Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes
Rachael F Grace, Kristin A Shimano, Rukhmi Bhat, Cindy Neunert, James B Bussel, Robert J Klaassen, Michele P Lambert, Jennifer A Rothman, Vicky R Breakey, Kerry Hege, Carolyn M Bennett, Melissa J Rose, Kristina M Haley, George R Buchanan, Amy Geddis, Adonis Lorenzana, Michael Jeng, Yves D Pastore, Shelley E Crary, Michelle Neier, Ellis J Neufeld, Nolan Neu, Peter W Forbes, Jenny M Despotovic, Rachael F Grace, Kristin A Shimano, Rukhmi Bhat, Cindy Neunert, James B Bussel, Robert J Klaassen, Michele P Lambert, Jennifer A Rothman, Vicky R Breakey, Kerry Hege, Carolyn M Bennett, Melissa J Rose, Kristina M Haley, George R Buchanan, Amy Geddis, Adonis Lorenzana, Michael Jeng, Yves D Pastore, Shelley E Crary, Michelle Neier, Ellis J Neufeld, Nolan Neu, Peter W Forbes, Jenny M Despotovic
Abstract
Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder with isolated thrombocytopenia and hemorrhagic risk. While many children with ITP can be safely observed, treatments are often needed for various reasons, including to decrease bleeding, or to improve health related quality of life (HRQoL). There are a number of available second-line treatments, including rituximab, thrombopoietin-receptor agonists, oral immunosuppressive agents, and splenectomy, but data comparing treatment outcomes are lacking. ICON1 is a prospective, multi-center, observational study of 120 children starting second-line treatments for ITP designed to compare treatment outcomes including platelet count, bleeding, and HRQoL utilizing the Kids ITP Tool (KIT). While all treatments resulted in increased platelet counts, romiplostim had the most pronounced effect at 6 months (P = .04). Only patients on romiplostim and rituximab had a significant reduction in both skin-related (84% to 48%, P = .01 and 81% to 43%, P = .004) and non-skin-related bleeding symptoms (58% to 14%, P = .0001 and 54% to 17%, P = .0006) after 1 month of treatment. HRQoL significantly improved on all treatments. However, only patients treated with eltrombopag had a median improvement in KIT scores at 1 month that met the minimal important difference (MID). Bleeding, platelet count, and HRQoL improved in each treatment group, but the extent and timing of the effect varied among treatments. These results are hypothesis generating and help to improve our understanding of the effect of each treatment on specific patient outcomes. Combined with future randomized trials, these findings will help clinicians select the optimal second-line treatment for an individual child with ITP.
Conflict of interest statement
Disclosures: EJN: Consultancy and Advisory Board for Novartis and Genentech. Research support and consultancy from Octopharma. JMD: Consultancy and honoraria from Sanofi-Genzyme. Research Funding from Novartis. RFG: Research funding from Novartis. JRR: Research funding and sdvisory Board for Novartis and Pfizer. YDP: Consultancy for Novartis. MPL: Consultancy for Novartis, Amgen, Shionogi, Kedrion, Shire, CSL Behring, Octapharma, Bayer, Sysmex, and EBSCO. Advisory Board for Amgen, Shionogi, CSL Behring, Octapharma and Novartis; Research support from Astra Zeneca, Quansys and PDSA. RJK: Consultancy for Amgen Inc., Hoffman-La Roche LTD and Speaker for Octapharma AG, Baxalta, and Biogen Canada Limited. JBB : Advisory Board and Research support from Amgen and Novartis; advisory board for Dova. CN: research support from the PDSA. The remaining authors have no relevant conflicts of interest.
© 2019 Wiley Periodicals, Inc.
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Source: PubMed