Publications by authors named "Daniela Cilloni"
Background: The response to ruxolitinib after 6 months (RR6) model allows early identification of ruxolitinib-treated myelofibrosis (MF) patients with poorer overall survival (OS); however, it is less applicable to lower-risk patients.
Methods: To further explore this, the authors performed a subanalysis of the "RUX-MF" study (NCT06516406) with an aim to validate the RR6 and to develop a score specific for intermediate-1 DIPSS/MYSEC-PM risk patients.
Results: Among the 776 evaluable patients, 34.
Background/objectives: The aim of this study was to assess the unmet needs of myelodysplastic neoplasm (MDS) patients and their caregivers, focusing on how these needs impact quality of life (QoL) and daily functioning. MDS predominantly affects older adults. It is often complicated by severe red blood cell transfusion-dependent anemia and may require frequent hospital visits, conferring a substantial burden on patients and caregivers.
View Article and Find Full Text PDFUpfront intensive treatment analysis of the Italian Cohort Study on FLT3-mutated AML patients (FLAM): The impact of a FLT3 inhibitor addition to standard chemotherapy in the real-life setting.
Cancer
April 2025
Background: The addition of a FLT3 inhibitor (FLT3i) to standard chemotherapy to treat fit newly diagnosed (ND) patients with FLT3-mutated acute myeloid leukemia (AML) represents the standard of care resulting from clinical trial results. However, evidence regarding FLT3i adoption in routine clinical practice is still scarce.
Methods: Clinical data are reported from 394 ND patients with FLT3-mutated AML enrolled in the retrospective observational Italian Cohort Study on FLT3-mutated patients with AML and treated with an upfront intensive regimen with (FLT3i group, n = 92) or without (CT group, n = 302) the addition of a FLT3i.
Disease Phenotype Significantly Influences the Outcome After Discontinuation of Ruxolitinib in Chronic Phase Myelofibrosis.
Clin Lymphoma Myeloma Leuk
July 2025
Introduction: In patients with myelofibrosis (MF), overall survival (OS) after ruxolitinib discontinuation is poor, with leukemic transformation, clonal evolution and thrombocytopenia as the main factors worsening prognosis.
Patients And Methods: To assess the impact of disease phenotype on outcome after ruxolitinib discontinuation in chronic phase patients, we performed a sub-analysis of the "RUX-MF" study (NCT06516406), which now includes 1055 MF patients who received ruxolitinib in a real-life context.
Results: After a median follow-up of 3.
: Treatment with tyrosine kinase inhibitors (TKIs) in chronic myeloid leukemia (CML) has revolutionized disease management and has transformed CML from a life-threatening disease to a chronic condition for many patients. However, overcoming resistance, particularly related to leukemic stem cells (LSC) that can persist even when the bulk of the leukemic cells are eliminated, remains a significant challenge. : K562 and KU812 cell lines were treated in vitro with the TKI Imatinib (IM).
View Article and Find Full Text PDFCalreticulin (CALR) mutations are detected in around 20% of patients with primary and post-essential thrombocythemia myelofibrosis (MF). Regardless of driver mutations, patients with splenomegaly and symptoms are generally treated with JAK2-inhibitors, most commonly ruxolitinib. Recently, new therapies specifically targeting the CALR mutant clone have entered clinical investigation.
View Article and Find Full Text PDFExpectation of survival of patients receiving HMA + VEN is influenced by pre-treatment comorbidity burden.
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- * The most common mutation is p.D816V, but there are other less frequent mutations that are typically undetectable with standard testing methods.
- * New, more sensitive detection methods are being developed, and the review discusses how to approach these tests for diagnosing and monitoring mastocytosis patients.
Deep Molecular Response Rate in Chronic Phase Chronic Myeloid Leukemia: Eligibility to Discontinuation Related to Time to Response and Different Frontline TKI in the Experience of the Gimema Labnet CML National Network.
Clin Lymphoma Myeloma Leuk
January 2025
Article Synopsis
- In the past decade, targeted kidney inhibitors (TKIs) have significantly improved the survival rates of chronic myeloid leukemia (CML) patients, particularly those achieving deep and sustained molecular response (DMR), allowing some to consider stopping treatment.
- A study analyzed responses from 1,777 CML patients in Italy, focusing on the impact of different TKIs (imatinib, dasatinib, nilotinib) and timing of responses (3, 6, and 12 months).
- Results showed that patients achieving certain molecular response benchmarks at 3 months were more likely to achieve DMR later, with significant differences in outcomes based on the specific TKI used.
Protein degradation is a biological phenomenon essential for cellular homeostasis and survival. Selective protein degradation is performed by the ubiquitination system which selectively targets proteins that need to be eliminated and leads them to proteasome degradation. In this narrative review, we focus on the ubiquitin-conjugating enzyme E2 O (UBE2O) and highlight the role of UBE2O in many biological and physiological processes.
View Article and Find Full Text PDFCerebral venous thrombosis (CVT) is a rare type of cerebrovascular event in which the thrombosis occurs in a vein of the cerebral venous system. The diagnosis could be challenging due to the great clinical variability, but the outcome is favourable in most cases, especially in the case of early diagnosis. Anticoagulant therapy is the core of CVT management and currently consists of heparin in the acute phase followed by vitamin K antagonists (VKAs) in the long term.
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- Ruxolitinib (RUX) is a treatment for myelofibrosis, mainly studied in high-risk patients, but is often given to intermediate-1 patients with limited data on its effects.
- In a study of 1,055 myelofibrosis patients, over half were classified as intermediate-1 risk, with notable symptoms and some having high-molecular-risk mutations.
- The study found that after 6 months of RUX treatment, a significant proportion of patients experienced improvements in spleen size and symptoms, with certain factors like the absence of high-molecular-risk mutations being linked to better treatment responses.
Article Synopsis
- Lymphoproliferative disorders, including lymphoma, can recur even years after treatment, so patients in remission need regular check-ups.
- There's debate over the use of imaging for these follow-ups, as traditional methods expose patients to harmful ionizing radiation, while whole-body MRI with diffusion-weighted imaging (WB-MRI-DWI) offers a radiation-free alternative.
- A study reviewing cases from 2010 to 2022 found WB-MRI-DWI highly effective for follow-ups, achieving 100% sensitivity and 98.6% specificity in detecting potential lymphoma relapses, although some false positives were noted.
Article Synopsis
- Acute myeloid leukemia (AML) is a severe blood cancer, with around 30% of cases linked to mutations in the NPM1 gene, leading to a specific classification of NPM1-mutated AML that generally has a favorable prognosis but still faces a high relapse rate of 30-50%.
- This study explored the use of total RNA sequencing (RNAseq) to better characterize NPM1-mutated AML, revealing complex molecular variations and different clonal types that previous methods might have missed, such as abnormal fusion transcripts.
- The findings suggest that advanced technologies like RNAseq could improve risk assessment and treatment planning for patients with NPM1-mutated AML, laying the groundwork for
Article Synopsis
- SETBP1 mutations are associated with various clonal myeloid disorders, but their role in initiating leukemia is uncertain, as they usually occur later in the progression of the disease.
- Researchers created a mouse model with SETBP1 mutations in blood-forming tissue, which resulted in significant changes in cell differentiation and the development of a serious myeloid neoplasm.
- In a study of triple-negative primary myelofibrosis patients, two groups were identified—those with SETBP1 mutations, who experienced more aggressive disease, and those without mutations, suggesting that SETBP1 mutations may act earlier in some clonal disorders than previously thought.
Article Synopsis
- Anemia is common in patients with myelofibrosis (MF) and is worsened by the treatment drug ruxolitinib (RUX), with new cases of blast phase (BP) emerging in anemic patients not previously on this treatment.
- A study of 886 MF patients treated with RUX found a BP incidence rate of 3.74 per 100 patient-years, with higher rates in patients who had varying levels of anemia; the most severe cases were in those dependent on transfusions.
- The findings suggest that both pre-existing and treatment-induced anemia significantly increase the risk of BP development, indicating a need for better anemia treatments alongside MF therapies.
Article Synopsis
- A significant number of myelofibrosis patients stop using ruxolitinib within the first 5 years due to treatment failure, highlighting the need for early identification of those at risk.* -
- A study analyzed data from 889 patients and found that factors like low platelet count, low hemoglobin, and certain disease types increase the likelihood of stopping ruxolitinib treatment early.* -
- A new prognostic model called STR-PM was developed to categorize patients into low, intermediate, and high-risk groups for early treatment failure, suggesting that those in higher risk categories might benefit from alternative treatments.*
In the era of personalized medicine greatly improved by molecular diagnosis and tailor-made therapies, the survival rate of acute myeloid leukemia (AML) at 5 years remains unfortunately low. Indeed, the high heterogeneity of AML clones with distinct metabolic and molecular profiles allows them to survive the chemotherapy-induced changes, thus leading to resistance, clonal evolution, and relapse. Moreover, leukemic stem cells (LSCs), the quiescent reservoir of residual disease, can persist for a long time and activate the recurrence of disease, supported by significant metabolic differences compared to AML blasts.
View Article and Find Full Text PDFUnmet needs remain in later lines chronic myeloid leukemia (CML): the response rate and the overall survival of resistant patients in the chronic phase who changed a second-generation TKI in the second line with another TKI with similar action are usually poor, while the off-target toxicities and the potential development of mutations increase. The recent approval of asciminib, a STAMP inhibitor, in the third line, has the potential to soon change the therapeutic algorithm for this subset of patients. Here, we report the results of a GIMEMA survey assessing the number of patients currently treated in the third line in Italy, the current approach in later lines by Italian physicians, and the future role of this drug according to the reason to switch to asciminib (resistance and/or intolerance), as well as the perceptions about the future position of this agent.
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- Patients with cytopenic myelofibrosis (MF) face more challenges in treatment options and prognosis compared to those with a myeloproliferative phenotype, with common definitions for cytopenia including low leukocyte, hemoglobin, or platelet counts.
- In a study of 886 patients treated with ruxolitinib, 45.9% had cytopenic MF and factors like high molecular risk mutations and high prognostic scores were linked to this phenotype.
- Patients with cytopenia received lower doses of ruxolitinib and had poorer response rates for symptoms and spleen size, along with a higher likelihood of treatment discontinuation over five years compared to those
Omidubicel is an umbilical cord blood (UCB)-derived ex vivo-expanded cellular therapy product that has demonstrated faster engraftment and fewer infections compared with unmanipulated UCB in allogeneic hematopoietic cell transplantation. Although the early benefits of omidubicel have been established, long-term outcomes remain unknown. We report on a planned pooled analysis of 5 multicenter clinical trials including 105 patients with hematologic malignancies or sickle cell hemoglobinopathy who underwent omidubicel transplantation at 26 academic transplantation centers worldwide.
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