Immune thrombocytopenia (ITP) secondary to chronic lymphoproliferative disorders (LPD) is poorly responsive to conventional treatments. We conducted a multicenter phase 2 prospective 24-week study in 18 patients with ITP secondary to LPD (sITP) to assess the safety and efficacy of eltrombopag. Responsive patients entered an extension study for up to 5 years. For inclusion patients should not necessitate cytotoxic treatment, and have platelet count < 30 x 109/L or bleeding. Eltrombopag was initiated at 50 mg/day, with a maximum of 150 mg/day. The primary endpoint was platelet response (R) after 4 weeks. Median age was 70 years (43-83), 14 patients had chronic lymphocytic leukemia (CLL), 2 classical Hodgkin's lymphoma, and 2 Waldenstrom macroglobulinemia. All patients had received previous ITP treatments. Response rate at week 4 was 78% (95% CI 58-97), with 50% of complete response (CR) (95% CI 43-57); respective results at week 24 were 59% (95% CI 36-82) with 30% of CR (95% CI 8-52). Median exposure time to eltrombopag was 16 months; median dose at week 4 and 24 was 50 mg (ranges 25-100 and 25-150). No > grade 2 adverse events were reported. Eltrombopag is active and well tolerated in ITP secondary to LPDs. ClinicalTrials.gov Identifier: NCT01610180.
Eltrombopag for immune thrombocytopenia secondary to chronic lymphoproliferative disorders: a phase 2 multicenter study
Visco, Carlo;Rambaldi, Alessandro;Ruggeri, Marco
2019-01-01
Abstract
Immune thrombocytopenia (ITP) secondary to chronic lymphoproliferative disorders (LPD) is poorly responsive to conventional treatments. We conducted a multicenter phase 2 prospective 24-week study in 18 patients with ITP secondary to LPD (sITP) to assess the safety and efficacy of eltrombopag. Responsive patients entered an extension study for up to 5 years. For inclusion patients should not necessitate cytotoxic treatment, and have platelet count < 30 x 109/L or bleeding. Eltrombopag was initiated at 50 mg/day, with a maximum of 150 mg/day. The primary endpoint was platelet response (R) after 4 weeks. Median age was 70 years (43-83), 14 patients had chronic lymphocytic leukemia (CLL), 2 classical Hodgkin's lymphoma, and 2 Waldenstrom macroglobulinemia. All patients had received previous ITP treatments. Response rate at week 4 was 78% (95% CI 58-97), with 50% of complete response (CR) (95% CI 43-57); respective results at week 24 were 59% (95% CI 36-82) with 30% of CR (95% CI 8-52). Median exposure time to eltrombopag was 16 months; median dose at week 4 and 24 was 50 mg (ranges 25-100 and 25-150). No > grade 2 adverse events were reported. Eltrombopag is active and well tolerated in ITP secondary to LPDs. ClinicalTrials.gov Identifier: NCT01610180.
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