Oral pulsed therapy of relapsing multiple sclerosis with cladribine tablets - expert opinion on issues in clinical practice.

BACKGROUND: Oral cladribine is the first oral pulsed therapy licensed for relapsing multiple sclerosis (RMS). Three years after the introduction into the European market, we evaluated practical aspects in the use of cladribine tablets, incorporating the experience gained in routine clinical practice and real-world studies. METHODS: Based on a structured review process, a panel of nine neurologists experienced in MS therapy discussed salient statements regarding the use of cladribine tables. For each statement the level of evidence was determined according to the levels of evidence recommended by the Centre for Evidence-Based Medicine, Oxford. The strength of each expert statement was then evaluated by means of a linear scale from 1 (very strong rejection) to 9 (very strong approval). Votes were collected by a formalized blinded process. Consent was considered to be reached if at least 75% of the experts agreed on a particular statement (i.e. voted for 7-9 points on the linear scale). RESULTS: . Statements include efficacy in early RMS, risk of side effects and infections, vaccination, pregnancy, and monitoring requirements. CONCLUSION: The consented recommendations summarize the practical experience inthe use of cladribine tablets in a real-world setting. These may provide guidance for unanswered questions arising with the introduction of new treatments such as cladribine tablets.

Prevention of episodic migraines with topiramate: results from a non-interventional study in a general practice setting.

The majority of patients with migraine headaches are treated in non-specialized institutions though data on treatment outcomes are largely derived from tertiary care centers. The current non-interventional study explores efficacy and tolerability outcomes of patients with episodic migraines receiving topiramate as preventive agent in a general practice setting. A total of 366 patients (87% female, mean age 41.8 +/- 11.6 years) were eligible for migraine prevention and treated with flexible dose topiramate for 6 months (core phase), and optionally for a total of 12 months (follow-up phase). Overall, 261 patients (77.7% of safety analysis set, SAF) completed the core phase. Reasons for discontinuation included adverse events (2.1%), lost to follow-up (1.8%), other reasons (1.5%), and end of therapy (0.3%) though in the majority of patients who discontinued no reasons were listed. The median daily dose at endpoint was 50 mg/day (range, 25-187.5 mg/day). The median days with migraine headaches decreased from 6.0 to 1.2 days (p < 0.001), median pain intensity score decreased from 17.0 to 3.2 points (p < 0.001). In women with reported menstruation-associated migraine, the median number of migraine attacks decreased from 4.0 to 0.9 (p < 0.001). Absenteeism as well as triptan use decreased significantly, and significant improvements in activities of daily living and quality of life were reported. The most frequently reported AEs were paraesthesia (4.2%) and nausea (3%). Results suggest that migraine prevention with topiramate in a general practice is generally well tolerated and associated with a significant improvement in migraine headaches and related functional impairment.

Patient satisfaction and healthcare services in specialized multiple sclerosis centres in Germany.

BACKGROUND: As patients with multiple sclerosis (MS) require lifelong treatment, optimization of therapy with respect to efficacy and safety is needed to limit long-term disease progression. Patients with MS also need a range of health-related services. Satisfaction with these as well as treatment is clinically relevant because satisfied patients are more likely to adhere to therapy. The aim of this study was to determine the status of patient satisfaction and of healthcare services in 70 specialized MS centres in Germany. METHODS: In 2011, patients with MS responded to a questionnaire, which solicited clinical and demographic information, as well as patients' perceptions of their overall situation and their satisfaction with treatment. RESULTS: Of 2791 patients surveyed, 81.9% had relapsing-remitting MS with mild disability [mean (standard deviation) Expanded Disability Status Scale score: 2.6 (1.8)]. Disease activity data were collected from 2205 patients, of whom 57.6% had remained relapse-free during the preceding 12 months. However, 38.9% had experienced one or more relapses, most of whom (67.3%) while receiving immunomodulatory treatment. About one-third of the patients indicated that they were more dissatisfied with their overall situation compared with the time before diagnosis. However, many patients (58.3%) were satisfied with their existing medication. Overall, 72.8% of patients would prefer oral to injectable treatments, assuming there was no difference in their efficacy. CONCLUSIONS: A substantial proportion of patients experienced breakthrough disease on treatment and may potentially benefit from a change of therapy. Although largely satisfied with treatment, most patients with MS would choose oral over injectable treatments.

Multiple sclerosis patients treated with intramuscular IFN-ß-1a autoinjector in a real-world setting: prospective evaluation of treatment persistence, adherence, quality of life and satisfaction.

OBJECTIVES: The 12-month observational PERSIST study (NCT01405872) evaluated adherence associated with the intramuscular IFNß-1a (i.m. IFN-ß-1a) autoinjector pen in multiple sclerosis (MS) patients. METHODS: MS patients initiating i.m. IFN-ß-1a autoinjector treatment were prospectively assessed for physician-reported persistence (percentage of patients remaining on therapy) and patient-reported outcomes, including adherence (percentage of unmissed injections), compliance (percentage of patients missing no injections), tolerability (injection-site reactions [ISRs] and pain) and satisfaction. RESULTS: The intent-to-treat population included 232 patients; of the 188 physician-reported 12-month completers, 182 patients remained on treatment (96.8% persistence). Monthly compliance rates were 87.5 - 96.2%. Mean monthly pain scores were 1.5 - 1.8 (scale: 0 = 'no pain'; 10 = 'extremely painful'). At 12 months, 73.5% of respondents reported no ISRs, 94.9% were satisfied/very satisfied with the autoinjector and 88.2% found using the device easy/very easy. Injection fear, injection anxiety and need for injection assistance by caregivers decreased from the initial visit to 12 months. No new safety signals were observed. CONCLUSIONS: The autoinjector pen is associated with high levels of persistence, compliance, adherence, and satisfaction, little-to-no pain and low need for caregiver assistance. Although these data are limited by reliance on patient questionnaires and the absence of a direct comparator group, this treatment may reduce barriers to injection therapy, while supporting long-term MS management.