Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia

Blood. 2016 Sep 22;128(12):1625-30. doi: 10.1182/blood-2016-03-704734. Epub 2016 Jun 27.

Abstract

Refractory immune thrombocytopenia (ITP) was previously defined as lack of a minimum response to splenectomy and the requirement for long-term treatment to reduce the risk of significant bleeding events. In this multicenter study, we included 37 patients with multirefractory ITP, defined as no response to splenectomy, rituximab, romiplostim, and eltrombopag. As compared with a historical cohort of 183 ITP patients, matched on the calendar year of ITP diagnosis with a 5:1 ratio, patients with multirefractory ITP were more likely to have secondary ITP (odds ratio [OR], 4.84; 95% confidence interval [CI], 1.31-17.86; P = .018) and monoclonal gammopathy of undetermined significance (OR, 5.94; 95% CI, 1.08-32.48; P = .04). The median duration of ITP before being recognized as multirefractory was 78 months (range, 6-450). The patients showed failure of a median of 10.5 prior treatment lines for ITP (range, 6-15). At the end of follow-up (median, 84 months; range, 12-455), only 1/14 patients achieved response with immunosuppressant therapy alone. By contrast, 7/10 patients achieved response with a combination of immunosuppressant therapy and thrombopoietin-receptor agonists that lasted for a median of 15 months (range, 6-32). Throughout the course of ITP, 5/37 patients died, 3 with ITP (bleeding, n = 2; sepsis n = 1); 15 (40%) had at least 1 bacterial infection and 9 (24%) at least 1 episode of thrombosis. In conclusion, multirefractory ITP was associated with high morbidity and mortality. Combining an immunosuppressant therapy with thrombopoietin-receptor agonists may be a good strategy for management for these patients with severe disease.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antineoplastic Agents / therapeutic use*
  • Case-Control Studies
  • Child
  • Chronic Disease
  • Combined Modality Therapy
  • Drug Resistance*
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Prognosis
  • Purpura, Thrombocytopenic, Idiopathic / therapy*
  • Receptors, Fc / therapeutic use*
  • Recombinant Fusion Proteins / therapeutic use*
  • Retrospective Studies
  • Rituximab / therapeutic use*
  • Splenectomy / adverse effects*
  • Survival Rate
  • Thrombopoietin / therapeutic use*
  • Young Adult

Substances

  • Antineoplastic Agents
  • Receptors, Fc
  • Recombinant Fusion Proteins
  • Rituximab
  • Thrombopoietin
  • romiplostim