Comparative effectiveness and cost-effectiveness of natalizumab and fingolimod in rapidly evolving severe relapsing-remitting multiple sclerosis in the United Kingdom.

AIM: To evaluate the real-world comparative effectiveness and the cost-effectiveness, from a UK National Health Service perspective, of natalizumab versus fingolimod in patients with rapidly evolving severe relapsing-remitting multiple sclerosis (RES-RRMS). METHODS: Real-world data from the MSBase Registry were obtained for patients with RES-RRMS who were previously either naive to disease-modifying therapies or had been treated with interferon-based therapies, glatiramer acetate, dimethyl fumarate, or teriflunomide (collectively known as BRACETD). Matched cohorts were selected by 3-way multinomial propensity score matching, and the annualized relapse rate (ARR) and 6-month-confirmed disability worsening (CDW6M) and improvement (CDI6M) were compared between treatment groups. Comparative effectiveness results were used in a cost-effectiveness model comparing natalizumab and fingolimod, using an established Markov structure over a lifetime horizon with health states based on the Expanded Disability Status Scale. Additional model data sources included the UK MS Survey 2015, published literature, and publicly available sources. RESULTS: In the comparative effectiveness analysis, we found a significantly lower ARR for patients starting natalizumab compared with fingolimod (rate ratio [RR] = 0.65; 95% confidence interval [CI], 0.57-0.73) or BRACETD (RR = 0.46; 95% CI, 0.42-0.53). Similarly, CDI6M was higher for patients starting natalizumab compared with fingolimod (hazard ratio [HR] = 1.25; 95% CI, 1.01-1.55) and BRACETD (HR = 1.46; 95% CI, 1.16-1.85). In patients starting fingolimod, we found a lower ARR (RR = 0.72; 95% CI, 0.65-0.80) compared with starting BRACETD, but no difference in CDI6M (HR = 1.17; 95% CI, 0.91-1.50). Differences in CDW6M were not found between the treatment groups. In the base-case cost-effectiveness analysis, natalizumab dominated fingolimod (0.302 higher quality-adjusted life-years [QALYs] and £17,141 lower predicted lifetime costs). Similar cost-effectiveness results were observed across sensitivity analyses. CONCLUSIONS: This MSBase Registry analysis suggests that natalizumab improves clinical outcomes when compared with fingolimod, which translates to higher QALYs and lower costs in UK patients with RES-RRMS.

There are several medications used to treat people with relapsing remitting multiple sclerosis, such as interferon-based therapies (Betaferon/Betaseron (US), Rebif, Avonex, Extavia), glatiramer acetate (Copaxone), teriflunomide (Aubagio), and dimethyl fumarate (Tecfidera), collectively named BRACETD. Other treatments for multiple sclerosis (MS) have a narrower use, such as natalizumab (Tysabri) or fingolimod (Gilenya), among others.This study objective was to assess how well natalizumab and fingolimod helped treating MS (clinical effectiveness) and subsequently estimate what the cost of these treatments is in comparison to the benefit they bring to people with rapidly evolving severe MS that use them in the United Kingdom (UK) (cost-effectiveness).We used an international disease registry (MSBase), which collects clinical data from people with MS in various centers around the world to compare the effectiveness of natalizumab, fingolimod and BRACETD treatments. We used a technique called propensity score matching to obtain results from comparable patient groups. People treated with natalizumab had better disease control, namely with fewer relapses and higher improvement on their disability level, than patients on fingolimod or BRACETD. Conversely, there were no differences between each group of people on a measure called disability worsening.Based on these clinical results, we built an economic model that simulates the lifetime costs and consequences of treating people with MS with natalizumab in comparison with fingolimod. We found that using natalizumab was less costly and was more effective compared to using fingolimod in UK patients.

Validation and comparison of Breast Graded Prognostic Assessment scores in patients with breast cancer and brain metastases.

PURPOSE: Brain metastases (BM) occur in 15-35% of patients with metastatic breast cancer, conferring poor prognosis and impairing quality of life. Clinical scores have been developed to classify patients according to their prognosis. We aimed to check the utility of the Breast Graded Prognostic Assessment (B-GPA) and its modified version (mB-GPA) and compare them in routine clinical practice. METHODS: This is an ambispective study including all patients with breast cancer BM treated in a single cancer comprehensive center. We analyzed the overall survival (OS) from BM diagnosis until death. The Kaplan-Meier method and Cox proportional hazard regression model were used in the analyses. ROC curves were performed to compare both scores. RESULTS: We included 169 patients; median age was 50 years. HER2-positive and triple negative patients were 33.7% and 20.7%, respectively. At the last follow-up, 90% of the patients had died. Median OS was 12 months (95% confidence interval 8.0-16.0 months). OS was worse in patients with > 3 BM and in patients with triple negative subtype. CONCLUSIONS: In our series, we confirm that B-GPA and mB-GPA scores correlated with prognosis. ROC curves showed that B-GPA and mB-GPA have similar prognostic capabilities, slightly in favor of mB-GPA.

[Diagnosis and assessment of therapeutic response of bone metastases by bone scintigraphy and PET study]. / Diagnóstico y valoración de la respuesta terapéutica de las metástasis óseas mediante gammagrafía ósea y estudio PET.

Nuclear medicine plays an important role in staging and evaluation of the initial extension and response to treatment of bone metastases. In order to accurately read Bone Scintigraphy (BS) and Positron Emission Tomography scan (PET) procedures, it is essential to understand the different behavior of these studies. We present a case report of a woman treated for breast cancer, with suspicion of recurrent disease due to increase of tumor markers. Initial BS showed non-conclusive findings, whereas PET study was consistent with a spread of bone metastases. The patient underwent both procedures again after a course of chemotherapy. Post-treatment BS showed progression of bone lesions, while PET showed good therapeutic response. The PET demonstrates lesions earlier and more effectively than the bone scintigraphy in the evaluation of the therapeutic response of bone metastases.

[An evaluation of growth tables]. / Una valoración de las tablas de crecimiento.

OBJECTIVE: To establish how suitable the growth tables we usually use are. DESIGN: A descriptive crossover study. SITE. Primary Care. Casalduch Health Centre in Castellón. PATIENTS AND OTHERS PARTICIPANTS: 120 boys and 158 girls, aged 6, who attended the Health Centre for routine School Health examinations. MEASUREMENTS AND MAIN RESULTS: The weight and height of the children was analysed. The percentil corresponding to each value was assigned in line with the growth tables in the Child Health Booklet. It was found that values higher than those expected were obtained, in both sexes and for weight and height. On comparing the proportion of girls with percentils above 5 and 75 with the theoretical proportions of 50 and 25% respectively, significant differences were obtained in all cases. CONCLUSIONS: Despite our only having studied exclusively 6 year old children in an extremely restricted geographical area, we can claim that this survey contributes to the revision and updating of the growth tables.

[Evaluation of transcutaneous oxygen pressure in adults with respiratory pathology]. / Valoración de la presión transcutánea de oxígeno en adultos con patología respiratoria.

The usefulness of the transcutaneous oxygen tension (tcPO2) in adults is under controversy. In a varied group of respiratory patients, results of the application of this method were compared with those from the arterial blood sampling method. Thirty-eight arterial oxygen tension (PaO2) and tcPO2 simultaneous determinations were made in a group of 22 patients, while in a sitting position; the tcPO2 measurements obtained (68 +/- 12.36 Torr) were significantly lower (p less than 0.05) than the PaO2 values (74 +/- 13.07 Torr). The correlation coefficient was 0.51 (p less than 0.01) with a regression line, tcPO2 = 31.58 + 0.48 PaO2. It is concluded that tcPO2 measurement does not correlate well with PaO2 and that this method cannot be always be safely applied and used in adults with respiratory diseases.