1.Ocular Involvement Occurs Frequently at All Stages of Amyotrophic Lateral Sclerosis: Preliminary Experience in a Large Italian Cohort
Federica COZZA ; Andrea LIZIO ; Lucia Catherine GRECO ; Stefania BONA ; Giordana DONVITO ; Elena CARRARO ; Silvia TAVAZZI ; Nicola TICOZZI ; Barbara POLETTI ; Valeria Ada SANSONE ; Christian LUNETTA
Journal of Clinical Neurology 2021;17(1):96-105
Background:
and Purpose The study aimed to obtain optometric findings of amyotrophic lateral sclerosis (ALS) patients in different stages of the disease, and to determine the relation between ocular data and ALS-related features; that is, functional and cognitive impairment and staging.
Methods:
The optometric protocol included tests of the ocular motility [broad-H test and Northeastern State University College of Optometry (NSUCO) test], near point of convergence (NPC), error refraction, best-corrected visual acuity, and binocular visual alignment, and an ocular symptoms questionnaire. The functional measures included the Amyotrophic Lateral Sclerosis Functional Rating Scale–revised (ALSFRS-r) and Milano-Torino staging (MiToS), and cognitive impairment was assessed using the Edinburgh Cognitive and Behavioural ALS Screen (ECAS). Demographic and clinical features were also collected, including whether the patients used an eye-tracking communication device (ETCD).
Results:
Two-hundred consecutive ALS patients (median age of 64 years, 118 males and 82 females) in different stages of disease were recruited. Nearly 70% of patients reported at least one ocular symptom, and the use of an ETCD was found to be significantly related to the presence of most symptoms. Moreover, the severely symptomatic group was characterized by significantly lower ALSFRS-r total and subscale scores, and higher MiToS. Abnormal NPC values were significantly related to lower ALSFRS-r total and bulbar-subscale scores. Patients with acceptable NSUCO test values exhibited significantly higher ECAS scores.
Conclusions
The presence of ocular alteration in patients in different stages of ALS supports the idea that this is a multisystem disorder and emphasizes the importance of optometric evaluations in multidisciplinary assessments to address ocular impairment early in the disease process.
2.Use of generic imatinib as first-line treatment in patients with chronic myeloid leukemia (CML): the GIMS (Glivec to Imatinib Switch) study
Maria GEMELLI ; Elena Maria ELLI ; Chiara ELENA ; Alessandra IURLO ; Tamara INTERMESOLI ; Margherita MAFFIOLI ; Ester PUNGOLINO ; Maria Cristina CARRARO ; Mariella D’ADDA ; Francesca LUNGHI ; Michela ANGHILERI ; Nicola POLVERELLI ; Marianna ROSSI ; Mattia BACCIOCCHI ; Elisa BONO ; Cristina BUCELLI ; Francesco PASSAMONTI ; Laura ANTOLINI ; Carlo GAMBACORTI-PASSERINI
Blood Research 2020;55(3):139-145
Background:
Generic formulations of imatinib mesylate have been introduced in Western Europe since 2017 to treat patients with chronic myeloid leukemia (CML). However, results on the safety and efficacy of generic formulations are contrasting. The aim of this study was to investigate the safety and efficacy of generic imatinib in CML patients treated in 12 Italian institutes.
Methods:
This is an observational, retro-prospective analysis of patients with CML for whom the treatment was switched from brand to generic imatinib. We analyzed and compared the variation in quantitative PCR values before and after the switch, and the proportion of patients who maintained molecular response after changing from brand to generic imatinib. Adverse events (AEs) were also evaluated.
Results:
Two hundred patients were enrolled. The median PCR value after the switch was reduced by 0.25 compared to the values before the switch. A significant difference was found between median PCR values before and after the switch in favor of generic imatinib (P = 0 .0 0 3 ). Molecular responses remained stable in 69.0%, improved in 25.5%, and worsened in 5.5% of patients. AEs were similar in the pre- and post-switch periods; however, a significant difference was found in favor of generic imatinib for muscular cramps (P < 0.0001), periorbital edema (P =0.0028), edema of the limbs (P <0.0001), fatigue (P=0.0482), and diarrhea (P =0.0027).
Conclusion
Our data indicate that generic imatinib does not have deleterious effects on CML control and present an acceptable safety profile, similar or better than brand imatinib.