Old and New Drugs for Chronic Lymphocytic Leukemia: Lights and Shadows of Real-World Evidence
Several novel treatments for chronic lymphocytic leukemia (CLL) have been recently approved based on the results of randomized clinical trials. However, real-world evidence (RWE) is also requested before and after drug authorization in order to confirm safety and to provide data for health technolog...
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| Format: | Article |
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MDPI AG
2022-04-01
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| Series: | Journal of Clinical Medicine |
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| Online Access: | https://www.mdpi.com/2077-0383/11/8/2076 |
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| author | Monia Marchetti Candida Vitale Gian Matteo Rigolin Alessandra Vasile Andrea Visentin Lydia Scarfò Marta Coscia Antonio Cuneo |
| author_facet | Monia Marchetti Candida Vitale Gian Matteo Rigolin Alessandra Vasile Andrea Visentin Lydia Scarfò Marta Coscia Antonio Cuneo |
| author_sort | Monia Marchetti |
| collection | DOAJ |
| description | Several novel treatments for chronic lymphocytic leukemia (CLL) have been recently approved based on the results of randomized clinical trials. However, real-world evidence (RWE) is also requested before and after drug authorization in order to confirm safety and to provide data for health technology assessments. We conducted a scoping review of the available RWE for targeted treatments of CLL, namely ibrutinib, acalabrutinib, idelalisib, and venetoclax, as well as for chemoimmunotherapy (CIT). In particular, we searched studies published since 1 January 2010 and reported outcomes of the above treatments based on health databases, registries, or phase IV studies, including named-patient programs. We included both full papers and abstracts of studies presented at major meetings. Overall, 110 studies were selected and analyzed: 28,880 patients were treated with ibrutinib, 1424 with idelalisib, 751 with venetoclax, 496 with acalabrutinib, and 14,896 with CIT. Reported discontinuation rates were higher than in clinical trials, while effectiveness could not be indirectly compared with clinical trials since a detailed case mix, including cytogenetic risk factors, was partially available and propensity scores rarely applied. RWE on CLL can help to set realistic outcomes with novel treatments, however, real-world studies should be fostered, and available data shared. |
| first_indexed | 2024-03-09T10:32:48Z |
| format | Article |
| id | doaj.art-2d01a46f576647848ae0b4d42eb2bda7 |
| institution | Directory Open Access Journal |
| issn | 2077-0383 |
| language | English |
| last_indexed | 2024-03-09T10:32:48Z |
| publishDate | 2022-04-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Clinical Medicine |
| spelling | doaj.art-2d01a46f576647848ae0b4d42eb2bda72023-12-01T21:06:20ZengMDPI AGJournal of Clinical Medicine2077-03832022-04-01118207610.3390/jcm11082076Old and New Drugs for Chronic Lymphocytic Leukemia: Lights and Shadows of Real-World EvidenceMonia Marchetti0Candida Vitale1Gian Matteo Rigolin2Alessandra Vasile3Andrea Visentin4Lydia Scarfò5Marta Coscia6Antonio Cuneo7Haematology and Transplant Unit, Azienda Ospedaliera SS Antonio e Biagio e Cesare Arrigo, 15121 Alessandria, ItalyHematology Unit, Città della Salute e della Scienza, University of Turin, 10126 Turin, ItalyHaematology Unit, Azienda Ospedaliera Universitaria di Ferrara, 44121 Ferrara, ItalyDepartment of Public Health, University of Eastern Pedemont, 28100 Novara, ItalyHematology and Clinical Immunology Unit, Department of Medicine, University of Padua, 35128 Padua, ItalyDivision of Experimental Oncology, Department of Onco-Hematology, IRCCS San Raffaele Hospital, 20132 Milan, ItalyHematology Unit, Città della Salute e della Scienza, University of Turin, 10126 Turin, ItalyHaematology Unit, Azienda Ospedaliera Universitaria di Ferrara, 44121 Ferrara, ItalySeveral novel treatments for chronic lymphocytic leukemia (CLL) have been recently approved based on the results of randomized clinical trials. However, real-world evidence (RWE) is also requested before and after drug authorization in order to confirm safety and to provide data for health technology assessments. We conducted a scoping review of the available RWE for targeted treatments of CLL, namely ibrutinib, acalabrutinib, idelalisib, and venetoclax, as well as for chemoimmunotherapy (CIT). In particular, we searched studies published since 1 January 2010 and reported outcomes of the above treatments based on health databases, registries, or phase IV studies, including named-patient programs. We included both full papers and abstracts of studies presented at major meetings. Overall, 110 studies were selected and analyzed: 28,880 patients were treated with ibrutinib, 1424 with idelalisib, 751 with venetoclax, 496 with acalabrutinib, and 14,896 with CIT. Reported discontinuation rates were higher than in clinical trials, while effectiveness could not be indirectly compared with clinical trials since a detailed case mix, including cytogenetic risk factors, was partially available and propensity scores rarely applied. RWE on CLL can help to set realistic outcomes with novel treatments, however, real-world studies should be fostered, and available data shared.https://www.mdpi.com/2077-0383/11/8/2076chronic lymphocytic leukemiaibrutinibidelalisibvenetoclaxacalabrutinibreal-world evidence |
| spellingShingle | Monia Marchetti Candida Vitale Gian Matteo Rigolin Alessandra Vasile Andrea Visentin Lydia Scarfò Marta Coscia Antonio Cuneo Old and New Drugs for Chronic Lymphocytic Leukemia: Lights and Shadows of Real-World Evidence Journal of Clinical Medicine chronic lymphocytic leukemia ibrutinib idelalisib venetoclax acalabrutinib real-world evidence |
| title | Old and New Drugs for Chronic Lymphocytic Leukemia: Lights and Shadows of Real-World Evidence |
| title_full | Old and New Drugs for Chronic Lymphocytic Leukemia: Lights and Shadows of Real-World Evidence |
| title_fullStr | Old and New Drugs for Chronic Lymphocytic Leukemia: Lights and Shadows of Real-World Evidence |
| title_full_unstemmed | Old and New Drugs for Chronic Lymphocytic Leukemia: Lights and Shadows of Real-World Evidence |
| title_short | Old and New Drugs for Chronic Lymphocytic Leukemia: Lights and Shadows of Real-World Evidence |
| title_sort | old and new drugs for chronic lymphocytic leukemia lights and shadows of real world evidence |
| topic | chronic lymphocytic leukemia ibrutinib idelalisib venetoclax acalabrutinib real-world evidence |
| url | https://www.mdpi.com/2077-0383/11/8/2076 |
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