Using Machine Learning to Leverage Biomarker Change and Predict Colorectal Cancer Recurrence.

PURPOSE: The risk of colorectal cancer (CRC) recurrence after primary treatment varies across individuals and over time. Using patients' most up-to-date information, including carcinoembryonic antigen (CEA) biomarker profiles, to predict risk could improve personalized decision making. METHODS: We used electronic health record data from an integrated health system on a cohort of patients diagnosed with American Joint Committee on Cancer stage I-III CRC between 2008 and 2013 (N = 3,970) and monitored until recurrence or end of follow-up. We addressed missingness in recurrence outcomes and longitudinal CEA measures, and engineered CEA features using current and past biomarker values for inclusion in a risk prediction model. We used a discrete time Superlearner model to evaluate various algorithms for predicting recurrence. We evaluated the time-varying discrimination and calibration of the algorithms and assessed the role of individual predictors. RESULTS: Recurrence was documented in 448 (11.3%) patients. XGBoost with depth = 1 (XGB-D1) predicted recurrence substantially better than all other algorithms at all time points, with AUC ranging from 0.87 (95% CI, 0.86 to 0.88) at 6 months to 0.94 (95% CI, 0.92 to 0.96) at 54 months. The only variable used by XGB-D1 was 6-month change in log CEA. Predicted 1-year risk of recurrence was nearly zero for patients whose log CEA did not increase in the last 6 months, between 12.2% and 34.1% for patients whose log CEA increased between 0.10 and 0.40, and 43.6% for those with a log CEA increase >0.40. Compared with XGB, penalized regression approaches (lasso, ridge, and elastic net) performed poorly, with AUCs ranging from 0.58 to 0.69. CONCLUSION: A flexible, machine learning approach that incorporated longitudinal CEA information yielded a simple and high-performing model for predicting recurrence on the basis of 6-month change in log CEA.

Racial Disparities in the Ascertainment of Cancer Recurrence in Electronic Health Records.

PURPOSE: There is growing interest in using computable phenotypes or proxies to identify important clinical outcomes, such as cancer recurrence, in rich electronic health records data. However, the race/ethnicity-specific accuracies of these proxies remain unclear. We examined whether the accuracy of a proxy for colorectal cancer (CRC) recurrence differed by race/ethnicity and the possible mechanisms that drove the differences. METHODS: Using data from a large integrated health care system, we identified a stratified random sample of 282 Black/African American (AA), Hispanic, and non-Hispanic White (NHW) patients with CRC who received primary treatment. Patient 5-year recurrence status was estimated using a utilization-based proxy and evaluated against the true recurrence status obtained using detailed chart review and by race/ethnicity. We used covariate-adjusted probit regression models to estimate the associations between race/ethnicity and misclassification. RESULTS: The recurrence proxy had excellent overall accuracy (positive predictive value [PPV] 89.4%; negative predictive value 96.5%; mean difference in timing 1.96 months); however, accuracy varied by race/ethnicity. Compared with NHW patients, PPV was 14.9% lower (95% CI, 2.53 to 28.6) among Hispanic patients and 4.3% lower (95% CI, -4.8 to 14.8) among Black/AA patients. The proxy disproportionately inflated the 5-year recurrence incidence for Hispanic patients by 10.6% (95% CI, 4.2 to 18.2). Compared with NHW patients, proxy recurrences for Hispanic patients were almost three times as likely to have been misclassified as positive (adjusted risk ratio 2.91 [95% CI, 1.21 to 8.31]). Higher false positives among racial/ethnic minorities may be related to higher prevalence of noncancerous lung-related problems and substantial delays in primary treatment because of insufficient patient-provider communication and abnormal treatment patterns. CONCLUSION: Using a proxy with worse accuracy among racial/ethnic minority patients to estimate population health may misdirect resources and support erroneous conclusions around treatment benefit for these patients.

Comparison of Teleintegrated Care and Telereferral Care for Treating Complex Psychiatric Disorders in Primary Care: A Pragmatic Randomized Comparative Effectiveness Trial.

Importance: Only one-third of patients with complex psychiatric disorders engage in specialty mental health care, and only one-tenth receive adequate treatment in primary care. Scalable approaches are critically needed to improve access to effective mental health treatments in underserved primary care settings. Objective: To compare 2 clinic-to-clinic interactive video approaches to delivering evidence-based mental health treatments to patients in primary care clinics. Design, Setting, and Participants: This pragmatic comparative effectiveness trial used a sequential, multiple-assignment, randomized trial (SMART) design with patient-level randomization. Adult patients treated at 24 primary care clinics without on-site psychiatrists or psychologists from 12 federally qualified health centers in 3 states who screened positive for posttraumatic stress disorder and/or bipolar disorder and who were not already receiving pharmacotherapy from a mental health specialist were recruited from November 16, 2016, to June 30, 2019, and observed for 12 months. Interventions: Two approaches were compared: (1) telepsychiatry/telepsychology-enhanced referral (TER), where telepsychiatrists and telepsychologists assumed responsibility for treatment, and (2) telepsychiatry collaborative care (TCC), where telepsychiatrists provided consultation to the primary care team. TER included an adaptive intervention (phone-enhanced referral [PER]) for patients not engaging in treatment, which involved telephone outreach and motivational interviewing. Main Outcomes and Measures: Survey questions assessed patient-reported outcomes. The Veterans RAND 12-item Health Survey Mental Component Summary (MCS) score was the primary outcome (range, 0-100). Secondary outcomes included posttraumatic stress disorder symptoms, manic symptoms, depressive symptoms, anxiety symptoms, recovery, and adverse effects. Results: Of 1004 included participants, 701 of 1000 (70.1%) were female, 660 of 994 (66.4%) were White, and the mean (SD) age was 39.4 (12.9) years. Baseline MCS scores were 2 SDs below the US mean; the mean (SD) MCS scores were 39.7 (14.1) and 41.2 (14.2) in the TCC and TER groups, respectively. There was no significant difference in 12-month MCS score between those receiving TCC and TER (ß = 1.0; 95% CI, -0.8 to 2.8; P = .28). Patients in both groups experienced large and clinically meaningful improvements from baseline to 12 months (TCC: Cohen d = 0.81; 95% CI, 0.67 to 0.95; TER: Cohen d = 0.90; 95% CI, 0.76 to 1.04). For patients not engaging in TER at 6 months, there was no significant difference in 12-month MCS score between those receiving PER and TER (ß = 2.0; 95% CI, -1.7 to 5.7; P = .29). Conclusions and Relevance: In this comparative effectiveness trial of patients with complex psychiatric disorders randomized to receive TCC or TER, significantly and substantially improved outcomes were observed in both groups. From a health care system perspective, clinical leadership should implement whichever approach is most sustainable. Trial Registration: ClinicalTrials.gov Identifier: NCT02738944.

Enteral Iron Supplementation in Infants Born Extremely Preterm and its Positive Correlation with Neurodevelopment; Post Hoc Analysis of the Preterm Erythropoietin Neuroprotection Trial Randomized Controlled Trial.

OBJECTIVES: To test whether an increased iron dose is associated with improved neurodevelopment as assessed by the Bayley Scales of Infant Development, third edition (BSID-III) among infants enrolled in the Preterm Erythropoietin (Epo) Neuroprotection Trial (PENUT). STUDY DESIGN: This is a post hoc analysis of a randomized trial that enrolled infants born at 24-28 completed weeks of gestation. All infants in PENUT who were assessed with BSID-III at 2 years were included in this study. The associations between enteral iron dose at 60 and 90 days and BSID-III component scores were evaluated using generalized estimating equations models adjusted for potential confounders. RESULTS: In total, 692 infants were analyzed (355 placebo, 337 Epo). Enteral iron supplementation ranged from 0 to 14.7 mg/kg/d (IQR 2.1-5.8 mg/kg/d) at day 60, with a mean of 3.6 mg/kg/d in infants treated with placebo and 4.8 mg/kg/d in infants treated with Epo. A significant positive association was seen between BSID-III cognitive scores and iron dose at 60 days, with an effect size of 0.77 BSID points per 50 mg/kg increase in cumulative iron dose (P = .03). Greater iron doses were associated with greater motor and language scores but did not reach statistical significance. Results at 90 days were not significant. The effect size in the infants treated with Epo compared with placebo was consistently greater. CONCLUSIONS: A positive association was seen between iron dose at 60 days and cognitive outcomes. Our results suggest that increased iron supplementation in infants born preterm, at the doses administered in the PENUT Trial, may have positive neurodevelopmental effects, particularly in infants treated with Epo. TRIAL REGISTRATION: Clinicaltrials.gov: NCT01378273.

Providing Epidemiological Data in Lumbar Spine Imaging Reports Did Not Affect Subsequent Utilization of Spine Procedures: Secondary Outcomes from a Stepped-Wedge Randomized Controlled Trial.

OBJECTIVE: To evaluate the effect of inserting epidemiological information into lumbar spine imaging reports on subsequent nonsurgical and surgical procedures involving the thoracolumbosacral spine and sacroiliac joints. DESIGN: Analysis of secondary outcomes from the Lumbar Imaging with Reporting of Epidemiology (LIRE) pragmatic stepped-wedge randomized trial. SETTING: Primary care clinics within four integrated health care systems in the United States. SUBJECTS: 238,886 patients ≥18 years of age who received lumbar diagnostic imaging between 2013 and 2016. METHODS: Clinics were randomized to receive text containing age- and modality-specific epidemiological benchmarks indicating the prevalence of common spine imaging findings in people without low back pain, inserted into lumbar spine imaging reports (the "LIRE intervention"). The study outcomes were receiving 1) any nonsurgical lumbosacral or sacroiliac spine procedure (lumbosacral epidural steroid injection, facet joint injection, or facet joint radiofrequency ablation; or sacroiliac joint injection) or 2) any surgical procedure involving the lumbar, sacral, or thoracic spine (decompression surgery or spinal fusion or other spine surgery). RESULTS: The LIRE intervention was not significantly associated with subsequent utilization of nonsurgical lumbosacral or sacroiliac spine procedures (odds ratio [OR] = 1.01, 95% confidence interval [CI] 0.93-1.09; P = 0.79) or any surgical procedure (OR = 0.99, 95 CI 0.91-1.07; P = 0.74) involving the lumbar, sacral, or thoracic spine. The intervention was also not significantly associated with any individual spine procedure. CONCLUSIONS: Inserting epidemiological text into spine imaging reports had no effect on nonsurgical or surgical procedure utilization among patients receiving lumbar diagnostic imaging.

A Randomized Trial Comparing Antibiotics with Appendectomy for Appendicitis.

BACKGROUND: Antibiotic therapy has been proposed as an alternative to surgery for the treatment of appendicitis. METHODS: We conducted a pragmatic, nonblinded, noninferiority, randomized trial comparing antibiotic therapy (10-day course) with appendectomy in patients with appendicitis at 25 U.S. centers. The primary outcome was 30-day health status, as assessed with the European Quality of Life-5 Dimensions (EQ-5D) questionnaire (scores range from 0 to 1, with higher scores indicating better health status; noninferiority margin, 0.05 points). Secondary outcomes included appendectomy in the antibiotics group and complications through 90 days; analyses were prespecified in subgroups defined according to the presence or absence of an appendicolith. RESULTS: In total, 1552 adults (414 with an appendicolith) underwent randomization; 776 were assigned to receive antibiotics (47% of whom were not hospitalized for the index treatment) and 776 to undergo appendectomy (96% of whom underwent a laparoscopic procedure). Antibiotics were noninferior to appendectomy on the basis of 30-day EQ-5D scores (mean difference, 0.01 points; 95% confidence interval [CI], -0.001 to 0.03). In the antibiotics group, 29% had undergone appendectomy by 90 days, including 41% of those with an appendicolith and 25% of those without an appendicolith. Complications were more common in the antibiotics group than in the appendectomy group (8.1 vs. 3.5 per 100 participants; rate ratio, 2.28; 95% CI, 1.30 to 3.98); the higher rate in the antibiotics group could be attributed to those with an appendicolith (20.2 vs. 3.6 per 100 participants; rate ratio, 5.69; 95% CI, 2.11 to 15.38) and not to those without an appendicolith (3.7 vs. 3.5 per 100 participants; rate ratio, 1.05; 95% CI, 0.45 to 2.43). The rate of serious adverse events was 4.0 per 100 participants in the antibiotics group and 3.0 per 100 participants in the appendectomy group (rate ratio, 1.29; 95% CI, 0.67 to 2.50). CONCLUSIONS: For the treatment of appendicitis, antibiotics were noninferior to appendectomy on the basis of results of a standard health-status measure. In the antibiotics group, nearly 3 in 10 participants had undergone appendectomy by 90 days. Participants with an appendicolith were at a higher risk for appendectomy and for complications than those without an appendicolith. (Funded by the Patient-Centered Outcomes Research Institute; CODA ClinicalTrials.gov number, NCT02800785.).

High-Dose Erythropoietin in Extremely Low Gestational Age Neonates Does Not Alter Risk of Retinopathy of Prematurity.

INTRODUCTION: The Preterm Erythropoietin (Epo) Neuroprotection (PENUT) Trial sought to determine the safety and efficacy of early high-dose Epo as a potential neuroprotective treatment. We hypothesized that Epo would not increase the incidence or severity of retinopathy of prematurity (ROP). METHODS: A total of 941 infants born between 24-0/7 and 27-6/7 weeks' gestation were randomized to 1,000 U/kg Epo or placebo intravenously for 6 doses, followed by subcutaneous or sham injections of 400 U/kg Epo 3 times a week through 32 weeks post-menstrual age. In this secondary analysis of PENUT trial data, survivors were evaluated for ROP. A modified intention-to-treat approach was used to compare treatment groups. In addition, risk factors for ROP were evaluated using regression methods that account for multiples and allow for adjustment for treatment and gestational age at birth. RESULTS: Of 845 subjects who underwent ROP examination, 503 were diagnosed with ROP with similar incidence and severity between treatment groups. Gestational age at birth, birth weight, prenatal magnesium sulfate, maternal antibiotic exposure, and presence of heart murmur at 2 weeks predicted the development of any ROP, while being on high-frequency oscillator or high-frequency jet ventilation (HFOV/HFJV) at 2 weeks predicted severe ROP. CONCLUSION: Early high-dose Epo followed by maintenance dosing through 32 weeks does not increase the risk of any or severe ROP in extremely low gestational age neonates. Gestational age, birth weight, maternal treatment with magnesium sulfate, antibiotic use during pregnancy, and presence of a heart murmur at 2 weeks were associated with increased risk of any ROP. Treatment with HFOV/HFJV was associated with an increased risk of severe ROP.

Study to promote innovation in rural integrated telepsychiatry (SPIRIT): Rationale and design of a randomized comparative effectiveness trial of managing complex psychiatric disorders in rural primary care clinics.

OBJECTIVE: Managing complex psychiatric disorders like PTSD and bipolar disorder is challenging in Federally Qualified Health Centers (FQHCs) delivering care to U.S residents living in underserved rural areas. This protocol paper describes SPIRIT, a pragmatic comparative effectiveness trial designed to compare two approaches to managing PTSD and bipolar disorder in FQHCs. INTERVENTIONS: Treatment comparators are: 1) Telepsychiatry Collaborative Care, which integrates consulting telepsychiatrists into primary care teams, and 2) Telepsychiatry Enhanced Referral, where telepsychiatrists and telepsychologists treat patients directly. METHODS: Because Telepsychiatry Enhanced Referral is an adaptive intervention, a Sequential, Multiple Assignment, Randomized Trial design is used. Twenty-four FQHC clinics without on-site psychiatrists or psychologists are participating in the trial. The sample is patients screening positive for PTSD and/or bipolar disorder who are not already engaged in pharmacotherapy with a mental health specialist. Intervention fidelity is measured but not controlled. Patient treatment engagement is measured but not required, and intent-to-treat analysis will be used. Survey questions measure treatment engagement and effectiveness. The Short-Form 12 Mental Health Component Summary (SF-12 MCS) is the primary outcome. RESULTS: A third (34%) of those enrolled (n = 1004) are racial/ethnic minorities, 81% are not fully employed, 68% are Medicaid enrollees, 7% are uninsured, and 62% live in poverty. Mental health related quality of life (SF-12 MCS) is 2.5 standard deviations below the national mean. DISCUSSION: We hypothesize that patients randomized to Telepsychiatry Collaborative Care will have better outcomes than those randomized to Telepsychiatry Enhanced Referral because a higher proportion will engage in evidence-based treatment.

Multi-site Pain Is Associated with Long-term Patient-Reported Outcomes in Older Adults with Persistent Back Pain.

OBJECTIVE: To estimate the prevalence of co-occurring pain sites among older adults with persistent back pain and associations of multisite pain with longitudinal outcomes. DESIGN: Secondary analysis of a cohort study. SETTING: Three integrated health systems in the United States. SUBJECTS: Eight hundred ninety-nine older adults with persistent back pain. METHODS: Participants reported pain in the following sites: stomach, arms/legs/joints, headaches, neck, pelvis/groin, and widespread pain. Over 18 months, we measured back-related disability (Roland Morris, scored 0-24), pain intensity (11-point numerical rating scale), health-related quality of life (EuroQol-5D [EQ-5D], utility from 0-1), and falls in the past three weeks. We used mixed-effects models to test the association of number and type of pain sites with each outcome. RESULTS: Nearly all (N = 839, 93%) respondents reported at least one additional pain site. There were 216 (24%) with one additional site and 623 (69%) with multiple additional sites. The most prevalent comorbid pain site was the arms/legs/joints (N = 801, 89.1%). Adjusted mixed-effects models showed that for every additional pain site, RMDQ worsened by 0.65 points (95% confidence interval [CI] = 0.43 to 0.86), back pain intensity increased by 0.14 points (95% CI = 0.07 to 0.22), EQ-5D worsened by 0.012 points (95% CI = -0.018 to -0.006), and the odds of falling increased by 27% (odds ratio = 1.27, 95% CI = 1.12 to 1.43). Some specific pain sites (extremity pain, widespread pain, and pelvis/groin pain) were associated with greater long-term disability. CONCLUSIONS: Multisite pain is common among older adults with persistent back pain. Number of pain sites was associated with all outcomes; individual pain sites were less consistently associated with outcomes.

Subsequent health-care utilization associated with early physical therapy for new episodes of low back pain in older adults.

BACKGROUND: The association between early physical therapy (PT) and subsequent health-care utilization following a new visit for low back pain is not clear, particularly in the setting of acute low back pain. PURPOSE: This study aimed to estimate the association between initiating early PT following a new visit for an episode of low back pain and subsequent back pain-specific health-care utilization in older adults. DESIGN/SETTING: This is a prospective cohort study. Data were collected at three integrated health-care systems in the United States through the Back Pain Outcomes using Longitudinal Data (BOLD) registry. PATIENT SAMPLE: We recruited 4,723 adults, aged 65 and older, presenting to a primary care setting with a new episode of low back pain. OUTCOME MEASURES: Primary outcome was total back pain-specific relative value units (RVUs), from days 29 to 365. Secondary outcomes included overall RVUs for all health care and use of specific health-care services including imaging (x-ray and magnetic resonance imaging [MRI] or computed tomography [CT]), emergency department visits, physician visits, PT, spinal injections, spinal surgeries, and opioid use. METHODS: We compared patients who had early PT (initiated within 28 days of the index visit) with those not initiating early PT using appropriate, generalized linear models to adjust for potential confounding variables. RESULTS: Adjusted analysis found no statistically significant difference in total spine RVUs between the two groups (ratio of means 1.19, 95% CI of 0.72-1.96, p=.49). For secondary outcomes, only the difference between total spine imaging RVUs and total PT RVUs was statistically significant. The early PT group had greater PT RVUs; the ratio of means was 2.56 (95% CI of 2.17-3.03, p<.001). The early PT group had greater imaging RVUs; the ratio of means was 1.37 (95% CI of 1.09-1.71, p=.01.) CONCLUSIONS: We found that in a group of older adults presenting for a new episode of low back pain, the use of early PT is not associated with any statistically significant difference in subsequent back pain-specific health-care utilization compared with patients not receiving early PT.

Effect of Comorbid Knee and Hip Osteoarthritis on Longitudinal Clinical and Health Care Use Outcomes in Older Adults With New Visits for Back Pain.

OBJECTIVE: To examine if a comorbid diagnosis of knee or hip osteoarthritis (OA) in older adults with new back pain visits is associated with long-term patient-reported outcomes and back-related health care use. DESIGN: Prospective cohort study. SETTING: Three integrated health systems forming the Back pain Outcomes using Longitudinal Data cohort. PARTICIPANTS: Participants (N=5155) were older adults (≥65y) with a new visit for back pain and a complete electronic health record data. INTERVENTIONS: Not applicable; we obtained OA diagnoses using diagnostic codes in the electronic health record 12 months prior to the new back pain visit. MAIN OUTCOME MEASURES: The Roland-Morris Disability Questionnaire (RDQ) and the EuroQol-5D (EQ-5D) were key patient-reported outcomes. Health care use, measured by relative-value units (RVUs), was summed for the 12 months after the initial visit. We used linear mixed-effects models to model patient-reported outcomes. We also used generalized linear models to test the association between comorbid knee or hip OA and total back-related RVUs. RESULTS: Of the 5155 participants, 368 (7.1%) had a comorbid knee OA diagnosis, and 94 (1.8%) had a hip OA diagnosis. Of the participants, 4711 (91.4%) had neither knee nor hip OA. In adjusted models, the 12-month RDQ score was 1.23 points higher (95% confidence interval [CI], 0.72-1.74) for patients with knee OA and 1.26 points higher (95% CI, 0.24-2.27) for those with hip OA than those without knee or hip OA, respectively. A lower EQ-5D score was found among participants with knee OA (.02 lower; 95% CI, -.04 to -.01) and hip OA diagnoses (.03 lower; 95% CI, -.05 to -.01) compared with those without knee or hip OA, respectively. Comorbid knee or hip OA was not significantly associated with total 12-month back-related resource use. CONCLUSIONS: Comorbid knee or hip OA in older adults with a new back pain visit was associated with modestly worse long-term disability and health-related quality of life.

Clinical outcomes of early and later physical therapist services for older adults with back pain.

BACKGROUND CONTEXT: The timing of physical therapy (PT) services and its association with later function and pain are not clear, especially in older adults. PURPOSE: The purpose of this study was to compare clinical outcomes of patients receiving early or later PT services with those not receiving PT among older adults presenting to primary care for a new visit for back pain. STUDY DESIGN/SETTING: Prospective cohort study using the Back Pain Outcomes Using Longitudinal Data registry. PATIENT SAMPLE: A total of 3,705 adults 65 years and older with a new visit for back pain were included. OUTCOME MEASURES: The outcome measures were Roland-Morris Disability Questionnaire (RMDQ), Pain Numerical Rating Scales, and EuroQol-5D. METHODS: We studied two phases of PT utilization: early (0-28 days) and later (3-6 months). At baseline, we selected the participants with complete 12 months of patient-reported outcomes and electronic medical record data. Early PT was defined as initiating PT less than or equal to 28 days from the index visit for back pain. The no early PT group consisted of patients with no PT, no injections, no surgery, and no chiropractic within 28 days. We restricted the later phase analysis to patients with pain greater than 2 of 10 and an RMDQ score greater than 4 to create a subsample of patients with continuing clinically important back pain. We defined later PT as initiating PT between 3 and 6 months after the index visit. The no later PT group consisted of patients without any PT during this time. We used propensity score matching followed by multiple linear regression to estimate the mean difference in outcome. Sensitivity analysis examined clinically important change and dose of PT use among the early PT group. RESULTS: The early PT group had better functional status with an adjusted mean RMDQ of 1.1 points less than the no early PT group (95% confidence interval: -2.2, -0.1) and less back pain of -0.5 (-0.9, -0.1) at 12 months. There was no difference between early PT groups at 3 and 6 months. The odds of a 30% improvement in function or pain were not different between these matched groups at 12 months, but the early PT group had increased odds of a 50% improvement in function at 12 months (odds ratio: 1.58, 95% confidence interval: 1.04, 2.40). There was no difference between later groups at 12 months. Greater dose of PT use within the early PT group was associated with better functional status (p= .01). CONCLUSIONS: We found that among older adults presenting to their primary care providers for a new episode of back pain, early referral to PT resulted in no or minimal differences in pain, function, or health-related quality at 3, 6, or 12 months compared with a matched group that did not receive early PT. Secondary analysis show that patients initiating early PT may be somewhat more likely to experience 50% improvement in function at 12 months.

The clinical course of pain and function in older adults with a new primary care visit for back pain.

OBJECTIVES: To report the clinical course of older adults presenting for a new primary care visit for back pain, no healthcare visit for back pain within the prior 6 months, by describing pain intensity, disability, pain interference, and resolution of back pain over 12 months. DESIGN: Prospective inception cohort study. SETTING: Primary care settings of three integrated healthcare systems in the United States that participated in the Back pain Outcomes using Longitudinal Data (BOLD) registry. PARTICIPANTS: Five thousand two hundred eleven (99.5%) of the 5,239 adults aged 65 and older who had reached their 12-month follow-up date. MEASUREMENTS: Baseline demographic characteristics, EQ-5D score, duration of back pain, expectation for recovery, depression, and anxiety. Participant-reported outcomes of back-related disability (Roland Morris Disability Questionnaire), numerical pain rating scale, pain interference, and resolution of back pain were collected at baseline and 3, 6, and 12 months. RESULTS: Most improvement occurred within the first 3 months. The number and proportion with 30% improvement in back pain increased from 1,950 (42.3%) at 3 months to 1,994 (44.8%) by 12 months, and 1,331 (28.8%) and 1,576 (35.4%) had 30% improvement in disability at 3 and 12 months. Only 23.0% reported that their back pain had resolved at 12 months. Improvements in disability and interference with activity over 12 months differed according to age, duration of back pain, symptoms of depression and anxiety, and expectation for recovery. CONCLUSION: The majority of older adults in primary care practice settings presenting with a new visit for back pain have persistent symptoms, disability, and interference over 12 months of follow-up. Future research is needed to identify risk factors for persistent symptoms and effective interventions.

Back pain in seniors: the Back pain Outcomes using Longitudinal Data (BOLD) cohort baseline data.

BACKGROUND: Back pain represents a substantial burden globally, ranking first in a recent assessment among causes of years lived with disability. Though back pain is widely studied among working age adults, there are gaps with respect to basic descriptive epidemiology among seniors, especially in the United States. Our goal was to describe how pain, function and health-related quality of life vary by demographic and geographic factors among seniors presenting to primary care providers with new episodes of care for back pain. METHODS: We examined baseline data from the Back pain Outcomes using Longitudinal Data (BOLD) registry, the largest inception cohort to date of seniors presenting to a primary care provider for back pain. The sample included 5,239 patients ≥ 65 years old with a new primary care visit for back pain at three integrated health systems (Northern California Kaiser-Permanente, Henry Ford Health System [Detroit], and Harvard Vanguard Medical Associates [Boston]). We examined differences in patient characteristics across healthcare sites and associations of patient sociodemographic and clinical characteristics with baseline patient-reported measures of pain, function, and health-related quality of life. RESULTS: Patients differed across sites in demographic and other characteristics. The Detroit site had more African-American patients (50%) compared with the other sites (7-8%). The Boston site had more college graduates (68%) compared with Detroit (20%). Female sex, lower educational status, African-American race, and older age were associated with worse functional disability as measured by the Roland-Morris Disability Questionnaire. Except for age, these factors were also associated with worse pain. CONCLUSIONS: Baseline pain and functional impairment varied substantially with a number of factors in the BOLD cohort. Healthcare site was an important factor. After controlling for healthcare site, lower education, female sex, African-American race, and older age were associated with worse physical disability and all of these factors except age were associated with worse pain. TRIAL REGISTRATION: Clinical Trials.gov NCT01776242; Registration date: June 13, 2012.

INvestigational Vertebroplasty Efficacy and Safety Trial (INVEST): a randomized controlled trial of percutaneous vertebroplasty.

BACKGROUND: The treatment of painful osteoporotic vertebral compression fractures has historically been limited to several weeks of bed rest, anti-inflammatory and analgesic medications, calcitonin injections, or external bracing. Percutaneous vertebroplasty (the injection of bone cement into the fractured vertebral body) is a relatively new procedure used to treat these fractures. There is increasing interest to examine the efficacy and safety of percutaneous vertebroplasty and to study the possibility of a placebo effect or whether the pain relief is from local anesthetics placed directly on the bone during the vertebroplasty procedure. METHODS/DESIGNS: Our goal is to test the hypothesis that patients with painful osteoporotic vertebral compression fractures who undergo vertebroplasty have less disability and pain at 1 month than patients who undergo a control intervention. The control intervention is placement of local anesthesia near the fracture, without placement of cement. One hundred sixty-six patients with painful osteoporotic vertebral compression fractures will be recruited over 5 years from US and foreign sites performing the vertebroplasty procedure. We will exclude patients with malignant tumor deposit (multiple myeloma), tumor mass or tumor extension into the epidural space at the level of the fracture. We will randomly assign participants to receive either vertebroplasty or the control intervention. Subjects will complete a battery of validated, standardized measures of pain, functional disability, and health related quality of life at baseline and at post-randomization time points (days 1, 2, 3, and 14, and months 1, 3, 6, and 12). Both subjects and research interviewers performing the follow-up assessments will be blinded to the randomization assignment. Subjects will have a clinic visit at months 1 and 12. Spine X-rays will be obtained at the end of the study (month 12) to determine subsequent fracture rates. Our co-primary outcomes are the modified Roland score and pain numerical rating scale at 1 month. DISCUSSION: Although extensively utilized throughout North America for palliation of pain, vertebroplasty still has not undergone rigorous study. The study outlined above represents the first randomized, controlled study that can account for a placebo effect in the setting of vertebroplasty. TRIAL REGISTRATION: Current Controlled Trials ISRCTN81871888.