Initial Evaluation of the Pediatric PROMIS Health Domains In Children and Adolescents with Sickle Cell Disease (SCD).

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3829-3829
Author(s):  
Carlton Dampier ◽  
Courtney Thornburg ◽  
Anthea Louise Greenway ◽  
Dana Faircloth ◽  
Heather Gross ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Low Energy ◽  
Pain Interference ◽  
Cell Disease ◽  
Clinical Events ◽  
Item Banks ◽  
Patient Reported ◽  
Health Domains

Abstract Abstract 3829 The Patient-Reported Outcomes Measurement Information System (PROMIS) project was developed to advance the science and application of patient-reported outcomes (PRO) associated with chronic diseases, and is supported as a trans-NIH initiative as part of the NIH Roadmap for Medical Research. One main PROMIS goal is to develop a set of item banks and computerized adaptive tests (CAT) that could be used by the clinical research community for clinical trials or health outcome studies. The PROMIS pediatric project is developing self-report PRO for youths aged 8–17 years across several health domains that are important across a variety of illnesses, including physical function, pain, fatigue, emotional distress, and social function. In addition to validation in a large sample of healthy children, these measures are being validated in a variety of pediatric chronic illnesses, including sickle cell disease (SCD). We hypothesized that the pediatric PROMIS measure would be sensitive to changes in health status of children with SCD. A one-time cross-sectional study was conducted of the pediatric PROMIS item banks in children with SCD. A convenience sample of SCD patients aged 8–17 years followed at 2 large Sickle Cell programs (Emory University and Duke University) was recruited from routine clinic visits. Individuals were eligible to participate if they could speak and read English and were able to complete a computer administered survey. Children completed the pediatric PROMIS survey over the internet using a computer adapted testing (CAT) protocol on the PROMIS Assessment Center secure website. Children were supervised by the research staff to ensure that the responses were self-reported. Parents also completed a short demographics questionnaire, and medical related information was verified from medical records. A total of 236 subjects (mean age 12.49 ± 2.82, 49.8% female) participated in the study (Emory 170, Duke 66) over an 8 month period from May 2009 to January 2010. Most participants had an SS genotype (76.5%), while 16.7% had SC, 4.7% had Sickle B+thalassemia, and 1.3% had Sickle B0 thalassemia. At the time of the study 19.1% were receiving chronic transfusions and 45.5% were taking hydroxyurea. In the prior 6 months, participants reported a mean (SD) of 4.93±9.08 home-managed painful episodes; 1.21±2.91 ED managed painful episodes; and 0.72±1.71 hospitalizations. Parents reported daily analgesic usage by 35%, and 17.1% with hip or joint problems. Scores on the pain interference domain increased with increasing age (r = .175, p=.007). Correlations between age and all other domains were not significant. Physical function dexterity and mobility, depression, pain interference, tired, and low energy domain scores were reduced in children with a reported hip or joint problem compared to for those without hip or joint problems (all p<.05). The occurrence of pain at home requiring treatment in the previous 7 days was associated with declines in functioning in the following domains: depression, anxiety, pain interference, fatigue, low energy, dexterity, and mobility (all p<0.01). Similarly, the number of pain episodes managed at home in the past 6 months was positively correlated with the following domains: pain interference (r = .21, p < .002), depression (r=-.6, p=.01), tired (r=-.26, p<.001), low energy (r=.17, p=.03), and mobility (r=-.23, p=.004). Initial evaluation of the the pediatric PROMIS item banks indicate that self-reported health domains vary with overall clinical severity and frequency of recent clinical events. Current longitudinal studies with these measures at the Emory Sickle Cell Consortium are designed to determine their sensitivity to specific clinical events (hospitalized painful episodes) and related minimally significant differences. These results suggest that the PROMIS pediatric measures should be considered for use in future pediatric SCD clinical trials. Supported by NIH U01-AR052181. Disclosures: Dampier: Anthera Pharmaceuticals Inc: Chair DSMB; Glycomimetics Inc: Chair DSMB.

scholarly journals End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain

2019 ◽  
Vol 3 (23) ◽  
pp. 3982-4001 ◽  
Author(s):  
Ann T. Farrell ◽  
Julie Panepinto ◽  
C. Patrick Carroll ◽  
Deepika S. Darbari ◽  
Ankit A. Desai ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Cell Disease ◽  
End Points ◽  
Additional Clinical Trial ◽  
Patient Reported ◽  
The Brain

Abstract To address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.

scholarly journals Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 196-205
Author(s):  
Sherif M. Badawy
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stakeholder Engagement ◽  
Patient Reported Outcomes ◽  
Outcomes Assessment ◽  
Cell Disease ◽  
Patient Centered ◽  
Patient Reported

Abstract Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.

scholarly journals Evaluation of Longitudinal Pain Study in Sickle Cell Disease (ELIPSIS) by Electronic Patient-Reported Outcomes, Actigraphy, and Biomarkers

Blood ◽  
2020 ◽  
Author(s):  
Debra Pittman ◽  
Patrick C Hines ◽  
David Roger Beidler ◽  
Denis Rybin ◽  
Andrew L Frelinger ◽  
...  
Keyword(s):  
Health Care ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Care Utilization ◽  
Cell Disease ◽  
Patient Reported ◽  
At Home

Clinical trials in sickle cell disease (SCD) often focus on health care utilization for painful vaso-occlusive crises (VOC). However, no objective, quantifiable pain biomarkers exist, pain is not specific to VOCs, health care utilization varies between patients, unreported at-home VOCs likely contribute to long-term outcomes, and patient-reported outcomes are seldom considered. This non-interventional, longitudinal, 6-month study aimed to develop tools to identify VOCs in SCD patients with or without health care utilization. Participants wore an actigraph device, tracking sleep and activity. SCD patients used an electronic patient-reported outcome (ePRO) tool collecting pain, medication, fatigue, and daily function. Patients self-reported when they experienced VOC pain (VOC day). Biomarkers were collected every 3 weeks (non-VOC). Self-reported VOCs triggered at-home or in-hospital blood collection. The study enrolled 37 participants with SCD; 35 completed the study. Participants reported 114 VOC events and 346 VOC days, of which 62.3% and 78.3%, respectively, were self-treated at home. The ePRO and actigraphy captured endpoints of pain, functionality, fatigue, activity, and sleep; each was significantly altered on VOC days compared with non-VOC days. Biomarkers collected at home or in hospital on VOC days were significantly altered compared with non-VOC baseline values, including leukocyte-platelet aggregates, microfluidic-based blood cell adhesion, interleukin-6, C-reactive protein, interleukin-10, tumor necrosis factor-alpha, and thrombin-antithrombin. ELIPSIS: demonstrates the feasibility of accurately monitoring out-of-hospital pain, using patient-reported VOC days as potential endpoints for clinical trials in SCD; showed changes in biomarkers and activity measured by actigraphy that may enable improved identification and assessment of VOCs.

scholarly journals Patient-Reported Outcomes Demonstrate The Value Of Non-Operative And Operative Orthopedic Treatments And Predict The Odds Of Improvement With Surgery

2017 ◽  
Vol 2 (3) ◽  
pp. 2473011417S0000
Author(s):  
Andrew Haskell ◽  
Todd S. Kim
Keyword(s):  
Pain Intensity ◽  
Patient Reported Outcomes ◽  
Pain Interference ◽  
Surgical Patients ◽  
Final Visit ◽  
Adaptive Tests ◽  
Item Banks ◽  
Patient Reported ◽  
Computer Adaptive Tests ◽  
Better Than

Category: Outcomes Measurement Introduction/Purpose: The importance of patient reported outcomes (PROs) has become increasingly recognized as an important tool to measure our clinical value. The National Institute of Health (NIH) created the Patient-Reported Outcomes Measurement Information System (PROMIS), a series of validated item banks, to help clinicians and researches measure key clinical domains. The PROMIS computer adaptive tests (CAT) may be administered with minimal resources or administrative burden. This study describes the results of administering computer adaptive tests (CAT) to every patient in a high volume Orthopedic Surgery practice. We test the hypotheses that both non-operative treatment and operative treatments improve PRO scores. Furthermore, we test the hypothesis that preoperative scores in these domains may be used to predict chances of improvement after surgery. Methods: The PROMIS CAT was administered prospectively for all patients as part of standard clinic intake and recorded in the patient’s electronic medical record (EMR) at each clinic visit. The PROMIS item banks are normalized to mean 50±10 for the US population. De-identified data was retrospectively extracted from the EMR including PROMIS scores, demographic information, as well as surgery specific information. As of this submission, 1688 PROMIS CATs from March 2015 to September 2016 have been analyzed. Data for initial and final clinic visits, as well as for the final preoperative visit for patients who had surgery, are compared using Wilcoxon Matched Pairs Test for paired samples and Mann-Whitney U Test for unpaired samples. Linear regression is used to assess the association of initial values to change in value after treatment. The effect of stratified initial clinic domain value on odds of improving with surgery is assessed using Analysis of Variance. Results: Non-surgical and surgical patients present with similar pain intensity (49.6±7.9 vs. 49.4±7.8). Surgical patients do not improve prior to surgery, but both improve by their final visit (45.7±7.5, 43.7±8.8, p<0.05). Non-surgical and surgical patients present with similar pain interference (60.0±8.4 vs. 60.3±8.9). Surgical patients do not improve prior to surgery, but both improve by their final visit (56.9±8.8, 54.3±9.4, p<0.05). For surgical patients, change in pain intensity and pain interference correlate with initial values (R2 0.32 and 0.27, p<0.05). The percentage whose pain intensity improves after surgery when initial value is over one SD worse than mean is 96%, within one SD worse than mean is 81%, within one SD better than mean is 56%, and over one SD better than mean is 40% (p<0.05). Conclusion: Both non-operative and operative orthopedic treatments improve patient reported pain intensity and pain interference. For patients that have surgery, patients that present with more severe symptoms tend to improve more with surgery. Furthermore, the odds of improving after surgery can be calculated based on preoperative PRO scores. This may allow surgeons to counsel patients about the potential benefits of surgery with personalized precision that is currently unavailable. Measuring PROs using PROMIS CATs demonstrates the value of both non-operative and operative Orthopedic Surgery care for our patients. Preoperative PRO scores may predict the odds of successful surgical intervention.

POST Marketing Observational Study of Children (6 years or older) Treated with Deferasirox (Exjade ICL 670)-One Year analysis

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 5295-5295
Author(s):  
Nagina Parmar ◽  
Manroop Singh ◽  
Isaac Odame ◽  
Melanie Kirby
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Chelation Therapy ◽  
Descriptive Statistics ◽  
Cell Disease ◽  
Outcomes Measures ◽  
Short Period ◽  
Patient Reported

Abstract Abstract 5295 In 2006, deferasirox was licensed in Canada for use in children over the age of 6 years. Deferasirox (Exjade â, ICL 670) is an orally active iron chelators which represents the new class of tridentate iron chelator. Deferasirox has been formulated as a dispersible tablet based on technical feasibility, stability, and adequate bioavailability. Deferasirox has been shown to be as effective as deferral (DFO) and has a clinically manageable safety profile (the most common side effects are gastrointestinal disturbances and rashes). Few studies have been conducted to compare the satisfaction, convenience, activity limitations, and patient preferences of deferasirox in comparison with DFO in sickle cell and thalassemia patients. The purpose of the present study was to evaluate the long-term response of deferosirox in iron-overloaded children aged 6 years or older treated with transfusion dependent anemia and secondary objective was to collect and analyze the following parameters such as serum ferrtin, SLT, AST, creatinine, audiology and ophthalmology, liver iron concentrations (LIC) and patient reported outcomes measures over a period of 2 years from the start of deferasirox. The study population comprised of male and female patients aged 6 years or <17.99 with chronic iron overload related to blood transfusions in patients with Thalassemia and Sickle cell disease. Fifty-one patients were enrolled into this study at the Hematology Clinic, Hospital for Sick Children, Toronto, ON Canada. This study was observational and does not impose a therapy protocol, diagnostic/therapeutic interventions or a strict visit schedule. Patients were treated with deferasirox according to the local physician's judgment and in accordance with the local (country-specific) deferasirox prescribing information. Data were collected at study entry (baseline), and at 12 months. Patients were approached for the study after the ethics approval was obtained. All variables were explored and summarized using descriptive statistics such as means, standard deviation, median and ranges, counts and proportions, graphs as appropriate. Patients reported outcomes measures questionnaires were given after the written consent/assent was obtained. Statistical analysis was done by descriptive statistics and paired t-test. Of the 51 subjects, 30 were females, 21 were males, and median age for the patients was 14 ± 5.3. Out of 51 patients, 49 patients had thalassemia and two had sickle cell disease. All of the subjects were on desferal chelation therapy before the start of the deferasirox, Analysis of patient reported outcomes measures showed that majority of the patients (73%) were very satisfied with deferasirox, when they started and after 12 months on the study, however 1 patient who reported dissatisfaction in 2009, reported satisfaction in year 2010. The main reason for the deferasirox, treatment preference were relief of pain associated with injections (30%) and more convenient (35%) in administration. Other reasons given were improved sleep patterns (2%) and less disruption to the family (6%). At baseline and 12 months laboratory evaluations were as follows: Mean Creatinine values (88% of the patients) (baseline- 34.41±11.49, 12 months – 52.41 ± 19.82), Mean ALT (65% of the patients) (baseline- 41.73 ± 7.32, 12 months – 30.94 ± 5.30) and serum ferritin (baseline- 1995± 276, 12 month, 1833 ± 203). Mean LIC (baseline-11.08 ± 1.29, 12 months- 7.87 ± 1.12) evaluations showed the incremental decrease over time with 14/51 showed the values of <3 over a 12 months period. Chelation therapy was held for some patients for a short period to prevent toxicity related to the chelator. Mild (4/51) and moderate (1/51) hearing loss were observed in subjects over period from 2009–2010. Data will be collected after 24 months to evaluate their long-term responses and for comparative analysis. Further, we can conclude that given the high levels of satisfaction, it is likely that the quality of life and adherence to chelation treatment have improved for patients who are receiving the deferasirox treatment compared with previous subcutaneous chelation treatment. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Psychometrics of three Swedish physical pediatric item banks from the Patient-Reported Outcomes Measurement Information System (PROMIS)®: pain interference, fatigue, and physical activity

2021 ◽  
Vol 5 (1) ◽  
Author(s):  
Frida Carlberg Rindestig ◽  
Marie Wiberg ◽  
John Eric Chaplin ◽  
Eva Henje ◽  
Inga Dennhag
Keyword(s):  
Physical Activity ◽  
Patient Reported Outcomes ◽  
School Health ◽  
Factor Models ◽  
Pain Interference ◽  
Measurement Information ◽  
Outcomes Measurement ◽  
Item Banks ◽  
Patient Reported ◽  
Measurement Information System

Abstract Background The Patient-Reported Outcomes Measurement Information System (PROMIS®) aims to provide self-reported item banks for several dimensions of physical, mental and social health. Here we investigate the psychometric properties of the Swedish pediatric versions of the Physical Health item banks for pain interference, fatigue and physical activity which can be used in school health care and other clinical pediatric settings. Physical health has been shown to be more important for teenagers’ well-being than ever because of the link to several somatic and mental conditions. The item banks are not yet available in Sweden. Methods 12- to 19-year-old participants (n = 681) were recruited in public school settings, and at a child- and psychiatric outpatient clinic. Three one-factor models using CFA were performed to evaluate scale dimensionality. We analyzed monotonicity and local independence. The items were calibrated by fitting the graded response model. Differential Item analyses (DIF) for age, gender and language were calculated. Results As part of the three one-factor models, we found support that each item bank measures a unidimensional construct. No monotonicity or local dependence were found. We found that 11 items had significant lack of fit in the item response theory (IRT) analyses. The result also showed DIF for age (seven items) and language (nine items). However, the differences on item fits and effect sizes of McFadden were negligible. After considering the analytic results, graphical illustration, item content and clinical relevance we decided to keep all items in the item banks. Conclusions We translated and validated the U.S. PROMIS item banks pain interference, fatigue and physical activity into Swedish by applying CFA, IRT and DIF analyses. The results suggest adequacy of the translations in terms of their psychometrics. The questionnaires can be used in school health and other pediatric care. Future studies can be to use Computerized Adaptive Testing (CAT), which provide fewer but reliable items to the test person compared to classical testing.

scholarly journals Can Patient Reported Outcomes Guide Therapy Needs in Foot and Ankle Patients?

2018 ◽  
Vol 3 (3) ◽  
pp. 2473011418S0025
Author(s):  
Jeff Houck ◽  
Jillian Santer ◽  
Judith Baumhauer
Keyword(s):  
Physical Function ◽  
Patient Reported Outcomes ◽  
Predictive Ability ◽  
Pain Interference ◽  
Measurement Information ◽  
Foot And Ankle ◽  
Data Set ◽  
T Score ◽  
Patient Reported ◽  
Health Domains

Category: Other Introduction/Purpose: The patient acceptable symptom state (PASS) is a validated question establishing if patients activity and symptoms are at a satisfactory low level for pain and function. Surprisingly, ~20% of foot and ankle patients at their initial visit present for care with an acceptable symptom state (i.e. PASS yes). These patients are important to identify to prevent over treatment and avoid excessive cost. It is also unclear what health domains (Pain Interference (PI), Physical Function (PF), or Depression (Dep)) influence a patients judgement of their PASS state (i.e. why they are seeking treatment). The purpose of this analysis is to document the prevalance of PASS state and determine the health domains that discriminate PASS patients and predict PASS state at the initiation of rehabilitation. Methods: Patient reported outcomes measurement information system (PROMIS) computer adaptive test (CAT) scales PF, pain PIand Dep and PASS ratings starting in summer 2017 were routinely collected for patient care. Of 746 unique patients in this data set, 114 patients had ICD-10 codes that were specific to the foot and ankle. Average age was 51years (±18) and 54.4% were female. Patients were seen an average of 19.8(±15.9) days from their referral and were billed as low (51.7%), moderate (44.7%) and high complexity (2.7%) evaluations per current procedural code (CPT) visits. ANOVA models were used to evaluate differences in PROMIS scales by PASS state (Yes/No). The area under receiver operator curve (AUC) was used to determine the predictive ability of each PROMIS scale to determine a PASS state. Thresholds for near 95% specificity were also calculated for a PASS Yes state for each PROMIS scale. Results: The prevalance of PASS Yes patients was 13.2% (15/114). Pass Yes patients were significantly better by an average of 7.2 to 8.0 points across all PROMIS health domains compared to PASS No patients (Table 1). ROC analysis suggested that Dep (AUC=0.73(0.07) p=0.005) was the highest predictor of PASS status followed by PI (AUC=0.70(0.08) p=0.012) and PF (AUC=0.69(0.07) p=0.18). The threshold PROMIS t-score values for determining PASS Yes with nearest 95% specificity were PF = 51.9, PI = 50.6, and Dep = 34. Conclusion: Surprising, yet consistent with previous data, 13.2% of patients at their initial physical therapy consultation rated themselves at an acceptable level of activity and symptoms. Health domains of physical function, pain interference, and depression were better in these patients and showed moderate ability (AUC~0.7) to identify these patients. The PROMIS thresholds suggest patients are identified by pain and physical function equal to the average of the US population (PROMIS T-Score ~50) and extremely low depression scores (34). Clinically it is important to recognize these patients and purposefully provide treatments that reinforce their self efficacy and prevent unnecessary costly treatments.

Standardizing Patient-Reported Outcomes Assessment in Cancer Clinical Trials: A Patient-Reported Outcomes Measurement Information System Initiative

2007 ◽  
Vol 25 (32) ◽  
pp. 5106-5112 ◽  
Author(s):  
Sofia F. Garcia ◽  
David Cella ◽  
Steven B. Clauser ◽  
Kathryn E. Flynn ◽  
Thomas Lad ◽  
...  
Keyword(s):  
Information System ◽  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Measurement Information ◽  
Outcomes Measurement ◽  
Oncology Research ◽  
Patient Reported ◽  
Health Domains ◽  
Measurement Information System ◽  
Reported Health

Patient-reported outcomes (PROs), such as symptom scales or more broad-based health-related quality-of-life measures, play an important role in oncology clinical trials. They frequently are used to help evaluate cancer treatments, as well as for supportive and palliative oncology care. To be most beneficial, these PROs must be relevant to patients and clinicians, valid, and easily understood and interpreted. The Patient-Reported Outcomes Measurement Information System (PROMIS) Network, part of the National Institutes of Health Roadmap Initiative, aims to improve appreciably how PROs are selected and assessed in clinical research, including clinical trials. PROMIS is establishing a publicly available resource of standardized, accurate, and efficient PRO measures of major self-reported health domains (eg, pain, fatigue, emotional distress, physical function, social function) that are relevant across chronic illnesses including cancer. PROMIS is also developing measures of self-reported health domains specifically targeted to cancer, such as sleep/wake function, sexual function, cognitive function, and the psychosocial impacts of the illness experience (ie, stress response and coping; shifts in self-concept, social interactions, and spirituality). We outline the qualitative and quantitative methods by which PROMIS measures are being developed and adapted for use in clinical oncology research. At the core of this activity is the formation and application of item banks using item response theory modeling. We also present our work in the fatigue domain, including a short-form measure, as a sample of PROMIS methodology and work to date. Plans for future validation and application of PROMIS measures are discussed.

Sign in / Sign up

Export Citation Format

Share Document