Should medical treatment options be exhausted before splenectomy is performed in adult ITP patients? A debate.
Patients with primary immune thrombocytopenia (ITP) may require treatment to reduce the risk of serious bleeding if platelets remain consistently below 30 × 10(9)/L. While approximately 70-80% of patients respond to an initial course of corticosteroids, relapse is common. For steroid-refractory patients, there is a choice between surgical splenectomy and further medical treatments, based on many factors including the patient's bleeding history, fitness for surgery, comorbidities, tolerance of adverse events, lifestyle and preferences. Treatments that have traditionally been used (corticosteroids, azathioprine, danazol) suppress the immune system, potentially predisposing patients to infection. Recent insights into the underlying pathophysiology of the disease have allowed the development of targeted therapies, including the thrombopoietin (TPO) receptor agonists, which enhance platelet production. Phase III trials have found romiplostim and eltrombopag to be well tolerated and effective in elevating platelet counts and reducing bleeding in both splenectomised and nonsplenectomised patients with chronic ITP. The B-cell targeted monoclonal antibody rituximab has also shown some potential in this setting, although data are currently limited and there are toxicity concerns. The decision whether to proceed to splenectomy or try other medical therapies in corticosteroid-refractory patients remains patient-specific. Splenectomy has its risks (including perioperative and long-term risks), and relapse/nonresponse are relatively common, but it offers the possibility of cure in the majority of patients. However, newer treatments may potentially allow splenectomy to be deferred for prolonged periods, as well as providing alternative treatment options for patients who fail splenectomy.
Stasi
Roberto
R
Department of Haematology, St. George's Hospital, London, UK. Roberto.Stasi@stgeorges.nhs.uk
Newland
Adrian
A
Thornton
Patrick
P
Pabinger
Ingrid
I
D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D001379:Azathioprine / Q000627:therapeutic use; D003613:Danazol / Q000627:therapeutic use; D006801:Humans; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000601:surgery*; D011961:Receptors, Fc / Q000627:therapeutic use; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use; D018570:Risk Assessment; D013156:Splenectomy / Q000379:methods*; D013926:Thrombopoietin / Q000627:therapeutic use; D013997:Time Factors
D000305:Adrenal Cortex Hormones; D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim; D001379:Azathioprine; D003613:Danazol