Safety and Efficacy of Turoctocog Alfa in the Prevention and Treatment of Bleeding Episodes in Previously Treated Patients from China with Severe Hemophilia A: Results from the Guardian 7 Trial.

PURPOSE: Hemophilia care in China is characterized by widespread use of on-demand regimens and low-dose prophylaxis. With a limited number of approved recombinant factor VIII (FVIII) products, the incidence of arthropathy and disability in hemophilia patients remains high in China. The purpose of this trial was to evaluate the safety and efficacy of turoctocog alfa for prophylaxis and treatment of bleeding episodes in patients from China with severe hemophilia A across all age groups. PATIENTS AND METHODS: In this Phase 3, open-label trial, previously treated males of all ages with severe hemophilia A from China received turoctocog alfa for prophylaxis or on-demand treatment of bleeds. The primary endpoint was hemostatic effect for the treatment of bleeds during the main phase of the trial. Secondary endpoints included annualized bleeding rate during prophylaxis and the frequency of FVIII inhibitor development. RESULTS: Overall, 42 pediatric patients (age <12 years) and 26 adolescent/adult patients (≥12 years) were dosed with turoctocog alfa; 51 patients initiated treatment with prophylaxis, while 17 patients initiated on-demand treatment. During the main phase of the trial (6 months), hemostatic success was 95.1%. During the full trial (up to 24 months), hemostatic success was 95.4%; the overall median ABR was 1.18 bleeds/patient/year for prophylaxis patients; and 25 (51.0%) of 49 patients with target joints at baseline had all target joints resolved. No FVIII inhibitors (≥0.6 BU) were reported. CONCLUSION: Turoctocog alfa was safe and effective for prophylaxis and treatment of bleeding episodes and for surgery in patients from China with severe hemophilia A across all ages.

The Cost-Effectiveness of Pregabalin Versus Gabapentin for Peripheral Neuropathic Pain (pNeP) and Postherpetic Neuralgia (PHN) in China.

INTRODUCTION: Postherpetic neuralgia (PHN), a type of peripheral neuropathic pain (pNeP), is the most common complication of herpes zoster. The objective of this analysis was to determine the cost-effectiveness of pregabalin compared with gabapentin in pNeP and PHN in China. METHODS: We developed a China-localized 12-week simulation model to determine the cost-effectiveness of pregabalin compared to gabapentin in 1000 patients with pNeP and PHN. We utilized a questionnaire of Chinese key opinion leaders to estimate the pre-treatment distribution of pain scores for pNeP and PHN. Treatment outcomes for pregabalin and gabapentin were acquired from the published literature. RESULTS: Treatment with pregabalin lead to 12-week decreases in pain scores of 0.6 (pNeP) and 0.7 (PHN) when compared to patients receiving gabapentin, at an incremental cost per additional day of mild/no pain of $45. The difference in mean days of no or mild pain, moderate pain, and severe pain was 8.8, -5.7, and -3.1, when comparing pregabalin and gabapentin, respectively. Pregabalin had more mean days with a >30% (7.71 days), 40% (8.97 days), and 50% reduction (9.97 days) in pain when compared with gabapentin. In the pNeP scenario, pregabalin was associated with a lower average pain score compared with gabapentin (3.91 vs. 4.55). The difference in mean days of no or mild pain, moderate pain, and severe pain was 9.39, -5.56, and -3.82, when comparing pregabalin and gabapentin, respectively. Pregabalin had more mean days with a >30% (8.77 days), 40% (9.81 days), and 50% reduction (10.55 days) in pain when compared with gabapentin. CONCLUSION: Pregabalin is an effective treatment for PHN and even for pNeP extensively, but at increased cost. It leads to improved outcomes including lower pain scores and more days with no or mild pain. FUNDING: Pfizer, China.

Remission of rheumatoid arthritis and potential determinants: a national multi-center cross-sectional survey.

The aim of this study is to investigate the remission rate of rheumatoid arthritis (RA) in China and identify its potential determinants. A multi-center cross-sectional study was conducted from July 2009 to January 2012. Data were collected by face-to-face interviews of the rheumatology outpatients in 28 tertiary hospitals in China. The remission rates were calculated in 486 RA patients according to different definitions of remission: the Disease Activity Score in 28 joints (DAS28), the Simplified Disease Activity Index (SDAI), the Clinical Disease Activity Index (CDAI), and the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Boolean definition. Potential determinants of RA remission were assessed by univariate and multivariate analyses. The remission rates of RA from this multi-center cohort were 8.6% (DAS28), 8.4% (SDAI), 8.2% (CDAI), and 6.8% (Boolean), respectively. Favorable factors associated with remission were: low Health Assessment Questionnaire (HAQ) score, absence of rheumatoid factor (RF) and anti-cyclic citrullinated peptide (anti-CCP), and treatment of methotrexate (MTX) and hydroxychloroquine (HCQ). Younger age was also predictive for the DAS28 and the Boolean remission. Multivariate analyses revealed a low HAQ score, the absence of anti-CCP, and the treatment with HCQ as independent determinants of remission. The clinical remission rate of RA patients was low in China. A low HAQ score, the absence of anti-CCP, and HCQ were significant independent determinants for RA remission.