| Primary | Percentage of Participants Who Achieved Remission Without Requirement of Therapeutic Plasma Exchange During Overall Study Period | Remission was defined as sustained clinical response with either no TPE and no anti- von Willebrand factor (vWF) therapy for >=30 days (clinical remission) or with attainment of a disintegrin and metalloproteinase with a thrombospondin type 1 motif13 (ADAMTS13) activity level >=50% (complete ADAMTS13 remission), whichever occurred first. Clinical response was defined as sustained platelet count >=150 × 10^9/liter (L) and lactate dehydrogenase (LDH) <1.5 × upper limit of normal (ULN) and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits. | The modified intent-to-treat (mITT) population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks) | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants Who Achieved Remission During Overall Study Period | Remission was defined as sustained clinical response with either no TPE and no anti- vWF therapy for >=30 days (clinical remission) or with ADAMTS13 activity level >=50% (complete ADAMTS13 remission), whichever occurred first. Clinical response was defined as sustained platelet count >=150 × 10^9/L and LDH <1.5 × ULN and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks) | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants Who Required Therapeutic Plasma Exchange During On-Treatment Period | TPE was a procedure in which blood of the participant was passed through a medical device which separated out plasma from other components of blood and the participant's plasma was removed and replaced with a replacement solution such as colloid solution (example, albumin and/or plasma) or a combination of crystalloid/colloid solution. TPE replenishes the ADAMTS13 enzyme and removes anti-ADAMTS13 antibodies, and ultra-large von Willebrand factor multimers gradually from the circulation. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), and Treatment-Emergent Adverse Events of Special Interest (TEAESI) | An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. TEAEs were defined as the AEs that developed, worsened or became serious during the treatment-emergent period. SAE: Any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect, or any other situation where medical or scientific judgment of investigator were exercised. An AESI is an AE (serious or nonserious) of scientific and medical concern specific to the Sponsor's product or program, for which ongoing monitoring and immediate notification by the Investigator to the Sponsor was required. | The safety population consisted of all enrolled participants who took at least 1 dose of the study treatment. | Posted | | Count of Participants | | Participants | | From first dose of study treatment (Day 1) up to last dose of study treatment + 28 days (approximately 16 weeks) | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants Who Achieved Clinical Response During On-Treatment Period | Clinical response was defined as sustained platelet count >=150 × 10^9/L and LDH <1.5 × ULN and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants Who Achieved Clinical Response During Overall Study Period | Clinical response was defined as sustained platelet count >=150 × 10^9/L and LDH <1.5 × ULN and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks) | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Time to Platelet Count Response | Time to platelet count response was defined as time from start of study treatment to initial platelet count >=150 × 10^9/L that was sustained for >=2 days. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Median | 95% Confidence Interval | days | | From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks) | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants Refractory to Therapy During On-Treatment Period | Refractory to therapy was defined as lack of sustained platelet count increment (over 2 consecutive days) or platelet counts <50 × 10^9/L and persistently elevated LDH (>1.5 × ULN) despite 5 days of treatment during the on-treatment period. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)-Related Death During On-Treatment Period | Percentage of participants with iTTP-related death during on-treatment period are reported. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants With Immune-mediated Thrombotic Thrombocytopenic Purpura-Related Death During Overall Study Period | Percentage of participants with iTTP-related death during overall study period are reported. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks) | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants With a Clinical Exacerbation of Immune-Mediated Thrombotic Thrombocytopenic Purpura During On-Treatment Period | Clinical exacerbation was defined as after a clinical response and before a clinical remission, platelet count decreased to <150 × 10^9/L (with other causes of thrombocytopenia excluded), with or without clinical evidence of new or progressive ischemic organ injury, within 30 days of stopping TPE or anti-vWF therapy. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants With a Clinical Exacerbation of Immune-Mediated Thrombotic Thrombocytopenic Purpura During Overall Study Period | Clinical exacerbation was defined as after a clinical response and before a clinical remission, platelet count decreased to <150 × 10^9/L (with other causes of thrombocytopenia excluded), with or without clinical evidence of new or progressive ischemic organ injury, within 30 days of stopping TPE or anti-vWF therapy. | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks) | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants With a Clinical Relapse of Immune-Mediated Thrombotic Thrombocytopenic Purpura During On-Treatment Period | Clinical relapse was defined as after a clinical remission, platelet count decreased to <150 × 10^9/L (with other causes of thrombocytopenia ruled out), with or without clinical evidence of new ischemic organ injury. A clinical relapse had to be confirmed by documentation of severe ADAMTS13 deficiency (<10%). | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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| Secondary | Percentage of Participants With a Clinical Relapse of Immune-Mediated Thrombotic Thrombocytopenic Purpura During Overall Study Period | Clinical relapse was defined as after a clinical remission, platelet count decreased to <150 × 10^9/L (with other causes of thrombocytopenia ruled out), with or without clinical evidence of new ischemic organ injury. A clinical relapse had to be confirmed by documentation of severe ADAMTS13 deficiency (<10%). | The mITT population consisted of all enrolled participants who received at least 1 dose of the study treatment with an evaluable primary endpoint. | Posted | | Number | 95% Confidence Interval | percentage of participants | | From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks) | | | | ID | Title | Description |
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| OG000 | Caplacizumab and IST Without First-Line TPE | Participants received open-label caplacizumab daily and IST (corticosteroid +/-anti-CD20 Ab therapy [rituximab or biosimilar]) without first-line TPE. Participant received caplacizumab 10 mg IV injection for a single dose followed by caplacizumab 10 mg SC injection 4 to 6 hours after the IV dose on Day 1. Then participants received caplacizumab 10 mg SC injection daily starting on Day 2 for a maximum treatment duration of 12 weeks. Participants also received corticosteroids (prednisone or prednisolone) 1 to 1.5 mg/kg/day, IV or oral for 1 week and taper over 3 to 4 weeks, along with anti-CD20 Ab therapy at the beginning of the study. Participants did not receive TPE as first-line therapy, however, they could receive TPE if it was determined that there was a lack of adequate response to study treatment or if there was any clinical deterioration at any time during the study, including during the first 24 hours. |
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