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

Publication ,  Journal Article
Grace, RF; Shimano, KA; Bhat, R; Neunert, C; Bussel, JB; Klaassen, RJ; Lambert, MP; Rothman, JA; Breakey, VR; Hege, K; Bennett, CM; Rose, MJ ...
Published in: Am J Hematol
July 2019

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.

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Published In

Am J Hematol

DOI

EISSN

1096-8652

Publication Date

July 2019

Volume

94

Issue

7

Start / End Page

741 / 750

Location

United States

Related Subject Headings

  • Time Factors
  • Thrombopoietin
  • Survival Rate
  • Rituximab
  • Recombinant Fusion Proteins
  • Receptors, Fc
  • Quality of Life
  • Purpura, Thrombocytopenic, Idiopathic
  • Prospective Studies
  • Platelet Count
 

Citation

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Grace, R. F., Shimano, K. A., Bhat, R., Neunert, C., Bussel, J. B., Klaassen, R. J., … Despotovic, J. M. (2019). Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol, 94(7), 741–750. https://doi.org/10.1002/ajh.25479
Grace, Rachael F., Kristin A. Shimano, Rukhmi Bhat, Cindy Neunert, James B. Bussel, Robert J. Klaassen, Michele P. Lambert, et al. “Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes.Am J Hematol 94, no. 7 (July 2019): 741–50. https://doi.org/10.1002/ajh.25479.
Grace RF, Shimano KA, Bhat R, Neunert C, Bussel JB, Klaassen RJ, et al. Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019 Jul;94(7):741–50.
Grace, Rachael F., et al. “Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes.Am J Hematol, vol. 94, no. 7, July 2019, pp. 741–50. Pubmed, doi:10.1002/ajh.25479.
Grace RF, Shimano KA, Bhat R, Neunert C, Bussel JB, Klaassen RJ, Lambert MP, Rothman JA, Breakey VR, Hege K, Bennett CM, Rose MJ, Haley KM, Buchanan GR, Geddis A, Lorenzana A, Jeng M, Pastore YD, Crary SE, Neier M, Neufeld EJ, Neu N, Forbes PW, Despotovic JM. Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019 Jul;94(7):741–750.
Journal cover image

Published In

Am J Hematol

DOI

EISSN

1096-8652

Publication Date

July 2019

Volume

94

Issue

7

Start / End Page

741 / 750

Location

United States

Related Subject Headings

  • Time Factors
  • Thrombopoietin
  • Survival Rate
  • Rituximab
  • Recombinant Fusion Proteins
  • Receptors, Fc
  • Quality of Life
  • Purpura, Thrombocytopenic, Idiopathic
  • Prospective Studies
  • Platelet Count