Background Chronic Myeloid Leukemia (CML) accounts for 10-15% of adult leukemias by incidence, with a rising prevalence of ~3/10000 and 2300 patients (pts) in Lombardy, Italy's largest region. Tyrosine kinase inhibitors have transformed CML into a chronic condition, with life expectancy comparable to the general population. It is, however, unclear whether the quality of life (QoL) of CML patients also approaches that of people without CML.

The organizational model of CML care may influence QoL and patient experience.

Aims The primary aim of the CML-OPERAP study (Outcome and PErformances Reported and Analyzed by Patients) is to assess the impact of hematology center organizational models on the QoL of CML patients.

Secondary aims include:

- Mapping and analyzing care models for CML across the Lombardy Hematologic Network (REL)

- Assessing the impact of CML and its treatment on daily life, symptom burden, emotional well-being, and social functioning

Methods This is an 18-month, cross-sectional, multicenter, prospective, observational study involving 22 REL centers.

Eligible pts are adults (≥18 years) with confirmed CML, followed for ≥6 months in a REL outpatient clinic, with sufficient knowledge of the Italian language and able to complete a 70-item questionnaire.

Data collection includes:

- Clinic organizational data

- Clinical and demographic data

- Patient-reported questionnaires

Administered questionnaires:

- EORTC QLQ-C30: general cancer pts QoL survey

- EORTC QLQ-CML24: CML-specific QoL survey

- Custom CML Hematology Center Questionnaire (CCHCQ) evaluating pts' perception of their center

Centers are classified into 4 categories:

- Large centers (>100 CML pts) with/without a dedicated clinic for CML (categories 1 and 2)

- Small centers (<100 CML pts) with/without dedicated time slots for CML (categories 3 and 4)

Questionnaires are anonymized, distributed during the visits, and returned by mail using prepaid envelopes.

Significance was assessed using Mann-Whitney U and one-sample t-tests.

Results The study opened in April 2025 and is ongoing: as of Jul 30, 2025, 350 pts have been enrolled (out of 2000 eligible) and 14 centers activated, with 221 questionnaires received.

Within REL, 3 of 6 large centers and 5 of 16 small centers have a dedicated organizational structure for CML pts.

QoL indicators were favorable across all domains. Functional scales scores were consistently high, indicating good functioning, whereas symptom scores and overall symptom burden were generally low. The only symptoms with a median score > 0 (indicating their presence) were fatigue and insomnia; insomnia was especially common in women over 45.

Both Global Health Status (GHS) score (mean: 74.7 ±19.4) and Summary score (mean: 86.5 ±11.0) were good. Satisfaction with care and information received at the center were also high (mean: 82.8 ±25.2), supported by excellent CCHCQ results.

Pts in treatment-free remission (n=43) reported slightly better QoL than those on active treatment (n=178), with higher GHS (mean: 78.7 vs. 73.8; median 83.3 vs. 75, p=0.0498), Summary (mean: 89.7 vs. 85.7; median 91.8 vs. 89.7, p=0.013) and lower Fatigue (mean: 20.6 vs. 27.9; median 22.2 vs. 33.3, p=0.049) and Symptom burden (mean: 11.4 vs. 20.1; median: 11.1 vs. 17.9, p<0.001) scores.

Scores were compared to reference values from the general Italian population (Pilz et al., 2022).

GHS score was 76.7 ±20.1 in males (n=134) vs. 67.6 in the reference cohort (p<0.001), and 71.6 ±17.9 in females (n=87) vs. 62.4 (p<0.001). Summary score was 87.5 ±11.2 in males vs. 85.5 in the reference cohort (p=0.039) and 84.8 ±11.5 in females vs. 82.9 (p=0.09). Findings remained consistent when focusing only on pts receiving treatment.

The QoL of patients with CML was therefore found to be equivalent to or better than reference values from the general Italian population, with no relevant impairments across functional or symptom scales.

Comparison across categories was limited by the small number of pts enrolled so far in centers without dedicated organizational structures (n=35). However, results between Category 1 (n=143) and 3 (n=43) were nearly identical, with similar and excellent CCHCQ scores. Thus, the QoL of patients with CML managed in centers with a CML dedicated structure appears to be unaffected by the center's size.

Conclusions The results of the CML-OPERAP study show that patients with CML report QoL scores comparable to those of the general population. Further results will be presented at the meeting.

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2025
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