Real-World Outcome of Treatment with Single-Agent Ibrutinib in Italian Patients with Chronic Lymphocytic Leukemia: Final Results of the EVIdeNCE Study

被引：2

作者：

Mauro, Francesca Romana ^{[1
]}

Scalzulli, Potito Rosario ^{[2
]}

Scarfo, Lydia ^{[3
,4
]}

Minoia, Carla ^{[5
]}

Murru, Roberta ^{[6
]}

Sportoletti, Paolo ^{[7
]}

Frigeri, Ferdinando ^{[8
]}

Albano, Francesco ^{[9
]}

Di Renzo, Nicola ^{[10
]}

Sanna, Alessandro ^{[11
]}

Laurenti, Luca ^{[12
]}

Massaia, Massimo ^{[13
,14
]}

Cassin, Ramona ^{[15
]}

Coscia, Marta ^{[14
,16
]}

Patti, Caterina ^{[17
]}

Pennese, Elsa ^{[18
]}

Tafuri, Agostino ^{[19
]}

Chiarenza, Annalisa ^{[20
]}

Galieni, Piero ^{[21
]}

Perbellini, Omar ^{[22
]}

Selleri, Carmine ^{[23
]}

Califano, Catello ^{[24
]}

Ferrara, Felicetto ^{[25
]}

Cuneo, Antonio ^{[26
]}

Murineddu, Marco ^{[27
]}

Palumbo, Gaetano ^{[28
]}

Scortechini, Ilaria ^{[29
]}

Tedeschi, Alessandra ^{[30
]}

Trentin, Livio ^{[31
]}

Varettoni, Marzia ^{[32
]}

Pane, Fabrizio ^{[33
]}

Liberati, Anna Marina ^{[34
]}

Merli, Francesco ^{[35
]}

Morello, Lucia ^{[36
]}

Musuraca, Gerardo ^{[37
]}

Tani, Monica ^{[38
]}

Ibatici, Adalberto ^{[39
]}

Regazzoni, Giulia ^{[40
]}

Di Candia, Michele ^{[40
]}

Palma, Maria ^{[40
]}

Arienti, Danilo ^{[40
]}

Molica, Stefano ^{[41
,42
]}

机构：

[1] Sapienza Univ Roma, Ematol, I-00185 Rome, Italy

[2] Osped Casa Sollievo Sofferenza, I-71013 San Giovanni Rotondo, Italy

[3] Univ Vita Salute San Raffaele, Milan, Italy

[4] IRCCS Osped San Raffaele, Milan, Italy

[5] IRCCS Ist Tumori Giovanni Paolo II, Hematol Unit, Bari, Italy

[6] Osped Oncol A Businco, ARNAS G Brotzu, Hematol & Stem Cell Transplantat Unit, Cagliari, Italy

[7] Univ Perugia, Sez Ematol, I-06123 Perugia, Italy

[8] AORN St Anna & San Sebastiano, I-81100 Caserta, Italy

[9] Univ Bari Aldo Moro, Dept Precis & Regenerat Med & Ionian Area DiMePRe, Hematol & Stem Cell Transplantat Unit, I-70121 Bari, Italy

[10] UOC Ematol & Trapianto Cellule Staminali PO Vito F, I-73100 Lecce, Italy

[11] AOU Careggi, Florence, Italy

[12] Policlin A Gemelli, I-00168 Rome, Italy

[13] SC Ematol AO S Croce & Carle, Cuneo, Italy

[14] Univ Torino, Dept Mol Biotechnol & Hlth Sci, I-10124 Turin, Italy

[15] Fdn IRCCS Ca Granda Osped Maggiore Policlin Milan, I-20122 Milan, Italy

[16] AOU Citta Salute & Sci Torino, SC Ematol, I-10126 Turin, Italy

[17] Osped Riuniti Villa Sofia Cervello, UOC Oncoematol, I-90146 Palermo, Italy

[18] ASL Pescara, UOC Ematol Clin, I-65124 Pescara, Italy

[19] Sapienza Univ Roma, Ematol, AOU St Andrea, I-00185 Rome, Italy

[20] AOU Policlin GRodol San Marco, UOC Ematol, I-95123 Catania, Italy

[21] Osped Mazzoni, UOC Ematol & Terapia Cellulare, I-63100 Ascoli Piceno, Italy

[22] Azienda ULSS 8 Ber, UOC Ematol, I-36100 Vicenza, Italy

[23] Univ Salerno, Dept Med & Surg, Hematol & Bone Marrow Transplant Ctr, Salerno, Italy

[24] PO Andrea Tortora Pagani, UOC Ematol, I-84016 Pagani, Italy

[25] AORN Antonio Cardarelli, I-80131 Naples, Italy

[26] Univ Ferrara, Dept Med Sci, I-44121 Ferrara, Italy

[27] Osped San Francesco ASL Nuoro, Ematol, I-08100 Nuoro, Italy

[28] AOU Policlin Foggia, SC Ematol, I-71122 Foggia, Italy

[29] Azienda Osped Univ Marche, I-60126 Ancona, Italy

[30] ASST GOM Niguarda, Div Ematol, I-20162 Milan, Italy

[31] Univ Padua, Dipartimento Med, UOC Ematol, I-35122 Padua, Italy

[32] Fdn IRCCS Policlin San Matteo, Div Ematol, I-27100 Pavia, Italy

[33] Univ Napoli Federico II, Dept Clin Med & Surg, Div Hematol, I-80131 Naples, Italy

[34] Univ Perugia, AO Santa Maria, Dept Gen Surg, I-05100 Terni, Italy

[35] Ematol AUSL IRCCS Reggio Emilia, I-42123 Reggio Emilia, Italy

[36] IRCCS Humanitas Res Hosp, Humanitas Canc Ctr, Milan, Italy

[37] IRCCS Ist Romagnolo Tumori IRST Dino Amadori, I-47014 Meldola, Italy

[38] Osped S Maria delle Croci, Dept Radiat Oncol, I-48121 Ravenna, Italy

[39] IRCCS Osped Policlin San Martino, Genoa, Italy

[40] Johnson & Johnson Innovat Med, Beerse, Belgium

[41] Azienda Osped Pugliese Ciaccio, Dipartimento Onco Ematol, Catanzaro, Italy

[42] Hull Univ NHS Trust, Castle Hill Hosp, Queens Ctr Oncol & Haematol, Kingston Upon Hull HU16 5JQ, England

来源：

CANCERS | 2024年 / 16卷 / 06期

关键词：

chronic lymphocytic leukemia; ibrutinib; real-world evidence; retention; clinical outcomes; effectiveness; DISCONTINUATION; PATTERNS; THERAPY;

D O I：

10.3390/cancers16061228

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Simple Summary In clinical trials, ibrutinib was found to be effective and well-tolerated in patients with chronic lymphocytic leukemia (CLL). To confirm these findings, data on unselected patients treated in clinical practice are necessary. The aim of our observational, prospective Italian cohort study was to describe the real-world persistence rate, patterns of use, and clinical outcomes in patients with CLL treated with single-agent ibrutinib across various treatment lines. We found that, despite the high burden of patient comorbidities and unfavorable genetic features, the majority of patients (217/309, 70%), especially those treated in first line (75%), continued ibrutinib treatment for >= 2 years. The most common reasons for treatment discontinuation were adverse events, primarily infections. We reported positive clinical and survival outcomes, especially in the first-line cohort, and a safety profile consistent with clinical trial data. Our data suggest that ibrutinib is a valuable option for both treatment-naive and previously treated patients with CLL.Abstract Real-world data in clinical practice are needed to confirm the efficacy and safety that ibrutinib has demonstrated in clinical trials of patients with chronic lymphocytic leukemia (CLL). We described the real-world persistence rate, patterns of use, and clinical outcomes in 309 patients with CLL receiving single-agent ibrutinib in first line (1L, n = 118), 2L (n = 127) and >= 3L (n = 64) in the prospective, real-world, Italian EVIdeNCE study. After a median follow-up of 23.9 months, 29.8% of patients discontinued ibrutinib (1L: 24.6%, 2L: 29.9%, >= 3L: 39.1%), mainly owing to adverse events (AEs)/toxicity (14.2%). The most common AEs leading to discontinuation were infections (1L, >= 3L) and cardiac events (2L). The 2-year retention rate was 70.2% in the whole cohort (1L: 75.4%, 2L: 70.1%, >= 3L: 60.9%). The 2-year PFS and OS were, respectively, 85.4% and 91.7% in 1L, 80.0% and 86.2% in 2L, and 70.1% and 80.0% in >= 3L. Cardiovascular conditions did not impact patients' clinical outcomes. The most common AEs were infections (30.7%), bleeding (12.9%), fatigue (10.0%), and neutropenia (9.7%), while grade 3-4 atrial fibrillation occurred in 3.9% of patients. No new safety signals were detected. These results strongly support ibrutinib as a valuable treatment option for CLL.

引用

页数：15

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