search for


Original Article
The Efficacy of High Dose Dexamethasone Therapy in Children with Immune Thrombocytopenic Purpura
Clin Pediatr Hematol Oncol 2018;25:102-7.
Published online October 31, 2018
© 2018 Korean Society of Pediatric Hematology-Oncology and Korean Society for Pediatric Neuro-Oncology

Hyun Ok Lee, M.D.1, Seong Hwan Chang, M.D.1, Hee Jo Baek, M.D., Ph.D.1, Ho Sung Kim, M.D.1, Su Min Park, M.D.1, Myung Geun Shin, M.D.2 and Hoon Kook, M.D.1

Departments of 1Pediatrics and 2Laboratory Medicine, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea
Correspondence to: Hee Jo Baek
Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, 322 Seoyang-ro, Hwasun-eup, Hwasun 58128, Korea
Tel: +82-61-379-7695
Fax: +82-61-379-7697
Received September 24, 2018; Revised September 26, 2018; Accepted October 10, 2018.
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License ( which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
Background: Few studies of high dose dexamethasone (HD-DXM) therapy in children with immune thrombocytopenic purpura (ITP) have been reported. The purpose of this study is to investigate efficacy and safety of repeated HD-DXM therapy as second-line treatment of ITP in childhood.
Methods: We retrospectively analyzed the medical records of patients <18 years of age with primary ITP who received more than 2 cycles of HD-DXM therapy from May 2004 to January 2018. HD-DXM was given orally in 4-day pulses every 28 days as a 20-40 mg/1.73 m2 daily dose.
Results: A total of 26 patients (male, 19; female, 7) were enrolled and their median age was 6 years (range, 1-15). All patients had received previous treatment for ITP. A median 6 cycles (range, 2-19) of HD-DXM was given. On the beginning of HD-DXM therapy, three patients satisfied the criteria for newly diagnosed ITP, 16 for persistent ITP and 7 for chronic ITP. Relapse-free survival (RFS) of responders (n=9) after the last HD-DXM cycle was estimated to be 38.1±17.2%, lasting for a median 9.1 months (range, 5.6-46.2). According to response after the 2nd cycle, RFS of responders (n=13) was significantly higher than non-responders (23.1±11.7% vs. 7.7%±7.4%, P=0.001). The most common adverse event was irritability (30.8%), followed by fatigue (19.2%).
Conclusion: HD-DXM therapy in children was relatively tolerated and response after therapy was acceptable. More courses of HD-DXM may be feasible in responders after two cycles of HD-DXM.
Keywords: Immune thrombocytopenic purpura, High dose dexamethasone
  1. Nomura S. Advances in diagnosis and treatments for immune thrombocytopenia. Clin Med Insights Blood Disord 2016;9:15-22.
    Pubmed KoreaMed CrossRef
  2. Neunert C, Lim W, Crowther M, et al. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood 2011;117:4190-207.
    Pubmed CrossRef
  3. Jang JH, Kim JY, Mun YC, et al. Management of immune thrombocytopenia: Korean experts recommendation in 2017. Blood Res 2017;52:254-63.
    Pubmed KoreaMed CrossRef
  4. Cuker A, Neunert CE. How I treat refractory immune thrombocytopenia. Blood 2016;128:1547-54.
    Pubmed CrossRef
  5. Hedlund-Treutiger I, Henter JI, Elinder G. Randomized study of IVIg and high-dose dexamethasone therapy for children with chronic idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol 2003;25:139-44.
    Pubmed CrossRef
  6. Kühne T, Freedman J, Semple JW, Doyle J, Butchart S, Blanchette VS. Platelet and immune responses to oral cyclic dexamethasone therapy in childhood chronic immune thrombocytopenic purpura. J Pediatr 1997;130:17-24.
  7. Borgna-Pignatti C, Rugolotto S, Nobili B, et al. A trial of high-dose dexamethasone therapy for chronic idiopathic thrombocytopenic purpura in childhood. J Pediatr 1997;130:13-6.
  8. Chen JS, Wu JM, Chen YJ, Yeh TF. Pulsed high-dose dexamethasone therapy in children with chronic idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol 1997;19:526-9.
    Pubmed CrossRef
  9. Mazzucconi MG, Fazi P, Bernasconi S, et al. Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience. Blood 2007;109:1401-7.
    Pubmed CrossRef
  10. Adams DM, Kinney TR, O'Branski-Rupp E, Ware RE. High-dose oral dexamethasone therapy for chronic childhood idiopathic thrombocytopenic purpura. J Pediatr 1996;128:281-3.
  11. Wali YA, Al Lamki Z, Shah W, Zacharia M, Hassan A. Pulsed high-dose dexamethasone therapy in children with chronic idiopathic thrombocytopenic purpura. Pediatr Hematol Oncol 2002;19:329-35.
    Pubmed CrossRef
  12. Andersen JC. Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy. N Engl J Med 1994;330:1560-4.
    Pubmed CrossRef
  13. Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood 2009;113:2386-93.
    Pubmed CrossRef

October 2018, 25 (2)
Full Text PDF
Send to a friend

Cited By Articles
  • CrossRef (0)

Author ORCID Information
  • Hee Jo Baek