Cindy E. Neunert

American Society of Hematology 2026 Guidelines for Immune Thrombocytopenia (ITP): Initial and Second-Line Therapy in Adults with Primary ITP

Adam Cuker, Deirdra R. Terrell, Honieh Sowdagar, Donald M. Arnold et autres

BACKGROUND: Since publication of the American Society of Hematology 2019 Guidelines for Immune Thrombocytopenia (ITP), new clinical trials have been completed and new treatments have become available. OBJECTIVE: These evidence-based guidelines from the American Society of Hematology (ASH) are a focused update …

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End-of-study results from the ICON3 pines trial, a phase 3, randomized trial of eltrombopag vs. standard first-line treatment for newly diagnosed immune thrombocytopenia in children

Kristin Ammon Shimano, Amanda Bell Grimes, Melissa J. Rose, Shipra M. Kaicker et autres

Abstract Background: The Pediatric ITP Newly diagnosed patients Eltrombopag vs Standard therapy (PINES) trial, NCT03939637, was an investigator-initiated, prospective, open label, randomized, multi-center trial sponsored by the ITP Consortium of North America (ICON) and funded by Novartis. Patients (pts) ages 1-<18 with …

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Shared decision making for immune thrombocytopenia (ITP) treatment: Data from the ITP natural history study registry

Jennifer MacWhirter - DiRaimo, Kevin Doyoon Won, Caroline Gentile Kruse, Cindy E. Neunert

Abstract Introduction: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder associated with an increased risk for bleeding. Various treatment strategies exist for ITP; however, it is unknown how frequently patients are involved in shared decision-making (SDM) related to their care. SDM is …

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Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment

Kristin Ammon Shimano, Amanda Bell Grimes, Shipra M. Kaicker, Sanjay J. Shah et autres

Importance: Eltrombopag, a thrombopoietin receptor agonist, is approved by the US Food and Drug Administration for children with chronic immune thrombocytopenia. Efficacy of eltrombopag during the newly diagnosed phase of pediatric immune thrombocytopenia is unknown. Objective: To determine if the proportion of …

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It Takes a Village and More: First Report of Successful Allogeneic Hematopoietic Cell Transplantation for Hepatopulmonary Syndrome Associated With Chronic Active Epstein–Barr Virus Disease

Prakash Satwani, Usha Krishnan, Alexis D. Boneparth, Lisa F. Imundo et autres

The data that support the findings of this study are available on request from the corresponding author. The data are not publicly available due to privacy or ethical restrictions.

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Treatment Landscape in Pediatric Immune Thrombocytopenia: Addressing Unmet Needs

Joana Azevedo, Jennifer MacWhirter - DiRaimo, Cindy E. Neunert, Nichola Cooper et autres

Pediatric immune thrombocytopenia (ITP) is associated with a multifaceted burden on children and their parents/caregivers due to bleeding, fatigue, activity restrictions, and psychological distress. Most children recover within 12 months, but up to 30% develop chronic ITP. While emergent therapies, such as …

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Working toward international consensus defining pediatric “refractory ITP”

Candelaria O’Farrell, Amanda Bell Grimes, Taizo A. Nakano, Robert J. Klaassen et autres

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Primary versus Secondary Immune Thrombocytopenia (ITP): A Meeting Report from the 2023 McMaster ITP Summit

Dimpy Modi, Saifur Rahman Chowdhury, Syed Mahamad, Douglas B. Cines et autres

Abstract The McMaster Immune Thrombocytopenia (ITP) Summit, held on October 27, 2023, was an educational seminar from leading experts in immune thrombocytopenia and related disorders geared toward hematologists, internists, immunologists, and clinical and translational scientists. The focus of the Summit was to …

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Complementary and Alternative Medicine (CAM) Use Reported Among Individuals with Immune Thrombocytopenia (ITP): Data from the ITP Natural History Study Registry

Jennifer MacWhirter - DiRaimo, Laura Bou-Maroun, Debbie Jiang, Caroline Gentile Kruse et autres

Background and Aims: CAM refers to non-traditional disease treatments, like supplements and dietary modifications, used either alone or alongside conventional medicine. The use of CAM in children and adults with immune thrombocytopenia (ITP), an autoimmune bleeding disorder, has not been well-studied or …

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Efficacy Findings in a Phase 3, Randomized Trial of Eltrombopag Vs. Standard First-Line Treatment for Newly Diagnosed Immune Thrombocytopenia in Children

Kristin Ammon Shimano, Amanda Bell Grimes, Melissa J. Rose, Shipra M. Kaicker et autres

Background: First-line treatments for patients (pts) with newly diagnosed immune thrombocytopenia (ITP) include corticosteroids, intravenous immunoglobulin (IVIg), and anti-D globulin. Eltrombopag (epag), a thrombopoietin receptor agonist, was FDA approved for children with chronic ITP in 2015. The efficacy of epag in the …

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

Taylor Olmsted Kim, Jennifer M. Geris, Jonathan Michael Flanagan, Rachael F. Grace et autres

ABSTRACT: Through the use of genetic sequencing, molecular variants driving autoimmunity are increasingly identified in patients with chronic and refractory immune cytopenias. With the goal of discovering genetic variants that predispose to pediatric immune thrombocytopenia (ITP) or increase risk for chronic disease, …

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Lightening the load for paediatric ITP : The importance of health‐related quality of life

Cindy E. Neunert, Michele P. Lambert

In their paper, Klaassen et al. present their findings on the revised Kids ITP Tools (KIT). This important work provides an update to the validated measure for evaluating health-related quality of life (HRQoL) in children with immune thrombocytopenia (ITP). ITP, an acquired …

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