4.8 Article

Thrombopoietin-Receptor Agonists for Primary Immune Thrombocytopenia

Journal

NEW ENGLAND JOURNAL OF MEDICINE
Volume 365, Issue 8, Pages 734-741

Publisher

MASSACHUSETTS MEDICAL SOC
DOI: 10.1056/NEJMct1014202

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Funding

  1. Amgen
  2. GlaxoSmithKline
  3. Boehringer Ingelheim
  4. Bristol-Myers Squibb
  5. Pfizer
  6. Leo

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A 26-year-old woman with a history of chronic primary immune thrombocytopenia presents for evaluation. Her condition was first diagnosed when she was 11 years of age. Her platelet count has typically been less than 10,000 per cubic millimeter. Her symptoms have not responded to glucocorticoids, and she has had only transient responses to intravenous immune globulin. Previous treatments have included a course of cyclosporine, a course of four doses of rituximab, and splenectomy. Her bleeding symptoms have consisted mainly of menorrhagia, resulting in iron-deficiency anemia. She is currently being treated with a thrombopoietin-receptor agonist, which has resulted in an increase in her platelet count to between 50,000 and 200,000 per cubic millimeter and resolution of her excessive menstrual bleeding, with normalization of her hemoglobin level after iron therapy. She is now inquiring about the long-term treatment of her condition.

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