Efficacy and Safety of Luspatercept in the Treatment of β-Thalassemia: A Systematic Review.

Autor:	Dighriri IM; Department of Pharmacy, King Abdulaziz Specialist Hospital, Taif, SAU., Alrabghi KK; Department of Pharmacy, Al Qurayyat General Hospital, Al Qurayyat, SAU., Sulaiman DM; College of Pharmacy, University of Duhok, Duhok, IRQ., Alruwaili AM; Department of Pharmacy, Al Dawaa Medical Services Company, Sakaka, SAU., Alanazi NS; Department of Pharmacy, King Salman Specialist Hospital, Hail, SAU., Al-Sadiq AA; Department of Pharmacy, Mouwasat Hospital, Jubail, SAU., Hadadi AM; Department of Pharmacy, Community Pharmacy, Jazan, SAU., Sahli BY; Department of Pharmacy, Community Pharmacy, Jazan, SAU., Qasem BA; College of Pharmacy, Jazan University, Jazan, SAU., Alotaibi MT; College of Pharmacy, Shaqra University, Al Dawadmi, SAU., Asiri TT; College of Pharmacy, King Khalid University, Abha, SAU., Majrashi SM; College of Pharmacy, Prince Sattam Bin Abdulaziz University, Al Kharj, SAU., Alotibia NT; College of Pharmacy, Shaqra University, Al Dawadmi, SAU., Alhamyani AT; College of Pharmacy, Taif University, Taif, SAU., Alharbi AA; Pharmaceutical Care Services, King Salman Specialist Hospital, Hail Health Cluster, Ministry of Health, Hail, SAU.
Jazyk:	angličtina
Zdroj:	Cureus [Cureus] 2022 Nov 16; Vol. 14 (11), pp. e31570. Date of Electronic Publication: 2022 Nov 16 (Print Publication: 2022).
DOI:	10.7759/cureus.31570
Abstrakt:	β-thalassemia is characterized by the faulty generation of hemoglobin resulting in an elevated α/β globin ratio; this led to several patients needing red blood cell (RBC) transfusions for the rest of their lives. Luspatercept is an erythroid maturation test for treating various types of anemia, including β-thalassemia. It inhibits the Smad2/3 cascade and treats β-thalassemia by downregulating the transforming growth factor-beta (TGF-β) pathway. Luspatercept was evaluated in randomized controlled trials (RCTs). However, there is still limited data. Therefore, the study aims to review the current literature to assess the efficacy of luspatercept in cure β-thalassemia and its safety. From 2015 to 2022, searches were undertaken in PubMed, Google Scholar, and Cochrane. Only RCTs published in English were eligible for inclusion. The Cochrane Collaboration tool for bias assessment was used to analyze the quality of the publications. Our search strategy revealed 94 publications, of which 12 full-text papers were read and five were chosen for this review.All five trials included 1161 participants. Of whom, 153 (13.18%) entered phase 2, and 1008 (86.82%) entered phase 3. Two articles included 153 participants, of whom 70 (45.75%) were transfusion-dependent beta-thalassemia (TD) and 83 (54.25%) were non-transfusion-dependent beta-thalassemia (NTD) of phase 2. Three articles included 1008 participants, of whom 672 (66.67%) were given luspatercept and 336 (33.33%) were given a placebo. All participants in RCTs were 18 years of age or older. In phase 2, 0.2 to 1.25 mg/kg of luspatercept was given, and in phase 3, 1.0 to 1.25 mg/kg of luspatercept was given once every three weeks. In beta-thalassemia patients, luspatercept was more effective than a placebo and well tolerated. The high dose has shown promising results in the erythroid response, measured by a drop in blood transfusions or an average rise in hemoglobin levels. Luspatercept might make patients less likely to need RBC transfusions, improve their clinical results, and improve their quality of life. Adverse events were hyperuricemia, arthralgia, dizziness, influenza hypertension, and bone pain, but they were manageable. Competing Interests: The authors have declared that no competing interests exist. (Copyright © 2022, Dighriri et al.)
Databáze:	MEDLINE
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