Efficacy and safety of recently approved drugs for sickle cell disease: a review of clinical trials

• Published in: Experimental hematology 2020-12, Vol.92, p.11-18.e1
• Main Authors: Ali, Muhammad Ashar, Ahmad, Asrar, Chaudry, Hafsa, Aiman, Wajeeha, Aamir, Sobia, Anwar, Muhammad Yasir, Khan, Anam
• Format: Article
• Language: English
• Subjects: Anemia, Sickle Cell - metabolism; Anemia, Sickle Cell - physiopathology; Anemia, Sickle Cell - therapy; Antisickling Agents - therapeutic use; Blood Transfusion; Clinical Trials as Topic; Drug Approval; Humans; Review
• ISSN: 0301-472X; 1873-2399
• DOI: 10.1016/j.exphem.2020.08.008

•VOC episodes and low hemoglobin levels are major complications of SCD.•L-Glutamine decreases the episodes of VOC.•Voxelotor increases hemoglobin levels and reduces hemolysis.•Crizanlizumab decreases the number of VOC episodes.•L-Glutamine, voxelotor, and crizanlizumab are well tolerated by SCD patients. Sickle cell disease is prevalent in several parts of the world. Most hospitalizations of these patients are related to pain crisis episodes. Moreover, levels of hemoglobin are lower in sickle cell disease patients as compared with the general population. Complications related to sickle cell disease are managed with blood transfusions, hydroxyurea, and opioids. Despite these therapies, patients with sickle cell disease experience multiple pain crisis episodes leading to hospitalizations and end-organ damage. The US Food and Drug Administration has approved three new drugs—L-glutamine, voxelotor, and crizanlizumab—for the prophylaxis and treatment of complications related to sickle cell disease. This review was aimed at assessing the efficacy and safety of recently approved drugs for the treatment of sickle cell disease. A comprehensive search was made on PubMed and clinicaltrials.gov to look for clinical trials reporting the efficacy and safety of recently approved drugs for sickle cell disease. Based on the results of clinical trials, L-glutamine, voxelotor, and crizanlizumab were well tolerated by sickle cell disease patients. L-Glutamine and crizanlizumab reduced the number of sickle cell crisis episodes, while voxelotor improved the level of hemoglobin in sickle cell disease patients. These drugs were effective alone and in combination with hydroxyurea. [Display omitted]