Reported Saturday, Mirum Pharmaceuticals' LIVMARLI Data Presented At ESPGHAN, Featuring Key Findings On ALGS And PFIC
Reported Saturday, Mirum Pharmaceuticals' LIVMARLI Data Presented At ESPGHAN, Featuring Key Findings On ALGS And PFIC
Presented by Professor Richard J. Thompson, MD, King's College London, United Kingdom
Patients with PFIC showed significant and sustained improvements in pruritus severity, serum bile acid (sBA) levels, total bilirubin and growth following up to two years of LIVMARLI treatment. Similar improvements in pruritus and sBA were seen in patients originally randomized to placebo who received LIVMARLI in the open-label study.
由英国伦敦国王学院医学博士理查德·汤普森教授主讲
在接受长达两年的LIVMARLI治疗后，PFIC患者的瘙痒严重程度、血清胆汁酸（SbA）水平、总胆红素和生长均有显著持续的改善。在开放标签研究中，最初随机分配到安慰剂的患者中也出现了类似的瘙痒和sbA改善。
Abstract 594: Maralixibat Leads to Significant Reductions in Bilirubin for Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH Trial
Presented by Lorenzo D'Antiga, MD, Papa Giovanni XXIII Hospital, Bergamo, Italy
Patients treated with LIVMARLI experienced significant decreases in both total and direct bilirubin compared to placebo. 40% of the patients with abnormal bilirubin at baseline treated with LIVMARLI achieved normalization versus none in the placebo group. Further, these reductions in bilirubin were consistent with reductions in sBAs.
摘要 594：Maralixibat 可显著降低进行性家族性肝内胆汁淤积患者的胆红素：来自 MARCH 试验的数据
由意大利贝加莫爸乔瓦尼二十三世医院医学博士洛伦佐·德安蒂加主持
与安慰剂相比，接受LIVMARLI治疗的患者的总胆红素和直接胆红素均显著降低。在基线接受LIVMARLI治疗的胆红素异常患者中，有40％实现正常化，而安慰剂组中没有一例。此外，胆红素的减少与SBA的减少是一致的。
Abstract 600: Maralixibat Impact on Concomitant Medication Use for the Treatment of Cholestatic Pruritus in Alagille Syndrome: Real-World Experience
Presented by Jolan Terner-Rosenthal, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA
Real-world evidence from 116 patients treated with LIVMARLI for at least one year showed that more than one-third of patients were able to discontinue ≥1 concomitant antipruritic medication, and one in five patients discontinued ≥2 medications. Reductions in concomitant medication usage were seen across all medication types and suggest that LIVMARLI may reduce the polypharmacy burden within the first year of treatment.
摘要 600：Maralixibat 对同时使用药物治疗阿拉吉尔综合征胆汁淤积性瘙痒的影响：现实世界的经验
由美国加利福尼亚州福斯特城 Mirum 制药公司的 Jolan Terner-Rosenthal 博士主持
来自116名接受LIVMARLI治疗至少一年的患者的真实证据表明，超过三分之一的患者能够停用≥1种伴随的止痒药物，五分之一的患者停用了≥2种药物。所有药物类型的伴随用药量均有所减少，这表明LIVMARLI可以在治疗的第一年内减轻多药负担。
Other presentations featured during ESPGHAN include:
ESPGHAN 期间的其他演讲包括：
Abstract 592: Maralixibat Leads to Significant Improvements in Cholestatic Pruritus for Children with Progressive Familial Intrahepatic Cholestasis Without a Genetic Diagnosis: Data from the MARCH Trial
Presented by Professor Richard J. Thompson, MD, King's College, London, United Kingdom
摘要 592：Maralixibat 可显著改善未经遗传诊断的进行性家族性肝内胆汁淤积患儿的胆汁淤积性瘙痒：来自 MARCH 试验的数据
由英国伦敦国王学院医学博士理查德·汤普森教授主讲
Abstract 599: Maralixibat Leads to Improvements in Cholestatic Pruritus for Children with Progressive Familial Intrahepatic Cholestasis Due to MDR3 Deficiency: Data From the MARCH/MARCH-ON Trials
Presented by Professor Richard J. Thompson, MD, King's College, London, United Kingdom
摘要 599：Maralixibat 改善了因 MDR3 缺乏导致进行性家族性肝内胆汁淤积症的患儿的胆汁淤积症状：来自 MARCH/MARCH-ON 试验的数据
由英国伦敦国王学院医学博士理查德·汤普森教授主讲
Abstract 595: Improvements in Pruritus with Maralixibat are Associated with Improved Quality of Life for Patients with Progressive Familial Intrahepatic Cholestasis: Data From the MARCH Trial
Presented by Douglas B. Mogul, MD, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA
摘要 595：Maralixibat 改善瘙痒症与进行性家族性肝内胆汁淤积患者的生活质量改善有关：来自 MARCH 试验的数据
由美国加利福尼亚州福斯特城 Mirum 制药公司医学博士、博士道格拉斯·莫格尔主持
Abstract 597: Maralixibat Can Improve Cholestatic Pruritus in Children with Progressive Familial Intrahepatic Cholestasis Who Previously Underwent a Surgical Biliary Diversion: Data From the MARCH/MARCH-ON Trials
摘要 597：Maralixibat 可以改善之前接受过胆道转移手术的进行性家族性肝内胆汁淤积患儿的胆汁淤积性瘙痒：来自 MARCH/MARCH-ON 试验的数据