Real-world Medical Practice Research Articles

Outcome Measures in Medication Trials for Substance Use Disorders

There are multiple therapeutic options to treat tobacco (e.g., nicotine replacement therapies, varenicline) and alcohol (e.g., naltrexone, acamprosate) use disorders. In contrast, there are currently no FDA-approved pharmacotherapies to treat stimulant (e.g., cocaine, methamphetamine) use disorders. Based on a commentary published by FDA staff, a period of sustained abstinence appears required for regulatory approval of a first-in-class medication to treat stimulant use disorders. Certainly, achieving abstinence remains the goal of treatment in both real world medical practice and clinical trials. However, if a medication can help patients to significantly reduce stimulant use (short of achieving a sustained abstinence) while attempting to quit, such reductions could confer meaningful benefit. The FDA has adopted the “percentage of subjects with no heavy drinking days” as an endpoint for pharmacotherapy trials in alcohol use disorders; this suggests there may be a potential path forward for developing analogous, non-abstinence endpoints for stimulant use disorder trials. However, reductions in drug use per se (short of abstinence) must have prognostic value in order to be considered an acceptable basis for FDA approval. Thus, even if a medication can provide sustained reductions in drug use, the challenge ahead is to demonstrate that this “success” is accompanied by benefits that accrue in dimensions readily understood by patients and their families, and of sufficient value to be reimbursed by third party payers. Emerging data sets discussed in this paper indicate that endpoints other than abstinence may ultimately be validated as outcome measures in pharmacotherapy trials for stimulant use disorders.

PCN23 - Bortezomib Re-treatment in Patients with Multiple Myeloma (MM). A Real World Medical Practice Experience from a Swedish National Registry

PCN23 - Bortezomib Re-treatment in Patients with Multiple Myeloma (MM). A Real World Medical Practice Experience from a Swedish National Registry

Prognostic factors in pulmonary arterial hypertension with Dana Point group 1

AimsTo clarify the prognosis and prognostic factors in pulmonary arterial hypertension (PAH) patients in real-world medical practice in the Tokai area in Japan. Main methodsWe conducted a retrospective, multicenter observational study. The data of 81 patients diagnosed with Dana Point group 1 or 1′ PAH was collected from January 2005 to January 2013. The primary outcome was all-cause death. Key findingsThe patients consisted of 34 cases of idiopathic PAH (IPAH), 28 of connective tissue-associated PAH (CTD-PAH), 16 of congenital heart disease-associated PAH (CHD-PAH) and others. Mean age was 51years and mean observation period was 46months. The systolic blood pressure (BPs) was 117±23mmHg. Pericardial effusion was observed in 27.0% of patients. The mean right atrial pressure (mRAP) was 10.2±7.3mmHg.In the univariate Cox regression analysis, WHO-FCS III & IV, a cardiac index (CI)<2.5L/min/m2, and the presence of pericardial effusion at baseline were significantly associated with all-cause death. In the multivariate analysis, the pericardial effusion (HR 3.3, 95% CI 1.03–10.63, p=0.04) and mRAP (HR 3.2, 95% CI 1.03–9.83, p=0.04) or CI<2.5L/min/m2 (HR 3.89, 95% CI 1.05–14.45, p=0.04) were the independent predictors of mortality. SignificanceThe presence of pericardial effusion and mRAP or CI<2.5L/min/m2 at diagnosis indicated high mortality.

Tapentadol Immediate Release Versus Oxycodone Immediate Release for Treatment of Acute Low Back Pain

Background: Tapentadol has demonstrated analgesic efficacy across a range of pain conditions. Objective: In a head-to-head study of up to 10 days in duration, the analgesic efficacy and tolerability of tapentadol immediate release (IR) versus oxycodone IR using a flexible dosing regimen were compared in patients with acute low back pain (LBP) and associated radicular leg pain. Study Design: Randomized (1:1), double-blind, parallel-group study (NCT00986180). Independent Ethics Committee/Institutional Review Board approval of the protocol was obtained. Setting: Ninety US outpatient treatment centers. Methods: Patients with moderate to severe, acute LBP received tapentadol IR (50, 75, or 100 mg) or oxycodone HCl IR (5, 10, or 15 mg) every 4 to 6 hours as needed for pain for up to 10 days. Patients reported current pain intensity twice daily (11-point numerical rating scale). The primary efficacy endpoint was the sum of pain intensity differences (SPID) over 120 hours for LBP. Tapentadol IR was considered non-inferior to oxycodone IR if the upper bound of the 95% confidence interval (CI) for the least-squares mean (LSM) difference in SPID120 was less than 120. Secondary efficacy endpoints included 2-, 3-, and 10-day SPID for LBP; 2-, 3-, 5-, and 10-day SPID for index leg pain; 30% and 50% responder rates; patient and clinician global impressions of change; and patient satisfaction. Results: The safety population included 645 patients, and the modified intent-to-treat population included 585 patients. In the tapentadol IR and oxycodone IR groups, respectively, 86.3% (277/321) and 82.7% (268/324) of patients completed the study. The most common reason for study withdrawal in both treatment groups was adverse events (tapentadol IR, 6.5% [21/321]; oxycodone IR, 7.1% [23/324]). The LSM (standard error) SPID120 for LBP was 264.6 (11.43) for tapentadol IR (n = 287) and 264.0 (11.22) for oxycodone IR (n = 298). The 95% CI for the LSM difference was −32.1 to 30.9; therefore, tapentadol IR was non-inferior to oxycodone IR for relief of LBP. No significant differences were observed between tapentadol IR and oxycodone IR for other SPID endpoints or for responder rates. At the end of the study, in the tapentadol IR and oxycodone IR treatment groups, respectively, approximately two-thirds of patients (66.2% vs 66.2%) and clinicians (67.9% vs 66.6%) rated patients’ overall condition as “very much improved” or “much improved,” and more than 75% of patients (79.3% vs 78.9%) were “very satisfied” or “somewhat satisfied” with their treatment. In the tapentadol IR and oxycodone IR groups, respectively, 52.3% (168/321) and 58.0% (188/324) of patients reported at least one treatment-emergent adverse event (TEAE); the most common (≥ 10%) TEAEs were vomiting (15.9% vs 24.7%), nausea (15.9% vs 20.7%), and dizziness (11.8% vs 10.5%). Vomiting (odds ratio [95% CI], 1.74 [1.17 - 2.57]) and constipation (3.43 [1.45 - 8.11]) were significantly more likely to occur in the oxycodone IR treatment group. Two (0.6%) patients in the tapentadol IR group and 3 (0.9%) patients in the oxycodone IR group experienced treatment-emergent serious adverse events. Limitations: Strict patient monitoring is generally not representative of real-world medical practice; consequently, higher incidences of TEAEs may have been reported than would be expected in a typical practice setting; it is anticipated that this bias would be similar for both treatment groups. Conclusions: This head-to-head study demonstrated that tapentadol IR had comparable analgesic efficacy and overall safety to that of oxycodone IR for the relief of moderate to severe, acute LBP and associated radicular leg pain when using flexible dosing regimens that reflect typical use in clinical practice; however, tapentadol IR demonstrated a better gastrointestinal tolerability profile, particularly for the common opioid-related TEAEs of vomiting and constipation. Clinical Trial Registration: NCT00986180 Key words: Tapentadol, oxycodone, acute low back pain, radicular leg pain, neuropathic pain, flexible dosing, radiculopathy, gastrointestinal tolerability

PSY77 - Quantifying the impact of differing measures used to study persistence and adherence with the infusible anti-inflammatory biologic infliximab: Utilization from real-world medical practice

PSY77 - Quantifying the impact of differing measures used to study persistence and adherence with the infusible anti-inflammatory biologic infliximab: Utilization from real-world medical practice

The Japan Thrombosis Registry for Atrial Fibrillation, Coronary or Cerebrovascular Events (J-TRACE) A Nation-Wide, Prospective Large Cohort Study; The Study Design

A previous history of myocardial infarction (MI), stroke, and the presence of atrial fibrillation (AF) are known risk factors for the onset of arterial thromboembolic events such as MI and ischemic stroke. To clarify the rate of incidence of such events for these high-risk patients in Japan, a nation-wide cohort study was conducted that was named the 'Japan Thrombosis Registry of Atrial Fibrillation, Coronary and Cerebrovascular Events' (ie, J-TRACE) [UMIN Registered ID C000000189]. In the J-TRACE registry, a total of 8,093 Japanese patients with either a history of stroke and/or MI or patients with non-valvular AF were registered. This registry was developed by specialists in cardiology and neurology, physicians working at general hospitals, as well as general practice physicians, from whole regions of Japan, possibly reflecting the real-world medical practice. Recruited patients will be followed up for 2-3 years. Medical history, accompanying risk factors, demographic characteristics, and information regarding the use of medications were collected for these patients at baseline. Cardiovascular ischemic events and serious adverse experiences, including cerebral bleedings, which occur during the follow-up period will be recorded over a maximum of 3 years. The J-TRACE offers an opportunity to provide fundamental information regarding the incidence of cardiovascular ischemic events by a stratum of the risk factor profile and current medical treatment for Japanese patients at high risk for thromboembolic diseases.

Medical practice and informatics tools: a vignette.

In this article the authors recreate an improvisational vignette originally presented at the Third Annual Information Connection in Burlington, Vermont, in January 1998. The vignette illustrates various state-of-the-art decision-support systems for clinical care and their promises and problems in real-world medical practice. The characters are Dr. Alex Grant, a rural physician in solo practice; Anna Everett, a patient suffering from chronic headaches; Bob, a hospital librarian; and Sarah, Dr. Grant's nurse. The short play centers on Dr. Grant's attempt to diagnose the cause of Anna's headaches (which he and she believe to be sinus-related) and the roles information technologies--Medline searching, Anna's own Internet searches, the use of MDConsult, which provides Internet access to standard medical texts, journals, etc., and a desktop decision-support tool called Problem Knowledge Couplers--play in this process. The vignette concludes with the realization that while computer technologies can be of great help in medicine "there is still a need for a good doctor to pull it all together."

Medical practice and informatics tools

In this article the authors recreate an improvisational vignette originally presented at the Third Annual Information Connection in Burlington, Vermont, in January 1998. The vignette illustrates various state-of-the-art decision-support systems for clinical care and their promises and problems in real-world medical practice. The characters are Dr. Alex Grant, a rural physician in solo practice; Anna Everett, a patient suffering from chronic headaches; Bob, a hospital librarian; and Sarah, Dr. Grant's nurse. The short play centers on Dr. Grant's attempt to diagnose the cause of Anna's headaches (which he and she believe to be sinus-related) and the roles information technologies--Medline searching, Anna's own Internet searches, the use of MDConsult, which provides Internet access to standard medical texts, journals, etc., and a desktop decision-support tool called Problem Knowledge Couplers--play in this process. The vignette concludes with the realization that while computer technologies can be of great help in medicine “there is still a need for a good doctor to pull it all together.”