Thrombopoietin receptor agonists for preparing adult patients with immune thrombocytopenia to splenectomy: results of a retrospective, observational GIMEMA study

Am J Hematol. 2016 May;91(5):E293-5. doi: 10.1002/ajh.24341. Epub 2016 Apr 4.

Abstract

In patients with immune thrombocytopenia (ITP) refractory to corticosteroids and intravenous immunoglobulins (IVIG), splenectomy may result at higher risk of peri-operative complications and, for this reason, potentially contraindicated. The thrombopoietin receptor agonists (TPO-RAs) romiplostim and eltrombopag have shown high therapeutic activity in primary ITP, but data of efficacy and safety regarding their use in preparation for splenectomy are missing. Thirty-one adult patients, median age 50 years, with corticosteroids and/or IVIG refractory persistent and chronic ITP who were treated with TPO-RAs (romiplostim= 24; eltrombopag= 7) with the aim to increase platelet count and allow a safer execution of splenectomy were retrospectively evaluated. Twenty-four patients (77%) responded to the use of TPO-RAs with a median platelet count that increased from 11 × 10(9) /L before starting TPO-RAs to 114 × 10(9) /L pre-splenectomy, but a concomitant treatment with corticosteroids and/or IVIG was required in 19 patients. Twenty-nine patients underwent splenectomy while two patients who responded to TPO-RAs subsequently refused surgery. Post-splenectomy complications were characterized by two Grade 3 thrombotic events (1 portal vein thrombosis in the patient with previous history of HCV hepatitis and 1 pulmonary embolism), with a platelet count at the time of thrombosis of 260 and 167 × 10(9) /L, respectively and one Grade 3 infectious event. TPO-RAs may represent a therapeutic option to improve platelet count and reduce the risk of peri-operative complications in ITP candidates to splenectomy. An increased risk of post-splenectomy thromboembolic events cannot be ruled out and thromboprophylaxis with low-molecular weight heparin is generally recommended.

Publication types

  • Multicenter Study
  • Observational Study

MeSH terms

  • Adrenal Cortex Hormones / pharmacology
  • Adrenal Cortex Hormones / therapeutic use
  • Adult
  • Aged
  • Aged, 80 and over
  • Benzoates / administration & dosage
  • Benzoates / adverse effects
  • Benzoates / therapeutic use*
  • Combined Modality Therapy
  • Drug Resistance
  • Female
  • Humans
  • Hydrazines / administration & dosage
  • Hydrazines / adverse effects
  • Hydrazines / therapeutic use*
  • Immunoglobulins, Intravenous / pharmacology
  • Immunoglobulins, Intravenous / therapeutic use
  • Italy / epidemiology
  • Male
  • Middle Aged
  • Platelet Count
  • Portal Vein
  • Postoperative Complications / chemically induced
  • Postoperative Complications / etiology
  • Postoperative Complications / prevention & control
  • Premedication*
  • Preoperative Care / methods*
  • Pulmonary Embolism / chemically induced
  • Pulmonary Embolism / etiology
  • Pulmonary Embolism / prevention & control
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy
  • Purpura, Thrombocytopenic, Idiopathic / surgery*
  • Pyrazoles / administration & dosage
  • Pyrazoles / adverse effects
  • Pyrazoles / therapeutic use*
  • Receptors, Fc / administration & dosage
  • Receptors, Fc / therapeutic use*
  • Receptors, Thrombopoietin / agonists*
  • Recombinant Fusion Proteins / administration & dosage
  • Recombinant Fusion Proteins / adverse effects
  • Recombinant Fusion Proteins / therapeutic use*
  • Retrospective Studies
  • Salvage Therapy
  • Splenectomy*
  • Thrombophilia / chemically induced
  • Thrombopoiesis / drug effects*
  • Thrombopoietin / administration & dosage
  • Thrombopoietin / adverse effects
  • Thrombopoietin / therapeutic use*
  • Venous Thrombosis / chemically induced
  • Venous Thrombosis / etiology
  • Venous Thrombosis / prevention & control
  • Young Adult

Substances

  • Adrenal Cortex Hormones
  • Benzoates
  • Hydrazines
  • Immunoglobulins, Intravenous
  • Pyrazoles
  • Receptors, Fc
  • Receptors, Thrombopoietin
  • Recombinant Fusion Proteins
  • MPL protein, human
  • Thrombopoietin
  • romiplostim
  • eltrombopag