Taliglucerase alfa is an enzyme replacement therapy (ERT) approved for treatment of adult and paediatric patients with Type 1 Gaucher disease (GD) in several countries and the first plant cell–expressed recombinant therapeutic protein approved by the US Food and Drug Administration for humans. Here, we review the findings across six key taliglucerase alfa clinical studies. A total of 33 treatment-naïve adult patients were randomized to taliglucerase alfa 30 U/kg or 60 U/kg in a 9-month, multicentre, randomized, double-blind, parallel-group, dose-comparison pivotal study, after which eligible patients continued into two consecutive extension studies; 17 treatment-naïve adult patients completed 5 total years of treatment with taliglucerase alfa. In the only ERT study focused on exclusively paediatric patients with GD, 11 treatment-naïve children were randomized to taliglucerase alfa 30 U/kg or 60 U/kg in a 12-month, multicentre, double-blind study; nine completed 3 total years of treatment in a dedicated paediatric extension study. The effect of switching patients from imiglucerase to taliglucerase alfa was also investigated in a separate 9-month study that included 26 adults and five children; 10 adults completed a total of 3 years and two children completed a total of 2.75 years of taliglucerase alfa treatment in the extension studies. All studies evaluated safety and spleen volume, liver volume, platelet count, haemoglobin concentration, and biomarkers as measures of efficacy. Detailed results from baseline through the end of these studies are presented. Taliglucerase alfa was well tolerated, and adverse events were generally mild/moderate in severity and transient. Treatment with taliglucerase alfa resulted in improvements (treatment-naïve patients) or stability (patients switched from imiglucerase) in visceral, haematologic, and biomarker parameters. Together, this comprehensive data set supports the treatment of adult and paediatric patients with GD who are naïve to ERT or who have previously been treated with imiglucerase.
Background Limited real-world data from routine clinical care are available on the safety and effectiveness of treatment with taliglucerase alfa in patients with Gaucher disease (GD). Methods Taliglucerase Alfa Surveillance (TALIAS), a multinational prospective Drug Registry of patients with GD, was established to evaluate the long-term safety (primary objective) and effectiveness (secondary objective) of taliglucerase alfa. We present an interim analysis of the data from the Drug Registry collected over the 5-year period from September 2013 to January 2019. Results A total of 106 patients with GD (15.1% children aged < 18 years; 53.8% females) treated with taliglucerase alfa have been enrolled in the Drug Registry, as of January 7, 2019. The median duration of follow-up was 795 days with quartiles (Q1, Q3) of 567 and 994 days. Fifty-three patients (50.0%) were from Israel, 28 (26.4%) were from the United States, and 25 (23.6%) were from Albania. At the time of enrollment, most patients (87.7%) had received prior enzyme replacement therapy (ERT). Thirty-nine of the 106 patients had treatment-emergent adverse events (AEs). Twelve of the 106 patients experienced serious AEs; two patients experienced four treatment-related serious AEs. Four patients died, although none of the deaths was considered to be related to taliglucerase alfa treatment by the treating physicians. Nine patients discontinued from the study, including the four who died. At baseline, patients with prior ERT had a higher mean hemoglobin concentration and platelet counts than treatment-naïve patients, likely reflecting the therapeutic effects of prior treatments. During follow-up, the hemoglobin concentration and platelet counts increased in the treatment-naïve patients and remained relatively constant or increased slightly in patients with prior ERT. Spleen and liver volumes decreased in treatment-naïve patients. Conclusions The interim data showed no new or emergent safety signals. The overall interim data are consistent with the clinical program experience and known safety and effectiveness profile of taliglucerase alfa.
Eur J Hosp Pharm 2013;20(Suppl 1):A1-238 A167 ConclusionsIn our study, ustekinumab demonstrated a rapid onset of action and a high effectiveness, stable safety and a great improvement in the quality of life in patients with moderate to severe plaque psoriasis on up to 34 months of continuous therapy.Abstract CPC-005 Background Natalizumab is a monoclonal antibody authorised as second-line treatment after failure with interferon beta or in rapidly evolving severe relapsing-remitting multiple sclerosis (RRMS). Due to its high cost and safety profile, the appropriate selection of patients who will benefit most is of paramount importance. Purpose To evaluate the adequacy of criteria for starting treatment with natalizumab in patients with multiple sclerosis (MS) based on the protocol approved in a tertiary hospital. Materials and Methods Observational, retrospective analysis of patients treated with natalizumab between 2008 and 2011. Study data were obtained from clinical records.Results 31 patients were treated with natalizumab, 26 women (83.9%) and 5 men (16.1%). Mean age was 38.8 years (SD = 9.1).Mean time between diagnosis and natalizumab start was 7.8 years (SD = 5.9). 29 patients (93.5%) had RRMS, 1 secondary-progressive MS (SPMS) and the other an intermediate disease between RRMS and SPMS. The mean number of relapses before treatment started was 3.7 (SD = 1.5) and the mean score for the expanded disability status scale was 3.3 (range 1-6). 27 patients (87.1%) had previously been treated with immunomodulatory drugs (interferon beta). In 4 patients (12.9%) natalizumab was first line treatment. All were diagnosed with rapidly evolving severe RRMS with gadoliniumenhancing lesions in brain magnetic resonance imaging and more than 2 disabling relapses in the previous year. At the end of the study 22 patients continued treatment and 9 had finished. These latter patients were categorised in two groups: short treatment duration (4 patients, median 5 months) and long treatment duration (5 patients, median 24 months). Conclusions In our population, adequacy of criteria for starting treatment with natalizumab is appropriate and the drug was used for the authorised indications in more than 90% of patients.
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