuo, MD MSc, FRCPC, D. Holmes Morton, MD, Wilma Barcellini, MD, Stefan W. Eber, MD, Bertil Glader, MD, Hassan M. Yaish, MD, Satheesh Chonat, MD, Nina Kollmar, MD, Jenny M. Despotovic, DO, MS, Dagmar Pospisilova, Prof. MD PhD, Christine M. Knoll, MD, Janet L. Kwiatkowski, MD, Yves D. Pastore, MD, Alexis A. Thompson, MD MPH, Winfred Wang, MD, Marcin W. Wlodarski, MD PhD, Peter E. Newburger, MD, Yaddanapudi Ravindranath, MBBS, Jennifer A. Rothman, MD, Heng Wang, MD PhD, Suzanne Holzhauer, MD MSc, Vicky R. Breakey, BSc, MD FRCPC, Madeleine M Verhovsek, MD FRCPC, Joachim B. Kunz, MD, Sujit Sheth, MD, Mukta Sharma, MD MPH, Melissa J. Rose, DO, Heather A. Bradeen, MD, Melissa A. McNaull, MD, Kathryn Addonizio, BA, David N Williams, Rachael F. Grace, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 4807.
https://doi.org/10.1182/blood-2018-99-113206
Published: 2018
Journal Articles

Genotype-Phenotype Correlation and Molecular Heterogeneity in Pyruvate Kinase Deficiency: Data from the PKD Natural History Study

Paola Bianchi, BSc, PhD, Elisa Fermo, PhD, Kimberly Lezon-Geyda, PhD, Patrick G. Gallagher, MD, Richard van Wijk, PhD, Eduard J. Van Beers, MD, Hassan M. Yaish, MD, Janet L. Kwiatkowski, MD, Peter E. Newburger, MD, Yaddanapudi Ravindranath, MBBS, Oliver Andres, MD, Wilma Barcellini, MD, D. Holmes Morton, MD, Bertil Glader, MD, Stefan W. Eber, MD, Satheesh Chonat, MD, Nina Kollmar, MD, Jenny M. Despotovic, DO, MS, Christine M. Knoll, MD, Kevin H.M. Kuo, MD MSc, FRCPC, Dagmar Pospisilova, Prof. MD PhD, Yves D. Pastore, MD, Alexis A Thompson, MD MPH, Winfred Wang, MD, Marcin W. Wlodarski, MD PhD, Jennifer A. Rothman, MD, Heng Wang, MD PhD, Suzanne Holzhauer, MD MSc, Vicky R. Breakey, BSc,MDFRCPC, Joachim B. Kunz, MD, Sujit Sheth, MD, Mukta Sharma, MD MPH, Melissa J. Rose, DO, Heather A. Bradeen, MD, Melissa N. McNaull, MD, Gabrielle P. Ooyama, Nolan Neu, BS, Hasan Al-Sayegh, Wendy B. London, PhD, Rachael F. Grace, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3479.
https://doi.org/10.1182/blood.V130.Suppl_1.3479.3479
Published: 2017
Journal Articles

G-CSF Vs Haematopoietic Stem Cell Transplantation in Severe Congenital Neutropenia with ELANE Mutation: Role of G-CSF Dose. a Retrospective Controlled Study on Behalf of Saawp (Severe Aplastic Anemia Working Party) of the EBMT, of the Stem Cell Transplant for Immunodeficiency Group in Europe (SCETIDE), of the Severe Chronic Neutropenia French Registry (SCNFR) and Italian Neutropenia Registry (INR)

Francesca Fioredda, MD, Filomena Pierri, Sabrina Zanardi, Bobby Gaspar, Phil Ancliff, Marc Bierings, MD PhD, Gulyuz Ozturk, Owen Smith, MD, Paul Veys, MD PhD, Per Ljungman, Regis Peffault De Latour, MD PhD, Reuven Or, MD, Robert Wynn, MD, Krzysztof Kalwak, MD, Giovanna Russo, MD, Fabio Tucci, MD, Christine M. Knoll, MD, Baldo Martire, Claudio Favre, MD, Christine bellanne Chantelot, MDPhD, Blandine Beaupain, MsC, Jean Donadieu, MD, Carlo Dufour, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 2267.
https://doi.org/10.1182/blood.V130.Suppl_1.2267.2267
Published: 2017
Journal Articles

Pegylated Full-Length Recombinant Factor VIII with Extended Half-Life in Previously Treated Pediatric Patients with Hemophilia Α: Efficacy of Prophylaxis and Treatment By Time (of Day/of Week) of Occurrence of Bleeding

Eric S. Mullins, MD, Christine M. Knoll, MD, Amy L. Dunn, MD, Werner Engl, PhD Cstat, Marlies Sharkhawy, MA, Brigitt E. Abbuehl, MD
Journal: Blood
Blood (2016) 128 (22): 1409.
https://doi.org/10.1182/blood.V128.22.1409.1409
Published: 2016
Journal Articles

Iron Overload Is Highly Prevalent in All Disease Severity States in Pyruvate Kinase Deficiency (PKD)

Eduard J van Beers, MD PhD, Wilma Barcellini, Stefan W. Eber, MD, Janet L Kwiatkowski, MD, Jennifer A Rothman, MD, Ellis J Neufeld, MD PhD, Mukta Sharma, MD MPH, D Holmes Morton, MD, Bertil Glader, MD PhD, Nina Kollmar, MD, Hassan M. Yaish, Jenny M. Despotovic, DO, MS, Christine M. Knoll, MD, Yves D. Pastore, MD, Kevin Kuo, BSc, MD, Peter E. Newburger, MD, Marcin W Wlodarski, MD, Alexis A. Thompson, MD MPH, Yaddanapudi Ravindranath, Winfred C. Wang, MD, Heng Wang, MD, Joachim B. Kunz, MD, Vicky R. Breakey, BSc, MD MD FRCPC, Melissa J. Rose, DO, Melissa Rhodes, MD, Heather A Bradeen, MD, Sujit Sheth, MD, Dongjing Guo, MS, Wendy B. London, PhD, Rachael F. Grace, MD
Journal: Blood
Blood (2016) 128 (22): 2430.
https://doi.org/10.1182/blood.V128.22.2430.2430
Published: 2016
Journal Articles

The Phenotypic Spectrum of Pyruvate Kinase Deficiency (PKD) from the PKD Natural History Study (NHS): Description of Four Severity Groups By Anemia Status

Rachael F. Grace, MD, D Holmes Morton, MD, Wilma Barcellini, MD, Stefan W. Eber, MD, Jenny M Depsotovic, DO, Christine M. Knoll, MD, Hassan M. Yaish, MD, Peter E. Newburger, MD, Jennifer Rothman, MD, Alexis A. Thompson, MD, Yaddanapudi Ravindranath, MBBS, Joachim Kunz, MD, Eduard J van Beers, MD PhD, Marcin W Wlodarski, MD, Kerri A Nottage, MD MPH, Heng Wang, MD, Mukta Sharma, MD MPH, Melissa J. Rose, DO, Yves D. Pastore, MD, Patricia J. Giardina, MD, Melissa Rhodes, MD, Kevin Kuo, BSc, MD, Vicky R. Breakey, BSc, MD MD FRCPC, Ellis J. Neufeld, MD PhD, Dongjing Guo, MS, Wendy B. London, PhD, Jill Falcone, Bertil Glader, MD PhD
Journal: Blood
Blood (2015) 126 (23): 2136.
https://doi.org/10.1182/blood.V126.23.2136.2136
Published: 2015
Journal Articles

Molecular Characterization of 140 Patients in the Pyruvate Kinase Deficiency (PKD) Natural History Study (NHS): Report of 20 New Variants

Paola Bianchi, BSC, PhD, Elisa Fermo, PhD, Kimberly Lezon-Geyda, PhD, Patrick G. Gallagher, MD, D Holmes Morton, MD, Wilma Barcellini, MD, Bertil Glader, MD PhD, Stefan W. Eber, MD, Jenny M. Despotovic, DO, MS, Christine M. Knoll, MD, Hassan M. Yaish, MD, Peter E. Newburger, MD, Jennifer Rothman, MD, Alexis A. Thompson, MD, Yaddanapudi Ravindranath, MBBS, Kerri A Nottage, MD MPH, Heng Wang, MD, Eduard J. van Beers, MD PhD, Joachim Kunz, MD, Marcin W Wlodarski, MD, Mukta Sharma, MD MPH, Melissa J. Rose, DO, Yves D. Pastore, MD, Patricia J. Giardina, MD, Melissa Rhodes, MD, Kevin Kuo, BSc, MD, Dongjing Guo, MS, Wendy B. London, PhD, Rachael F. Grace, MD
Journal: Blood
Blood (2015) 126 (23): 3337.
https://doi.org/10.1182/blood.V126.23.3337.3337
Published: 2015