Health Care Utilization Databases obtained from health system inform outcome for ruxolitinib treatment in patients with myelofibrosis Barbara Mora, Matteo Franchi, Ludovica Margotto, Olivia Leoni, Daniela D'ippoliti, et al. Hemasphere, 2026 Ruxolitinib (RUX) is a JAK1/2 inhibitor widely used in patients with myelofibrosis (MF). Here, we provided real‐world data on 652 intermediate‐2 and high‐risk MF patients receiving RUX, by analyzing electronic Health Care Utilization Databases (HCUD) of all individuals that started RUX in three Italian regions (Lombardy, Lazio, and Tuscany) between October 2014 and December 2017. Over 9 years of observation, the median follow‐up of the cohort was 36.8 months. HCUD of this cohort provided relevant information, (1) contemporary rate of patients on RUX receiving stem cell transplantation: 10.9%; (2) median time to RUX discontinuation: 31.2 months (95% confidence interval [CI]: 26.4–36); (3) transfusions need in the first 6 months of RUX: no red blood cell (RBC) units in 408 (69%), 1–5 in 172 (29%), ≥6 in 14 (2%); (4) events' incidence rate (×100 person‐years) that led to hospital admission: 10.3 for infections, 5.47 for solid tumors, 3.47 for bleeding, 1.56 for thrombosis, and 5.22 for accelerated/blast phase; (5) RUX individual average cost rate in Italy: 30,675 €/year, increasing with worsening Multisource Comorbidity Score (MCS). Finally, the median survival was 48 months (95% CI: 43.2–51.6). In a multivariable Cox model, together with patient‐related factors, starting RUX doses below 20 mg every 12 h (BID) were associated with increased mortality (P from 0.007 to <0.001). We report a novel HCUD‐based approach to provide critical healthcare information in the field of MF, based on large populations of patients with documented follow‐up.
Genomic profiling for decision-making in post–polycythemia vera and post–essential thrombocythemia myelofibrosis Barbara Mora, Francesca Palandri, Paola Guglielmelli, Andrew T. Kuykendall, Margherita Maffioli, et al. Blood, 2026 Secondary myelofibrosis (SMF) represents a late stage of polycythemia vera and essential thrombocythemia, with overall survival (OS) currently defined by the MYelofibrosis SECondary to PV and ET (MYSEC) Prognostic Model (MYSEC-PM). To identify additional myeloid neoplasm-associated cancer gene variants (CGVs) associated with SMF outcome, we evaluated next-generation sequencing panel testing in 644 patients within the MYSEC cohort. Overall, 429 (66.6%) subjects reported at least one CGV, with ASXL1, TET2 and DNMT3A being the most frequently involved. Specific molecular profiles affected OS (p < .001): U2AF1, TP53 or SRSF2 variants (UTS, 9.3% of cases, median OS 4.1 years) and ASXL1 without UTS (25.3%, median OS 8.4 years). By integrating these genetic signatures within the MYSEC-PM through penalized Cox regressions, we identified the following independent predictors (p from < .0001 to .02) and weighted: hemoglobin <11 g/dl (1 point), circulating blasts ⩾3% (2), platelets <150 × 109/l (2), age (0.21 points/year), ASXL1 without UTS mutations (1) and any UTS mutations (3). Finally, we developed the MYSEC-molecular prognostic model (MYSEC-mPM) allocating 582 SMF patients into four categories with different OS (p < .001): low (median OS 18.0 years, 95%CI: 14.2-not reached; score <14), intermediate-1 (8.8. years, 95%CI: 7.7-9.7; score 14-16), intermediate-2 (4.6 years, 95%CI: 3.1-7.2; score 17-18), and high risk (1.9 years, 95%CI: 1.2-2.5; score ⩾19). Additionally, in 381 SMF with available cytogenetics, the MYSEC-mPM was implemented with complex/monosomal karyotype, generating the karyotype-enhanced MYSEC-kmPM. Our study shows that genomic and cytogenetic profiling improve survival prediction in SMF, outperforming the MYSEC-PM.
CALR-mutated myeloproliferative neoplasms Valentina Bellani, Barbara Mora, Alessandra Iurlo, Francesco Passamonti Leukemia and Lymphoma, 2025 Calreticulin (CALR) is a chaperone protein that plays a crucial role in protein folding quality control and calcium homeostasis. Mutations in CALR result in a mutated protein lacking key calcium-binding sites and the KDEL sequence, leading to a constitutive activation of the MPL-JAK2-STAT5 pathway, which is involved in the pathogenesis of essential thrombocythemia (ET) and primary myelofibrosis (PMF). Despite advancements in understanding the role of CALR mutations, current therapeutic strategies remain focused on managing symptoms and complications, with allogeneic stem cell transplantation (alloHSCT) as the only curative option. Emerging research is exploring novel immunotherapeutic approaches targeting mutant CALR, including the development of anti-CALR antibodies and T-cell receptor-mediated therapies, offering potential new avenues for treatment in CALR-mutated MPNs. In this review, we will discuss on the role of CALR in MPNs, focusing on its biological structure and its implications on the prognosis and treatment of essential thrombocythemia and primary myelofibrosis.
Validity and Reliability of the Self-Care in MyeloProliferative Neoplasms Inventory (SC-MPNI) Valentina Biagioli, Alessandro Inzoli, Antonella Barone, Alessandra Iurlo, Paola Guglielmelli, et al. European Journal of Cancer Care, 2025 ObjectiveThis study aimed to develop and psychometrically test a self‐report questionnaire for measuring self‐care behaviors in patients with myeloproliferative neoplasms (MPNs): the Self‐Care in MyeloProliferative Neoplasms Inventory (SC‐MPNI).MethodsA cross‐sectional validation study was conducted in 9 Italian hematology centers from November 2021 to January 2024. Adult patients with myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) were asked to complete a paper‐and‐pencil questionnaire during their outpatient visit or at home. The SC‐MPNI was developed according to the Middle‐Range Theory of Self‐Care of Chronic Illness. This 30‐item questionnaire includes three scales: self‐care maintenance, self‐care monitoring, and self‐care management. The construct validity was tested using confirmatory factor analysis (CFA). The reliability of each scale was evaluated using McDonald’s Omega composite reliability.ResultsOverall, 285 patients with MPNs (53% male; mean age = 60 years ± 13) were included. They were diagnosed with MF (43%), PV (29%), or ET (28%). The self‐care maintenance scale (13 items and 3 factors: adherence, healthy lifestyle, and prevention) fit well when tested with a three‐factor model, and its reliability was 0.87. The self‐care monitoring scale (9 items and 2 factors: symptom monitoring and parameter and test monitoring) fit well when tested with a two‐factor model, and its reliability was 0.85. The self‐care management scale (8 items and 2 factors: provider‐directed behaviors and spontaneous behaviors) fit well when tested with a two‐factor model, and its reliability was 0.79.ConclusionThe SC‐MPNI is a valid and reliable self‐report instrument to measure self‐care behaviors in people living with MPNs.
Using real-world evidence in haematology Francesco Passamonti, Giovanni Corrao, Gastone Castellani, Barbara Mora, Giulia Maggioni, et al. Best Practice and Research Clinical Haematology, 2024
Prognostication in myelofi-brosis Francesco Passamonti, Domenica Caramazza, Barbara Mora, Margherita Maffioli Novel Insights into Myelofibrosis Pathophysiology and Treatment, 2015
