Comparison of second-line treatments with regard to dosing, monitoring, and populations for consideration
| . | Dosing/administration . | In support . | In opposition . | Recommended screening and monitoring . | Populations for consideration . |
|---|---|---|---|---|---|
| Splenectomy | Laparoscopic or open total splenectomy | Possibility of permanent remission or response that would alleviate the need for pharmacologic treatment | Risk for perisurgical complications | Presplenectomy and postsplenectomy vaccinations and boosters (http://www.cdc.gov/vaccines/); lifetime fever guidelines and antibiotic prophylaxis | Primary ITP |
| Immediate response | Permanent immunocompromise/risk for infection | Life-threatening bleeding (ie, intracranial hemorrhage) | |||
| Possible increased risk for thrombosis | Fully immunized children age >5 y with chronic ITP refractory to medical therapy | ||||
| 40% long-term risk for failure | Fully immunized adults who have failed steroids | ||||
| Rituximab | Intravenous infusion in the physician office setting; Optimal dosing regimen in ITP is uncertain, and multiple dosing regimens are reported (eg, 375 mg/m2 IV weekly for 4 wk) | Infusion with a potential platelet effect for a median of 11 mo | Long-term immunosuppression even in nonresponders, in whom rescue therapies may further immune suppress | B/T subsets, immunoglobulin levels, HIV, and hepatitis B/C screening before start; consider vaccinations before treatment; consider immunoglobulin G levels every few months after infusion | Primary or secondary ITP, but must be cautious in patients with an immunodeficiency |
| Possibility of long-term remission in up to 20-25% of patients | Risk for infusion-related adverse effects | Fully immunized children with chronic ITP | |||
| Risk for infection, including PML | Fully immunized adults with ITP who have failed steroids | ||||
| Risks for recurrent, repeated courses are unknown | |||||
| Expensive | |||||
| Delayed response (1 wk-3 mo) | |||||
| Romiplostim | Weekly subcutaneous 1-10 µg/kg injection, mainly only available for injection in physician’s office | Does not suppress immune system | Expensive | Monitor weekly complete blood count; consideration of annual bone marrow exams | Bridge therapy (eg, before surgery or chance of remission) |
| Steady and predictable platelet count | Weekly injection at a physician’s office | Patients who need to avoid immune suppression (eg, failure of splenectomy or rituximab) | |||
| Straightforward to wean off | Requires regular monitoring | Children or adults who have failed steroids | |||
| Not curative therapy | |||||
| Slightly delayed response (1-4 wk, depending on starting dose and weekly dose increases) | |||||
| Risk for thrombosis and bone marrow reticulin | |||||
| Eltrombopag | Oral medication, 12.5-75 mg daily, decreased efficacy if taken with food | Does not suppress immune system | Expensive | Monitor monthly complete blood count and LFTs; annual ophthalmologic exam; consideration of annual bone marrow exams | Bridge therapy (eg, before surgery or chance of remission) |
| Steady and predictable platelet count | Requires regular monitoring | Patients who need to avoid immune suppression (eg, failure of splenectomy or rituximab) | |||
| Straightforward to wean off | Not curative therapy | Children or adults who have failed steroids | |||
| Slightly delayed response (1-4 wk) | |||||
| Risk for thrombosis, bone marrow reticulin, hepatotoxicity, and cataracts | |||||
| Mycophenolyate mofetil | Oral medication 400 mg/m2 twice daily, maximum 1 g twice daily | Straightforward to wean off | Risk for infection | Monthly complete blood count/differential, LFTs, creatinine | Patients with a likelihood of remission |
| Inexpensive | Requires regular monitoring | Primary or secondary ITP | |||
| Delayed response (2 wk-3 mo) | Patients who fail other agents | ||||
| Dapsone | 1-2 mg/kg by mouth daily | Straightforward to wean off | Risk for hemolysis and methemoglobinuria | Baseline glucose-6-phosphate dehydrogenase screen; monthly complete blood count; monitor methemoglobin levels | Patients who fail other agents |
| Inexpensive | Requires regular monitoring | ||||
| Delayed response (2 wk-3 mo) | |||||
| Sirolimus | Loading dose 3 mg/m2, then 1-2 mg daily maintenance, adjust to target trough levels (5-15 ng/mL) | Straightforward to wean off | Risk for infection, hypertriglyceridemia, hyperlipidemia, mucositis | Regular trough levels; | Immune cytopenias associated with immunodeficiency, particularly ALPs |
| Inexpensive | Requires regular laboratory monitoring | Monthly complete blood count, LFTs, cholesterol, triglycerides, creatinine, urinary protein; | Patients with primary ITP who fail other agents | ||
| Delayed response (2 wk-3 mo) | Monitor blood pressure |
| . | Dosing/administration . | In support . | In opposition . | Recommended screening and monitoring . | Populations for consideration . |
|---|---|---|---|---|---|
| Splenectomy | Laparoscopic or open total splenectomy | Possibility of permanent remission or response that would alleviate the need for pharmacologic treatment | Risk for perisurgical complications | Presplenectomy and postsplenectomy vaccinations and boosters (http://www.cdc.gov/vaccines/); lifetime fever guidelines and antibiotic prophylaxis | Primary ITP |
| Immediate response | Permanent immunocompromise/risk for infection | Life-threatening bleeding (ie, intracranial hemorrhage) | |||
| Possible increased risk for thrombosis | Fully immunized children age >5 y with chronic ITP refractory to medical therapy | ||||
| 40% long-term risk for failure | Fully immunized adults who have failed steroids | ||||
| Rituximab | Intravenous infusion in the physician office setting; Optimal dosing regimen in ITP is uncertain, and multiple dosing regimens are reported (eg, 375 mg/m2 IV weekly for 4 wk) | Infusion with a potential platelet effect for a median of 11 mo | Long-term immunosuppression even in nonresponders, in whom rescue therapies may further immune suppress | B/T subsets, immunoglobulin levels, HIV, and hepatitis B/C screening before start; consider vaccinations before treatment; consider immunoglobulin G levels every few months after infusion | Primary or secondary ITP, but must be cautious in patients with an immunodeficiency |
| Possibility of long-term remission in up to 20-25% of patients | Risk for infusion-related adverse effects | Fully immunized children with chronic ITP | |||
| Risk for infection, including PML | Fully immunized adults with ITP who have failed steroids | ||||
| Risks for recurrent, repeated courses are unknown | |||||
| Expensive | |||||
| Delayed response (1 wk-3 mo) | |||||
| Romiplostim | Weekly subcutaneous 1-10 µg/kg injection, mainly only available for injection in physician’s office | Does not suppress immune system | Expensive | Monitor weekly complete blood count; consideration of annual bone marrow exams | Bridge therapy (eg, before surgery or chance of remission) |
| Steady and predictable platelet count | Weekly injection at a physician’s office | Patients who need to avoid immune suppression (eg, failure of splenectomy or rituximab) | |||
| Straightforward to wean off | Requires regular monitoring | Children or adults who have failed steroids | |||
| Not curative therapy | |||||
| Slightly delayed response (1-4 wk, depending on starting dose and weekly dose increases) | |||||
| Risk for thrombosis and bone marrow reticulin | |||||
| Eltrombopag | Oral medication, 12.5-75 mg daily, decreased efficacy if taken with food | Does not suppress immune system | Expensive | Monitor monthly complete blood count and LFTs; annual ophthalmologic exam; consideration of annual bone marrow exams | Bridge therapy (eg, before surgery or chance of remission) |
| Steady and predictable platelet count | Requires regular monitoring | Patients who need to avoid immune suppression (eg, failure of splenectomy or rituximab) | |||
| Straightforward to wean off | Not curative therapy | Children or adults who have failed steroids | |||
| Slightly delayed response (1-4 wk) | |||||
| Risk for thrombosis, bone marrow reticulin, hepatotoxicity, and cataracts | |||||
| Mycophenolyate mofetil | Oral medication 400 mg/m2 twice daily, maximum 1 g twice daily | Straightforward to wean off | Risk for infection | Monthly complete blood count/differential, LFTs, creatinine | Patients with a likelihood of remission |
| Inexpensive | Requires regular monitoring | Primary or secondary ITP | |||
| Delayed response (2 wk-3 mo) | Patients who fail other agents | ||||
| Dapsone | 1-2 mg/kg by mouth daily | Straightforward to wean off | Risk for hemolysis and methemoglobinuria | Baseline glucose-6-phosphate dehydrogenase screen; monthly complete blood count; monitor methemoglobin levels | Patients who fail other agents |
| Inexpensive | Requires regular monitoring | ||||
| Delayed response (2 wk-3 mo) | |||||
| Sirolimus | Loading dose 3 mg/m2, then 1-2 mg daily maintenance, adjust to target trough levels (5-15 ng/mL) | Straightforward to wean off | Risk for infection, hypertriglyceridemia, hyperlipidemia, mucositis | Regular trough levels; | Immune cytopenias associated with immunodeficiency, particularly ALPs |
| Inexpensive | Requires regular laboratory monitoring | Monthly complete blood count, LFTs, cholesterol, triglycerides, creatinine, urinary protein; | Patients with primary ITP who fail other agents | ||
| Delayed response (2 wk-3 mo) | Monitor blood pressure |
LFTs, liver function tests.