Table 6. Second-line treatments of... | American Society of Hematology

Table 6.

Second-line treatments of secondary ITP associated with predominantly antibody deficiencies

Section	Rituximab (Gobert et al¹⁶)	Splenectomy (Wong et al¹⁷)	TPO-RAs
Population	Adults with secondary ITP N = 25 CVID, criteria from Conley et al 1999³²	Adults with secondary ITP N = 14 CVID, criteria of ESID in 2013.	Adults with secondary ITP N = 28 Predominantly antibody deficiencies
Concurrent treatments	At baseline, concurrent treatments (IVIG, steroids, romiplostim) were ongoing in 27 of 33 cases.	No data available apart from that a number of patients still required long-term oral corticosteroids (prednisolone 4-15 mg).	At baseline, second-line treatments were ongoing in 10 of 28 cases.
Criteria for response	Platelet count >30 × 10/L with at least a twofold increase, without any other medication but substitutive IVIG and/or corticosteroids at stable or decreasing doses	Complete or partial responses according to treating physician’s judgment, without any other medication but low-dose maintenance corticosteroid	Platelet count >30 × 10/L without bleeding events with a Khellaf score >8 without any other medication but substitutive IVIG and/or corticosteroids at stable or decreasing doses or low-dose maintenance corticosteroid
Initial outcome	After a median delay of 4 wk (range 2-8): initial response in 21 of 25 (84%).		After 6 wk of treatment: response in 24 of 28 patients (85.7%).
End of study outcome	After a mean follow-up of 38 ± 26 mo: response in 13 of 25 patients (52%); 4 were successfully retreated with rituximab.	After a median follow-up of >100 mo, 11 patients (78.6%) were considered responders.	After a median follow-up of 33 mo: response in 20 of 28 patients (71.4%).
Safety	(Among adult ITP cases) Severe infections: 8 episodes in 6 patients (24%). Cytopenias: 1 severe pancytopenia and neutropenia of mild severity in 1 patient Mortality: 2 deaths (8%; 1 pulmonary aspergilloma and postoperative pulmonary hemorrhage; 1 Streptococcus pneumonia sepsis).	(Among the 45 patients with PID) Infections: 14 severe infections (6 cytomegalovirus reactivations and 5 fungal infections). Surgical complications: 1 subdiaphragmatic abscess and 1 portal vein thrombosis Mortality: 10 deaths (25%). 4 patients died from unusual infections. The remaining deaths are thought to be unrelated to splenectomy.	Thrombotic events: 3 episodes requiring therapeutic anticoagulation or dual antiplatelet therapy. Hepatobiliary adverse events: 3 mild episodes. Inherent risk of interfering in hepatic monitoring. Mortality: 1 death (3.6%) (severe infection after use of rituximab).
Other indications in PIDs	AIHA, autoimmune diseases Lymphoproliferative diseases Granulomatous diseases	AIHA Suspicion of lymphoma	None

Section	Rituximab (Gobert et al¹⁶)	Splenectomy (Wong et al¹⁷)	TPO-RAs
Population	Adults with secondary ITP N = 25 CVID, criteria from Conley et al 1999³²	Adults with secondary ITP N = 14 CVID, criteria of ESID in 2013.	Adults with secondary ITP N = 28 Predominantly antibody deficiencies
Concurrent treatments	At baseline, concurrent treatments (IVIG, steroids, romiplostim) were ongoing in 27 of 33 cases.	No data available apart from that a number of patients still required long-term oral corticosteroids (prednisolone 4-15 mg).	At baseline, second-line treatments were ongoing in 10 of 28 cases.
Criteria for response	Platelet count >30 × 10/L with at least a twofold increase, without any other medication but substitutive IVIG and/or corticosteroids at stable or decreasing doses	Complete or partial responses according to treating physician’s judgment, without any other medication but low-dose maintenance corticosteroid	Platelet count >30 × 10/L without bleeding events with a Khellaf score >8 without any other medication but substitutive IVIG and/or corticosteroids at stable or decreasing doses or low-dose maintenance corticosteroid
Initial outcome	After a median delay of 4 wk (range 2-8): initial response in 21 of 25 (84%).		After 6 wk of treatment: response in 24 of 28 patients (85.7%).
End of study outcome	After a mean follow-up of 38 ± 26 mo: response in 13 of 25 patients (52%); 4 were successfully retreated with rituximab.	After a median follow-up of >100 mo, 11 patients (78.6%) were considered responders.	After a median follow-up of 33 mo: response in 20 of 28 patients (71.4%).
Safety	(Among adult ITP cases) Severe infections: 8 episodes in 6 patients (24%). Cytopenias: 1 severe pancytopenia and neutropenia of mild severity in 1 patient Mortality: 2 deaths (8%; 1 pulmonary aspergilloma and postoperative pulmonary hemorrhage; 1 Streptococcus pneumonia sepsis).	(Among the 45 patients with PID) Infections: 14 severe infections (6 cytomegalovirus reactivations and 5 fungal infections). Surgical complications: 1 subdiaphragmatic abscess and 1 portal vein thrombosis Mortality: 10 deaths (25%). 4 patients died from unusual infections. The remaining deaths are thought to be unrelated to splenectomy.	Thrombotic events: 3 episodes requiring therapeutic anticoagulation or dual antiplatelet therapy. Hepatobiliary adverse events: 3 mild episodes. Inherent risk of interfering in hepatic monitoring. Mortality: 1 death (3.6%) (severe infection after use of rituximab).
Other indications in PIDs	AIHA, autoimmune diseases Lymphoproliferative diseases Granulomatous diseases	AIHA Suspicion of lymphoma	None