Function Change Measurements Research Articles

Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study.

Evaluations of treatment efficacy in Duchenne muscular dystrophy (DMD), a rare genetic disease that results in progressive muscle wasting, require an understanding of the 'meaningfulness' of changes in functional measures. We estimated the minimal detectable change (MDC) for selected motor function measures in ambulatory DMD, i.e., the minimal degree of measured change needed to be confident that true underlying change has occurred rather than transient variation or measurement error. MDC estimates were compared across multiple data sources, representing >1000 DMD patients in clinical trials and real-world clinical practice settings. Included patients were ambulatory, aged ≥4 to <18 years and receiving steroids. Minimal clinically important differences (MCIDs) for worsening were also estimated. Estimated MDC thresholds for >80% confidence in true change were 2.8 units for the North Star Ambulatory Assessment (NSAA) total score, 1.3 seconds for the 4-stair climb (4SC) completion time, 0.36 stairs/second for 4SC velocity and 36.3 meters for the 6-minute walk distance (6MWD). MDC estimates were similar across clinical trial and real-world data sources, and tended to be slightly larger than MCIDs for these measures. The identified thresholds can be used to inform endpoint definitions, or as benchmarks for monitoring individual changes in motor function in ambulatory DMD.

A novel tool for characterising upper limb function in progressive multiple sclerosis through kinematic assessment

IntroductionCurrent upper limb assessment methods in MS rely on measuring duration in tasks like the nine-hole peg test (9HPT). Kinematic techniques may provide a more useful measure of functional change in clinical and research practice. The aim of this study was to assess upper limb function prospectively in people with progressive MS using a kinematic 3D motion capture system and compare with current measures. Methods42 people with progressive MS (PwPMS) and 15 healthy controls reached-and-grasped different objects whilst recorded by a kinematic assessment system. 9HPT, Expanded Disability Status Scale (EDSS), and patient reported outcome measures (PROs) were collected. All measures were taken at baseline for PwPMS and controls, and again at six months for PwPMS. ResultsRelative to controls, PwPMS had significantly longer reaction (0.11 s, p < 0.05) and reach (0.25 s, p < 0.05) times. PwPMS took longer to pick-up (0.34 s, p < 0.05), move (0.14 s, p < 0.05), and place (0.18 s, p < 0.05) objects. PwPMS had lower peak velocities when reaching (7.4 cm/s, p < 0.05) and moving (7.3 cm/s, p < 0.05) objects. Kinematic assessment demonstrated consistent differences between PwPMS with mild and severe upper limb dysfunction as defined by PROs, which were not captured by 9HPT or EDSS in this group. PwPMS demonstrated altered grip apertures profiles, as measured by their ability to complete individual parts of the reach and grasp task, between the baseline and follow-up timepoints. ConclusionsWe have created and tested a novel upper limb function assessment tool which has detected changes and characteristics in hand function, not currently captured by the EDSS and 9HPT.

In Vitro Patch-Clamp.

The patch-clamp technique is one of the most useful tools to analyze the function of electrically active cells such as neurons. This technique allows for the analysis of proteins (ion channels and receptors), cells (neurons), and synapses that are the building blocks of neuronal networks. Cortical development involves coordinated changes in functional measures at each of these levels of analysis that reflect both cellular and circuit maturation. This chapter explains the technical and theoretical basis of patch-clamp methodology and introduces several examples of how this technique can be applied in the context of cortical development.

Left ventricular remodelling and changes in functional measurements in patients undergoing transcatheter vs surgical aortic valve replacement: a head-to-head comparison.

Left ventricular remodelling and changes in functional measurements in patients undergoing transcatheter vs surgical aortic valve replacement: a head-to-head comparison.

A Convolutional Neural Network Approach to Quantify Lung Disease Progression in Patients with Fibrotic Hypersensitivity Pneumonitis (HP)

Rationale and Objectives To evaluate associations between longitudinal changes of quantitative CT parameters and spirometry in patients with fibrotic hypersensitivity pneumonitis (HP). Materials and Methods Serial CT images and spirometric data were retrospectively collected in a group of 25 fibrotic HP patients. Quantitative CT analysis included histogram parameters (median, interquartile range, skewness, and kurtosis) and a pretrained convolutional neural network (CNN)-based textural analysis, aimed at quantifying the extent of consolidation (C), fibrosis (F), ground-glass opacity (GGO), low attenuation areas (LAA) and healthy tissue (H). Results At baseline, FVC was 61(44-70) %pred. The median follow-up period was 1.4(0.8-3.2) years, with 3(2-4) visits per patient. Over the study, 8 patients (32%) showed a FVC decline of more than 5%, a significant worsening of all histogram parameters (p≤0.015) and an increased extent of fibrosis via CNN (p=0.038). On histogram analysis, decreased skewness and kurtosis were the parameters most strongly associated with worsened FVC (respectively, r2=0.63 and r2=0.54, p<0.001). On CNN classification, increased extent of fibrosis and consolidation were the measures most strongly correlated with FVC decline (r2=0.54 and r2=0.44, p<0.001). Conclusion CT histogram and CNN measurements provide sensitive measures of functional changes in fibrotic HP patients over time. Increased fibrosis was associated with FVC decline, providing index of disease progression. CNN may help improve fibrotic HP follow-up, providing a sensitive tool for progressive interstitial changes, which can potentially contribute to clinical decisions for individualizing disease management.

Inpatient Rehabilitation Outcomes After Severe COVID-19 Infections: A Retrospective Cohort Study.

The aim of the study was to describe the characteristics and functional outcomes of patients undergoing acute inpatient rehabilitation after hospitalization for COVID-19. Using a retrospective chart review, patients were identified who were admitted to inpatient rehabilitation after COVID-19. Patient information collected included sociodemographic characteristics, comorbidities, length of stay, discharge disposition, self-care, mobility, and cognitive functioning. These patients were compared with patients (controls) without COVID-19 with similar impairment codes treated at the same facility before the COVID-19 pandemic. There were 43 patients who were admitted to the inpatient rehabilitation hospital after COVID-19 infection and 247 controls. Patients who had COVID-19 were significantly more likely to be African American and to have been admitted to a long-term acute care hospital. They also had a longer length of rehabilitation stay. The groups did not differ by age, sex, or insurance. Functionally, although presenting with significantly worse mobility, self-care, and motor scores, the patients previously infected with COVID-19 had similar functional outcomes at time of discharge to the control group. Although patients with a history of COVID-19 had worse function at time of admission to acute rehabilitation, inpatient rehabilitation significantly improved their function to comparable levels as patients who did not have COVID-19. Complete the self-assessment activity and evaluation online at http://www.physiatry.org/JournalCME. Upon completion of this article, the reader should be able to: (1) Identify how characteristics of patients with COVID-19 admitted to acute rehabilitation differ from those with similar admission codes but without COVID-19; (2) Describe changes in functional measures at admission and discharge of COVID-19 patients compared with patients without COVID-19; and (3) Recognize how inpatient rehabilitation may help reduce inequities in outcomes after severe COVID-19 infection. Advanced. The Association of Academic Physiatrists is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.The Association of Academic Physiatrists designates this Journal-based CME activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should only claim credit commensurate with the extent of their participation in the activity.

DMD/BMD – OUTCOME MEASURES: EP.125 Minimal detectable changes in functional measures in Duchenne muscular dystrophy (DMD): A study of multiple centers, networks and trial arms

DMD/BMD – OUTCOME MEASURES: EP.125 Minimal detectable changes in functional measures in Duchenne muscular dystrophy (DMD): A study of multiple centers, networks and trial arms

Benefits of blood transfusions in palliative care patients with advanced cancer.

e24090 Background: Anemia is highly prevalent in patients with advanced cancer and adversely affects quality of life. There is limited data on the frequency, clinical utility and effectiveness of red blood cell transfusions (RBC), and no randomized controlled trials or clinical practice guidelines on this subject are available. The aim of this study was to evaluate clinician practices on RBC transfusion in an oncologic palliative care unit (PCU), its impact on patients’ symptoms, overall survival and to identify predictive factors for survival. Methods: Retrospective cohort study of all advanced cancer patients who had received RBC transfusions over a 3-year period, after admission to the PCU for symptomatic control, as inpatients or outpatients. All had histologically confirmed malignant tumors and were not under anti-cancer treatments. Patients’demographics, clinical and laboratory features, symptoms and mortality were reviewed. Survival analysis was estimated using the Kaplan-Meier method and Cox's regression was used for multivariate analysis. Results: We identified 179 patients with a median age of 68 years [30-93], 60% were male, with a mean Charlson comorbidity index of 8.9 (SD ±2.3). The majority had gastrointestinal (42%) and genitourinary (35%) malignancies. A total of 435 RBC units, during 301 transfusion episodes were recorded. The majority (58%) were performed as inpatients. A combination of symptoms with low haemoglobin (Hb) levels was the main reason for transfusion (80%). Asthenia/fatigue was the most frequent symptom (68%). Prior to transfusion, the majority (73%) had an ECOG-performance status (ECOG-PS) greater than 2. The mean pretransfusion Hb was 6.9 g/dL and 48% patients had an Hb above 7 g/dL. Symptomatic benefit post-transfusion was achieved in 36% of patients. A statistically significant association between ECOG-PS and symptomatic benefit was found (p = 0.005). Median overall survival post-transfusion was 41 days (IC95% 30.6-51.4). On multivariate analysis, Hb level pre-transfusion, ECOG-PS and symptomatic benefit with transfusions were significantly associated with survival. Conclusions: Transfusion practices are more liberal in palliative care, increasing iatrogenic risk, while consuming a valuable and limited resource. However, transfusion does provide symptom relief, and should be offered to advanced cancer patients with a higher level of functioning. Post-transfusion symptomatic benefit, and pre-transfusion ECOG-PS and hemoglobin levels seem to be independent predictors of survival. Further high-quality trials are needed to develop validated measures of objective functional changes to evaluate the clinical impact of transfusions and to identify patients most likely to be positively impacted by transfusion.

Identifying the Drivers of Spatial Taxonomic and Functional Beta-Diversity of British Breeding Birds

Spatial variation in community composition may be driven by a variety of processes, including environmental filtering and dispersal limitation. While work has been conducted on the relative importance of these processes on various taxa and at varying resolutions, tests using high-resolution empirical data across large spatial extents are sparse. Here, we use a dataset on the presence/absence of breeding bird species collected at the 10 km × 10 km scale across the whole of Britain. Pairwise spatial taxonomic and functional beta diversity, and the constituent components of each (turnover and nestedness/richness loss or gain), were calculated alongside two other measures of functional change (mean nearest taxon distance and mean pairwise distance). Predictor variables included climate and land use measures, as well as a measure of elevation, human influence, and habitat diversity. Generalized dissimilarity modeling was used to analyze the contribution of each predictor variable to variation in the different beta diversity metrics. Overall, we found that there was a moderate and unique proportion of the variance explained by geographical distance per se, which could highlight the role of dispersal limitation in community dissimilarity. Climate, land use, and human influence all also contributed to the observed patterns, but a large proportion of the explained variance in beta diversity was shared between these variables and geographical distance. However, both taxonomic nestedness and functional nestedness were uniquely predicted by a combination of land use, human influence, elevation, and climate variables, indicating a key role for environmental filtering. These findings may have important conservation implications in the face of a warming climate and future land use change.

Inner retinal thickening affects microperimetry thresholds in the presence of photoreceptor thinning in patients with RPGR retinitis pigmentosa

BackgroundLoss of photoreceptors cause degeneration in areas of the retina beyond the photoreceptors. The pattern of changes has implications for disease monitoring and measurement of functional changes. The aim of...

Pathophysiology of Breast Cancer-Related Lymphoedema

To review the literature regarding the pathophysiological changes found in BCRL and to link them to the cancer and the type and sequencing of treatments and raise attention to the fact that not all parts of the arm at risk or with lymphoedema may have underlying changes which are similar or are progressing at the same rate. There still remains a lack of awareness regarding BCRL-related pathophysiology and of the range of reasons for it. Linked to this is a lack of appropriate in-clinic assessment of these changes and of the use of this information to better target and sequence treatment. We are refining and improving our consensus documents related to lymphoedema and the impact of the pathophysiology on its staging which will help in this. We should link the pathophysiological changes we find and record with the functional changes which are signs of that underlying structural change through increased use of a range of assessment tools such as indurometry, bio-impedance spectroscopy, tissue dielectric constants, ICG, lympho-scintigraphy, etc. We must better target and sequence our treatments and relate them to our measures of pathophysiological and functional changes.

Initial functional recovery as a measure for rehabilitation outcome in post-acute hip fractured patients

The assessment of immediate functional change after hip fracture surgery may assist in predicting rehabilitation outcome and improve program efficiency. To evaluate the potential of the initial functional recovery measure in predicting rehabilitation outcome of hip fractured patients. Retrospective cohort study. Post-acute geriatric rehabilitation center. A total of 630 hip fractured patients admitted from 1/2016-6/2019. The initial functional recovery, a measure of functional change from the time of surgery to rehabilitation was calculated for each patient. The association between initial functional recovery and clinical and demographic variables was assessed by the Mann-Whitney and chi-square tests. A multiple linear regression model evaluated the association between initial functional recovery and the discharge functional independence measure score. Regression analysis estimated the association between initial functional recovery and the achievement of a favorable outcome measure. A good correlations were found between initial functional recovery and rehabilitation outcomes but not length of stay (p < 0.001). Multiple linear regression analysis showed that after adjustment for potential confounding variables, the higher the initial functional recovery, the better the discharge functional independence measure score (r2 = 0.783; p < 0.001). Logistic regression revealed that a high initial functional recovery was significantly associated with a higher probability of achieving a favorable functional gain (OR = 1.093; 95% CI = 1.04-1.15, P < 0.001). Initial functional recovery is a good predictor of rehabilitation outcomes in hip fractured patients, and may be utilized in decision-making regarding the rehabilitation program in addition to coordinating expectations with patients and their caregivers.

Rotating frame MRI relaxations as markers of diffuse white matter abnormalities in multiple sclerosis

Even though MRI visualization of white matter lesions is pivotal for the diagnosis and management of multiple sclerosis (MS), the issue of detecting diffuse brain tissue damage beyond the apparent T2-hyperintense lesions continues to spark considerable interest. Motivated by the notion that rotating frame MRI methods are sensitive to slow motional regimes critical for tissue characterization, here we utilized novel imaging protocols of rotating frame MRI on a clinical 3 Tesla platform, including adiabatic longitudinal, T1ρ, and transverse, T2ρ, relaxation methods, and Relaxation Along a Fictitious Field (RAFF) in the rotating frame of rank 4 (RAFF4), in 10 relapsing-remitting multiple sclerosis patients and 10 sex- and age-matched healthy controls. T1ρ, T2ρ and RAFF4 relaxograms extracted from the whole white matter exhibited a significant shift towards longer relaxation time constants in MS patients as compared to controls. T1ρ and RAFF4 detected alterations even when considering only regions of normally appearing white matter (NAWM), while other MRI metrics such as T1w/T2w ratio and diffusion tensor imaging measures failed to find group differences. In addition, RAFF4, T2ρ and, to a lesser extent, T1ρ showed differences in subcortical grey matter structures, mainly hippocampus, whereas no functional changes in this region were detected in resting-state functional MRI metrics. We conclude that rotating frame MRI techniques are exceptionally sensitive methods for the detection of subtle abnormalities not only in NAWM, but also in deep grey matter in MS, where they surpass even highly sensitive measures of functional changes, which are often suggested to precede detectable structural alterations. Such abnormalities are consistent with a wide spectrum of different, but interconnected pathological features of MS, including the loss of neuronal cells and their axons, decreased levels of myelin even in NAWM, and altered iron content.

Staged residential post-acute rehabilitation for adults following acquired brain injury: A comparison of functional gains rated on the UK Functional Assessment Measure (UK FIM+FAM) and the Mayo-Portland Adaptability Inventory (MPAI-4)

ABSTRACTObjective: To compare the UK Functional Assessment Measure (UK FIM+FAM) and Mayo-Portland Adaptability Inventory (MPAI-4) as measures of functional change in patients with brain injury receiving a staged residential post-acute community-based rehabilitation programme. Research design: Longitudinal cohort study of consecutive admissions (N = 42) over 3 years.Methods: Patients were assessed at admission and discharge/annual review. We examined groups according to stage of independence on admission: Maximum support (stages 1 and 2: N = 17); moderate/maximum self-care/household support (stage 3: N = 15); minimal self-care and moderate household/community support (stages 4–6: N = 10).Results: Median (IQR) age: 50 (37–56) years. Male:female ratio: (71%:29%). Aetiology: stroke (50%), traumatic (36%) and other brain injuries (14%). Both tools demonstrated significant gains in overall scores and all subscales (p < 0.01). However, the UK FIM+FAM provides more detailed evaluation of personal activities of daily living and mobility, which were most relevant in clients admitted in graduation stages 1 and 2 of the programme, whereas the MPAI-4 was more sensitive to changes in adjustment and participation for clients admitted in the later stages (4–6).Conclusions: The UK FIM+FAM and MPAI-4 provide complementary evaluation across functional tasks ranging from self-care to participation. This study supports their use for longitudinal outcome evaluation in community residential rehabilitation services that take patients at different stages of recovery.

The Test of Masticating and Swallowing Solids (TOMASS): reliability, validity and international normative data.

Clinical swallowing assessment is largely limited to qualitative assessment of behavioural observations. There are limited quantitative data that can be compared with a healthy population for identification of impairment. The Test of Masticating and Swallowing Solids (TOMASS) was developed as a quantitative assessment of solid bolus ingestion. This research programme investigated test development indices and established normative data for the TOMASS to support translation to clinical dysphagia assessment. A total of 228 healthy adults (ages 20-80+ years) stratified by age and sex participated in one or more of four consecutive studies evaluating test-retest and interrater reliability and validity to instrumental assessment. For each study the test required participants to ingest a commercially available cracker with instructions to 'eat this as quickly as is comfortably possible'. Further averaged measures were derived including the number of masticatory cycles and swallows per bite, and time per bite, masticatory cycle and swallow. Initial analyses identified significant differences on salient measures between two commercially available crackers that are nearly identical in shape, size and ingredients, suggesting the need for separate normative samples for specific regional products. Additional analyses on a single cracker identified that the TOMASS was sensitive at detecting changes in performance based on age and sex. Test-retest reliability across days and interrater reliability between clinicians was high, as was validation of observational measures to instrumental correlates of the same behaviours. Therefore, normative data are provided for the TOMASS from a minimum of 80 healthy controls, stratified by age and sex, for each of seven commercially available crackers from broad regions worldwide. Analyses on a single cracker identified Arnott's Salada, and that TOMASS measures were sensitive for detecting changes in performance based on age and sex. Interrater and test-retest reliability across days were high, as was validation of observational measures to instrumental correlates of the same behaviours. Significant differences were identified between two commercially available crackers, nearly identical in shape, size and ingredients, thus normative samples for specific regional products were required. Normative data were then acquired for the TOMASS from a minimum of 80 healthy controls, stratified by age and sex, for each of seven commercially available crackers from broad regions worldwide. The TOMASS is presented as a valid, reliable and broadly normed clinical assessment of solid bolus ingestion. Clinical application may help identify dysphagic patients at bedside and provide a non-invasive, but sensitive, measure of functional change in swallowing.

Cognitive Decline in Relation to Psychological Wellbeing and HIV Disease- and Treatment Characteristics in HIV-Infected Patients on cART: A One-Year Follow-Up Study

The objectives of the current study were to examine cognitive decline in relation to psychological wellbeing, HIV disease and treatment characteristics and baseline variables over a one-year period of time in a group of HIV-infected patients on long term cART with undetectable viral load in comparison to a HIV-negative control group. Eighty-two of 95 patients and 43 of 55 controls who completed a baseline assessment for the Art-NeCo study underwent a follow-up neuropsychological assessment. A repeated-measure general linear model analysis was performed to compare the performance at follow-up in comparison to baseline between the patients and controls. Reliable change indices were computed as a measure of significant change in cognitive function. Compared to controls, patients overall performed worse on the domain speed of information processing. In the patient group a worse performance at follow-up was present for the verbal fluency domain compared to the controls, in the absence of a baseline group difference. For the executive function domain, no group differences were found at follow-up, but the patients performed worse than the controls at baseline. We found that cognitive decline was related to more frequent use of recreational drugs and a somewhat heightened level of irritability and more somatic complaints at baseline. However, the decliners did not differ from the non-decliners on any of the HIV-related variables.

Efficacy of Inpatient Rehabilitation After Left Ventricular Assist Device Implantation

BackgroundLeft ventricular assist devices (LVADs) have become an increasingly popular and effective means for treating advanced heart failure. LVAD implantation requires extensive surgery and postoperative rehabilitation. The Functional Independence Measure (FIM) has been used to quantify functional gains in numerous patient populations, including those with stroke and spinal cord injury. This study investigated functional improvements in patients undergoing LVAD implantation using the FIM score. ObjectiveTo assess functional improvements in patients with advanced heart failure who underwent LVAD implantation. DesignRetrospective. SettingInpatient rehabilitation unit. SubjectsNinety consecutive patients who received acute inpatient rehabilitation after continuous flow LVAD implantation. MethodsDemographic, laboratory, and functional outcomes data including inpatient rehabilitation unit (IRU) length of stay (LOS), discharge disposition, and FIM score were collected for all patients. Paired t-tests were used to assess change in functional measures and laboratory data. Main Outcome MeasuresPrimary outcome measures included FIM gain, FIM efficiency, discharge disposition, rates of readmission after discharge from rehabilitation, and LOS in the rehabilitation unit. ResultsThe FIM gain was statistically significant at 28.4 ± 12.3 (P < .001) and compared favorably with benchmarks for mean FIM gains at our facility (26.4), regionally (21.5), and nationally (22.7) for patients admitted to IRUs with a cardiac diagnosis. FIM efficiency (FIM gain/IRU LOS) was 1.9 ± 1.0 compared with the mean FIM efficiency at our facility (2.2), regionally (2.1), and nationally (2.2). Seventy-four percent (n = 67) of patients were discharged directly home after inpatient rehabilitation, 17% (n = 16) were readmitted to the acute hospital service, and 8% (n = 7) required additional rehabilitation at a subacute rehabilitation facility. The IRU LOS was 16.2 ± 6.9 days. ConclusionsOur study indicates that most patients with an LVAD achieve clinically meaningful functional gains from acute inpatient rehabilitation, with the majority of patients being discharged home. Further studies need to be performed to analyze clinical outcomes after acute inpatient rehabilitation. Level of EvidenceIV

Six-Minute Walk Test As a Measure of Functional Change after Chemotherapy in Cardiac AL Amyloidosis

Background: Light chain amyloidosis (AL) is characterized by tissue deposition of misfolded proteins that cause multisystem organ dysfunction. Current treatments, including chemotherapy and autologous hematopoietic cell transplantation (AHCT), target plasma cells producing pathogenic amyloid proteins. The development of amyloid protein targeted monoclonal antibody therapy has led to an increased need for objective measurements of clinical response. The 6-minute walk test (6MWT) has been used for years as the primary outcome measure to monitor disease severity in clinical trials of heart failure (HF). For example, HF trials with cardiac devices MIRACLE (Abraham, 2002) and MUSTIC (Cazeau, 2001) demonstrated important therapeutic effects based on an improved 6MWT distance of 39 meters and a change of 23%, respectively. These findings, in conjunction with improvement in other markers of disease activity, lead to approval of cardiac resynchronization for the management of HF. Our primary objective was to determine the impact of chemotherapy on changes in 6MWT in patients with AL cardiac amyloid and correlate these changes with cardiac biomarkers.Patients and Methods: We retrospectively analyzed outcomes of 22 AL amyloid patients with cardiac involvement. Subjects performed 6MWT at diagnosis and at the end of planned initial chemotherapy. All patients received bortezomib (B)-based chemotherapy. About half received chemotherapy alone (45%) and the others received B-based induction chemotherapy followed by AHCT (55%). To identify factors associated with changes in 6MWT, we analyzed cardiac response and changes in New York Heart Association (NYHA) class, troponin I, brain natriuretic peptide (BNP) and left ventricular ejection fraction (LV EF). Cardiac response was defined as BNP decrease of 30% or NYHA class decrease ≥ 2 in subjects with baseline NYHA class 3 or 4 (adapted from Comenzo, 2012).Results: At baseline, 59% (n=13) of patients were modified AL amyloid cardiac stage I/II and 41% (n=9) stage III. The median percent change in 6MWT was an increase in 26.5% with a median change of 90 meters (range, -120 to 365), p < 0.001. Overall, 81% (n=18) had improvement in 6MWT distance, 9% (n=2) declined and 9% (n=2) were unchanged. Patients had reassessment of 6MWT a median of 13.5 months from diagnosis. Hematological responses (HR) included CR (36%), VGPR (36%), PR (23%) and SD (5%). HR was not associated with change in 6MWT. Fifty percent (n=11) experienced a cardiac response. By Wilcoxon rank sum test, variables that were associated with improvement in 6MWT included improved BNP, LV EF, troponin I, NYHA class and cardiac response (all p<0.001). The median follow-up of patients was 2.15 years (range, 1.10 - 7.20).By multivariate analysis, patients with a cardiac response had significantly better improvement in 6MWT disease compared with patients without cardiac response (p=0.02). A cardiac response was associated with a median increase of 170 meters of distance traveled by 6MWT when compared with patients having no cardiac response. All other variables tested were not statistically significant.Conclusion: In AL amyloid patients with cardiac involvement, the 6MWT can be used as an objective marker of functional improvement complementing biochemical and imaging parameters of response. Furthermore, treatment of cardiac amyloidosis with contemporary standard regimens has a major impact on the disease process by a number of objective parameters. Patients experiencing a cardiac response had substantial improvements in 6MWT. The 6MWT should be incorporated in trials of patients with cardiac amyloid. [Display omitted] DisclosuresCornell:Prothena: Research Funding.

Exercises Including Weight Vests and a Patient Education Program for Women With Osteopenia: A Feasibility Study of the OsteoACTIVE Rehabilitation Program

Prospective case series with 1-year follow-up. The primary aim was to describe the OsteoACTIVE rehabilitation program and evaluate its feasibility in terms of progression, adherence, and adverse events in patients with low bone mineral density (BMD) and a healed forearm fracture. The secondary aim was to assess changes in measures of function and quality of life. Previous studies have shown benefits of weight-bearing activities, resistance exercises, and balance and coordination training for women with low BMD and older adults. However, no studies, to our knowledge, have described or examined a rehabilitation program combining the use of weight vests and patient education in patients with low BMD. Forty-two postmenopausal women with osteopenia and a healed forearm fracture attended the OsteoACTIVE program for 6 months (3 sessions of 60 minutes per week). Feasibility was assessed by documenting training progression (load and exercises), program adherence (aiming for greater than 80%), and adverse events (joint pain, muscle soreness, and falls). Secondary measures included quadriceps strength, BMD, dynamic balance, walking ability, and self-report functional outcome measures. All outcome measures were recorded preintervention (baseline), postintervention, and at 1-year follow-up. Thirty-five women (83%) completed the 6-month program and 31 women (74%) attended all the follow-up measurement sessions. All participants progressed during the rehabilitation program for both load and type of exercises. Furthermore, 87% of the participants met the a priori goal of 80% adherence, and no participants reported adverse events. Improvements in quadriceps strength and BMD of the femoral trochanter were noted at the end of the 6-month training period (P<.05). At 1-year follow-up, there were significant improvements in quadriceps strength and dynamic balance compared to baseline (P<.05). The OsteoACTIVE rehabilitation program was feasible and achieved progression of training level, had high adherence, and had no adverse events. Positive improvements were established in lower extremity function and femoral trochanter BMD. Clinical trial registered at ClinicalTrials.gov (NCT01357278). Therapy, level 4.

Longitudinal measurements of MRI-T2 in boys with Duchenne muscular dystrophy: Effects of age and disease progression

Duchenne muscular dystrophy (DMD) is characterized by an increased muscle damage and progressive replacement of muscle by noncontractile tissue. Both of these pathological changes can lengthen the MRI transverse proton relaxation time (T2). The current study measured longitudinal changes in T2 and its distribution in the lower leg of 16 boys with DMD (5–13years, 15 ambulatory) and 15 healthy controls (5–13years). These muscles were chosen to allow extended longitudinal monitoring, due to their slow progression compared with proximal muscles in DMD. In the soleus muscle of boys with DMD, T2 and the percentage of pixels with an elevated T2 (⩾2SD above control mean T2) increased significantly over 1year and 2years, while the width of the T2 histogram increased over 2years. Changes in soleus T2 variables were significantly greater in 9–13years old compared with 5–8years old boys with DMD. Significant correlations between the change in all soleus T2 variables over 2years and the change in functional measures over 2years were found. MRI measurement of muscle T2 in boys with DMD is sensitive to disease progression and shows promise as a clinical outcome measure.