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Multicenter Study
. 2025 Jul 1;131(13):e35963.
doi: 10.1002/cncr.35963.

Chronic myeloid leukemia outcomes according to baseline risk and first-line treatment in real-world settings: Data from the Italian Network/CML Campus

Valentina Giai  1 , Tiziana Rosso  2 , Fausto Castagnetti  3 , Patrizia Pregno  1 , Massimiliano Bonifacio  4 , Isabella Capodanno  5 , Mario Tiribelli  6 , Fabio Stagno  7 , Giovanni Caocci  8 , Antonella Gozzini  9 , Luigiana Luciano  10 , Sara Galimberti  11 , Monica Bocchia  12 , Andrea Patriarca  13 , Giuseppe Lanzarone  14 , Bruno Martino  15 , Anna Guella  16 , Anna Rita Scortechini  17 , Carmen Fava  18 , Claudio Fozza  19 , Simona Sica  20 , Nicola Di Renzo  21 , Pellegrino Musto  22 , Domenico Pastore  23 , Alessandro Maggi  24 , Michele Pizzuti  25 , Lorella Maria Antonia Melillo  26 , Angelo Michele Carella  27 , Giuseppe Tarantini  28 , Anna Mele  29 , Elisabetta Calistri  30 , Maria Rosaria Coppi  31 , Fabio Saccona  2 , Emilia Scalzulli  32 , Beatrice Battaglio  4 , Miriam Iezza  33 , Cinzia Bitetti  34 , Roberto Freilone  1 , Gianantonio Rosti  35 , Francesco Albano  36 , Fabrizio Pane  37 , Giorgina Specchia  36 , Giovannino Ciccone  2 , Giuseppe Saglio  38 , Massimo Breccia  32
Affiliations
Multicenter Study

Chronic myeloid leukemia outcomes according to baseline risk and first-line treatment in real-world settings: Data from the Italian Network/CML Campus

Valentina Giai et al. Cancer. .

Abstract

Background: Improved outcome has been reported in chronic myeloid leukemia (CML) patients treated with tyrosine kinase inhibitors (TKIs) in sponsored trials.

Methods: This is a multicenter prospective cohort study of consecutive patients with newly diagnosed chronic phase CML from 19 regions in Italy. Baseline treatments and prognostic factors on time to first optimal molecular response (≥ molecular response 3, MR3), time to disease progression, time to death from CML, and overall survival (OS) were analyzed using multivariable Fine and Gray models.

Results: The authors included 1433 CML patients: 49% (median age, 70 years) treated with frontline imatinib (IMA), and 51% treated with second-generation TKIs (2G-TKIs; median age, 52 years). EUTOS long-term survival (ELTS) was low in 68.1% of 2G-TKIs patients, compared to 50.4% of IMA patients. Faster molecular responses were observed with 2G-TKIs within the first 6 months and maintained thereafter (subhazard ratio [sHR], 1.31; 95% confidence interval [CI], 1.15-1.50). Female gender and low ELTS risk had faster time of response. Achieving major molecular response (MMR or MR3) was associated with reduced risk of progression at 6 and 12 months. Overall, 41 patients progressed without differences between IMA and 2G-TKIs. Intermediate and high risk ELTS showed higher risk of progression and death from CML. Twenty-two CML-related deaths (16.5%) occurred mostly in the first 2 years from diagnosis, higher in 2G-TKIs patients (sHR, 1.75; 95% CI, 0.52-5.87). OS at 5 years was 88% with no clear differences between IMA and 2G-TKIs treatment after adjustment for potential confounders.

Conclusions: The study confirms faster responses with 2G-TKIs compared to IMA but similar clinical outcomes and a strong prognostic effect of ELTS.

Keywords: TKI; chronic myeloid leukemia; first‐line treatment; imatinib; real‐life study; risk factors; second generation TKI.

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Conflict of interest statement

Valentina Giai served on advisory board for Novartis, Pfizer, and Incyte and received honoraria from Alexion, Sobi, and Roche. Massimiliano Bonifacio received honoraria from Amgen and served on advisory board for Incyte, Novartis, Bristol‐Myers Squibb, and Pfizer. Elisabetta Capodanno received honoraria from Incyte, Novartis, Bristol‐Myers Squibb, and Celgene and served on advisory board for Pfizer. Fabio Stagno received honoraria for Speakers Bureau from Incyte and Novartis. Sara Galimberti received honoraria for supporting meetings from Novartis, AbbVie, Pfizer, Incyte, Astra Zeneca, Jazz, Celgene, Roche, and Janssen. Andrea Patriarca received honoraria from Sobi, Novartis, Roche, Incyte, Alexion, Takeda, Pfizer, Sanofi, and Bristol‐Myers Squibb. Bruno Martino received honoraria for Speakers Bureau from Incyte, Astra Zeneca, Novartis, and Janssen. Claudio Fozza received research funding from Sanofi, Amgen, Bristol‐Myers Squibb, and Sobi. Pellegrino Musto received consulting fees from Beigene. Gianantonio Rosti received honoraria for Speakers Bureau from Novartis, Incyte, and Pfizer. Fabrizio Pane received fees for consulting, membership on an entity's Board of Directors or advisory committees and Speakers Bureau from Incyte, Takeda, AbbVie, Amgen, Alexion, GSK, Bristol‐Myers Squibb, Janssen, Jazz, Novartis, and Pfizer. Massimo Breccia received honoraria from Novartis, Incyte, Pfizer, AbbVie, Bristol‐Myers Squibb, and AOP. Giuseppe Saglio received consulting fees and Speakers Bureau from Novartis, Hikma, and Ascentage Pharma. Monica Bocchia received consulting fees from Incyte Corporation and Novartis. Fausto Castagnetti received consulting fees from Bristol‐Myers Squibb, Incyte Corporation, Novartis, and Pfizer. Maria Rosaria Coppi received fees for professional activities from the American Cancer Society. Anna Guella received fees for professional activities from APSS Trento. Giuseppe Lanzarone received travel fees from AbbVie, Jazz Pharmaceuticals, and Novartis. Fabio Saccona received fees for professional activities from AOU Città della Salute e della Scienza di Torino. Anna Rita Scortechini holds a copyright for “Chronic Myeloid Leukemia Outcomes According to Baseline Risk and First‐Line Treatment in Real‐World.” Mario Tiribelli received consulting fees from Novartis; and received fees for professional activities from Bristol‐Myers Squibb and Incyte Corporation. The others declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Cumulative incidence of time to molecular response (≥MR3) (A) since diagnosis of chronic myeloid leukemia (CML), estimated with the method by Gooley, considering deaths from any causes as competing events. Cumulative incidence of time to CML progression (B) and to CML‐related deaths (C) since diagnosis of CML, estimated with the method by Gooley, considering the other causes of deaths as competing events.
FIGURE 2
FIGURE 2
Description of the duration of the chronic and blastic phases in the 22 patients deceased from chronic myeloid leukemia, according to first‐line treatment, EUTOS long‐term survival (ELTS) risk, and age at diagnosis. Looking at ELTS risk, in the 2G‐TKI group, seven were high risk (HR), five were intermediate risk (IR), and one was low risk (LR); in the IMA group, four were HR, three were IR, and two were LR.
See this image and copyright information in PMC

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