Alignment of preferences in the treatment of multiple myeloma - a discrete choice experiment of patient, carer, physician, and nurse preferences.

BACKGROUND: Multiple Myeloma (MM) is a cancer characterised by the proliferation of malignant plasma cells in the bone marrow. This study examined the treatment preferences of people living with MM compared to the treatment preferences of other groups involved in treatment decision making, including carers, as well as physicians and nurses who treat people living with MM in Australia. METHODS: Data were collected using discrete choice experiments (DCEs) through an online survey. The DCEs presented participants with a traditional treatment generic choice experiment (e.g., treatment A vs treatment B), focusing on the clinical benefits of treatments and the associated risks. The attributes and levels of the attributes were selected based on previous research, literature review, qualitative research and expert opinion. The DCE data were modelled using a Latent Class Model (LCM). RESULTS: The model revealed significant heterogeneity in preferences for treatment attributes. In particular, overall survival, remission period and annual out of pocket cost were the attributes with the most variation. In comparison to people living with MM, carers were less cost-sensitive and more concerned with quality of life (remission period). Physicians and nurses were generally more concerned with overall survival and more cost sensitive than people living with MM. CONCLUSIONS: This study demonstrated that not all people living with MM valued the same treatment attributes equally. Further, not all groups involved in MM treatment decision making had preference alignment on all treatment attributes. This has important implications for healthcare policy decisions and shared decision making. Results from this study could be used to guide decisions around the value of new MM medicines or the medical plan surrounding the needs of those living with MM, as well as those caring for them.

Asian cerebral venous thrombosis registry: study protocol.

INTRODUCTION: Cerebral venous thrombosis (CVT) is a well known but poorly reported entity. Most of the studies and registries related to CVT are reported from European countries. No large multi-center or multi- national data base or registry has been reported from Asian countries. CVT is not uncommon in Asia especially in south Asian subcontinent including India, Pakistan and Bangladesh. One study reported from India that CVT accounted for half of all strokes in the young and 40% of strokes in women. Review of CVT cases from Asian countries is suggestive of differences in risk factors profile and outcome in these patients as compared with European studies. These findings from multi- center data base in Asian countries will be extremely important in identifying risk factors for CVT in these countries. STUDY DESIGN: This is a prospective observational study. We plan to enroll more than 1000 patients from at least ten Asian countries (about 40-50 centers). Patients will be enrolled prospectively and followed for six months. Primary outcome would be death or dependence as assessed by modified Rankin scale (mRS). Data will be collected on a pre-defined data form. There will not be any laboratory test, investigation or treatment specified by the study. Only results of routinely performed studies and treatments will be recorded. Patient (aged 16 or above) will only be included in study if they have diagnosis of CVT proven by magnetic resonance imaging (MRI), magnetic resonance venography (MRV), computed tomography (CT) venography and cerebral venography according to established criteria. Follow up visits will be performed at 6 months, 12 months, and yearly thereafter, preferably by direct interview and observations by the local investigators. OUTCOME: Primary outcome is death or dependence (mRS >2) at the end of the follow-up period. Secondary outcomes are death and dependence at 6 months. Patients will be enrolled from January 2009 to June 2010.

Neurological aspects of tuberous sclerosis in relation to MRI/MR spectroscopy findings in children with epilepsy.

Our aim was to evaluate the relationship between the neurological outcome of tuberous sclerosis complex (TSC) and the findings obtained from both cranial magnetic resonance imaging (MRI) and single voxel proton spectroscopy (SVPS). MRIs of 13 children who met the diagnostic criteria for TSC were taken. Eleven of these children also underwent a prospective analysis of SVPS. Fisher's exact test and Mann-Whitney U test were used, where applicable, to detect any signs of the imaging data that would indicate poor outcome, or in other words, poor seizure control and/ or high degree of mental retardation. Poor seizure control was seen in eight of the patients and multiple seizure types in seven. Mental retardation was severe in six patients and mild/moderate in seven. MRI revealed multiple bilateral tubers and subependymal nodules (13/13), confluence of tubers (8/13), subcortical linear heterotopias (7/13), gyral cores (3/13) and cortical atrophy (3/13). SVPS findings of tubers were characterized by decreased NAA/Cr (1.43 +/- 0.33, p<0.001), increased Cho/Cr (0.91 +/- 0.082, p< 0.05) and mI/Cr (0.97 +/- 0.19, p<0.01) ratios when compared with those of the control group. Lactate peak was detected in six patients. Unfavorable outcome in TSC can be predicted with the help of the following: multiple seizure types, a number of confluent appearances of the tubers and cortical atrophy. SVPS could be a useful clue to understand the pathophysiologic function of the tubers, especially in children with refractory epilepsy along with TSC.

Analysis of NAMCS data for multiple sclerosis, 1998-2004.

BACKGROUND: To our knowledge, no study to date has investigated the prescribing patterns of immunomodulatory agents (IMAs) in an outpatient setting in the United States. To address this issue, we performed retrospective data analyses on National Ambulatory Medical Care Survey (NAMCS) data for MS patient visits between 1998 and 2004. METHODS: NAMCS data are a weighted estimate of the nationwide frequency of patients' outpatient clinic visits. We analyzed NAMCS data in the following categories: (1) the proportion of MS patient visits to neurologists, family practitioners or internists, (2) age/gender/race/geographical distribution patterns in patient visits, and (3) the proportion of patients on IMA treatment among established MS patients. RESULTS: There were an estimated 6.7 million multiple sclerosis (MS) patient visits to the clinics between 1998-2004. Neurologists recorded the most patient visits, 50.7%. Patient visits were mostly in the fourth and fifth decade age group (57.9%). The male to female ratio was 1:4. No statistical evidence was observed for a decline or increase in IMA usage. About 62% patients visiting neurologists and 92% seen by family practitioners/internists were not using IMAs. Our results suggest that between the years 1998-2003, the use of interferon-1a tended to decline while the use of interferon-1b and glatiramer acetate, increased. CONCLUSION: Strategies that lead to improved use of IMAs in the management of MS in the outpatient setting are needed.

Local thrombolytic treatment of cerebral venous thrombosis in three paediatric patients.

There is increasing evidence that local thrombolysis can be used with relative safety and efficacy in adults with superior sagittal sinus thrombosis (SSST). However, little data is available on the use of local thrombolysis in children with SSST. We report three patients who received local thrombolysis for dural sinus thrombosis. Two patients received urokinase and one patient received urokinase followed by local TPA infusion. Recanalization was achieved in two patients.

Can peak systolic velocities be used for prediction of stroke in sickle cell anemia?

BACKGROUND AND PURPOSE: Ischemic stroke occurs in at least 11% of patients with homozygous sickle cell anemia (SCD) by the time they turn 20 years old. High risk associated with distal intracranial internal carotid (ICA) and proximal middle cerebral artery (MCA) stenosis can be detected by transcranial Doppler (TCD). TCD screening offers the possibility of reducing the risk of first stroke significantly based on a paradigm tested and proven to be effective in a stroke prevention trial in sickle cell anemia (STOP). Children with high flow velocity in the ICA and MCA of 200 cm/s time average mean of the maximum (TAMM) or higher had a 10% per year risk of first stroke that was reduced to <1% with regular red cell transfusion (reduction of hemoglobin S <30%). The clinical application of the STOP results could be enhanced if criteria for treatment could be found that are based on peak systolic velocity (PSV), the measure more commonly used in vascular ultrasound practice. OBJECTIVE: To compare PSV and end diastolic velocity (EDV) with TAMM for prediction of stroke and to derive PSV cutpoints for STOP protocol definitions of conditional and abnormal TCD. Using the STOP TCD and stroke outcome data to compare PSV and TAMM in terms of stroke prediction, PSV cutpoints comparable to those based on TAMM and used in STOP were derived. Because of their familiarity to the vascular ultrasound community, PSV cutpoints should be an important alternative to TAMM and may increase availability of screening and risk stratification for children with this disease. MATERIALS AND METHODS: Data from 1,937 baseline TCD studies from STOP were correlated with stroke outcome in those not treated with transfusion. Stroke prediction was assessed with survival analysis using TAMM, PSV and EDV as continuous variables individually and then pair-wise in the same model, which contained 53 stroke events. RESULTS: PSV and EDV were highly correlated to the TAMM velocity (r=0.94). The multivariate model for prediction indicated that TAMM velocity was a better predictor than EDV, and PSV and TAMM were approximately equivalent. PSV cutpoints defining the two relevant STOP risk categories--"conditional," which should lead to increased TCD surveillance, and "abnormal," which should lead to strong consideration for treatment according to STOP--were derived taking into consideration known differences in measurements between the dedicated Doppler systems (TCD) used in STOP and the transcranial Doppler imaging (TCDI) systems commonly used in clinical practice. The recommended PSV cutpoint for conditional TCD is 200 cm/s, and for abnormal TCD triggering consideration for treatment is 250 cm/s. CONCLUSION: Assuming TCDI equipment is used and the STOP protocol is applied, a PSV cutpoint of 200 cm/s is recommended as the threshold for increased TCD surveillance (comparable to a TCD TAMM of 170 cm/s in STOP); a PSV of 250 cm/s is recommended as the cutpoint at which, if confirmed in a second examination, chronic transfusion should be considered. Assuming the STOP scanning protocol is used, PSV is at least as good as TAMM and can be used to select children with SCD for treatment or increased surveillance to prevent first stroke.