Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes

• Type of Material: Article
• Authors: Rachael F. Grace, Dana-Farber/Boston Children’s Cancer and Blood Disorder Center; Kristin A. Shimano, UCSF Benioff Childrens Hospital; Rukhmi Bhat, Northwestern University; Cindy Neunert, Columbia University; James B. Bussel, Weill Cornell Medical College; Robert J. Klaassen, Childrens Hospital Eastern Ontario; Michele P. Lambert, Childrens Hospital of Philadelphia; Jennifer A. Rothman, Duke University; Vicky R. Breakey, McMaster University; Kerry Hege, Indiana University; Carolyn Bennett, Emory University; Melissa J. Rose, Ohio State University; Kristina M. Haley, Oregon Health and Science University; George R. Buchanan, University of Texas Southwestern Medical Center; Amy Geddis, University of Washington; Adonis Lorenzana, St. John Hospital & Medical Center; Michael Jeng, Stanford University; Yves D. Pastore, CHU Sainte-Justine; Shelley E. Crary, University of Arkansas; Michelle Neier, Goryeb Childrens Hospital; Ellis J. Neufeld, St Jude Childrens Research Hospital; Nolan Neu, Dana-Farber/Boston Children’s Cancer and Blood Disorder Center; Peter W. Forbes, Boston Childrens Hospital; Jenny M. Despotovic, Baylor College of Medicine
• Language: English
• Date: 2019-07-01
• Publisher: Wiley
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2019 Wiley Periodicals, Inc.
• Final Published Version (URL): http://dx.doi.org/10.1002/ajh.25479
• Title of Journal or Parent Work: American Journal of Hematology
• Volume: 94
• Issue: 7
• Start Page: 741
• End Page: 750
• Grant/Funding Information: Grant support from NHLBI 4K12HL087164-10.
• Keywords: Purpura; Adults; ITP; Science & Technology; Quality of life; Life Sciences & Biomedicine; Hematology
• Research Categories: Health Sciences, Health Care Management; Health Sciences, Human Development; Health Sciences, Medicine and Surgery; Health Sciences, Immunology

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.