ObjectiveTo evaluate the efficacy of a prolonged-release formulation of a porcine adrenocorticotropic hormone analogue (repository corticotropin injection (RCI)) added to standard of care in patients requiring moderate-dose corticosteroids for symptomatic SLE.MethodsThis prospective, randomised, double-blind, phase 4, pilot study (NCT01753401) enrolled 38 patients with persistently active SLE involving skin and/or joints. Enrolled patients received RCI, 40 U daily or 80 U every other day, or volume-matched placebo gel, for 8 weeks, with dose tapering to twice weekly during weeks 5–8. Efficacy endpoints included proportion of responders at week 4 based on a novel composite measure that included resolution of rash or arthritis measured using the hybrid SLE Disease Activity Index (hSLEDAI) without worsening British Isles Lupus Assessment Group (BILAG) scores in other organ systems at week 4 (primary), as well as improvements in total hSLEDAI and BILAG scores and other measures of skin and joint disease activity over the 8-week treatment period.ResultsResponse, as defined for the primary endpoint, did not differ significantly between the combined placebo and RCI-treated groups at week 4. At week 8, the proportion of responders was higher in RCI-treated patients but did not statistically differ between groups (RCI 40 U (53.8%), RCI 80 U (33.3%), combined placebo (27.3%)). However, RCI treatment was associated with statistically significant improvements in several secondary endpoints, including total hSLEDAI, total BILAG and Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity scores within 8 weeks. Treatment was well tolerated.ConclusionsAlthough the primary endpoint was not met in this pilot study, secondary and post hoc analyses suggested that RCI was associated with improvements in SLE disease activity in a select patient population with steroid-dependent persistent disease.Trial registration numberNCT01753401; results.
The etiology of most cases of acute chest syndrome (ACS) in sickle cell disease (SCD) is unknown. Although pulmonary fat embolism (PFE) is frequently found on autopsy, it is rarely considered in the differential diagnosis in pediatric patients. We conducted a study to determine if we could identify PFE in SCD patients with ACS, define the clinical and laboratory course of PFE, and determine if bronchoalveolar lavage is safe and useful in diagnosis of PFE. Twenty-seven SCD patients with ACS were evaluated and compared with 43 control patients. Serial tests (complete blood count, platelet count, nucleated red blood cells [NRBCs], chest x-ray, and oxygen saturations) were compared with steady-state results. Diagnosis of PFE was made by quantitative evaluation of pulmonary macrophages for intracellular fat. No serious complications from bronchoscopy were observed. In the SCD patients with ACS, 12 were PFE+ and 15 were PFE-. The clinical course of the two groups was quite different. All PFE+ patients experienced bone pain and 11 of 12 had chest pain. In contrast, only 6 of 15 of PFE- patients had bone or chest pain. Neurologic symptoms developed in 6 of 12 of the PFE+ group and in none of the PFE- group. Mean hospital days for PFE+ was 13 compared with 7 for PFE-. Laboratory studies in PFE+ showed a significant decrease in hemoglobin (-2 g, P < .05), platelet count (- 293,000, P < .001), and an increase in NRBCs/100 white blood cells (+8.3, P < .001) compared with PFE-. These results indicate that when PFE is associated with ACS, it is characterized by a distinct clinical course, and that bronchial lavage is a safe and useful test in diagnosing PFE in patients with ACS.
Aims-To test aripiprazole for efficacy in decreasing use in methamphetamine-dependent adults, compared to placebo.Design-Participants were randomized to receive 12 weeks of aripiprazole or placebo, with a 3 month follow-up and a platform of weekly 30-minute substance abuse counseling. Setting-The trial was conducted from January 2009 to March 2012 at the San Francisco Department of Public Health.Participants-Ninety actively-using, methamphetamine-dependent, sexually active, adults were recruited from community venues.Measurements-The primary outcome was regression estimated reductions in weekly methamphetamine-positive urines. Secondary outcomes were study medication adherence (by self-report and medication event monitoring systems [MEMS]), sexual risk behavior, and abstinence from methamphetamine. COMPETING INTERESTSAll authors declare that they have no competing interests. AUTHORS' CONTRIBUTIONSPC directed study activities, participated in the interpretation of data, and drafted the manuscript. GS participated in study design, performed the statistical analysis and interpretation of data, and participated in drafting of the manuscript. MD participated in study design and coordination, and interpretation of data. DS coordinated the study, participated in study design, acquisition and interpretation of data, and participated in study activities. SH participated in the acquisition and interpretation of data. TM participated in study design and in study activities. JG participated in the design of the study. EV participated in the design of the study and the statistical analysis and interpretation of data. GC conceived and designed the study and participated in the conduct of the study and interpretation of data. All authors participated in the revision of the manuscript for important intellectual content and provided approval of the final version of the manuscript. Declarations of interest:The study was funded by a National Institute on Drug Abuse grant, 1 R01 DA023387-01. The funder did not play any role in study design; in the collection, analysis, and interpretation of data; in the writing of the report; nor in the preparation, review, or approval of the manuscript. Conclusion-Compared with placebo, aripiprazole did not significantly reduce methamphetamine use among actively-using, dependent adults. NIH Public Access
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