Profile of Luspatercept in the Treatment of Anemia in Adults with Non-Transfusion-Dependent f3-Thalassemia (NTDT): Design, Development and Potential Place in Therapy

被引：5

作者：

Musallam, Khaled M. ^{[1
]}

Taher, Ali T. ^{[2
]}

Kattamis, Antonis ^{[3
]}

Kuo, Kevin H. M. ^{[4
]}

Sheth, Sujit ^{[5
]}

Cappellini, Maria Domenica ^{[6
,7
]}

机构：

[1] Burjeel Med City, Thalassemia Ctr, Abu Dhabi, U Arab Emirates

[2] Amer Univ Beirut, Med Ctr, Dept Internal Med, Beirut, Lebanon

[3] Natl & Kapodistrian Univ Athens, Dept Pediat 1, Athens, Greece

[4] Univ Toronto, Div Hematol, Toronto, ON, Canada

[5] Weill Cornell Med, Dept Pediat, Div Hematol & Oncol, New York, NY USA

[6] Univ Milan, Ca Granda Fdn IRCCS Maggiore Policlin Hosp, Dept Clin Sci & Community, Milan, Italy

[7] Univ Milan, Ca Granda Fdn IRCCS Maggiore Policlin Hosp, Dept Clin Sci & Community, I-20122 Milan, Italy

来源：

DRUG DESIGN DEVELOPMENT AND THERAPY | 2023年 / 17卷

关键词：

anemia; ineffective erythropoiesis; iron; management; thalassemia; transfusion; PULMONARY ARTERIAL-HYPERTENSION; BETA-THALASSEMIA PATIENTS; LIVER IRON CONCENTRATION; INDEPENDENT PATIENTS; INTERMEDIA; MORBIDITY; DISEASE; HYPERCOAGULABILITY; DIFFERENTIATION; COMPLICATIONS;

D O I：

10.2147/DDDT.S368584

中图分类号：

R914 [药物化学];

学科分类号：

100701 ;

摘要：

Over the past decade, evidence has been mounting on the detrimental clinical sequelae of untreated anemia in patients with non-transfusion-dependent f3-thalassemia (NTDT). There are no pharmacologic agents that are specifically approved for the manage-ment of anemia in NTDT, and available options such as splenectomy, transfusion therapy, and hydroxyurea each come with their own shortcomings, especially for long-term use. Luspatercept is an erythroid maturation agent that has been evaluated in a Phase 2, randomized trial and showed a significant benefit in raising hemoglobin level by at least 1 g/dL in adults with NTDT and a baseline hemoglobin level <= 10 g/dL. These data led to luspatercept's approval by the European Commission for the treatment of anemia in adults with NTDT and presents the first evidence-based approach for a novel agent that is able to ameliorate anemia in this patient population.

引用

页码：1583 / 1591

页数：9

共 63 条

[1] Factors Impacting Quality of Life in Thalassemia Patients; Results from the Intercontinenthal Collaborative Study [J].

Amid, Ali ;

Leroux, Rebecca ;

Merelles-Pulcini, Manuela ;

Yassobi, Saeed ;

Saliba, Antoine N. ;

Ward, Richard ;

Karimi, Mehran ;

Taher, Ali T. ;

Klaassen, Robert J. ;

Kirby-Allen, Melanie .

BLOOD, 2016, 128 (22)

[2] A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers [J].

Attie, Kenneth M. ;

Allison, Mark J. ;

McClure, Ty ;

Boyd, Ingrid E. ;

Wilson, Dawn M. ;

Pearsall, Amelia E. ;

Sherman, Matthew L. .

AMERICAN JOURNAL OF HEMATOLOGY, 2014, 89 (07) :766-770

[3] The impact of illness perception and socio-clinico-demographic factors on perceived quality of life in children and adolescents with thalassemia intermedia [J].

Atwa, Zeze T. H. ;

Wahed, Wafaa Y. Abdel .

PEDIATRIC BLOOD & CANCER, 2019, 66 (07)

[4] Systematic Literature Review of the Burden of Disease and Treatment for Transfusion-dependent β-Thalassemia [J].

Betts, Marissa ;

Flight, Patrick A. ;

Paramore, L. Clark ;

Tian, Li ;

Milenkovic, Dusan ;

Sheth, Sujit .

CLINICAL THERAPEUTICS, 2020, 42 (02) :322-+

[5]

Bristol Myers Squibb, BRIST MYERS SQUIBB W

[6] A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia [J].

Cappellini, M. D. ;

Viprakasit, V. ;

Taher, A. T. ;

Georgiev, P. ;

Kuo, K. H. M. ;

Coates, T. ;

Voskaridou, E. ;

Liew, H. -K. ;

Pazgal-Kobrowski, I. ;

Forni, G. L. ;

Perrotta, S. ;

Khelif, A. ;

Lal, A. ;

Kattamis, A. ;

Vlachaki, E. ;

Origa, R. ;

Aydinok, Y. ;

Bejaoui, M. ;

Ho, P. J. ;

Chew, L. -P. ;

Bee, P. -C. ;

Lim, S. -M. ;

Lu, M. -Y. ;

Tantiworawit, A. ;

Ganeva, P. ;

Gercheva, L. ;

Shah, F. ;

Neufeld, E. J. ;

Thompson, A. ;

Laadem, A. ;

Shetty, J. K. ;

Zou, J. ;

Zhang, J. ;

Miteva, D. ;

Zinger, T. ;

Linde, P. G. ;

Sherman, M. L. ;

Hermine, O. ;

Porter, J. ;

Piga, A. .

NEW ENGLAND JOURNAL OF MEDICINE, 2020, 382 (13) :1219-1231

[7] Quality of life in patients with β-thalassemia: A prospective study of transfusion-dependent and non-transfusion-dependent patients in Greece, Italy, Lebanon, and Thailand [J].

Cappellini, Maria Domenica ;

Kattamis, Antonis ;

Viprakasit, Vip ;

Sutcharitchan, Pranee ;

Pariseau, Joseph ;

Laadem, Abderrahmane ;

Jessent-Ciaravino, Valerie ;

Taher, Ali .

AMERICAN JOURNAL OF HEMATOLOGY, 2019, 94 (10) :E261-E264

[8] Venous thromboembolism and hypercoagulability in splenectomized patients with thalassaemia intermedia [J].

Cappellini, MD ;

Robbiolo, L ;

Bottasso, BM ;

Coppola, R ;

Fiorelli, G ;

Mannucci, PM .

BRITISH JOURNAL OF HAEMATOLOGY, 2000, 111 (02) :467-473

[9] An update on the US adult thalassaemia population: a report from the CDC thalassaemia treatment centres [J].

Chapin, John ;

Cohen, Alan R. ;

Neufeld, Ellis J. ;

Vichinsky, Elliott ;

Giardina, Patricia J. ;

Boudreaux, Jeanne ;

Le, Binh C. ;

Kenney, Kristy ;

Trimble, Sean ;

Thompson, Alexis A. .

BRITISH JOURNAL OF HAEMATOLOGY, 2022, 196 (02) :380-389

[10] Prevalence and Risk Factors for Pulmonary Arterial Hypertension in a Large Group of β-Thalassemia Patients Using Right Heart Catheterization A Webthal Study [J].

Derchi, Giorgio ;

Galanello, Renzo ;

Bina, Patrizio ;

Cappellini, Maria Domenica ;

Piga, Antonio ;

Lai, Maria-Eliana ;

Quarta, Antonella ;

Casu, Gavino ;

Perrotta, Silverio ;

Pinto, Valeria ;

Musallam, Khaled M. ;

Forni, Gian Luca .

CIRCULATION, 2014, 129 (03) :338-345

← 1 2 3 4 5 6 7 →