Publications by authors named "M Bocchia"
Background: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) patients are not responsive to standard rituximab in approximately 10% to 15% of cases, and oral immunosuppressants showed controversial results with significant toxicity. Targeting plasma cells with bortezomib appears promising, but the available evidence is scarce and stems only from isolated reports in the precaplacizumab era.
Objectives: To evaluate the safety and efficacy of bortezomib in rituximab-refractory iTTP patients.
Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) is a haemato-inflammatory syndrome genetically defined by somatic mutations in the X-linked UBA1 gene, typically Val/Thr/Leu substitutions at the Met41 hotspot. Clinical manifestations are heterogeneous and refractory to most haemato-rheumatological treatments. To date, no guidelines exist for the management of VEXAS, and scarce is the evidence on methodology and clinical significance of longitudinal UBA1 clonal burden evaluation upon therapy.
View Article and Find Full Text PDFUnraveling vulnerabilities in chronic lymphocytic leukemia (CLL) represents a key approach to understand molecular basis for its indolence and a path toward developing tailored therapeutic approaches. In this study, we found that CLL cells are particularly sensitive to the inhibitory action of abundant serum protein, apolipoprotein E (ApoE). Physiological concentrations of ApoE affect CLL cell viability and inhibit CD40-driven proliferation.
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- The study evaluated the use of frontline TKI therapy in elderly patients (75 years or older) with chronic phase chronic myeloid leukemia (CP-CML) among a large cohort of 332 patients.
- Results showed that 85.8% of patients received imatinib (IM), while 14.2% were treated with second-generation TKIs (2G-TKI) like dasatinib and nilotinib, with a notable percentage starting on reduced doses.
- The findings indicated increased usage of IM after generic versions became available in Italy, but significant discontinuation rates due to resistance and toxicities were observed, highlighting the need for personalized treatment assessments and further studies on lower TKI doses.
Article Synopsis
- 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.