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Am J Hematol. 2019 Jul;94(7):741-750. doi: 10.1002/ajh.25479. Epub 2019 Apr 29.

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

Author information

1
Division of Hematology/Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center, Boston, Massachusetts.
2
Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, UCSF Benioff Children's Hospital, San Francisco, California.
3
Center for Cancer & Blood Disorders, Ann and Robert H. Lurie Childrens Hospital of Chicago, Feinberg School of Medicine, Northwestern University, Chicago, Illinois.
4
Division of Hematology, Oncology, and Stem Cell Transplant, Columbia University Medical School, New York, New York.
5
Department of Pediatrics, Weill Cornell Medicine, New York, New York.
6
Division of Hematology/Oncology, Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada.
7
Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
8
Division of Pediatric Hematology/Oncology, Duke University Medical Center, Durham, North Carolina.
9
Division of Pediatric Hematology/Oncology, McMaster University, Hamilton, Ontario, Canada.
10
Division of Pediatric Hematology/Oncology, Riley Hospital at IU Health, Indiana University School of Medicine, Indianapolis, Indiana.
11
Division of Hematology/Oncology, Aflac Cancer and Blood Disorders Center, Emory University School of Medicine, Children's Healthcare of Atlanta, Atlanta, Georgia.
12
Division of Hematology, Oncology, and Bone Marrow Transplant, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, Ohio.
13
Division of Pediatric Hematology, Oregon Health & Science University, Portland, Oregon.
14
Division of Hematology-Oncology, University of Texas Southwestern Medical Center, Dallas, Texas.
15
Division of Pediatric Hematology/Oncology, University of Washington, Seattle Children's Hospital, Seattle, Washington.
16
Division of Pediatric Hematology/Oncology, St. John Ascension Hospital, Detroit, Michigan.
17
Department of Pediatrics, Stanford School of Medicine, Palo Alto, California.
18
Division of Hematology/Oncology, CHU Sainte-Justine, Montreal, Québec, Canada.
19
Department of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, Arkansas.
20
Division of Pediatric Hematology/Oncology, Goryeb Children's Hospital, Morristown, New Jersey.
21
Division of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee.
22
Clinical Research Center, Boston Children's Hospital, Boston, Massachusetts.
23
Department of Pediatrics, Hematology/Oncology Section, Baylor College of Medicine, Houston, Texas.

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.

PMID:
30945320
PMCID:
PMC6527349
[Available on 2020-07-01]
DOI:
10.1002/ajh.25479

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