Comparison between splenectomy, thrombopoietic agents, and rituximab
| Therapy . | Efficacy and response prediction . | Safety . | Contraindications . | Mode of application and follow-up . | Grade of ASH 2011 guidelines recommendation . |
|---|---|---|---|---|---|
| Splenectomy | Highest cure rate; short-term response 80% and long-term response 60%-70% at 5-10 y Response hard to predict | Surgery-related mortality and morbidity (bleeding, infections, thrombosis); lifetime risk of overwhelming infection | Patients with comorbid conditions that increase the risk of complications | Invasive procedure usually performed laparoscopically requires preoperative and postoperative preparation and care and regular vaccination | Well-established treatment for ITP |
| Possible AE: venous thrombosis, pulmonary hypertension, atherosclerosis, dementia | Relative: elderly patients over 60-70 because high rate of complication and lower response; patients with immunodeficiency and secondary ITP eg, CVID, hepatitis C, neutropenia, possibly SLE | ASH: 1B after failure of steroids | |||
| TPO-RA | |||||
| Romiplostim | A maintenance treatment; 60%-80% achieve platelet elevation; sustained response in 70%-90% in those entering long-term treatment studies | Headache, rebound thrombocytopenia, weekly injection | Pregnancy and lactation, MDS | Weekly subcutaneous injections; requires dose adjustment and regular CBC | Approved treatment for ITP |
| Possible AE: bone marrow reticulin fibrosis, arterial and venous thrombosis, risk of malignancy (if MDS) | Relative: past history of venous or arterial thrombosis | ASH: 2C after failure of steroids before splenectomy | |||
| Eltrombopag | A maintenance treatment; 60%-80% achieve platelet elevation; sustained response in 70%-90% in those entering long-term treatment studies | Headache, rebound thrombocytopenia, elevated liver enzymes | As with romiplostim | Daily ingestions; requires dose adjustment and regular CBC and liver tests | ASH: 2C after failure of steroids before splenectomy |
| Possible AE: bone marrow reticulin fibrosis, arterial and venous thrombosis; nausea, vomiting in small percent; cataracts (very infrequent if at all) | Requires monitoring of liver tests but used successfully in large studies of patients with liver disease secondary to hepatitis C | ||||
| Anti-CD20 | |||||
| Rituximab | May be curative treatment; initial response in 50%-60%; sustained response 3-5 y in 20%; retreatment gives the same pattern of response as observed after the first course in complete responders cannot predict response | Infusion-related side effects (chills, fever, dyspnea), neutropenia, | Active hepatitis B virus, known clinically significant allergy, including past serum sickness with anti-CD20, or antimouse antibody | Weekly intravenous infusions for 4 wks; CBC required depending on the response | Not approved for ITP, only off-label use |
| Possible AE: increased risk of infection and viral reactivation, hypogammaglobulinemia, serum sickness (especially in children), multifocal leukoencephalopathy (PML) | Pregnancy and lactation | ASH: 2C after failure of steroids |
| Therapy . | Efficacy and response prediction . | Safety . | Contraindications . | Mode of application and follow-up . | Grade of ASH 2011 guidelines recommendation . |
|---|---|---|---|---|---|
| Splenectomy | Highest cure rate; short-term response 80% and long-term response 60%-70% at 5-10 y Response hard to predict | Surgery-related mortality and morbidity (bleeding, infections, thrombosis); lifetime risk of overwhelming infection | Patients with comorbid conditions that increase the risk of complications | Invasive procedure usually performed laparoscopically requires preoperative and postoperative preparation and care and regular vaccination | Well-established treatment for ITP |
| Possible AE: venous thrombosis, pulmonary hypertension, atherosclerosis, dementia | Relative: elderly patients over 60-70 because high rate of complication and lower response; patients with immunodeficiency and secondary ITP eg, CVID, hepatitis C, neutropenia, possibly SLE | ASH: 1B after failure of steroids | |||
| TPO-RA | |||||
| Romiplostim | A maintenance treatment; 60%-80% achieve platelet elevation; sustained response in 70%-90% in those entering long-term treatment studies | Headache, rebound thrombocytopenia, weekly injection | Pregnancy and lactation, MDS | Weekly subcutaneous injections; requires dose adjustment and regular CBC | Approved treatment for ITP |
| Possible AE: bone marrow reticulin fibrosis, arterial and venous thrombosis, risk of malignancy (if MDS) | Relative: past history of venous or arterial thrombosis | ASH: 2C after failure of steroids before splenectomy | |||
| Eltrombopag | A maintenance treatment; 60%-80% achieve platelet elevation; sustained response in 70%-90% in those entering long-term treatment studies | Headache, rebound thrombocytopenia, elevated liver enzymes | As with romiplostim | Daily ingestions; requires dose adjustment and regular CBC and liver tests | ASH: 2C after failure of steroids before splenectomy |
| Possible AE: bone marrow reticulin fibrosis, arterial and venous thrombosis; nausea, vomiting in small percent; cataracts (very infrequent if at all) | Requires monitoring of liver tests but used successfully in large studies of patients with liver disease secondary to hepatitis C | ||||
| Anti-CD20 | |||||
| Rituximab | May be curative treatment; initial response in 50%-60%; sustained response 3-5 y in 20%; retreatment gives the same pattern of response as observed after the first course in complete responders cannot predict response | Infusion-related side effects (chills, fever, dyspnea), neutropenia, | Active hepatitis B virus, known clinically significant allergy, including past serum sickness with anti-CD20, or antimouse antibody | Weekly intravenous infusions for 4 wks; CBC required depending on the response | Not approved for ITP, only off-label use |
| Possible AE: increased risk of infection and viral reactivation, hypogammaglobulinemia, serum sickness (especially in children), multifocal leukoencephalopathy (PML) | Pregnancy and lactation | ASH: 2C after failure of steroids |
AE indicates adverse event; MDS, myelodysplastic syndrome; SLE, systemic lupus erythematosus; CBC, complete blood count; and PML, progressive multifocal leukoencephalopathy.