Gaucher disease prevalence in 600 patients affected by monoclonal gammopathy of undetermined significance.

Background: Gaucher disease (GD) is a rare autosomal recessive inherited disorder caused by the lysosomal enzyme acid β-glucosidase deficiency. Many patients experience a critical delay in the diagnosis of up to 8-10 years due to its rarity and variability in signs and symptoms, with the consultation of several specialists.

Patients And Methods: This prospective observational study analyzed the prevalence of GD in 600 patients with monoclonal gammopathy of uncertain significance (MGUS) from January 2018 until February 2022.

Efficacy and safety of tixagevimab-cilgavimab versus SARS-CoV-2 breakthrough infection in the hematological conditions.

Background: Managing SARS-CoV-2 infection in frail and immunosuppressed patients still represents an open challenge, but, starting from the phase 3 PROVENT study, prophylaxis with tixagevimab-cilgavimab has improved the approach in this category of patients, guaranteeing a better outcome and inferior mortality. Real-life data in a heterogeneous cohort are few.

Methods: The aim of this study is to evaluate the benefit of prophylaxis with tixagevimab-cilgavimab in a cohort of 202 patients affected by different hematological diseases (lymphoproliferative, myeloproliferative, autoimmune, patients recently receiving a bone marrow transplant), active (with ongoing treatment), or in watch-and-wait strategy, followed in our center, during a median follow-up of 249 (45-325) days.

AIPSS-MF machine learning prognostic score validation in a cohort of myelofibrosis patients treated with ruxolitinib.

Background: In myelofibrosis (MF), new model scores are continuously proposed to improve the ability to better identify patients with the worst outcomes. In this context, the Artificial Intelligence Prognostic Scoring System for Myelofibrosis (AIPSS-MF), and the Response to Ruxolitinib after 6 months (RR6) during the ruxolitinib (RUX) treatment, could play a pivotal role in stratifying these patients.

Aims: We aimed to validate AIPSS-MF in patients with MF who started RUX treatment, compared to the standard prognostic scores at the diagnosis and the RR6 scores after 6 months of treatment.

Severe Gastrointestinal Toxicity Following the Use of Gilteritinib: A Case Series and Analysis of Postmarketing Surveillance Data.

Gilteritinib has been approved as monotherapy in adults with acute myeloid leukemia (AML) FLT3 mutated with relapsed or refractory disease, in light of its advantages in terms of survival and the favorable safety profile. Hepatobiliary disorders and musculoskeletal and connective tissue disorders represent the most frequent adverse reactions associated with gilteritinib, whereas the most frequent serious adverse reaction is acute kidney injury. In the summary of product characteristics, gastrointestinal (GI) events are indicated as very common, in particular diarrhea, nausea and stypsis.

Myelofibrosis and Survival Prognostic Models: A Journey between Past and Future.

Among the myeloproliferative diseases, myelofibrosis is a widely heterogeneous entity characterized by a highly variable prognosis. In this context, several prognostic models have been proposed to categorize these patients appropriately. Identifying who deserves more invasive treatments, such as bone marrow transplantation, is a critical clinical need.

Prediction of Survival and Prognosis Migration from Gold-Standard Scores in Myelofibrosis Patients Treated with Ruxolitinib Applying the RR6 Prognostic Model in a Monocentric Real-Life Setting.

The wide use of ruxolitinib, approved for treating primary and secondary myelofibrosis (MF), has revolutionized the landscape of these diseases. This molecule can reduce spleen volume and constitutional symptoms, guaranteeing patients a better quality of life and survival or even a valid bridge to bone marrow transplantation. Despite a rapid response within the first 3 to 6 months of treatment, some patients fail to achieve a significant benefit or lose early response.