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Background: Anemia is frequently present in patients with myelofibrosis (MF), and it may be exacerbated by treatment with the JAK2‐inhibitor ruxolitinib (RUX). Recently, a relevant blast phase (BP) incidence has been reported in anemic MF patients unexposed to RUX. Methods: The authors investigated the incidence of BP in 886 RUX‐treated MF patients, included in the "RUX‐MF" retrospective study. Results: The BP incidence rate ratio (IRR) was 3.74 per 100 patient‐years (3.74 %p‐y). At therapy start, Common Terminology Criteria for Adverse Events grade 3‐4 anemia (hemoglobin [Hb] <8 g/dL) and severe sex/severity‐adjusted anemia (Hb <8/<9 g/dL in women/men) were present in 22.5% and 25% patients, respectively. IRR of BP was 2.34 in patients with no baseline anemia and reached respectively 4.22, 4.89, and 4.93 %p‐y in patients with grade 1, 2, and 3–4 anemia. Considering the sex/severity‐adjusted Hb thresholds, IRR of BP was 2.85, 4.97, and 4.89 %p‐y in patients with mild/no anemia, moderate, and severe anemia. Transfusion‐dependent patients had the highest IRR (5.03 %p‐y). Progression‐free survival at 5 years was 70%, 52%, 43%, and 27% in patients with no, grade 1, 2, and 3–4 anemia, respectively (p <.001). At 6 months, 260 of 289 patients with no baseline anemia were receiving ruxolitinib, and 9.2% had developed a grade 3–4 anemia. By 6‐month landmark analysis, BP‐free survival was significantly worse in patients acquiring grade 3–4 anemia (69.3% vs. 88.1% at 5 years, p <.001). Conclusions: This study highlights that anemia correlates with an increased risk of evolution into BP, both when present at baseline and when acquired during RUX monotherapy. Innovative anemia therapies and disease‐modifying agents are warranted in these patients. Anemia correlates with an increased risk of evolution into blast phase, both when present at baseline and when acquired during ruxolitinib monotherapy. Innovative anemia therapies and disease‐modifying agents are warranted in these patients. [ABSTRACT FROM AUTHOR]

Background: The authors assessed the clinical utility of patient‐reported symptom monitoring in the setting of newly diagnosed chronic myeloid leukemia (CML). The primary objective was to evaluate adherence to therapy. Methods: The authors conducted an international prospective study that included patients with newly diagnosed, chronic‐phase CML. Before clinical consultation, patients were provided a tablet computer to self‐rate their symptoms, and the results were available in real time to each physician during the patient's visit. Adherence was assessed by pill count and with a validated self‐reported questionnaire. The proportions of optimal responders at 3 and 6 months were assessed according to the European LeukemiaNet criteria. Results: Between July 2020 and August 2021, 94 patients with a median age of 57 years were enrolled. Pill count adherence analysis indicated that 86 of 93 evaluable patients (92.5%) took at least 90% of prescribed tyrosine kinase inhibitor therapy during the 6‐month observation period. The online platform was well accepted by patients and physicians. An optimal response was achieved by 69 of 79 patients (87.3%) at 3 months and by 61 of 81 patients (75.3%) at 6 months. Conclusions: Patient‐reported symptom monitoring from the beginning of therapy in patients with CML may be critical to improve adherence to therapy and early molecular response rates (ClinicalTrials.gov identifier NCT04384848). Optimal adherence to tyrosine kinase inhibitor therapy in patients with chronic myeloid leukemia is critical to attain and maintain an optimal clinical response. The current findings suggest that systematic monitoring of patient‐reported symptoms is associated with high adherence rates. [ABSTRACT FROM AUTHOR]

Background Methods Results Conclusions Ruxolitinib (RUX) is a JAK1/2 inhibitor approved for the therapy of myelofibrosis (MF) based on clinical trials including only intermediate2‐high risk (INT2/HIGH) patients. However, RUX is commonly used in intermediate‐1 (INT1) patients, with scarce information on responses and outcome.The authors investigated the benefit of RUX in 1055 MF patients, included in the “RUX‐MF” retrospective study.At baseline (BL), 595 (56.2%) patients were at INT1‐risk according to DIPSS (PMF) or MYSEC‐PM (SMF). The spleen was palpable at <5 cm, between 5 and 10 cm, and >10 cm below costal margin in 5.9%, 47.4%, and 39.7% of patients, respectively; 300 (54.1%) were highly symptomatic (total symptom score ≥20). High‐molecular‐risk (HMR) mutations (IDH1/2, ASXL‐1, SRSF2, EZH2, U2AF1Q157) were detected in 77/167 patients. A total of 101 (19.2%) patients had ≥1 cytopenia (Hb < 10 g/dL: n.36; PLT <100 x 109/L: n = 43; white blood cells <4 x 109/L: n = 40). After 6 months on RUX, IWG‐MRT–defined spleen and symptoms response rates were 26.8% and 67.9%, respectively. In univariate analysis, predictors of SR at 6 months were no HMR mutations odds ratio [OR], 2.0, p = .05], no cytopenia (OR, 2.10; p = .01), and blasts <1% (OR, 1.91; p = .01). In multivariate analysis, absence of HMR maintained a significant association (OR, 2.1 [1.12–3.76]; p = .01).In INT1 patients, responses were more frequent and durable, whereas toxicity rates were lower compared to INT2/high‐risk patients. Presence of HMR mutations, cytopenia, and peripheral blasts identified less‐responsive INT1 patients, who may benefit for alternative therapeutic strategies. [ABSTRACT FROM AUTHOR]