Development and validation of the safe transfusion assessment tool.

BACKGROUND: Given the prevalence and risks of blood transfusion, it is essential that trainees and practicing clinicians have a thorough understanding of relevant transfusion medicine competencies. The aim of this research was to develop and gather validity evidence for an instrument to assess knowledge of core transfusion-related competencies. METHODS: We developed the safe transfusion assessment tool (STAT) using a multistep process. Initially, 20 core competencies in transfusion medicine were identified through a consensus-driven Delphi process. Learning objectives and assessment items pertinent to each competency were created. Next, a 13-item assessment tool was piloted with multidisciplinary experts and trainees. Multiple iterative revisions were made based on feedback. Finally, the 12-item STAT was administered to 100 participants of varying training level and specialty to establish validity, difficulty and item discrimination indices, and perceived utility. RESULTS: Analysis of instrument item difficulty and item discrimination indices demonstrated the ability of the STAT to assess essential knowledge in transfusion medicine relevant to trainees and clinicians in multiple programs and practice settings. Eight of twelve items discriminated between learners with varying degrees of expertise. Hundred percent of students and trainees rated the STAT as Extremely Helpful or Somewhat Helpful and the majority planned to utilize the answer guide as a study aid. CONCLUSION: The STAT is a concise, valid, and reliable knowledge assessment tool that may be used by researchers and educators to augment transfusion medicine curricula (www.safetransfusion.ucsf.edu). Scores can help inform departments on areas in which trainees require additional support and areas of potential educational interventions.

Defining core competencies in transfusion medicine for resident physicians: A multi-specialty Delphi consensus study.

BACKGROUND: Although resident physicians across disciplines are responsible for ordering blood products and managing sequelae of blood product transfusion, no designated set of competencies in transfusion medicine has been established for postgraduate trainees. The primary goal of this study was to determine core transfusion-related competencies that such residents should possess. STUDY DESIGN AND METHODS: A modified Delphi method was used to achieve consensus among a panel of clinical faculty and program leadership in six medical specialties to establish core transfusion-related competencies for resident physicians. Review of transfusion education literature, relevant clinical responsibilities, and specialty licensing requirements facilitated generation of an initial transfusion medicine topic list and additional topics were considered if suggested by experts. In two Delphi rounds, experts rated the clinical significance of initial topics on a 5-point Likert scale. Select topics were deemed core competencies if identified as Extremely Important or Moderately Important by at least 75% of panelists to meet a minimum content validity index (CVI) of 0.75 and if topics achieved a minimum content validity ratio (CVR) of 0.5. RESULTS: Nineteen invited clinical experts completed both Delphi rounds with 100% completion across the two rounds. Twenty transfusion medicine topics achieved minimum CVI 0.75 and minimum CVR 0.5. Highest-ranked topics by level of importance include Red Blood Cell (RBC) Transfusion Indications, Platelet Transfusion Indications, and Pulmonary Reactions. CONCLUSIONS: Multispecialty panelists across six medical specialties reached consensus in identification of core transfusion-related competencies for resident physicians. Such consensus-driven core competencies may inform the development of transfusion medicine curricula and assessments to improve transfusion safety.

Long-term evolution of multiple sclerosis disability in the treatment era.

OBJECTIVE: To characterize the accrual of long-term disability in a cohort of actively treated multiple sclerosis (MS) patients and to assess whether clinical and magnetic resonance imaging (MRI) data used in clinical trials have long-term prognostic value. METHODS: This is a prospective study of 517 actively managed MS patients enrolled at a single center. RESULTS: More than 91% of patients were retained, with data ascertained up to 10 years after the baseline visit. At this last assessment, neurologic disability as measured by the Expanded Disability Status Scale (EDSS) was stable or improved compared to baseline in 41% of patients. Subjects with no evidence of disease activity (NEDA) by clinical and MRI criteria during the first 2 years had long-term outcomes that were no different from those of the cohort as a whole. 25-OH vitamin D serum levels were inversely associated with short-term MS disease activity; however, these levels had no association with long-term disability. At a median time of 16.8 years after disease onset, 10.7% (95% confidence interval [CI] = 7.2-14%) of patients reached an EDSS ≥ 6, and 18.1% (95% CI = 13.5-22.5%) evolved from relapsing MS to secondary progressive MS (SPMS). INTERPRETATION: Rates of worsening and evolution to SPMS were substantially lower when compared to earlier natural history studies. Notably, the NEDA 2-year endpoint was not a predictor of long-term stability. Finally, the data call into question the utility of annual MRI assessments as a treat-to-target approach for MS care. Ann Neurol 2016;80:499-510.

Clinical Features and Outcomes of Pediatric Monophasic and Recurrent Idiopathic Optic Neuritis.

Limited data exist on isolated optic neuritis in children. We report the clinical features and treatment of pediatric subjects with monophasic and recurrent idiopathic optic neuritis. This retrospective cohort study of patients with isolated optic neuritis identified 10 monophasic and 7 recurrent optic neuritis cases. Monophasic optic neuritis patients were older (mean 13.3 ± 4.22) than those with recurrent idiopathic optic neuritis (9.86 ± 3.63). Females represented 50% of monophasic and 85.7% of recurrent idiopathic optic neuritis cases. Patients with monophasic optic neuritis were less likely to have a bilateral onset than recurrent idiopathic optic neuritis (40% vs 57.1%). Only 1 case had oligoclonal bands in the cerebrospinal fluid CSF. Most recurrent idiopathic optic neuritis cases had evidence of anti-myelin oligodendrocyte glycoprotein (MOG) antibodies (5/7). Treatment of recurrent idiopathic optic neuritis cases included intravenous pulse glucocorticosteroids and immunotherapy. We observed differences between recurrent and monophasic idiopathic optic neuritis. Immunosuppression appeared to prevent further relapses in recurrent idiopathic optic neuritis patients. Weaning immunotherapies after several years of quiescence in recurrent idiopathic optic neuritis may be possible, but larger studies are needed.

Subclinical Saccadic Eye Movement Dysfunction in Pediatric Multiple Sclerosis.

BACKGROUND: Efferent visual dysfunction in children could lead to impaired quality of life at home and school. Eye-tracking can detect subtle efferent dysfunction missed on bedside examination but has not been validated in the pediatric multiple sclerosis population. OBJECTIVE: We sought to determine the feasibility of eye-tracking in children and associations with multiple sclerosis. METHODS: Participants meeting criteria for pediatric multiple sclerosis without acute efferent vision abnormalities and healthy controls were recruited. Multiple sclerosis participants underwent a clinical assessment and saccade and antisaccade testing paradigms. Intraclass correlation coefficients were generated for intertest repeatability. Adjusting for age and intereye correlations, generalized estimating equations compared latencies with case status, Expanded Disability Status Scale and Symbol Digit Modalities Test (SDMT) scores. RESULTS: We eye-tracked 15 children with multiple sclerosis (n = 30 eyes, mean age 15.6 ± 2.1, mean disease duration 3.9 years, median Expanded Disability Status Scale 1.5) compared to 6 healthy controls (n = 12 eyes, age 14.3 ± .95). The intraclass correlation coefficient for repeated trials was 0.85. Adjusting for age, saccadic latency was 60 milliseconds (ms) longer for cases than controls (95% confidence interval = 26.4, 93.8; P = .0005). For antisaccadic latency, we observed a similar trend of 60 ms longer for cases than controls ( P = .06). CONCLUSION: Eye-tracking is a short noninvasive examination, and high intertest repeatability supports use of eye-tracking technology in pediatric multiple sclerosis. Longer saccadic latencies were seen in children with multiple sclerosis despite short disease duration and low Expanded Disability Status Scale scores.

Early infectious exposures are not associated with increased risk of pediatric-onset multiple sclerosis.

OBJECTIVE: We sought to determine if early infectious exposures such as daycare, early use of antibiotics, vaccinations and other germ exposures including pacifier use and playing on grass are associated with multiple sclerosis (MS) risk in children. METHODS: This was a case-control study of children with MS or clinically isolated syndrome (CIS) and healthy controls enrolled at sixteen clinics participating in the US Network of Pediatric MS Centers. Parents completed a comprehensive environmental questionnaire that captured early infectious exposures, habits, and illnesses in the first five years of life. A panel of at least two pediatric MS specialists confirmed diagnosis of participants. Association of early infectious variables with diagnosis was assessed via multivariable logistic regression analyses, adjusting for age, sex, race, ethnicity, US birth region, and socioeconomic status (SES). RESULTS: Questionnaire responses for 326 eligible cases (mean age 14.9, 63.5% girls) and 506 healthy pediatric subjects (mean age 14.4, 56.9% girls) were included in analyses. History of flu with high fever before age five (p = 0.01), playing outside in grass and use of special products to treat head lice or scabies (p = 0.04) were associated with increased risk of MS in unadjusted analyses. In the multivariable model adjusted for age, sex, race, ethnicity, and mother's highest educational attainment, these results were not statistically significant. Notably, antibiotic use (p = 0.22) and regular daycare attendance before age 6 (p = 0.09) were not associated with odds of developing MS. CONCLUSION: Early infectious factors investigated in this study were not associated with MS risk.

Biosensing in multiple sclerosis.

INTRODUCTION: The goal of using wearable biosensors in multiple sclerosis (MS) is to provide outcome metrics with higher sensitivity to deficits and better inter-test and inter-rater reliability than standard neurological exam bedside maneuvers. A wearable biosensor not only has the potential to enhance physical exams, but also offers the promise of remote evaluations of the patient either at home or with local non-specialist providers. Areas covered: We performed a structured literature review on the use of wearable biosensors in studies of multiple sclerosis. This included accelerometers, gyroscopes, eye-trackers, grip sensors, and multi-sensors. Expert commentary: Wearable sensors that are sensitive to change in function over time have great potential to serve as outcome metrics in clinical trials. Key features of generalizability are simplicity in the application of the device and delivery of data to the provider. Another important feature to establish is best sampling rate. Having too high of a sampling rate can lead to over-interpretation of noisy data On the other hand, a low sampling rate can result in an insensitive test thus missing subtle changes of clinical interest. Of most importance is to establish metrics derived from wearable devices that provide meaningful data in longitudinal studies.