Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated ... more Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated hemolysis. Pegcetacoplan is the first C3-targeted therapy approved for adults with PNH (United States), adults with PNH with inadequate response or intolerance to a C5 inhibitor (Australia), and adults with anemia despite C5-targeted therapy for ≥3 months (European Union). PRINCE was a phase 3, randomized, multicenter, open-label, controlled study to evaluate the efficacy and safety of pegcetacoplan vs control (supportive care only; eg, blood transfusions, corticosteroids, and supplements) in complement inhibitor–naive patients with PNH. Eligible adults receiving supportive care only for PNH were randomly assigned and stratified based on their number of transfusions (<4 or ≥4) 12 months before screening. Patients received pegcetacoplan 1080 mg subcutaneously twice weekly or continued supportive care (control) for 26 weeks. Coprimary end points were hemoglobin stabilization (avoidance ...
Background In RERISE phase 3 study, radotinib demonstrated significantly higher and faster rates ... more Background In RERISE phase 3 study, radotinib demonstrated significantly higher and faster rates of major molecular response (MMR) than imatinib in patients with newly diagnosed CML-CP. By 12 and 24 months follow up, MMR (BCR-ABL1IS≤ 0.1%) and MR4.5 (BCR-ABL1IS ≤ 0.0032%) in radotinib 300 mg twice daily (bid) were higher than imatinib group. Also, early molecular response (EMR) at 3- or 6- months could predict better outcomes in both radotinib or imatinib groups. To confirm the long-term benefits and risks of radotinib 300mg bid and imatinib 400mg qd, we update the results from RERISE phase 3 study based on a minimum follow-up of 36 months. Methods 241 patients were randomized 1:1:1 to radotinib 300 mg bid (n=79), radotinib 400 mg bid (n=81), or imatinib 400 mg once daily (qd) (n=81). Methods have been previously reported (Blood 2015 126:476). We evaluated MMR and MR4.5, overall survival (OS), and progression-free survival (PFS) by 36 months. Also, we analyzed the clinical impacts of early and deeper molecular response in radotinib 300mg bid and imatinib 400mg qd groups. Results By 36 months, MMR was significantly higher in patients receiving radotinib 300 mg bid compared with imatinib 400mg qd (Table). The MR4.5 rate by 36 months was also higher for radotinib compared to imatinib (43% vs 28%; P=0.0538). More patients treated with radotinib achieved additional MMR and MR4.5 since 12 months and time to MR4.5 was faster in radotinib than imatinib (median 924 vs 1,095 days; P=0.2534). Of 59 patients who had MMR by 36 months, 18 patients achieved MMR, and of 34 patients who had MR4.5 by 36 months, 22 patients achieved MR4.5 since 12 months. Estimated OS and PFS rate at 36 months were not significantly different in two groups (98% vs 97%; P=0.0554, 99% vs 95%; P=0.4707). Treatment failure was lower in radotinib group compared with imatinib group (Table). The safety profiles were consistent with those previously reported and most of adverse events (AEs) have developed within 12 months. Since 12 months, newly developed AEs such as rash, nausea/vomiting, pruritis, musculoskeletal pain, fatigue, hyperbilirubinemia, and ALT elevation, etc have shown minimal increase by 36 months FU. Conclusions With a minimum 36 months follow-up, radotinib continued to demonstrate significantly higher rates of MMR and MR4.5 than imatinib in newly diagnosed CML-CP. Also, these responses with radotinib were earlier and deeper compared with imatinib. These results still demonstrate that radotinib can be one of the standards of care in newly diagnosed CML-CP and support the higher possibility of treatment- free remission (TFR) on frontline therapy with radotinib. Disclosures Kim: Il-Yang: Consultancy, Honoraria, Membership on an entity9s Board of Directors or advisory committees, Research Funding; BMS: Honoraria, Membership on an entity9s Board of Directors or advisory committees, Research Funding; Takeda: Membership on an entity9s Board of Directors or advisory committees, Research Funding; Novartis: Honoraria, Membership on an entity9s Board of Directors or advisory committees, Research Funding; Pfizer: Honoraria, Membership on an entity9s Board of Directors or advisory committees, Research Funding.
Achievement of MMR by 12 months according to 3-month EMR (&lt; 10% of BCR-ABL1IS). (b) Imatin... more Achievement of MMR by 12 months according to 3-month EMR (&lt; 10% of BCR-ABL1IS). (b) Imatinib 400 mg qd (n = 78 patients with evaluable molecular data at 3 months).
Achievement of MMR by 12 months according to 3-month EMR (&lt; 10% of BCR-ABL1IS).EMR at 3 mo... more Achievement of MMR by 12 months according to 3-month EMR (&lt; 10% of BCR-ABL1IS).EMR at 3 months.
Major molecular response by 12 months, by Sokal risk group. bid, twice daily; MMR, major molecula... more Major molecular response by 12 months, by Sokal risk group. bid, twice daily; MMR, major molecular response (defined as BCR-ABL1 transcript level {less than or equal to}0.1% [MR3.0]); qd, daily.
Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (c) Radotinib ... more Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (c) Radotinib 300 mg bid (n = 79 patients with evaluable molecular data at 3 months).
Purpose: Radotinib is a second-generation BCR-ABL1 tyrosine kinase inhibitor (TKI) approved in Ko... more Purpose: Radotinib is a second-generation BCR-ABL1 tyrosine kinase inhibitor (TKI) approved in Korea for chronic phase chronic myeloid leukemia (CML-CP) in patients newly diagnosed or with insufficient response to other TKIs. This study was conducted to evaluate the efficacy and safety of radotinib as first-line therapy for CML-CP.Experimental Design: This multinational, open-label study assigned patients (1:1:1) to one of two twice-daily radotinib doses, or imatinib daily. The primary endpoint was major molecular response (MMR) by 12 months.Results: Two hundred forty-one patients were randomized to receive radotinib 300 mg (n = 79) or 400 mg twice-daily (n = 81), or imatinib 400 mg daily (n = 81). MMR rates by 12 months were higher in patients receiving radotinib 300 mg (52%) or radotinib 400 mg twice-daily (46%) versus imatinib (30%; P = 0.0044 and P = 0.0342, respectively). Complete cytogenetic response (CCyR) rates by 12 months were higher for radotinib 300 mg (91%) versus imati...
Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (b) Imatinib 4... more Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (b) Imatinib 400 mg qd (n = 78 patients with evaluable molecular data at 3 months).
Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (d) Radotinib ... more Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (d) Radotinib 400 mg bid (n = 79 patients with evaluable molecular data at 3 months).
Background: In RERISE phase 3 study, radotinib demonstrated significantly higher and faster rates... more Background: In RERISE phase 3 study, radotinib demonstrated significantly higher and faster rates of major molecular response (MMR) than imatinib in patients with newly diagnosed CML-CP. By 36 months follow up, MMR (BCR-ABL1IS ≤ 0.1%) and MR4.5 (BCR-ABL1IS ≤ 0.0032%) in radotinib 300 mg twice daily (bid) were higher than imatinib group. Also, early molecular response (EMR) at 3 months could predict better long term outcomes in both radotinib and imatinib groups. Here, authors updated 48 months long-term benefits and risks of 300mg bid and imatinib 400mg qd from RERISE phase 3 study (NCT01511289). Methods: RERISE study was randomized trial of radotinib 300 mg bid (n=79), radotinib 400 mg bid (n=81), or imatinib 400 mg once daily (qd) (n=81) in patients with newly diagnosed CML-CP. We evaluated long-term MMR and MR4.5, overall survival (OS), and progression-free survival (PFS) including safety data by 48 months. Results: At the study completion, 53% of patients with radotinib and 44% ...
Major molecular response by 12 months, by Sokal risk group. bid, twice daily; MMR, major molecula... more Major molecular response by 12 months, by Sokal risk group. bid, twice daily; MMR, major molecular response (defined as BCR-ABL1 transcript level {less than or equal to}0.1% [MR3.0]); qd, daily.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated ... more Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated hemolysis. Pegcetacoplan is the first C3-targeted therapy approved for adults with PNH (United States), adults with PNH with inadequate response or intolerance to a C5 inhibitor (Australia), and adults with anemia despite C5-targeted therapy for ≥3 months (European Union). PRINCE was a phase 3, randomized, multicenter, open-label, controlled study to evaluate the efficacy and safety of pegcetacoplan vs control (supportive care only; eg, blood transfusions, corticosteroids, and supplements) in complement inhibitor–naive patients with PNH. Eligible adults receiving supportive care only for PNH were randomly assigned and stratified based on their number of transfusions (<4 or ≥4) 12 months before screening. Patients received pegcetacoplan 1080 mg subcutaneously twice weekly or continued supportive care (control) for 26 weeks. Coprimary end points were hemoglobin stabilization (avoidance ...
Background In RERISE phase 3 study, radotinib demonstrated significantly higher and faster rates ... more Background In RERISE phase 3 study, radotinib demonstrated significantly higher and faster rates of major molecular response (MMR) than imatinib in patients with newly diagnosed CML-CP. By 12 and 24 months follow up, MMR (BCR-ABL1IS≤ 0.1%) and MR4.5 (BCR-ABL1IS ≤ 0.0032%) in radotinib 300 mg twice daily (bid) were higher than imatinib group. Also, early molecular response (EMR) at 3- or 6- months could predict better outcomes in both radotinib or imatinib groups. To confirm the long-term benefits and risks of radotinib 300mg bid and imatinib 400mg qd, we update the results from RERISE phase 3 study based on a minimum follow-up of 36 months. Methods 241 patients were randomized 1:1:1 to radotinib 300 mg bid (n=79), radotinib 400 mg bid (n=81), or imatinib 400 mg once daily (qd) (n=81). Methods have been previously reported (Blood 2015 126:476). We evaluated MMR and MR4.5, overall survival (OS), and progression-free survival (PFS) by 36 months. Also, we analyzed the clinical impacts of early and deeper molecular response in radotinib 300mg bid and imatinib 400mg qd groups. Results By 36 months, MMR was significantly higher in patients receiving radotinib 300 mg bid compared with imatinib 400mg qd (Table). The MR4.5 rate by 36 months was also higher for radotinib compared to imatinib (43% vs 28%; P=0.0538). More patients treated with radotinib achieved additional MMR and MR4.5 since 12 months and time to MR4.5 was faster in radotinib than imatinib (median 924 vs 1,095 days; P=0.2534). Of 59 patients who had MMR by 36 months, 18 patients achieved MMR, and of 34 patients who had MR4.5 by 36 months, 22 patients achieved MR4.5 since 12 months. Estimated OS and PFS rate at 36 months were not significantly different in two groups (98% vs 97%; P=0.0554, 99% vs 95%; P=0.4707). Treatment failure was lower in radotinib group compared with imatinib group (Table). The safety profiles were consistent with those previously reported and most of adverse events (AEs) have developed within 12 months. Since 12 months, newly developed AEs such as rash, nausea/vomiting, pruritis, musculoskeletal pain, fatigue, hyperbilirubinemia, and ALT elevation, etc have shown minimal increase by 36 months FU. Conclusions With a minimum 36 months follow-up, radotinib continued to demonstrate significantly higher rates of MMR and MR4.5 than imatinib in newly diagnosed CML-CP. Also, these responses with radotinib were earlier and deeper compared with imatinib. These results still demonstrate that radotinib can be one of the standards of care in newly diagnosed CML-CP and support the higher possibility of treatment- free remission (TFR) on frontline therapy with radotinib. Disclosures Kim: Il-Yang: Consultancy, Honoraria, Membership on an entity9s Board of Directors or advisory committees, Research Funding; BMS: Honoraria, Membership on an entity9s Board of Directors or advisory committees, Research Funding; Takeda: Membership on an entity9s Board of Directors or advisory committees, Research Funding; Novartis: Honoraria, Membership on an entity9s Board of Directors or advisory committees, Research Funding; Pfizer: Honoraria, Membership on an entity9s Board of Directors or advisory committees, Research Funding.
Achievement of MMR by 12 months according to 3-month EMR (&lt; 10% of BCR-ABL1IS). (b) Imatin... more Achievement of MMR by 12 months according to 3-month EMR (&lt; 10% of BCR-ABL1IS). (b) Imatinib 400 mg qd (n = 78 patients with evaluable molecular data at 3 months).
Achievement of MMR by 12 months according to 3-month EMR (&lt; 10% of BCR-ABL1IS).EMR at 3 mo... more Achievement of MMR by 12 months according to 3-month EMR (&lt; 10% of BCR-ABL1IS).EMR at 3 months.
Major molecular response by 12 months, by Sokal risk group. bid, twice daily; MMR, major molecula... more Major molecular response by 12 months, by Sokal risk group. bid, twice daily; MMR, major molecular response (defined as BCR-ABL1 transcript level {less than or equal to}0.1% [MR3.0]); qd, daily.
Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (c) Radotinib ... more Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (c) Radotinib 300 mg bid (n = 79 patients with evaluable molecular data at 3 months).
Purpose: Radotinib is a second-generation BCR-ABL1 tyrosine kinase inhibitor (TKI) approved in Ko... more Purpose: Radotinib is a second-generation BCR-ABL1 tyrosine kinase inhibitor (TKI) approved in Korea for chronic phase chronic myeloid leukemia (CML-CP) in patients newly diagnosed or with insufficient response to other TKIs. This study was conducted to evaluate the efficacy and safety of radotinib as first-line therapy for CML-CP.Experimental Design: This multinational, open-label study assigned patients (1:1:1) to one of two twice-daily radotinib doses, or imatinib daily. The primary endpoint was major molecular response (MMR) by 12 months.Results: Two hundred forty-one patients were randomized to receive radotinib 300 mg (n = 79) or 400 mg twice-daily (n = 81), or imatinib 400 mg daily (n = 81). MMR rates by 12 months were higher in patients receiving radotinib 300 mg (52%) or radotinib 400 mg twice-daily (46%) versus imatinib (30%; P = 0.0044 and P = 0.0342, respectively). Complete cytogenetic response (CCyR) rates by 12 months were higher for radotinib 300 mg (91%) versus imati...
Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (b) Imatinib 4... more Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (b) Imatinib 400 mg qd (n = 78 patients with evaluable molecular data at 3 months).
Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (d) Radotinib ... more Achievement of MMR by 12 months according to 3-month EMR (< 10% of BCR-ABL1IS). (d) Radotinib 400 mg bid (n = 79 patients with evaluable molecular data at 3 months).
Background: In RERISE phase 3 study, radotinib demonstrated significantly higher and faster rates... more Background: In RERISE phase 3 study, radotinib demonstrated significantly higher and faster rates of major molecular response (MMR) than imatinib in patients with newly diagnosed CML-CP. By 36 months follow up, MMR (BCR-ABL1IS ≤ 0.1%) and MR4.5 (BCR-ABL1IS ≤ 0.0032%) in radotinib 300 mg twice daily (bid) were higher than imatinib group. Also, early molecular response (EMR) at 3 months could predict better long term outcomes in both radotinib and imatinib groups. Here, authors updated 48 months long-term benefits and risks of 300mg bid and imatinib 400mg qd from RERISE phase 3 study (NCT01511289). Methods: RERISE study was randomized trial of radotinib 300 mg bid (n=79), radotinib 400 mg bid (n=81), or imatinib 400 mg once daily (qd) (n=81) in patients with newly diagnosed CML-CP. We evaluated long-term MMR and MR4.5, overall survival (OS), and progression-free survival (PFS) including safety data by 48 months. Results: At the study completion, 53% of patients with radotinib and 44% ...
Major molecular response by 12 months, by Sokal risk group. bid, twice daily; MMR, major molecula... more Major molecular response by 12 months, by Sokal risk group. bid, twice daily; MMR, major molecular response (defined as BCR-ABL1 transcript level {less than or equal to}0.1% [MR3.0]); qd, daily.
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