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1. Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia

Author

Kim, Taylor Olmsted, Geris, Jennifer M., Flanagan, Jonathan M., Grace, Rachael F., Lambert, Michele P., O’Farrell, Candelaria, Rose, Melissa J., Shimano, Kristin A., Niss, Omar, Neunert, Cindy, Nakano, Taizo A., MacMath, Derek, Dinu, Bogdan, Kirk, Susan E., Neufeld, Ellis J., Despotovic, Jenny M., Scheurer, Michael E., and Grimes, Amanda B.

Published
2024
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4. What is in a name: defining pediatric refractory ITP

Author

Nakano, Taizo A., Grimes, Amanda B., Klaassen, Robert J., Lambert, Michele P., Neunert, Cindy, Rothman, Jennifer A., Shimano, Kristin A., Amend, Christina, Askew, Megan, Badawy, Sherif M., Baker, Jillian M., Breakey, Vicky, Crary, Shelley, Davini, Monica, Fritch Lilla, Stephanie, Gilbert, Megan, Hays, Taru, Hege, Kerry, Hillier, Kirsty, Jacobson-Kelly, Amanda, Kaicker, Shipra, Kim, Taylor O., Kochhar, Manpreet, Leblanc, Thierry, Martinelli, Marie, Nunez, Mara, Remiker, Allison, Schultz, Corrina, Sharma, Ruchika, and Grace, Rachael F.

Abstract

There are no agreed upon terminology to define “refractory” pediatric immune thrombocytopenia (ITP). Guidelines are therefore limited to arbitrary and outdated definitions. The Pediatric ITP Consortium of North America held a meeting in 2023 to define this entity. With 100% agreement, the faculty established that pediatric ITP that is refractory to emergent therapy could be defined as no platelet response after treatment with all eligible emergent pharmacotherapies. With 100% agreement, the working group established that pediatric patients with ITP that continue to demonstrate high disease burden and/or no platelet response despite treatment with multiple classes of disease-modifying therapies represent a challenging subset of ITP. These patients are at higher risk of ongoing disease burden and merit additional investigation as well as consideration for clinical trials or novel therapies. Future efforts to define disease burden and disease response will be completed in collaboration with the ITP International Working Group.

Published
2024
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7. Pediatric refractory immune thrombocytopenia: A systematic review

Author

Ibrahim, Layan, primary, Dong, Selina X., additional, O'Hearn, Katie, additional, Grimes, Amanda B., additional, Kaicker, Shipra, additional, FritchLilla, Stephanie, additional, Breakey, Vicky R., additional, Grace, Rachael F., additional, Lebensburger, Jeffrey D., additional, Klaassen, Robert J., additional, and Lambert, Michele, additional

Published
2022
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12. Premedication with Hydrocortisone Increases Risk of Adverse Drug Events and Return to Medical Care Among Pediatric Patients with ITP Treated with IVIG

Author

Gilbert, Megan M, primary, Kim, Taylor Olmsted, additional, Kirk, Susan E, additional, Han, Hyojeong, additional, Huang, Xiaofan, additional, Staggers, Kristen A, additional, Grace, Rachael F., additional, Neunert, Cindy, additional, Lambert, Michele P., additional, Despotovic, Jenny M., additional, and Grimes, Amanda B, additional

Published
2021
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18. A Phase 3 Study of Eltrombopag Vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia in Children: Trial in Progress Update

Author

Grimes, Amanda B., Grace, Rachael F., Rose, Melissa J., Shah, Sanjay J., Kaicker, Shipra, Bennett, Carolyn, Briones, Michael, Nakano, Taizo A., Fritch Lilla, Stephanie A., Lebensburger, Jeffrey D., Thompson, Alexis A., Bhat, Rukhmi, Jesudas, Rohith, Lambert, Michele P., Crary, Shelley E., Hege, Kerry, Lee-Miller, Cathy A., Majumdar, Suvankar, Ford, James B., Bies, Joshua J., Black, Vandy, Fort, John, Kochhar, Manpreet, Wong, Julia MW, Dinu, Bogdan, Kao, Pei-Chi, London, Wendy B., Despotovic, Jenny M., Klaassen, Robert J., Neufeld, Ellis J., Neunert, Cindy, and Shimano, Kristin A.

Published
2023
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22. Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use

Author

Gilbert,Megan M, Grimes,Amanda B, Kim,Taylor Olmsted, and Despotovic,Jenny M

Subjects
Patient Preference and Adherence, hemic and lymphatic diseases
Abstract

Megan M Gilbert, Amanda B Grimes, Taylor Olmsted Kim, Jenny M Despotovic Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, TX, USACorrespondence: Megan M GilbertDepartment of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Texas Children’s Hospital, 6701 Fannin Suite 1510, Houston, TX 77030, USATel +1 (832) 824-4736Fax +1 (832) 825-4846Email megan.gilbert@bcm.eduAbstract: Immune thrombocytopenia (ITP) is an immune-mediated disorder resulting in platelet destruction and subsequent thrombocytopenia. Bleeding symptoms range from mild cutaneous bleeding to life-threatening hemorrhage. Romiplostim, a peptide-antibody fusion product, is a thrombopoietin receptor agonist (TPO-RA) indicated for use in patients with ITP. Romiplostim is US Food and Drug Administration (FDA) approved in children ≥ 1 year of age with ITP of > 6 months’ duration who have had an inadequate response to first-line therapies or splenectomy. FDA approval in adults with chronic ITP was expanded in October 2019 to include adults with newly diagnosed (< 3 months’ duration) and persistent (3– 12 months’ duration) ITP who demonstrated an inadequate response to first-line therapies, including corticosteroids and immunoglobulins, or splenectomy. The newly published 2019 American Society of Hematology ITP Guidelines place TPO-RAs, including romiplostim, as second-line therapies in both children and adults. Here, we review the use of romiplostim as second-line therapy with a spotlight on health-related quality of life, ease of use, and patient preference.Keywords: romiplostim, immune thrombocytopenia, quality of life, patient preference

Published
2020

29. Pediatric eosinophilia: A review and multiyear investigation into etiologies.

Author

Ness, Tara E., Erickson, Timothy A., Diaz, Veronica, Grimes, Amanda B., Rochat, Ryan, Anvari, Sara, Hajjar, Joud, and Weatherhead, Jill

Abstract

Objectives: To identify the etiology of peripheral eosinophilia in a large pediatric population and to develop a diagnostic algorithm to help guide diagnosis and management of peripheral eosinophilia in the outpatient pediatric population.Study Design: We performed a retrospective chart review of children presenting to Texas Children's Hospital in Houston with peripheral eosinophilia between January 1, 2011 and December 31, 2019. Eosinophilia was classified as mild (absolute eosinophil count [AEC] >500 and <1500 cells/μL), moderate (AEC >1500 and <4500 cells/μL), or severe (AEC >4500 cells/μL). Demographic information and diagnostic workup data were collected.Results: A total of 771 patients aged <18 years were evaluated. The most common cause of eosinophilia was allergy (n = 357; 46%), with atopy (n = 296) and drug reaction (n = 54) the most common subcauses. This was followed by unknown etiology (n = 274; 36%), infectious causes (n = 72; 9%), and eosinophilic disorders (n = 47; 6%). Many patients with an unknown cause (n = 202; 74%) had limited or no follow-up testing.Conclusions: More information on the etiology of pediatric eosinophilia and workup data could help identify the causes. This study provides important information on the evaluation of eosinophilia in the US pediatric population, including a diagnostic algorithm to guide primary care pediatricians. [ABSTRACT FROM AUTHOR]

Published
2023
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